packet_row_id,priority,evidence_domain,entity_table,evidence_id,benchmark_eligible,evidence_grade,molecule_id,canonical_name,modality,target_gene_symbol,disease_context,evidence_label,source_document_id,pmid,pmcid,doi,source_title,source_url,source_location,assay_id,organism,model_system,cell_line_or_tissue,dose_value,dose_unit,exposure_time_value,exposure_time_unit,replicate_count,sense_sequence,antisense_sequence,guide_sequence,passenger_sequence,seed_region,backbone_chemistry,sugar_modification,base_modification,conjugate_delivery,sequence_annotation_status,modification_annotation_status,missing_sequence,missing_seed,missing_modification,missing_delivery,missing_dose,missing_exposure,missing_model,field_source_location,field_source_quote_or_table_id,source_location_verified,curator_decision,field_curator_id,independent_reviewer_id,review_status,audit_note,do_not_fill_without_source,updated_at
core-field-00001,P0,offtarget,offtarget_evidence,1,yes,A,4,SNA-modified GalNAc-siRNA panel from PMID 41994868,siRNA,TTR,,seed-mediated off-target effect,1031,41994868,PMC13096799,10.1093/nar/gkag345,Acyclic serinol nucleic acid modification of siRNAs overcomes seed region mediated off-target effects while maintaining potency.,https://pubmed.ncbi.nlm.nih.gov/41994868/,Results > Analysis of miRNA-like off-target activity in a luciferase reporter assay; paragraph 3,4,human,COS-7 cells; GalNAc-siRNA targeting Ttr,COS-7 cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00002,P0,offtarget,offtarget_evidence,2,yes,A,5,siVIM-270 chemical modification panel from PMID 41467115,siRNA,VIM,,seed-mediated off-target effect,1157,41467115,PMC12744844,10.1016/j.omtn.2025.102693,An siRMSD parameter of structural distortion induced by chemical modification is predictive of the off-target effect of siRNA.,https://pubmed.ncbi.nlm.nih.gov/41467115/,Results > RNAi and off-target activities of siRNAs with chemical modifications; paragraph 10,5,human,HeLa cells and luciferase reporter constructs,HeLa cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00003,P0,offtarget,offtarget_evidence,4,yes,A,7,APP-AON/ATXN3-AON/HTT-AON panel from PMID 41066632,ASO,,,hybridization/mismatch off-target effect,1442,41066632,PMC12581821,10.1093/hmg/ddaf153,Determining off-target effects of splice-switching antisense oligonucleotides using short read RNAseq in neuronally differentiated human induced pluripotent stem cells.,https://pubmed.ncbi.nlm.nih.gov/41066632/,Results > Hybridization-dependent off-target splicing events do not play a major role after transfection; paragraph 19,7,human,neuronally differentiated human induced pluripotent stem cells,neuronally differentiated hiPSCs,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00004,P0,offtarget,offtarget_evidence,5,yes,A,8,PNA-braced ASO panel from PMID 38042877,ASO,,,hybridization/mismatch off-target effect,1136,38042877,PMC10693639,10.1038/s41467-023-43714-0,Dynamic and static control of the off-target interactions of antisense oligonucleotides using toehold chemistry.,https://pubmed.ncbi.nlm.nih.gov/38042877/,Results > Hybridization-dependent mechanism: complementary PNA strand mitigates off-target hybridizations; paragraph 31,8,human,Huh-7 cells and in-cuvette hybridization assay,Huh-7 cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00005,P0,offtarget,offtarget_evidence,6,yes,A,9,DM1 ASO panel from PMID 37200862,ASO,DMPK,,transcriptome-level off-target effect,1481,37200862,PMC10185704,10.1016/j.omtn.2023.04.010,Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1.,https://pubmed.ncbi.nlm.nih.gov/37200862/,Results > RNA-seq reveals activation of the interferon response by all ASOs and additional off-target effects on gene expression by the repeat gapmer; paragraph 3,9,human,DM1 patient-derived immortalized myoblasts,DM1 myoblasts,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00006,P0,offtarget,offtarget_evidence,7,yes,A,10,ALN-HBV/ALN-HBV02 GalNAc-siRNA panel from PMID 35736224,siRNA,HBV,,seed-mediated off-target effect,1411,35736224,PMC9262600,10.1093/nar/gkac539,From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization.,https://pubmed.ncbi.nlm.nih.gov/35736224/,RESULTS > Application of ESC+ design to improve the specificity and safety of ALN-HBV; paragraph 46,10,human; mouse,HepG2.2.15 cells and HBV-AAV mouse model,HepG2.2.15 cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00007,P0,offtarget,offtarget_evidence,8,yes,A,11,GalNAc-siRNA panel from PMID 29459660,siRNA,,,seed-mediated off-target effect,2058,29459660,PMC5818625,10.1038/s41467-018-02989-4,Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity.,https://pubmed.ncbi.nlm.nih.gov/29459660/,Results > siRNA off-targets are enriched for seed complementarity; paragraph 17,11,rat,rat hepatocytes and rat liver after GalNAc-siRNA exposure,rat hepatocytes; rat liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00008,P0,offtarget,offtarget_evidence,10,yes,A,13,GSK2910546A ASO gapmer panel from PMID 26338776,ASO,BACH1,,hybridization/mismatch off-target effect,2813,26338776,PMC4605310,10.1093/nar/gkv857,In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization.,https://pubmed.ncbi.nlm.nih.gov/26338776/,RESULTS > Intronic regions are markedly more susceptible to ASO-silencing than exonic sequence; paragraph 24,13,human,normal human bronchial epithelial cells,NHBE cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00009,P0,offtarget,offtarget_evidence,59,yes,A,989,"ASO 6 (recovered B2 from PMID:40672232, curator:CM)",ASO,,,transcriptome-level off-target effect,1824,40672232,PMC12265680,10.1101/2025.07.11.664364,Targeted RNA editing by direct delivery of an adenosine deaminase-antisense oligo conjugate.,https://pubmed.ncbi.nlm.nih.gov/40672232/,RESULTS > RNP delivery reduces transcriptome-wide off-target editing; paragraph 22,536,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00010,P0,offtarget,offtarget_evidence,60,yes,A,434,siRNA-based,siRNA,,,seed-mediated off-target effect,9324,40503175,PMC12156227,10.1016/j.omtn.2025.102567,Targeting CIDEB alleviates liver steatosis and fibrosis in mouse MASH models.,https://pubmed.ncbi.nlm.nih.gov/40503175/,Results > Minimal off-target activity of GalNAc-siCIDEB; paragraph 8,537,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00011,P0,offtarget,offtarget_evidence,62,yes,A,435,siRNAs,siRNA,,,seed-mediated off-target effect,2309,34648028,PMC8565336,10.1093/nar/gkab916,Overcoming GNA/RNA base-pairing limitations using isonucleotides improves the pharmacodynamic activity of ESC+ GalNAc-siRNAs.,https://pubmed.ncbi.nlm.nih.gov/34648028/,RESULTS > Impact of GNA isonucleotides on GalNAc-siRNA in vitro activity; paragraph 49,539,,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00012,P0,offtarget,offtarget_evidence,63,yes,A,435,siRNAs,siRNA,,,seed-mediated off-target effect,12558,33664996,PMC7900643,10.1016/j.omtn.2021.01.018,AGO-accessible anticancer siRNAs designed with synergistic miRNA-like activity.,https://pubmed.ncbi.nlm.nih.gov/33664996/,Results > Transcriptome-wide analyses of mi/siRNAs targeting E6/E7; paragraph 17,540,cell line,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00013,P0,offtarget,offtarget_evidence,64,yes,A,990,unspecified siRNA (PMID:31375812),siRNA,,,seed-mediated off-target effect,4213,31375812,PMC6879195manuscript-id: NIHMS1533065,10.1038/s41587-019-0205-0,A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.,https://pubmed.ncbi.nlm.nih.gov/31375812/,Results > Di-siRNA modulates HTT expression with minimal off-target events; paragraph 33,541,rat,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00014,P0,offtarget,offtarget_evidence,65,yes,A,991,unspecified ASO (PMID:29390093),ASO,,,transcriptome-level off-target effect,2060,29390093,PMC5861398,10.1093/nar/gky060,Acute hepatotoxicity of 2' fluoro-modified 5-10-5 gapmer phosphorothioate oligonucleotides in mice correlates with intracellular protein binding and the loss of DBHS proteins.,https://pubmed.ncbi.nlm.nih.gov/29390093/,RESULTS > Hybridization-dependent toxicity is not likely to account for the 2′ F PS-ASO-induced hepatotoxicity; Figure 5. caption,542,,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00015,P0,offtarget,offtarget_evidence,66,yes,A,992,"Sequence 1 (recovered B2 from PMID:28918038, curator:CM)",ASO,,,transcriptome-level off-target effect,2064,28918038,PMC5537172,10.1016/j.omtn.2017.07.003,Strategies for In Vivo Screening and Mitigation of Hepatotoxicity Associated with Antisense Drugs.,https://pubmed.ncbi.nlm.nih.gov/28918038/,Results > High Confirmation Rate of In Silico Predicted OTEs in Mouse Liver; paragraph 14,543,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00016,P0,offtarget,offtarget_evidence,67,yes,A,438,LNA ASO,ASO,,,hybridization/mismatch off-target effect,2062,26553810,PMC4797265,10.1093/nar/gkv1210,Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts.,https://pubmed.ncbi.nlm.nih.gov/26553810/,RESULTS > Hepatotoxicity of LNA ASOs is RNase H1 dependent; paragraph 35,544,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00017,P0,offtarget,offtarget_evidence,78,yes,A,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,transcriptome-level off-target effect,1035,41918821,PMC13034028,10.1093/narmme/ugag016,Integrating transcriptomics and proteomics to assess antisense oligonucleotide safety and efficacy: a time-resolved approach.,https://pubmed.ncbi.nlm.nih.gov/41918821/,Results > ASO off-target RNA reduction correlates well with off-target protein reduction in HepG2 cells; paragraph 48,555,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00018,P0,offtarget,offtarget_evidence,82,yes,A,993,"siRNA-IGF2BP2 (recovered B2 from PMID:41859644, curator:CM)",siRNA,,,transcriptome-level off-target effect,1732,41859644,PMC12996677,10.1016/j.mtbio.2026.102999,An exosome-biomimetic photothermal nanocarrier for IGF2BP2 siRNA delivery and enhanced ferroptosis in head and neck squamous cell carcinoma.,https://pubmed.ncbi.nlm.nih.gov/41859644/,Results > IGF2BP2 Knockdown promotes ferroptosis in HNSCC via reduced NRF2 mRNA stability; paragraph 10,559,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00019,P0,offtarget,offtarget_evidence,91,yes,A,994,"S9 (recovered B2 from PMID:41552385, curator:CM)",siRNA,,,hybridization/mismatch off-target effect,3301,41552385,PMC12804371,10.1016/j.omtn.2025.102802,An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment.,https://pubmed.ncbi.nlm.nih.gov/41552385/,Results > Screening of SNA-modified siRNAs targeting CAG repeats; paragraph 8,568,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00020,P0,offtarget,offtarget_evidence,113,yes,A,995,unspecified siRNA (PMID:40385635),siRNA,,,seed-mediated off-target effect,1477,40385635,PMC12083919,10.1016/j.omtn.2025.102542,"Design, pharmacology, and toxicology of a novel chemically modified siRNA targeting hepatic angiotensinogen.",https://pubmed.ncbi.nlm.nih.gov/40385635/,Results > In silico off-target analysis and qPCR validation; paragraph 10,590,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00021,P0,offtarget,offtarget_evidence,115,yes,A,435,siRNAs,siRNA,,,off-target evidence,1788,41373822,PMC12692593,10.3390/ijms262311675,Respiratory Delivery of Highly Conserved Antiviral siRNAs Suppress SARS-CoV-2 Infection.,https://pubmed.ncbi.nlm.nih.gov/41373822/,2. Results > 2.4. Gene Silencing Effect of Chemically Modified siRNAs; paragraph 14,592,cell line,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00022,P0,offtarget,offtarget_evidence,116,yes,A,996,unspecified siRNA (PMID:41341748),siRNA,,,seed-mediated off-target effect,1789,41341748,PMC12670904,10.1016/j.omtn.2025.102768,A novel bispecific siRNA concept: Efficient dual knockdown of YAP1 and WWTR1 with a single guide strand.,https://pubmed.ncbi.nlm.nih.gov/41341748/,Results > Characterization of off-target activities of bsYW-61; paragraph 14,593,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00023,P0,offtarget,offtarget_evidence,135,yes,A,468,siNTC,siRNA,,,transcriptome-level off-target effect,8415,40928446,PMC12790151,10.1016/j.jacbts.2025.101364,Small Interfering RNA Therapy Targeting the Long Noncoding RNA SMILR for Therapeutic Intervention in Coronary Artery Bypass Graft Failure.,https://pubmed.ncbi.nlm.nih.gov/40928446/,Results > Transcriptomic analyses of BHF7-transfected vSMCs demonstrates expected changes to known SMILR regulated genes without apparent toxic effect; paragraph 43,612,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00024,P0,offtarget,offtarget_evidence,157,yes,A,997,"Anti-N1 (recovered B2 from PMID:38379891, curator:CM)",ASO,,,transcriptome-level off-target effect,1592,38379891,PMC10878212,10.1177/26331055241233596,Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene.,https://pubmed.ncbi.nlm.nih.gov/38379891/,Results; paragraph 9,634,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00025,P0,offtarget,offtarget_evidence,164,yes,A,998,"PH-762 (recovered B2 from PMID:39697325, curator:CM)",siRNA,,,transcriptome-level off-target effect,10539,39697325,PMC11652358,10.3389/fimmu.2024.1501679,Self-delivering RNAi immunotherapeutic PH-762 silences PD-1 to generate local and abscopal antitumor efficacy.,https://pubmed.ncbi.nlm.nih.gov/39697325/,Results > PH-762 is a stabilized self-delivering siRNA compound designed to potently silence PDCD1 mRNA with high specificity; paragraph 10,641,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00026,P0,offtarget,offtarget_evidence,188,yes,A,999,"Glu-CT102MOE5 (recovered B2 from PMID:37251692, curator:CM)",ASO,,,hybridization/mismatch off-target effect,14112,37251692,PMC10220282,10.1016/j.omtn.2023.04.028,Modified ASO conjugates encapsulated with cytidinyl/cationic lipids exhibit more potent and longer-lasting anti-HCC effects.,https://pubmed.ncbi.nlm.nih.gov/37251692/,Results > Transcriptomics and proteomics analysis of modified CT102s; paragraph 23,665,,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00027,P0,offtarget,offtarget_evidence,190,yes,A,1000,"si-S1A3-Chol (recovered B2 from PMID:36618268, curator:CM)",siRNA,,,seed-mediated off-target effect,14301,36618268,PMC9813581,10.1016/j.omtn.2022.12.008,Chemically modified small interfering RNA targeting Hedgehog signaling pathway for rheumatoid arthritis therapy.,https://pubmed.ncbi.nlm.nih.gov/36618268/,Results > Off-target effect analysis; paragraph 18,667,,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00028,P0,offtarget,offtarget_evidence,193,yes,A,1001,unspecified ASO (PMID:36511872),ASO,,,transcriptome-level off-target effect,2253,36511872,PMC9825156,10.1093/nar/gkac1108,G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects.,https://pubmed.ncbi.nlm.nih.gov/36511872/,RESULTS > Indirect FXN activation is unlikely to be mediated by a mutual hybridization-dependent off-target transcript; paragraph 49,670,,,,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00029,P0,offtarget,offtarget_evidence,207,yes,A,1002,"T39 ASO (recovered B2 from PMID:32180554, curator:CM)",ASO,,,off-target evidence,2770,32180554,PMC7036698,10.1016/j.molmet.2020.01.010,Antisense oligonucleotide treatment produces a type I interferon response that protects against diet-induced obesity.,https://pubmed.ncbi.nlm.nih.gov/32180554/,Results > ASOs targeting human mRNAs can also induce a type I IFN response in cultured THP1 cells; paragraph 31,684,human,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00030,P0,offtarget,offtarget_evidence,217,yes,A,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,transcriptome-level off-target effect,12621,30114557,PMC6092551,10.1016/j.omtn.2018.07.010,Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models.,https://pubmed.ncbi.nlm.nih.gov/30114557/,"Results > QR-110 Treatment Reduces Aberrant Splicing, Increases Wild-Type CEP290 in Homozygous Retinal Organoids, and Shows No Off-Target Pharmacology; paragraph 15",694,,,,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00031,P0,offtarget,offtarget_evidence,220,yes,A,1003,unspecified siRNA (PMID:27461380),ASO,,,transcriptome-level off-target effect,2061,27461380,PMC4961955,10.1038/srep30377,Ribonuclease H1-dependent hepatotoxicity caused by locked nucleic acid-modified gapmer antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/27461380/,Results > Off-target knockdown by the Acsl1 gapmer; paragraph 15,697,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00032,P0,offtarget,offtarget_evidence,222,yes,A,1004,"FM10 (recovered B2 from PMID:27378237, curator:CM)",PMO,,,transcriptome-level off-target effect,15206,27378237,PMC5023379,10.1038/mt.2016.111,Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics.,https://pubmed.ncbi.nlm.nih.gov/27378237/,Results; paragraph 9,699,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00033,P0,toxicity,toxicity_endpoint,1,yes,A,14,phosphorothioate ASO panel from PMID 41760661,ASO,,,genotoxicity: DNA damage response,1002,41760661,PMC12954066,10.1038/s41467-026-69980-2,Dysregulation of the DNA damage response by phosphorothioate antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/41760661/,Results > ASO treatment cause activation of DNA repair enzymes and these proteins are enriched in ASO-seeded PS bodies; paragraph 15,14,human; mouse,U2OS cells and mouse brain after ASO exposure,U2OS cells; brain,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00034,P0,toxicity,toxicity_endpoint,2,yes,A,15,tool ASO safety panel from PMID 41918821,ASO,,,hepatic: hepatotoxicity,1035,41918821,PMC13034028,10.1093/narmme/ugag016,Integrating transcriptomics and proteomics to assess antisense oligonucleotide safety and efficacy: a time-resolved approach.,https://pubmed.ncbi.nlm.nih.gov/41918821/,Results > Tool ASO safety profile characterization in vitro and in vivo; paragraph 32,15,mouse; human,mouse high-dose ASO exposure and HepG2 transfection time course,HepG2 cells; plasma,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00035,P0,toxicity,toxicity_endpoint,4,yes,A,17,5?-CP-modified PS-ASO panel from PMID 41467114,ASO,,,neurological: neurotoxicity,1028,41467114,PMC12744863,10.1016/j.omtn.2025.102692,Unraveling and controlling late-onset neurotoxicity of antisense oligonucleotides through strategic chemical modifications.,https://pubmed.ncbi.nlm.nih.gov/41467114/,Results > Gap modification of 5′-CP mitigates cytotoxicity and in vivo neurotoxicity across various PS-ASOs; paragraph 25,17,mouse; rat,"Neuro-2a cells, mouse ICV dosing, and rat intrathecal dosing",Neuro-2a cells; CNS tissue,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00036,P0,toxicity,toxicity_endpoint,9,yes,A,22,LEM-S401,siRNA,CTGF,,general safety: safety/tolerability,2285,40160160,PMC11955805,10.1111/cts.70207,"A First-In-Human Phase 1 Study to Evaluate the Safety and Tolerability of LEM-S401, a Novel siRNA-DegradaBALL Drug Targeting CTGF in Healthy Adults.",https://pubmed.ncbi.nlm.nih.gov/40160160/,Results > Safety and Tolerability; paragraph 24,22,human,healthy adult volunteers receiving single-dose LEM-S401,healthy volunteers,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00037,P0,toxicity,toxicity_endpoint,10,yes,A,24,hypoxanthine-substituted LNA-gapmer ASO1 panel from PMID 41928909,ASO,Mapt,,neurological: neurotoxicity,1413,41928909,PMC13040562,10.1039/d5cb00316d,Guanine base modifications in antisense oligonucleotides mitigate acute central nervous system toxicity.,https://pubmed.ncbi.nlm.nih.gov/41928909/,Result > Nucleobase-dependent effects of hypoxanthine substitution on in vivo CNS toxicity; Fig. 3 caption,24,mouse,,CNS,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00038,P0,toxicity,toxicity_endpoint,12,yes,A,26,RGLS4326 anti-miR-17 ASO,ASO,MIR17,,neurological: neurotoxicity,1410,41315228,PMC12663328,10.1038/s41467-025-65799-5,The nucleobase guanine at the 3'-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity.,https://pubmed.ncbi.nlm.nih.gov/41315228/,Results > CNS toxicity after RGLS4326 treatment in nonclinical toxicity studies; paragraph 7,26,mouse; cynomolgus monkey,,CNS,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00039,P0,toxicity,toxicity_endpoint,13,yes,A,27,SNS812,siRNA,SARS-CoV-2 RdRP,,general safety: safety/tolerability,2286,40116355,PMC11926758,10.1111/cts.70202,"Pharmacokinetics and Safety Profile of SNS812, a First in Human Fully Modified siRNA Targeting Wide-Spectrum SARS-CoV-2, in Healthy Subjects.",https://pubmed.ncbi.nlm.nih.gov/40116355/,Results > Primary Efficacy: Safety; paragraph 27,27,human,,healthy subjects,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00040,P0,toxicity,toxicity_endpoint,24,yes,A,38,G-clamp/BNAP-AEO modified Malat1 ASO panel from PMID 38978695,ASO,MALAT1,,neurological: neurotoxicity,1110,38978695,PMC11229412,10.1016/j.omtn.2024.102161,"Favorable efficacy and reduced acute neurotoxicity by antisense oligonucleotides with 2',4'-BNA/LNA with 9-(aminoethoxy)phenoxazine.",https://pubmed.ncbi.nlm.nih.gov/38978695/,Results > G-clamp modification in ASO targeting Malat1 RNA mitigates acute CNS toxicity of ASO; paragraph 11,38,mouse,,CNS,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00041,P0,toxicity,toxicity_endpoint,25,yes,A,39,aCSF+-formulated CNS ASO/di-siRNA panel from PMID 39554992,ASO/siRNA mixed context,,,neurological: neurotoxicity,1115,39554992,PMC11567125,10.1016/j.omtn.2024.102359,Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca(2+) and Mg(2+) in the formulation.,https://pubmed.ncbi.nlm.nih.gov/39554992/,Results > Using aCSF+ reduces the acute neurotoxicity of CNS-administered ASOs; paragraph 26,39,mouse,,CNS,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00042,P0,toxicity,toxicity_endpoint,28,yes,A,42,CT102,ASO,IGF1R,,general safety: safety/tolerability,2600,38573183,PMC11534120,10.2217/fon-2023-0872,Preclinical and phase I studies of an antisense oligonucleotide drug targeting IGF-1R in liver cancer.,https://pubmed.ncbi.nlm.nih.gov/38573183/,Results > Participants in phase I clinical trial; paragraph 55,42,human,,primary liver cancer participants,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00043,P0,toxicity,toxicity_endpoint,38,yes,A,52,ASO1-ASO3 modified ASO panel from PMID 36700050,ASO,,,neurological: neurotoxicity,12067,36700050,PMC9843489,10.1016/j.omtn.2022.12.010,Change of intracellular calcium level causes acute neurotoxicity by antisense oligonucleotides via CSF route.,https://pubmed.ncbi.nlm.nih.gov/36700050/,Results > Effect of ASO oligonucleotide sequence and modifications on neurotoxicity; paragraph 16,52,mouse; rat primary neurons,,CNS; primary cortical neurons,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00044,P0,toxicity,toxicity_endpoint,39,yes,A,53,TS1-ASO nucleobase modification panel from PMID 35801870,ASO,TTR,,hepatic: hepatotoxicity,1409,35801870,PMC9303313,10.1093/nar/gkac562,Identification of nucleobase chemical modifications that reduce the hepatotoxicity of gapmer antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/35801870/,RESULTS > Identification of potential nucleobase modifications that reduce LNA gapmer hepatotoxicity; Figure 2. caption,53,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00045,P0,toxicity,toxicity_endpoint,40,yes,A,54,ESC+ GalNAc-siRNA D1/D4/D6/D9 panel from PMID 35736224,siRNA,TTR; HAO1,,hepatic: hepatotoxicity,1411,35736224,PMC9262600,10.1093/nar/gkac539,From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization.,https://pubmed.ncbi.nlm.nih.gov/35736224/,RESULTS > GNA improves the therapeutic index in rats; paragraph 33,54,rat,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00046,P0,toxicity,toxicity_endpoint,42,yes,A,56,siLuc-ALC-0315 LNP from PMID 35642083,siRNA,LUC,,hepatic: hepatotoxicity,1979,35642083,PMC9621687manuscript-id: NIHMS1814479,10.1021/acs.molpharmaceut.2c00033,Comparison of DLin-MC3-DMA and ALC-0315 for siRNA Delivery to Hepatocytes and Hepatic Stellate Cells.,https://pubmed.ncbi.nlm.nih.gov/35642083/,Results > A high intravenous dose of ALC-0315 LNPs increases some markers of liver toxicity.; paragraph 21,56,mouse,,liver; serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00047,P0,toxicity,toxicity_endpoint,48,yes,A,62,HsPCSK9-1811-LNA/GalNAc ASO panel from PMID 34760338,ASO,PCSK9,,renal; hepatic: renal and hepatic safety,1632,34760338,PMC8560717,10.1016/j.omtn.2021.10.008,Drug discovery and development scheme for liver-targeting bridged nucleic acid antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/34760338/,Results > Evaluating the safety of the GalNAc conjugate in rat; paragraph 15,62,rat; cynomolgus monkey,,liver; kidney,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00048,P0,toxicity,toxicity_endpoint,58,yes,A,72,H51(+67)W tcDNA-ASO from PMID 31881528,ASO,DMD,,hepatic; renal: hepatic and renal safety,2225,31881528,PMC7063478,10.1016/j.omtn.2019.11.020,Identifying and Avoiding tcDNA-ASO Sequence-Specific Toxicity for the Development of DMD Exon 51 Skipping Therapy.,https://pubmed.ncbi.nlm.nih.gov/31881528/,Results > Safety Biomarkers at the End of H51(+67)W Treatment; paragraph 14,72,mouse,,liver; kidney,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00049,P0,toxicity,toxicity_endpoint,59,yes,A,73,GalNAc AON-B/D/E panel from PMID 30583097,ASO,,,renal: renal safety,2059,30583097,PMC6305803,10.1016/j.omtn.2018.11.005,GalNAc Conjugation Attenuates the Cytotoxicity of Antisense Oligonucleotide Drugs in Renal Tubular Cells.,https://pubmed.ncbi.nlm.nih.gov/30583097/,Results > GalNAc Conjugation Masked the In Vivo Nephrotoxicity Potential of AON Drug Candidates; paragraph 7,73,rat; human cell model,,kidney; primary human proximal tubule epithelial cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00050,P0,toxicity,toxicity_endpoint,60,yes,A,74,GalNAc-siRNA hepatotoxicity panel with REVERSIR from PMID 29459660,siRNA,HAO1; TTR,,hepatic: hepatotoxicity,2058,29459660,PMC5818625,10.1038/s41467-018-02989-4,Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity.,https://pubmed.ncbi.nlm.nih.gov/29459660/,Results > Blocking siRNA-loaded RISC activity mitigates hepatotoxicity; paragraph 12,74,rat,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00051,P0,toxicity,toxicity_endpoint,61,yes,A,75,236-LNA-ASO hepatotoxicity screening panel from PMID 29499955,ASO,,,hepatic: hepatotoxicity,2319,29499955,PMC5725219,10.1016/j.omtn.2017.11.004,A Sensitive In Vitro Approach to Assess the Hybridization-Dependent Toxic Potential of High Affinity Gapmer Oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/29499955/,Results > Apoptosis Induction by Hepatotoxic LNA-ASOs In Vitro Is RNase H1 Dependent; paragraph 8,75,mouse cell line; mouse,,liver; cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00052,P0,toxicity,toxicity_endpoint,62,yes,A,76,nephrotoxic AON A/B/C panel from PMID 28325303,ASO,,,renal: renal safety,2106,28325303,PMC5363415,10.1016/j.omtn.2016.11.006,Inhibition of EGF Uptake by Nephrotoxic Antisense Drugs In Vitro and Implications for Preclinical Safety Profiling.,https://pubmed.ncbi.nlm.nih.gov/28325303/,Results > Identification of Extracellular EGF as an In Vitro Marker of AON-Induced Tubulotoxicity; paragraph 9,76,human cell model,,primary human proximal tubule epithelial cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00053,P0,toxicity,toxicity_endpoint,63,yes,A,77,Acsl1/GR LNA gapmer panel from PMID 27461380,ASO,ACSL1; NR3C1,,hepatic: hepatotoxicity,2061,27461380,PMC4961955,10.1038/srep30377,Ribonuclease H1-dependent hepatotoxicity caused by locked nucleic acid-modified gapmer antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/27461380/,Results > In vivo hepatotoxicity of gapmers and ‘non-gapmers’; paragraph 8,77,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00054,P0,toxicity,toxicity_endpoint,64,yes,A,78,toxic LNA/MOE gapmer ASO panel from PMID 26553810,ASO,long pre-mRNA transcripts,,hepatic: hepatotoxicity,2062,26553810,PMC4797265,10.1093/nar/gkv1210,Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts.,https://pubmed.ncbi.nlm.nih.gov/26553810/,RESULTS > Targeted down-regulation of several long pre-mRNA transcripts by mixtures of MOE ASOs mimics LNA ASOs hepatotoxicity; paragraph 39,78,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00055,P0,toxicity,toxicity_endpoint,66,yes,A,80,3-8-3 LNA gapmer hepatotoxicity panel from PMID 24550163,ASO,,,hepatic: hepatotoxicity,2063,24550163,PMC4005641,10.1093/nar/gku142,Sequence motifs associated with hepatotoxicity of locked nucleic acid--modified antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/24550163/,RESULTS > Evaluation of hepatotoxicity in mice; paragraph 14,80,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00056,P0,toxicity,toxicity_endpoint,68,yes,A,83,siRNA candidate from PMID 41076269,siRNA,,,immunotoxicity: immune activation,1476,41076269,PMC12766900manuscript-id: NIHMS2123465,10.1016/j.jtha.2025.09.012,Preclinical safety and bleeding evaluation in swine for a small interfering RNA-lipid nanoparticle that prevents excess fibrinogen synthesis.,https://pubmed.ncbi.nlm.nih.gov/41076269/,RESULTS > siFibrinogen did not cause adverse effects; paragraph 23,83,swine,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00057,P0,toxicity,toxicity_endpoint,70,yes,A,85,siRNA candidate from PMID 40692538,siRNA,,,hematologic: platelet/bleeding safety,1242,40692538,PMC12277184,10.2147/IJN.S510937,Protective Effects of GalNac-Modified Red Blood Cell-Derived Extracellular Vesicles Against Liver Diseases.,https://pubmed.ncbi.nlm.nih.gov/40692538/,Results > Drugs-Loaded GalNac-RBC-EVs Were Non-Toxic to Mice; paragraph 42,85,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00058,P0,toxicity,toxicity_endpoint,71,yes,A,86,Plozasiran,ASO/siRNA mixed context,,,hematologic: platelet/bleeding safety,1532,41094680,PMC12523034,10.1186/s12933-025-02929-9,"Safety, pharmacokinetics and pharmacodynamics of Plozasiran in Chinese healthy volunteers.",https://pubmed.ncbi.nlm.nih.gov/41094680/,Results > Safety; paragraph 28,86,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00059,P0,toxicity,toxicity_endpoint,78,yes,A,93,Inclisiran,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,2609,38371290,PMC10866732,10.1016/j.jacasi.2023.09.006,Efficacy and Safety of Inclisiran in Asian Patients: Results From ORION-18.,https://pubmed.ncbi.nlm.nih.gov/38371290/,Results; paragraph 18,93,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00060,P0,toxicity,toxicity_endpoint,79,yes,A,94,ASO candidate from PMID 37365583,ASO,,,hematologic: platelet/bleeding safety,1167,37365583,PMC10294514,10.1186/s13045-023-01468-8,"Safety and activity of the first-in-class locked nucleic acid (LNA) miR-221 selective inhibitor in refractory advanced cancer patients: a first-in-human, phase 1, open-label, dose-escalation study.",https://pubmed.ncbi.nlm.nih.gov/37365583/,Results > Safety of LNA-i-miR-221; paragraph 28,94,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00061,P0,toxicity,toxicity_endpoint,84,yes,A,99,Inotersen,ASO/siRNA mixed context,,,hematologic: platelet/bleeding safety,1658,32343462,PMC7496583,10.1111/ene.14285,Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial.,https://pubmed.ncbi.nlm.nih.gov/32343462/,Results > Safety and tolerability > Thrombocytopenia and glomerulonephritis; paragraph 30,99,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00062,P0,toxicity,toxicity_endpoint,96,yes,A,114,Angptl4-targeting ASO from PMID 39259836,ASO,,,immunotoxicity: immune activation,1565,39259836,PMC11687395,10.1093/cvr/cvae195,Liver-targeted Angptl4 silencing by antisense oligonucleotide treatment attenuates hyperlipidaemia and atherosclerosis development in APOE*3-Leiden.CETP mice.,https://pubmed.ncbi.nlm.nih.gov/39259836/,Results > Short-term liver-targeted Angptl3 and Angptl4 silencing does not induce hepatic inflammation; paragraph 27,114,non-human primate,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00063,P0,toxicity,toxicity_endpoint,100,yes,A,118,ASO candidate from PMID 36631935,ASO,,,general safety: safety/tolerability,1148,36631935,PMC10087069,10.1111/cts.13476,Correlations between preclinical BJAB assay ranking of antisense drugs and clinical trial adverse events.,https://pubmed.ncbi.nlm.nih.gov/36631935/,RESULTS > Correlations identified using adverse event network analysis; FIGURE 1 caption,118,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00064,P0,toxicity,toxicity_endpoint,110,yes,A,130,Nedosiran,ASO/siRNA mixed context,,,renal: renal safety,10207,39875734,PMC12031765,10.1007/s00467-025-06675-8,"Nedosiran in pediatric patients with PH1 and relatively preserved kidney function, a phase 2 study (PHYOX8).",https://pubmed.ncbi.nlm.nih.gov/39875734/,Results > Safety; paragraph 24,131,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00065,P0,toxicity,toxicity_endpoint,111,yes,A,130,Nedosiran,ASO/siRNA mixed context,,,renal: renal safety,2597,38707801,PMC11068990,10.1016/j.ekir.2024.02.1439,Nedosiran Safety and Efficacy in PH1: Interim Analysis of PHYOX3.,https://pubmed.ncbi.nlm.nih.gov/38707801/,Results > Participants; paragraph 20,132,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00066,P0,toxicity,toxicity_endpoint,113,yes,A,132,Viltolarsen,ASO,,,renal: renal safety,1602,37005891,PMC10200237,10.3233/JND-221656,"Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.",https://pubmed.ncbi.nlm.nih.gov/37005891/,RESULTS > Safety; paragraph 21,134,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00067,P0,toxicity,toxicity_endpoint,114,yes,A,132,Viltolarsen,ASO,,,general safety: safety/tolerability,1621,35634851,PMC9398057,10.3233/JND-220811,Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.,https://pubmed.ncbi.nlm.nih.gov/35634851/,RESULTS > Safety; paragraph 21,135,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00068,P0,toxicity,toxicity_endpoint,116,yes,A,93,Inclisiran,ASO/siRNA mixed context,,,general safety: safety/tolerability,2299,37850379,PMC10815002,10.1161/CIRCULATIONAHA.122.063460,"Efficacy, Safety, and Tolerability of Inclisiran in Patients With Homozygous Familial Hypercholesterolemia: Results From the ORION-5 Randomized Clinical Trial.",https://pubmed.ncbi.nlm.nih.gov/37850379/,RESULTS > Safety Evaluation; paragraph 46,138,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00069,P0,toxicity,toxicity_endpoint,147,yes,A,152,Nusinersen,ASO/siRNA mixed context,,,hematologic: platelet/bleeding safety,12141,40011938,PMC11866593,10.1186/s13023-025-03603-9,Real-world analysis of the efficacy and safety of nusinersen in pediatric patients with spinal muscular atrophy.,https://pubmed.ncbi.nlm.nih.gov/40011938/,Results > Safety of nusinersen sodium injection for the treatment of SMA; paragraph 41,178,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00070,P0,toxicity,toxicity_endpoint,150,yes,A,174,Olezarsen,ASO/siRNA mixed context,,,hematologic: platelet/bleeding safety,1286,39363329,PMC11448427,10.1186/s12944-024-02297-5,Efficacy and safety of olezarsen in lowering apolipoprotein C-III and triglycerides in healthy Japanese Americans.,https://pubmed.ncbi.nlm.nih.gov/39363329/,Results > Primary endpoint: safety results; paragraph 19,181,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00071,P0,toxicity,toxicity_endpoint,151,yes,A,175,ASO/siRNA mixed context candidate from PMID 38271438,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,1290,38271438,PMC10810432,10.1371/journal.pone.0294847,A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice.,https://pubmed.ncbi.nlm.nih.gov/38271438/,Results > Clinical trial results > Safety; paragraph 32,182,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00072,P0,toxicity,toxicity_endpoint,154,yes,A,93,Inclisiran,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,2621,38220186,PMC11150722,10.5551/jat.64454,"Efficacy, Safety, and Pharmacokinetics of Inclisiran in Japanese Patients: Results from ORION-15.",https://pubmed.ncbi.nlm.nih.gov/38220186/,Results; paragraph 46,185,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00073,P0,toxicity,toxicity_endpoint,163,yes,A,185,unspecified siRNA,siRNA,,,general safety: cell viability/cytotoxicity,12521,37110552,PMC10144622,10.3390/molecules28083318,A Straightforward Method for the Development of Positively Charged Gold Nanoparticle-Based Vectors for Effective siRNA Delivery.,https://pubmed.ncbi.nlm.nih.gov/37110552/,2. Results > 2.5. Cell Viability; paragraph 14,194,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00074,P0,toxicity,toxicity_endpoint,167,yes,A,188,inotersen,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,1616,35908242,PMC9618524,10.1007/s00415-022-11276-8,Long-term efficacy and safety of inotersen for hereditary transthyretin amyloidosis: NEURO-TTR open-label extension 3-year update.,https://pubmed.ncbi.nlm.nih.gov/35908242/,Results > Safety and tolerability; paragraph 28,198,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00075,P0,toxicity,toxicity_endpoint,174,yes,A,195,HepG2; LO2,siRNA,,,hepatic: hepatotoxicity,1641,34042551,PMC8168781,10.1080/10717544.2021.1928794,cRGD peptide-conjugated polyethylenimine-based lipid nanoparticle for intracellular delivery of siRNA in hepatocarcinoma therapy.,https://pubmed.ncbi.nlm.nih.gov/34042551/,Results > Cytotoxicity tests; paragraph 28,205,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00076,P0,toxicity,toxicity_endpoint,181,yes,A,201,radavirsen,ASO,,,hepatic: hepatotoxicity,2230,28929521,PMC5736848,10.1111/bcp.13405,"Safety, tolerability, and pharmacokinetics of radavirsen (AVI-7100), an antisense oligonucleotide targeting influenza a M1/M2 translation.",https://pubmed.ncbi.nlm.nih.gov/28929521/,Abstract > Results; paragraph 3,212,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00077,P0,toxicity,toxicity_endpoint,187,yes,A,205,drisapersen,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,2328,24321374,PMC4145871manuscript-id: NIHMS592631,10.1016/j.nmd.2013.09.004,Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial.,https://pubmed.ncbi.nlm.nih.gov/24321374/,3. Results > 3.2. Safety results; paragraph 15,218,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00078,P0,toxicity,toxicity_endpoint,192,yes,A,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,renal: renal safety,1629,34788571,PMC8817703,10.1089/nat.2021.0043,"Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.",https://pubmed.ncbi.nlm.nih.gov/34788571/,Results > Safety; paragraph 23,223,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00079,P0,toxicity,toxicity_endpoint,194,yes,A,220,MC3-based,siRNA,,,hepatic: hepatotoxicity,1196,41907143,PMC13021479,10.3389/fmolb.2026.1785592,Efficiency and safety of five different agents for in vivo delivery of novel bioengineered RNAi molecules.,https://pubmed.ncbi.nlm.nih.gov/41907143/,Results > Tolerability and safety profiles; paragraph 24,237,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00080,P0,toxicity,toxicity_endpoint,210,yes,A,232,fitusiran,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,2522,40053895,PMC12824673,10.1182/blood.2024027008,Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study.,https://pubmed.ncbi.nlm.nih.gov/40053895/,Results > Integrated safety analysis: AEs; paragraph 26,253,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00081,P0,toxicity,toxicity_endpoint,229,yes,A,244,inclisiran,ASO/siRNA mixed context,,,general safety: safety/tolerability,2676,37252442,PMC10209488,10.1016/j.ajpc.2023.100503,"Efficacy and safety of inclisiran in patients with cerebrovascular disease: ORION-9, ORION-10, and ORION-11.",https://pubmed.ncbi.nlm.nih.gov/37252442/,Results > Safety outcomes; paragraph 13,272,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00082,P0,toxicity,toxicity_endpoint,235,yes,A,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,2755,33540294,PMC8264460manuscript-id: NIHMS1711667,10.1016/j.thromres.2021.01.006,Antisense oligonucleotides and nucleic acids generate hypersensitive platelets.,https://pubmed.ncbi.nlm.nih.gov/33540294/,Results > PS- and ME/PS- modified oligonucleotides decrease platelet concentration in vitro; paragraph 29,278,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00083,P0,toxicity,toxicity_endpoint,239,yes,A,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,2116,29565739,PMC5899290,10.1089/nat.2017.0705,Intracerebroventricular Administration of a 2'-O-Methyl Phosphorothioate Antisense Oligonucleotide Results in Activation of the Innate Immune System in Mouse Brain.,https://pubmed.ncbi.nlm.nih.gov/29565739/,Materials and Methods > AON administration; FIG. 1. caption,282,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00084,P0,toxicity,toxicity_endpoint,244,yes,A,252,nusinersen,ASO/siRNA mixed context,,,general safety: safety/tolerability,2611,38361750,PMC10864329,10.1016/j.lanepe.2024.100862,Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study.,https://pubmed.ncbi.nlm.nih.gov/38361750/,Results; paragraph 15,287,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00085,P0,toxicity,toxicity_endpoint,414,yes,A,371,Inclisiran,siRNA,PCSK9,,general safety: clinical laboratory safety,2498,40391436,PMC12180692,10.1161/CIRCULATIONAHA.124.073233,"Efficacy and Safety of Inclisiran in Adolescents With Genetically Confirmed Homozygous Familial Hypercholesterolemia: Results From the Double-Blind, Placebo-Controlled Part of the ORION-13 Randomized Trial.",https://pubmed.ncbi.nlm.nih.gov/40391436/,RESULTS > Safety Analysis; paragraph 36,473,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00086,P0,toxicity,toxicity_endpoint,416,yes,A,373,Nusinersen,ASO,SMN2,,general safety: adverse event reporting,1571,39060346,PMC11282055,10.1038/s41598-024-67627-0,Unveiling the adverse events of Nusinersen in spinal muscular atrophy management based on FAERS database.,https://pubmed.ncbi.nlm.nih.gov/39060346/,Results > Risk signal detection; paragraph 11,475,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00087,P0,toxicity,toxicity_endpoint,417,yes,A,371,Inclisiran,siRNA,PCSK9,,general safety: treatment-emergent adverse events,2632,36550348,PMC11101568,10.1007/s10557-022-07413-0,"Efficacy and Safety of Inclisiran in Patients with Polyvascular Disease: Pooled, Post Hoc Analysis of the ORION-9, ORION-10, and ORION-11 Phase 3 Randomized Controlled Trials.",https://pubmed.ncbi.nlm.nih.gov/36550348/,Results > Safety; paragraph 23,476,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00088,P0,toxicity,toxicity_endpoint,419,yes,A,375,phosphorothioate ASO panel,ASO,,,neurological: acute neuronal inhibition,1044,41494985,PMC12865454,10.1093/nar/gkaf1333,Acute neuronal inhibition response caused by phosphorothioate antisense oligonucleotides following local delivery to the central nervous system.,https://pubmed.ncbi.nlm.nih.gov/41494985/,"Results > Acute inhibition is transient, reversible, and dose-dependent following CNS ASO administration; paragraph 26",478,rat/mouse/non-human primate,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00089,P0,toxicity,toxicity_endpoint,421,yes,A,377,TET3 siRNA lipid nanoparticles,siRNA,TET3,,general safety: hemolysis and cytotoxicity,1192,41923247,PMC13169958,10.1186/s12951-026-04312-6,Dual-targeted lipid nanoparticles for TET3 siRNA delivery: nanobiotechnology strategy to remodel tumor immune microenvironment in hepatocellular carcinoma.,https://pubmed.ncbi.nlm.nih.gov/41923247/,Results > Preparation and characterization of TET3 siRNA@αmp-lipo; paragraph 95,480,cell line,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00090,P0,toxicity,toxicity_endpoint,424,yes,A,380,FW-LP@siIRF5,siRNA,IRF5,,general safety: hematology/clinical chemistry/histology safety,1741,41776767,PMC13001080,10.1021/acsnano.5c18044,Reprogramming Lesional Macrophage Homeostasis via Interferon Regulatory Factor 5 Targeted siRNA Nanoimmunotherapy for Atherosclerosis.,https://pubmed.ncbi.nlm.nih.gov/41776767/,Results > In Vivo Biosafety of FW-LP@siIRF5; paragraph 46,483,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00091,P0,toxicity,toxicity_endpoint,425,yes,A,381,DCA-JAK13033 siRNA,siRNA,JAK1,,general safety: systemic exposure and immunogenicity safety,1750,41634015,PMC12976281,10.1038/s41467-026-68993-1,Intradermal delivery of lipophilic siRNAs enables prolonged skin retention and sustained gene silencing in a porcine model.,https://pubmed.ncbi.nlm.nih.gov/41634015/,"Results > Intradermal administration of conjugated JAK13033 siRNAs in pigs demonstrates durable skin retention for at least 2 months, minimal systemic exposure, and a favorable safety profile; paragraph 12",484,pig,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00092,P0,toxicity,toxicity_endpoint,426,yes,A,382,siRNA nanocarriers/lipoplexes,siRNA,,,general safety: cell viability/cytotoxicity,2428,41274361,PMC12700850,10.1016/j.ejps.2025.107386,Pulmonary delivery of siRNA lipoplexes and lipid nanoparticles using a vibrating mesh nebuliser.,https://pubmed.ncbi.nlm.nih.gov/41274361/,Results > Cytotoxicity of siRNA nanocarriers; paragraph 24,485,cell line,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00093,P0,toxicity,toxicity_endpoint,427,yes,A,383,DP-EVs/siPD-L1,siRNA,CD274,,general safety: systemic toxicity/histology/body weight,3228,41815210,PMC12972210,10.1002/mco2.70673,An Engineered Extracellular Vesicle With Enhanced Tumor and Lymph Nodes Targeting as siRNA Delivery System for Robust Tumor Immunotherapy.,https://pubmed.ncbi.nlm.nih.gov/41815210/,Results > In Vivo Antitumor Efficacy Evaluation of DP‐EVs/siPD‐L1; paragraph 28,486,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00094,P0,toxicity,toxicity_endpoint,432,yes,A,388,siRNA-loaded CaP-NP/cGM formulation,siRNA,Chordin,,general safety: cell viability/biocompatibility,3276,41647289,PMC12869795,10.1093/rb/rbaf115,A smart nanocomposite bioactive ink for controlled siRNA delivery in calvarial mesenchymal stromal cells as a minimally invasive treatment for craniosynostosis.,https://pubmed.ncbi.nlm.nih.gov/41647289/,Results > PLGA NPs are suitable biocompatible nanocarriers for siRNAs delivery in CMSCs; paragraph 53,491,3D in vitro human cell model,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00095,P0,toxicity,toxicity_endpoint,436,yes,A,392,siRNA@VG-Exos,siRNA,IL6,,hepatic/renal/general safety: histology and serum biochemistry safety,3325,41491215,PMC12781530,10.1186/s12951-025-03954-2,Endothelial-targeted modification of ginseng-derived exosomes for IL-6 SiRNA delivery ameliorates hepatic ischemia-reperfusion injury.,https://pubmed.ncbi.nlm.nih.gov/41491215/,Result > In vivo safety assessment of siRNA@VG-Exos; paragraph 31,495,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00096,P0,toxicity,toxicity_endpoint,439,yes,A,395,pMSNC@ISG15 siRNA,siRNA,ISG15,,general safety: in vivo biocompatibility/clinical chemistry,6684,41560785,PMC12813121,10.1016/j.mtbio.2025.102631,Interferon-stimulated gene 15 small interfering RNA-loaded polarized mesoporous silica nanocarriers remodel the immune microenvironment to ameliorate post-myocardial infarction heart failure.,https://pubmed.ncbi.nlm.nih.gov/41560785/,Results > pMSNC@ISG15 siRNA exhibits favorable in vivo biocompatibility and cardiac tissue targeting; paragraph 78,498,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00097,P0,toxicity,toxicity_endpoint,440,yes,A,396,SANG-siRNA,siRNA,,,general safety: systemic toxicity/repeat-dose tolerability,6833,41500988,PMC12780282,10.1038/s41467-025-66788-4,Tumor agnostic drug delivery with dynamic nanohydrogels.,https://pubmed.ncbi.nlm.nih.gov/41500988/,Results > Systemic delivery of SANGs is minimally toxic; paragraph 20,499,mouse/rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00098,P0,toxicity,toxicity_endpoint,441,yes,A,397,cationic/anionic siRNA lipoplexes,siRNA,,,general safety: cell viability/cytotoxicity,12337,41282509,PMC12635908,10.3892/br.2025.2085,Assessment of anionic siRNA lipoplexes prepared via modified ethanol injection for tumor cell delivery.,https://pubmed.ncbi.nlm.nih.gov/41282509/,Results > Cytotoxicity of cationic and anionic siRNA lipoplexes; paragraph 27,500,cell line,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00099,P0,toxicity,toxicity_endpoint,449,yes,A,405,BE-ST@siBACE1 / BE-ST@siRNA,siRNA,BACE1,,immunotoxicity: immune activation,9961,40064874,PMC11893799,10.1038/s41467-025-57488-0,Berberine-inspired ionizable lipid for self-structure stabilization and brain targeting delivery of nucleic acid therapeutics.,https://pubmed.ncbi.nlm.nih.gov/40064874/,Results > Brain-targeted delivery of siBACE1 by BE-ST for AD treatment; paragraph 18,508,mouse,,blood; major organs; brain disease model,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00100,P0,toxicity,toxicity_endpoint,450,yes,A,406,PEI/PHD2 siRNA nanocomplexes,siRNA,PHD2,,general safety: cell viability/cytotoxicity,12938,41266661,PMC12634668,10.1038/s41598-025-24808-9,Optimized delivery of RNA silencing prolyl hydroxylase domain 2 for enhanced angiogenesis and osteogenesis using bioactive glass scaffolds.,https://pubmed.ncbi.nlm.nih.gov/41266661/,Results > Biological effects of PEI/PHD2 SiRNA complexes on HUVECs; paragraph 36,509,human cell line,,HUVEC,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00101,P0,toxicity,toxicity_endpoint,451,yes,A,407,PLGA-CpG@ID8-M nanovaccine,CpG oligodeoxynucleotide,,,general safety: cell viability/cytotoxicity,13359,39985265,PMC12005807,10.1002/advs.202412881,CpG-Based Nanovaccines Enhance Ovarian Cancer Immune Response by Gbp2-Mediated Remodeling of Tumor-Associated Macrophages.,https://pubmed.ncbi.nlm.nih.gov/39985265/,"Results and Discussion > Synthesis, Characterization, and Safety Assessment of PLGA-CpG@ID8-M Nanovaccine; paragraph 9",510,mouse; cell line,,RAW264.7; BMDM; major organs,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00102,P0,toxicity,toxicity_endpoint,452,yes,A,408,siRNA SORT LNPs,siRNA,,,general safety: systemic safety/tolerability,2577,38973655,PMC11823468manuscript-id: NIHMS2006301,10.1002/adma.202313791,"Expanding RNAi to Kidneys, Lungs, and Spleen via Selective ORgan Targeting (SORT) siRNA Lipid Nanoparticles.",https://pubmed.ncbi.nlm.nih.gov/38973655/,Results > SORT LNPs enable robust silencing of tissue-enriched endogenous gene targets and are well tolerated in vivo.; paragraph 12,511,mouse,,liver; kidney; lung; spleen,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00103,P0,toxicity,toxicity_endpoint,453,yes,A,409,Ube3a-AS ASO,ASO,Ube3a-AS,,hepatic: hepatotoxicity,2614,38327047,PMC11163203,10.1016/j.ymthe.2024.02.004,Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.,https://pubmed.ncbi.nlm.nih.gov/38327047/,Results > Analysis of potential toxicities of ASO exposure in dams; paragraph 15,512,mouse,,maternal liver; fetal/pup liver; maternal serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00104,P0,toxicity,toxicity_endpoint,454,yes,A,410,CD44 siRNA nanoplexes,siRNA,CD44,,general safety: cell viability/cytotoxicity,10973,38998694,PMC11243722,10.3390/nano14131089,Development of Focused Ultrasound-Assisted Nanoplexes for RNA Delivery.,https://pubmed.ncbi.nlm.nih.gov/38998694/,3. Results > 3.4. siRNA Delivery Cytotoxicity of Complexes Developed in This Study; paragraph 24,513,human cell line,,MV3; ARPE-19,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00105,P0,toxicity,toxicity_endpoint,455,yes,A,411,Phe-NPs/ASO,ASO,,,general safety: cell viability/cytotoxicity,12161,39411247,PMC11474373,10.1016/j.omtn.2024.102340,l-Type amino acid transporter 1-targeting nanoparticles for antisense oligonucleotide delivery to the CNS.,https://pubmed.ncbi.nlm.nih.gov/39411247/,"Results > In vitro characterization of Phe-NPs/ASO: Cellular uptake, endosomal escape, cell viability, and cytotoxicity; paragraph 11",514,mouse cell line,,GL261-Luc,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00106,P0,toxicity,toxicity_endpoint,456,yes,A,412,DOTAP/siSmad4@EM,siRNA,Smad4,,immunotoxicity: immune activation,12475,39258001,PMC11385781,10.1016/j.ajps.2024.100929,Pulmonary fibroblast-specific delivery of siRNA exploiting exosomes-based nanoscaffolds for IPF treatment.,https://pubmed.ncbi.nlm.nih.gov/39258001/,Results and discussion > Safety profiles; paragraph 46,515,mouse,,lung; BALF,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00107,P0,toxicity,toxicity_endpoint,457,yes,A,413,TPNs-siRNA,siRNA,MMP-2/MMP-9,,general safety: systemic safety/tolerability,12485,39118143,PMC11308685,10.1186/s12951-024-02756-2,Tea polyphenol nanoparticles enable targeted siRNA delivery and multi-bioactive therapy for abdominal aortic aneurysms.,https://pubmed.ncbi.nlm.nih.gov/39118143/,Results and discussion > Safety assessment of TPNs-siRNA; paragraph 67,516,mouse,,blood; heart; liver; spleen; lung; kidney,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00108,P0,toxicity,toxicity_endpoint,459,yes,A,415,AA-T3A-C12/siHSP47 LNP,siRNA,HSP47,,hepatic: hepatotoxicity,4035,36650129,PMC9845313,10.1038/s41467-022-35637-z,Ligand-tethered lipid nanoparticles for targeted RNA delivery to treat liver fibrosis.,https://pubmed.ncbi.nlm.nih.gov/36650129/,Results > Safety evaluation; paragraph 19,518,mouse,,liver; heart; spleen; lung; kidney; serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00109,P0,toxicity,toxicity_endpoint,461,yes,A,417,MPLN siRNA nanoparticles,siRNA,,,general safety: cell viability/cytotoxicity,12533,36755201,PMC10074117,10.1002/advs.202300282,Lymphocyte Membrane- and 12p1-Dual-Functionalized Nanoparticles for Free HIV-1 Trapping and Precise siRNA Delivery into HIV-1-Infected Cells.,https://pubmed.ncbi.nlm.nih.gov/36755201/,Results > Cytotoxicity; paragraph 19,520,human cell line; human primary cells,,TZM-bl; MT2; PBMC,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00110,P0,toxicity,toxicity_endpoint,462,yes,A,418,ZNF416 siRNA-loaded liposomes,siRNA,ZNF416,,general safety: systemic safety/tolerability,12545,36371191,PMC9652794,10.1186/s12967-022-03740-w,Targeted delivery of ZNF416 siRNA-loaded liposomes attenuates experimental pulmonary fibrosis.,https://pubmed.ncbi.nlm.nih.gov/36371191/,Results > Administration of ZNF416 siRNA-loaded liposomes attenuates pulmonary fibrosis in experimental mouse models; paragraph 41,521,mouse; cell line,,fibroblasts; serum; major organs,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00111,P0,toxicity,toxicity_endpoint,463,yes,A,419,CS-OA coated PLGA siRNA nanoparticles,siRNA,,,general safety: cell viability/cytotoxicity,4114,34684009,PMC8539707,10.3390/pharmaceutics13101716,Chitosan Oleate Coated PLGA Nanoparticles as siRNA Drug Delivery System.,https://pubmed.ncbi.nlm.nih.gov/34684009/,3. Results and Discussion > 3.4. Cytotoxicity Test on Human CD14+ Monocytes from Peripheral Blood; paragraph 23,522,human primary cells,,CD14+ peripheral-blood monocytes,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00112,P0,toxicity,toxicity_endpoint,465,yes,A,421,siSTAT3/DoCh LNP,siRNA,STAT3,,hepatic: hepatotoxicity,4197,32059541,PMC7072202,10.3390/biom10020285,Silencing of STAT3 via Peptidomimetic LNP-Mediated Systemic Delivery of RNAi Downregulates PD-L1 and Inhibits Melanoma Growth.,https://pubmed.ncbi.nlm.nih.gov/32059541/,3. Results and Discussion; paragraph 27,524,mouse,,liver; lung; serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00113,P0,toxicity,toxicity_endpoint,470,yes,A,426,Br-ApoE(K->A)-PMO and related P-PMO conjugates,PMO,,,general safety: cell viability/cytotoxicity,12658,28118087,PMC5467147,10.1089/nat.2016.0652,Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy.,https://pubmed.ncbi.nlm.nih.gov/28118087/,Results > PMO conjugation and cellular assays as measures of cell uptake ability; paragraph 25,529,human cell line,,Huh7 hepatocytes,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00114,P0,toxicity,toxicity_endpoint,475,yes,A,494,unspecified ASO,ASO,,,hepatic: hepatotoxicity,1055,42095135,PMC13140060,10.1016/j.omtn.2026.102930,Development of an exon 27-skipping antisense oligonucleotide as a targeted therapy for refractory skin ulcers in Werner syndrome.,https://pubmed.ncbi.nlm.nih.gov/42095135/,Results > Safety evaluation; paragraph 25,752,cell line,,cell line,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00115,P0,toxicity,toxicity_endpoint,476,yes,A,1005,unspecified ASO (PMID:41981306),ASO,,,general safety: systemic safety/tolerability,1058,41981306,PMC13099374,10.1038/s41591-026-04314-9,Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy.,https://pubmed.ncbi.nlm.nih.gov/41981306/,Results > Safety characterization and IND-enabling studies; paragraph 15,753,rat,,brain/CNS,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00116,P0,toxicity,toxicity_endpoint,477,yes,A,1006,"TLK2 ASO (recovered B2 from PMID:41878536, curator:CM)",ASO,,,hepatic: hepatotoxicity,1062,41878536,PMC13006219,10.3389/fonc.2026.1659341,Targeting TLK2 with antisense oligonucleotides as a new strategy in acute myeloid leukemia.,https://pubmed.ncbi.nlm.nih.gov/41878536/,Results; paragraph 24,754,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00117,P0,toxicity,toxicity_endpoint,478,yes,A,1007,unspecified ASO (PMID:42033225),ASO,,,hepatic: hepatotoxicity,1185,42033225,PMC13109722,10.1093/nar/gkag343,Direct targeting of C9ORF72 repeat RNA with fluorinated antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/42033225/,Results > F-ASO gapmers reduce disease-associated repeat RNA foci in patient-derived NSCs; paragraph 32,755,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00118,P0,toxicity,toxicity_endpoint,479,yes,A,507,siRNA-Lipid,siRNA,,,hepatic: hepatotoxicity,1193,41919978,PMC13085844,10.1021/acsnano.5c21452,DNA Repair Enzyme Regulation Strategy for Enhanced Pancreatic Neuroendocrine Tumor Therapy via Targeting siRNA-Lipid Nanoparticles.,https://pubmed.ncbi.nlm.nih.gov/41919978/,Results > In Vivo Therapeutic Efficacy Evaluation in Various Mouse Models; paragraph 39,756,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00119,P0,toxicity,toxicity_endpoint,480,yes,A,508,CO2,ASO,,,general safety: cell viability/cytotoxicity,1210,41704705,PMC12907196,10.3389/fmed.2026.1719202,Investigation of the activity of phosphothioate and phosphothioate-LNA-modified oligonucleotides against HIV-1.,https://pubmed.ncbi.nlm.nih.gov/41704705/,Materials and methods > Assessment of oligonucleotide toxicity (LD50) using the MTT assay; paragraph 13,757,cell line,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00120,P0,toxicity,toxicity_endpoint,481,yes,A,1008,"siRNA2 (recovered B2 from PMID:42005705, curator:CM)",siRNA,,,hepatic: hepatotoxicity,1368,42005705,PMC13091633,10.2147/IJN.S588018,Preparation and Antitumor Activity of METTL3 SiRNA Nanoparticles Carried by Hypoxia Sensitive Polymer (mPEG-Azo-PEI).,https://pubmed.ncbi.nlm.nih.gov/42005705/,Results > Cytotoxicity of mPEG-Azo-PEI; paragraph 50,758,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00121,P0,toxicity,toxicity_endpoint,483,yes,A,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,2412,41587091,PMC12994142manuscript-id: NIHMS2150465,10.1182/blood.2025029834,Silencing of PAI-1 using siRNA-lipid nanoparticles reduces thrombosis and prolongs life span in murine models.,https://pubmed.ncbi.nlm.nih.gov/41587091/,RESULTS > siPAI-1 does not elicit overt toxicity in vivo.; paragraph 22,760,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00122,P0,toxicity,toxicity_endpoint,485,yes,A,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,3294,41572432,PMC13042977,10.1002/advs.202510547,PARPi Combining Nanoparticle LIN28B siRNA for the Management of Malignant Ascites.,https://pubmed.ncbi.nlm.nih.gov/41572432/,Results > The siLin28b Plus BMN673 Combination Inhibits the Deterioration of Vascular Permeability; paragraph 44,762,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00123,P0,toxicity,toxicity_endpoint,492,yes,A,1009,"eteplirsen (recovered B2 from PMID:41961051, curator:CM)",PMO,,,hepatic: hepatotoxicity,5375,41961051,PMC13154584,10.57264/cer-2025-0108,"Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD.",https://pubmed.ncbi.nlm.nih.gov/41961051/,Results > Treatment continuation; paragraph 33,769,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00124,P0,toxicity,toxicity_endpoint,499,yes,A,514,tofersen,ASO,,,general safety: systemic safety/tolerability,6390,41661214,PMC12723595,10.1001/jamaneurol.2025.4946,Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis.,https://pubmed.ncbi.nlm.nih.gov/41661214/,Results > Safety; paragraph 35,776,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00125,P0,toxicity,toxicity_endpoint,500,yes,A,1010,unspecified ASO (PMID:41630990),ASO,,,hepatic: hepatotoxicity,6475,41630990,PMC12860987,10.1016/j.omtn.2026.102834,Improving angiogenesis ameliorates the efficacy of ASO-based exon skipping for the treatment of Duchenne muscular dystrophy.,https://pubmed.ncbi.nlm.nih.gov/41630990/,Results > Safety evaluation through serum biochemistry; paragraph 22,777,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00126,P0,toxicity,toxicity_endpoint,511,yes,A,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,general safety: cell viability/cytotoxicity,7336,40762902,PMC12893402manuscript-id: NIHMS2134215,10.1007/s13346-025-01920-x,Genetically bio-engineered PD-L1 targeted exosomes for immunotherapy of resistant triple negative breast cancer.,https://pubmed.ncbi.nlm.nih.gov/40762902/,Results > PDL E inhibits viability of MDA-MB-231 DOX RT cells in 2D and 3D cultures; paragraph 36,788,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00127,P0,toxicity,toxicity_endpoint,513,yes,A,1011,"ASO 8–3 (recovered B2 from PMID:40098305, curator:CM)",ASO,,,hepatic: hepatotoxicity,1020,40098305,PMC12044654,10.1111/cas.70024,AmNA-Modified Antisense Oligonucleotide Targeting MCM8 as a Cancer-Specific Chemosensitizer for Platinum Compounds.,https://pubmed.ncbi.nlm.nih.gov/40098305/,Results > ASO 8–3419 Enhances the Therapeutic Effect of Cisplatin in Xenografted HCT116 Tumors in Nude Mice; paragraph 26,790,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00128,P0,toxicity,toxicity_endpoint,514,yes,A,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic: hepatotoxicity,1233,40926414,PMC12703169,10.1016/j.ymthe.2025.08.045,Restoring chloride efflux in cystic fibrosis with TMEM16a antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/40926414/,Results > Subcutaneous administration allows for optimal distribution and is not toxic to mice; paragraph 14,791,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00129,P0,toxicity,toxicity_endpoint,517,yes,A,299,nusinersen,ASO,,,general safety: systemic safety/tolerability,1256,40367190,PMC12423764manuscript-id: NIHMS2101949,10.1126/scitranslmed.adv4656,Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy.,https://pubmed.ncbi.nlm.nih.gov/40367190/,RESULTS > Prenatal IA ASO administration enhances therapeutic efficacy in SMAΔ7 mice; paragraph 10,794,human,,brain/CNS,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00130,P0,toxicity,toxicity_endpoint,518,yes,A,522,AZD8701,ASO,,,hepatic: hepatotoxicity,1270,39937271,PMC11995004,10.1158/1078-0432.CCR-24-1818,"AZD8701, an Antisense Oligonucleotide Targeting FOXP3 mRNA, as Monotherapy and in Combination with Durvalumab: A Phase I Trial in Patients with Advanced Solid Tumors.",https://pubmed.ncbi.nlm.nih.gov/39937271/,Results > Safety > AZD8701 monotherapy; paragraph 39,795,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00131,P0,toxicity,toxicity_endpoint,520,yes,A,1012,"Chol-HDO[PMO] (recovered B2 from PMID:41429785, curator:CM)",PMO,,,hepatic: hepatotoxicity,1783,41429785,PMC12722745,10.1038/s41467-025-66765-x,"Morpholino-RNA duplex exhibits robust, sustained, and safe steric-block antisense activity by intracerebroventricular and intrathecal injection.",https://pubmed.ncbi.nlm.nih.gov/41429785/,Results > Safety evaluation of Chol-HDO[PMO]; paragraph 27,797,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00132,P0,toxicity,toxicity_endpoint,522,yes,A,1013,"iExoKrasG12D (recovered B2 from PMID:41027940, curator:CM)",siRNA,,,hepatic: hepatotoxicity,1803,41027940,PMC12485160,10.1038/s41467-025-63718-2,Engineered exosomes with Kras(G12D) specific siRNA in pancreatic cancer: a phase I study with immunological correlates.,https://pubmed.ncbi.nlm.nih.gov/41027940/,Results > GMP grade exosomes for siRNA delivery showed no toxicity in preclinical studies; paragraph 6,799,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00133,P0,toxicity,toxicity_endpoint,529,yes,A,1014,"D-siRNA (recovered B2 from PMID:41473322, curator:CM)",siRNA,,,hepatic: hepatotoxicity,3402,41473322,PMC12746015,10.64898/2025.12.16.694641,Albumin-binding dendrimer-conjugated siRNA enables safe and effective gene silencing throughout the central nervous system.,https://pubmed.ncbi.nlm.nih.gov/41473322/,"RESULTS > D-siRNA supports safe, potent and durable activity in the CNS; paragraph 24",806,mouse,,brain/CNS,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00134,P0,toxicity,toxicity_endpoint,530,yes,A,435,siRNAs,siRNA,,,hepatic: hepatotoxicity,3407,41432033,PMC12723224,10.1093/nar/gkaf1374,Potent and durable gene modulation in heart and muscle with chemically defined lipophilic siRNAs.,https://pubmed.ncbi.nlm.nih.gov/41432033/,Results > Muscle-optimal siRNA scaffold achieves systemic muscle effects and has a favorable safety profile; Figure 6. caption,807,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00135,P0,toxicity,toxicity_endpoint,531,yes,A,525,elebsiran,siRNA,,,hepatic: hepatotoxicity,3426,41326382,PMC12780110,10.1038/s41467-025-66876-5,An RNA interference therapeutic potentially achieves functional cure of chronic hepatitis B virus infection.,https://pubmed.ncbi.nlm.nih.gov/41326382/,Results > Safety profiles of KC13-M2G2 in SD rats and cynomolgus monkeys; paragraph 19,808,rat,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00136,P0,toxicity,toxicity_endpoint,533,yes,A,1015,"KFFK (FFK)2-conjugated PNA (recovered B2 from PMID:41096791, curator:CM)",ASO,,,general safety: cell viability/cytotoxicity,3472,41096791,PMC12525168,10.3390/ijms26199526,Targeting carA Using Optimized Antisense Peptide Nucleic Acid-Cell-Penetrating Peptide Conjugates in Acinetobacter baumannii: A Novel Antibacterial Approach.,https://pubmed.ncbi.nlm.nih.gov/41096791/,2. Results > 2.2. Cytotoxicity of CPP–PNA Conjugates in Mammalian Cells; paragraph 7,810,human,,lung,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00137,P0,toxicity,toxicity_endpoint,540,yes,A,1016,"L2-siRNA (recovered B2 from PMID:40598893, curator:CM)",siRNA,,,hepatic: hepatotoxicity,3619,40598893,PMC12214009,10.1093/nar/gkaf600,Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid-siRNA conjugate administered into cerebrospinal fluid.,https://pubmed.ncbi.nlm.nih.gov/40598893/,Results > L2-siRNA does not induce hallmarks of toxicity; paragraph 54,817,rat,,brain/CNS,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00138,P0,toxicity,toxicity_endpoint,543,yes,A,1017,"HSA@ICG/siRNA NPs (recovered B2 from PMID:40259913, curator:CM)",siRNA,,,hepatic: hepatotoxicity,3710,40259913,PMC12010081,10.2147/IJN.S511655,NIR-Triggered siRNA Release and Lysosomal Escape for Synergistic Photothermal Tumor Therapy.,https://pubmed.ncbi.nlm.nih.gov/40259913/,Results > Anti-Tumor Efficacy and Safety Evaluation in Vivo; paragraph 63,820,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00139,P0,toxicity,toxicity_endpoint,544,yes,A,1018,unspecified siRNA (PMID:40143003),siRNA,,,general safety: cell viability/cytotoxicity,3725,40143003,PMC11946471,10.3390/pharmaceutics17030339,Analysis of Polymer/siRNA Nanoparticle Efficacy and Biocompatibility in 3D Air-Liquid Interface Culture Compared to 2D Cell Culture.,https://pubmed.ncbi.nlm.nih.gov/40143003/,2. Materials and Methods > 2.8. Acute Toxicity (LDH Release); paragraph 25,821,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00140,P0,toxicity,toxicity_endpoint,546,yes,A,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,renal: renal safety,3771,39860035,PMC11766699,10.3390/life15010095,A Novel Approach for In Vitro Testing and Hazard Evaluation of Nanoformulated RyR2-Targeting siRNA Drugs Using Human PBMCs.,https://pubmed.ncbi.nlm.nih.gov/39860035/,2. Materials and Methods > 2.8. Toxicity Pathways Evaluation; paragraph 22,823,cell line,,kidney,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00141,P0,toxicity,toxicity_endpoint,548,yes,A,435,siRNAs,siRNA,,,hepatic: hepatotoxicity,3801,39632755,PMC11704396,10.4062/biomolther.2024.071,"Design, Screening and Development of Asymmetric siRNAs Targeting the MYC Oncogene in Triple-Negative Breast Cancer.",https://pubmed.ncbi.nlm.nih.gov/39632755/,RESULTS > Reduced 5’ end- and -2’- fluoro modification design; paragraph 37,825,cell line,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00142,P0,toxicity,toxicity_endpoint,556,yes,A,1019,"siPCSK9 (recovered B2 from PMID:41290746, curator:CM)",siRNA,,,hepatic: hepatotoxicity,7717,41290746,PMC12749358,10.1038/s41467-025-66630-x,Silencing PCSK9 reshapes the spatiotemporal activation of STING for safe and effective cancer immunotherapy.,https://pubmed.ncbi.nlm.nih.gov/41290746/,Results > NP containing PCSK9 siRNA and STING agonists enable safe and effective cancer immunotherapy; paragraph 17,833,mouse,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00143,P0,toxicity,toxicity_endpoint,560,yes,A,1020,unspecified siRNA (PMID:41226635),siRNA,,,hepatic: hepatotoxicity,7835,41226635,PMC12607363,10.3390/ijms262110582,Anti-Angiogenic RNAi-Based Treatment of Endometriosis in a Rat Model Using CXCR4-Targeted Peptide Nanoparticles.,https://pubmed.ncbi.nlm.nih.gov/41226635/,2. Results > 2.4. Evaluation of the Cytotoxic Effects of Ternary Polyplexes; paragraph 22,837,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00144,P0,toxicity,toxicity_endpoint,566,yes,A,531,siPD-L1,siRNA,,,hepatic: hepatotoxicity,8508,40885724,PMC12398490,10.1038/s41467-025-63415-0,Modulating tumor collagen fiber alignment for enhanced lung cancer immunotherapy via inhaled RNA.,https://pubmed.ncbi.nlm.nih.gov/40885724/,Results > Inhalation of mscFv/siPD-L1@LNP drives TME reconfiguration in orthotopic lung tumors; paragraph 15,843,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00145,P0,toxicity,toxicity_endpoint,578,yes,A,1021,"DG9-PMO (recovered B2 from PMID:40368879, curator:CM)",PMO,,,hepatic: hepatotoxicity,9560,40368879,PMC12078682,10.1038/s41467-025-59494-8,DG9 boosts PMO nuclear uptake and exon skipping to restore dystrophic muscle and cardiac function.,https://pubmed.ncbi.nlm.nih.gov/40368879/,Results > DG9-PMO facilitates enhanced cellular uptake and efficacious nuclear localization; paragraph 9,855,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00146,P0,toxicity,toxicity_endpoint,581,yes,A,1022,unspecified siRNA (PMID:40297405),siRNA,,,hepatic: hepatotoxicity,9662,40297405,PMC12036690,10.2147/IJN.S514375,ROS-Responsive Biomimetic Nanocomplexes of Liposomes and Macrophage-Derived Exosomes for Combination Breast Cancer Therapy.,https://pubmed.ncbi.nlm.nih.gov/40297405/,Results > In vitro Antitumor Effect of E-cLip-DTX / Si; paragraph 46,858,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00147,P0,toxicity,toxicity_endpoint,587,yes,A,299,nusinersen,ASO,,,renal: renal safety,9966,40060931,PMC11889396,10.1016/j.omtn.2025.102479,Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2S.,https://pubmed.ncbi.nlm.nih.gov/40060931/,Results > Three-month rat toxicology study; paragraph 38,864,human,,kidney,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00148,P0,toxicity,toxicity_endpoint,589,yes,A,1023,unspecified CpG (PMID:39838063),ASO,,,hepatic: hepatotoxicity,10245,39838063,PMC11751084,10.1038/s43856-024-00712-7,Preclinical assessment of splicing modulation therapy for ABCA4 variant c.768G>T in Stargardt disease.,https://pubmed.ncbi.nlm.nih.gov/39838063/,Results > Safety aspects of the lead candidate AON; paragraph 43,866,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00149,P0,toxicity,toxicity_endpoint,598,yes,A,1024,unspecified ASO (PMID:39460376),ASO,,,hepatic: hepatotoxicity,1282,39460376,PMC11638874,10.1016/j.ymthe.2024.10.024,Quantifying and mitigating motor phenotypes induced by antisense oligonucleotides in the central nervous system.,https://pubmed.ncbi.nlm.nih.gov/39460376/,Results > Pre-saturation with calcium reduces acute neurotoxicity; paragraph 25,875,,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00150,P0,toxicity,toxicity_endpoint,600,yes,A,1025,"ION-827359 (recovered B2 from PMID:39286058, curator:CM)",ASO,,,hepatic: hepatotoxicity,1562,39286058,PMC11403593,10.1183/23120541.00986-2023,"Randomised, phase 1/2a trial of ION-827359, an antisense oligonucleotide inhibitor of ENaC.",https://pubmed.ncbi.nlm.nih.gov/39286058/,Methods > End-points > Safety; paragraph 13,877,,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00151,P0,toxicity,toxicity_endpoint,602,yes,A,472,siRNA-mediated,siRNA,,,hepatic: hepatotoxicity,2588,38779336,PMC11109470,10.1016/j.omtm.2024.101258,siRNA-mediated reduction of a circulating protein in swine using lipid nanoparticles.,https://pubmed.ncbi.nlm.nih.gov/38779336/,Results > siRNA-LNPs displayed a low risk of CARPA and other toxicities; paragraph 9,879,cell line,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00152,P0,toxicity,toxicity_endpoint,607,yes,A,1026,"aODN-Survivin (recovered B2 from PMID:38672408, curator:CM)",ASO,,,hepatic: hepatotoxicity,3903,38672408,PMC11048556,10.3390/biom14040390,Synthesis and Validation of TRIFAPYs as a Family of Transfection Agents for Therapeutic Oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/38672408/,3. Results > 3.3. Intrinsic Toxicity and Transfection Efficiency of TRIFAPYs; paragraph 31,884,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00153,P0,toxicity,toxicity_endpoint,608,yes,A,1027,unspecified siRNA (PMID:38617800),siRNA,,,general safety: cell viability/cytotoxicity,3912,38617800,PMC11015852,10.2147/IJN.S443413,Microdroplets Encapsulated with NFATc1-siRNA and Exosomes-Derived from MSCs Onto 3D Porous PLA Scaffold for Regulating Osteoclastogenesis and Promoting Osteogenesis.,https://pubmed.ncbi.nlm.nih.gov/38617800/,Results > Biocompatibility Test of Different Scaffolds; paragraph 34,885,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00154,P0,toxicity,toxicity_endpoint,616,yes,A,299,nusinersen,ASO,,,hepatic: hepatotoxicity,11036,38921951,PMC11206794,10.3390/neurolint16030047,Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait.,https://pubmed.ncbi.nlm.nih.gov/38921951/,2. Materials and Methods > 2.3. Functional and Safety Assessments; paragraph 10,893,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00155,P0,toxicity,toxicity_endpoint,623,yes,A,551,fitusiran,siRNA,,,hepatic: hepatotoxicity,11416,38452197,PMC11181353,10.1182/blood.2023021864,Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial.,https://pubmed.ncbi.nlm.nih.gov/38452197/,Results > Safety; paragraph 27,900,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00156,P0,toxicity,toxicity_endpoint,628,yes,A,1028,"cRGD-GR9G-(LLHH)3/siEGFR/siP65 (recovered B2 from PMID:39539970, curator:CM)",siRNA,,,hepatic: hepatotoxicity,12446,39539970,PMC11559232,10.2147/IJN.S483252,Stimuli-Responsive Peptide/siRNA Nanoparticles as a Radiation Sensitizer for Glioblastoma Treatment by Co-Inhibiting RELA/P65 and EGFR.,https://pubmed.ncbi.nlm.nih.gov/39539970/,Results > Influence of Nanoparticles on Tumor Growth and Survival Time in Orthotopic Glioblastoma Mice and Its Evaluation of Immunogenicity and Toxicity; paragraph 53,905,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00157,P0,toxicity,toxicity_endpoint,639,yes,A,1029,unspecified ASO (PMID:38042877),ASO,,,hepatic: hepatotoxicity,1136,38042877,PMC10693639,10.1038/s41467-023-43714-0,Dynamic and static control of the off-target interactions of antisense oligonucleotides using toehold chemistry.,https://pubmed.ncbi.nlm.nih.gov/38042877/,Results > Optimized BROTHERS architectures mitigate hepatotoxicity without affecting antisense activity; paragraph 11,916,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00158,P0,toxicity,toxicity_endpoint,640,yes,A,185,unspecified siRNA,siRNA,,,general safety: cell viability/cytotoxicity,1292,37834294,PMC10573891,10.3390/ijms241914846,Antiviral Efficacy of RNase H-Dependent Gapmer Antisense Oligonucleotides against Japanese Encephalitis Virus.,https://pubmed.ncbi.nlm.nih.gov/37834294/,4. Materials and Methods > 4.8. Cytotoxicity Assay; paragraph 37,917,cell line,,cell line,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00159,P0,toxicity,toxicity_endpoint,642,yes,A,1030,unspecified siRNA (PMID:36727438),ASO,,,hepatic: hepatotoxicity,1958,36727438,PMC9976930,10.1093/nar/gkad023,Simultaneous inhibition of endocytic recycling and lysosomal fusion sensitizes cells and tissues to oligonucleotide therapeutics.,https://pubmed.ncbi.nlm.nih.gov/36727438/,RESULTS > SH-BC-893 increases ASO delivery and activity in vitro; paragraph 20,919,human,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00160,P0,toxicity,toxicity_endpoint,644,yes,A,1031,unspecified ASO (PMID:38015828),ASO,,,hepatic: hepatotoxicity,11827,38015828,PMC10683999,10.1371/journal.pone.0294731,Targeted ASO-mediated Atp1a2 knockdown in astrocytes reduces SOD1 aggregation and accelerates disease onset in mutant SOD1 mice.,https://pubmed.ncbi.nlm.nih.gov/38015828/,Results > Design of locked nucleic acid (LNA) ASO gapmers targeting Atp1a2; Fig 1 caption,921,mouse,,,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00161,P0,toxicity,toxicity_endpoint,646,yes,A,1032,"9b2KC-23b (recovered B2 from PMID:37744174, curator:CM)",PMO,,,hepatic: hepatotoxicity,11931,37744174,PMC10514136,10.1016/j.omtn.2023.09.001,Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression.,https://pubmed.ncbi.nlm.nih.gov/37744174/,Results > CPP-PMO antimiRs improve histopathology signs and muscle function in HSALR; paragraph 15,923,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00162,P0,toxicity,toxicity_endpoint,648,yes,A,1033,unspecified ASO (PMID:37704069),ASO,,,hepatic: hepatotoxicity,12175,37704069,PMC10591917manuscript-id: NIHMS1931813,10.1016/j.bone.2023.116898,Antisense oligonucleotides targeting a NOTCH3 mutation in male mice ameliorate the cortical osteopenia of lateral meningocele syndrome.,https://pubmed.ncbi.nlm.nih.gov/37704069/,RESULTS > Effect of Notch3 ASOs on Notch3 Expression in Cells of the Osteoblast Lineage; paragraph 17,925,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00163,P0,toxicity,toxicity_endpoint,650,yes,A,1034,"iLANDsiPAK4 (recovered B2 from PMID:37719367, curator:CM)",siRNA,,,hepatic: hepatotoxicity,13969,37719367,PMC10501866,10.1016/j.apsb.2023.03.020,Antitumor synergism between PAK4 silencing and immunogenic phototherapy of engineered extracellular vesicles.,https://pubmed.ncbi.nlm.nih.gov/37719367/,Results > Synergistic antitumor effects in vivo; paragraph 29,927,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00164,P0,toxicity,toxicity_endpoint,654,yes,A,1035,"RO7062931 (recovered B2 from PMID:37147890, curator:CM)",ASO,,,hepatic: hepatotoxicity,14152,37147890,PMC10339693,10.1111/cts.13531,A single ascending dose study of single-stranded oligodeoxyribonucleotide RO7062931 in Chinese healthy volunteers.,https://pubmed.ncbi.nlm.nih.gov/37147890/,RESULTS > Safety; paragraph 27,931,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00165,P0,toxicity,toxicity_endpoint,658,yes,A,1036,"DG9-PMO (recovered B2 from PMID:36719755, curator:CM)",PMO,,,immunotoxicity: immune activation,14276,36719755,PMC10077475,10.1172/jci.insight.160516,DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration.,https://pubmed.ncbi.nlm.nih.gov/36719755/,Results > DG9-PMO treatment rescues the SMA phenotype without apparent toxicity.; paragraph 19,935,mouse,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00166,P0,toxicity,toxicity_endpoint,659,yes,A,1037,unspecified siRNA (PMID:36614194),siRNA,,,hepatic: hepatotoxicity,14302,36614194,PMC9821664,10.3390/ijms24010752,Target-Specific Nanoparticle Polyplex Down-Regulates Mutant Kras to Prevent Pancreatic Carcinogenesis and Halt Tumor Progression.,https://pubmed.ncbi.nlm.nih.gov/36614194/,2. Results > 2.6. CCK-BR Targeted NPs Exhibit Broad Safety Profile and Lack off Target Toxicity; paragraph 15,936,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00167,P0,toxicity,toxicity_endpoint,660,yes,A,1038,unspecified ASO (PMID:35861704),ASO,,,hepatic: hepatotoxicity,1485,35861704,PMC9595634,10.1089/nat.2022.0005,The Combination of Mesyl-Phosphoramidate Inter-Nucleotide Linkages and 2'-O-Methyl in Selected Positions in the Antisense Oligonucleotide Enhances the Performance of RNaseH1 Active PS-ASOs.,https://pubmed.ncbi.nlm.nih.gov/35861704/,"Results > MsPA backbone improves potency, whereas gap2 OMe reduces toxicity in cytotoxic PS ASOs; FIG. 1. caption",937,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00168,P0,toxicity,toxicity_endpoint,661,yes,A,1039,"ASO-001933 (recovered B2 from PMID:36090761, curator:CM)",ASO,,,hepatic: hepatotoxicity,1615,36090761,PMC9424863,10.1016/j.omtn.2022.07.027,Identification and characterization of a MAPT-targeting locked nucleic acid antisense oligonucleotide therapeutic for tauopathies.,https://pubmed.ncbi.nlm.nih.gov/36090761/,Results > Initial tiling screen identified a parental ASO targeting the 3′ UTR of MAPT with high cross-species reactivity; paragraph 6,938,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00169,P0,toxicity,toxicity_endpoint,662,yes,A,561,single-dose,ASO,,,hepatic: hepatotoxicity,1617,35860385,PMC9287140,10.1016/j.omtn.2022.06.015,"Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models.",https://pubmed.ncbi.nlm.nih.gov/35860385/,Results > Single-dose tolerability study of lead ASO candidates in cynomolgus monkeys; paragraph 17,939,human,,brain/CNS,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00170,P0,toxicity,toxicity_endpoint,664,yes,A,1040,"LNP-S60-siRNA (recovered B2 from PMID:36520934, curator:CM)",siRNA,,,hepatic: hepatotoxicity,1964,36520934,PMC9754606,10.1371/journal.pone.0276905,Inhibition of c-Rel expression in myeloid and lymphoid cells with distearoyl -phosphatidylserine (DSPS) liposomal nanoparticles encapsulating therapeutic siRNA.,https://pubmed.ncbi.nlm.nih.gov/36520934/,Results > LNP containing DSPS can reach the cytoplasm of cells without detectable cytotoxicity; paragraph 14,941,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00171,P0,toxicity,toxicity_endpoint,666,yes,A,1041,"IONIS-FXIRx (recovered B2 from PMID:35155859, curator:CM)",ASO,,,hepatic: hepatotoxicity,1981,35155859,PMC8820988,10.1016/j.ekir.2021.11.011,Phase 2 Study of the Factor XI Antisense Inhibitor IONIS-FXI(Rx) in Patients With ESRD.,https://pubmed.ncbi.nlm.nih.gov/35155859/,Results > Safety; paragraph 28,943,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00172,P0,toxicity,toxicity_endpoint,668,yes,A,1042,"ASO 5–2 (recovered B2 from PMID:34949835, curator:CM)",ASO,,,hepatic: hepatotoxicity,2727,34949835,PMC8861976manuscript-id: NIHMS1779498,10.1038/s41591-021-01557-6,Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.,https://pubmed.ncbi.nlm.nih.gov/34949835/,Results > PS reduction optimizes ASO5 safety and activity in mice; paragraph 15,945,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00173,P0,toxicity,toxicity_endpoint,670,yes,A,1043,unspecified siRNA (PMID:36457549),siRNA,,,hepatic: hepatotoxicity,4051,36457549,PMC9707323,10.2147/IJN.S383862,Combined Self-Assembled iRGD Polymersomes for Effective Targeted siRNA Anti-Tumor Therapy.,https://pubmed.ncbi.nlm.nih.gov/36457549/,Materials and Methods > The Cytotoxicity Studies of iRGD Functionalized Lipopolysaccharide-Amine Nanopolymersomes to Mesenchymal Stem Cells; paragraph 11,947,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00174,P0,toxicity,toxicity_endpoint,676,yes,A,1044,unspecified ASO (PMID:34921016),ASO,,,hepatic: hepatotoxicity,14660,34921016,PMC8898261manuscript-id: NIHMS1767244,10.1158/0008-5472.CAN-20-0948,ASO-Based PKM Splice-Switching Therapy Inhibits Hepatocellular Carcinoma Growth.,https://pubmed.ncbi.nlm.nih.gov/34921016/,Results > ASO1-cEt/DNA inhibits HCC cell growth; paragraph 29,953,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00175,P0,toxicity,toxicity_endpoint,679,yes,A,299,nusinersen,ASO,,,hepatic: hepatotoxicity,1645,33860461,PMC8609055,10.1007/s13311-021-01045-2,Reconditioning the Neurogenic Niche of Adult Non-human Primates by Antisense Oligonucleotide-Mediated Attenuation of TGFβ Signaling.,https://pubmed.ncbi.nlm.nih.gov/33860461/,Results > In Vivo Effects of NVP-13 on Target Regulation and Intracellular Pathways: NVP-13 is a Safe and Effective Compound in Cynomolgus Monkeys; paragraph 8,956,non-human primate,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00176,P0,toxicity,toxicity_endpoint,686,yes,A,1045,"CpG2018B (recovered B2 from PMID:34566407, curator:CM)",CpG oligodeoxynucleotide,,,hepatic: hepatotoxicity,14741,34566407,PMC8459178,10.2147/DDDT.S325790,CpG Oligodeoxynucleotide Developed to Activate Primate Immune Responses Promotes Antitumoral Effects in Combination with a Neoantigen-Based mRNA Cancer Vaccine.,https://pubmed.ncbi.nlm.nih.gov/34566407/,Results > Safety of Combined Therapy with CpG and the mRNA Vaccine; paragraph 28,963,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00177,P0,toxicity,toxicity_endpoint,693,yes,A,563,Site-specific,ASO,,,hepatic: hepatotoxicity,14905,33544849,PMC7913697,10.1093/nar/gkab047,Site-specific incorporation of 5'-methyl DNA enhances the therapeutic profile of gapmer ASOs.,https://pubmed.ncbi.nlm.nih.gov/33544849/,"RESULTS > Effect of site-specific incorporation of R- and S-5′-methyl DNA on duplex thermal stability, ASO activity and toxicity in cells; Figure 3. caption",970,cell line,,cell line,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00178,P0,toxicity,toxicity_endpoint,694,yes,A,1046,unspecified PMO (PMID:33337582),PMO,,,hepatic: hepatotoxicity,14926,33337582,PMC7863382,10.15252/emmm.202012993,MOTS-c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice.,https://pubmed.ncbi.nlm.nih.gov/33337582/,Results > PMO‐M improves muscle pathologies without detectable toxicity in mdx mice; paragraph 20,971,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00179,P0,toxicity,toxicity_endpoint,695,yes,A,1047,"NVP-13 (recovered B2 from PMID:32178467, curator:CM)",ASO,,,hepatic: hepatotoxicity,1492,32178467,PMC7139664,10.3390/ijms21061952,Antisense Oligonucleotide in LNA-Gapmer Design Targeting TGFBR2-A Key Single Gene Target for Safe and Effective Inhibition of TGFβ Signaling.,https://pubmed.ncbi.nlm.nih.gov/32178467/,2. Results > 2.1. Discovery Process; paragraph 7,972,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00180,P0,toxicity,toxicity_endpoint,696,yes,A,1048,"LNAc/2′OMe18h (recovered B2 from PMID:31873063, curator:CM)",ASO,,,hepatic: hepatotoxicity,1660,31873063,PMC7133450,10.1089/nat.2019.0811,Systemic Evaluation of Chimeric LNA/2'-O-Methyl Steric Blockers for Myotonic Dystrophy Type 1 Therapy.,https://pubmed.ncbi.nlm.nih.gov/31873063/,Results > Toxicity status of chimeric LNA/2′OMe ASOs in vivo; paragraph 34,973,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00181,P0,toxicity,toxicity_endpoint,700,yes,A,1049,unspecified ASO (PMID:32805488),ASO,,,hepatic: hepatotoxicity,14986,32805488,PMC7452286,10.1016/j.omtn.2020.07.008,MYC ASO Impedes Tumorigenesis and Elicits Oncogene Addiction in Autochthonous Transgenic Mouse Models of HCC and RCC.,https://pubmed.ncbi.nlm.nih.gov/32805488/,"Results > MYC ASO Treatment Is Not Associated with Morbidity, Mortality, or Toxicity; paragraph 12",977,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00182,P0,toxicity,toxicity_endpoint,702,yes,A,1050,unspecified ASO (PMID:30988454),ASO,,,hepatic: hepatotoxicity,2782,30988454,PMC6465270,10.1038/s41598-019-42523-0,Systematic evaluation of 2'-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping in vitro.,https://pubmed.ncbi.nlm.nih.gov/30988454/,Results > Evaluation of in vitro cytotoxicity of the 2′-F modified AOs; paragraph 10,979,mouse,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00183,P0,toxicity,toxicity_endpoint,703,yes,A,563,Site-specific,ASO,,,hepatic: hepatotoxicity,3148,31034558,PMC6582325,10.1093/nar/gkz247,Site-specific replacement of phosphorothioate with alkyl phosphonate linkages enhances the therapeutic profile of gapmer ASOs by modulating interactions with cellular proteins.,https://pubmed.ncbi.nlm.nih.gov/31034558/,RESULTS > A single MOP linkage at position 2 in the DNA gap reduces toxicity for multiple ASOs; paragraph 46,980,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00184,P0,toxicity,toxicity_endpoint,705,yes,A,990,unspecified siRNA (PMID:31375812),siRNA,,,hepatic: hepatotoxicity,4213,31375812,PMC6879195manuscript-id: NIHMS1533065,10.1038/s41587-019-0205-0,A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.,https://pubmed.ncbi.nlm.nih.gov/31375812/,Results > Di-siRNA are safe and well-tolerated in mice; paragraph 21,982,mouse,,blood/serum,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00185,P0,toxicity,toxicity_endpoint,706,yes,A,1051,"IONIS-PKKRx (recovered B2 from PMID:30817230, curator:CM)",ASO,,,hepatic: hepatotoxicity,12610,30817230,PMC6461157,10.1089/nat.2018.0754,IONIS-PKK(Rx) a Novel Antisense Inhibitor of Prekallikrein and Bradykinin Production.,https://pubmed.ncbi.nlm.nih.gov/30817230/,Results > Phase I study results > Safety and tolerability; paragraph 45,983,,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00186,P0,toxicity,toxicity_endpoint,708,yes,A,1052,"BMSP-PMO-GF (recovered B2 from PMID:31629961, curator:CM)",PMO,,,hepatic: hepatotoxicity,15084,31629961,PMC6807288,10.1016/j.omtn.2019.09.012,Hexose Potentiates Peptide-Conjugated Morpholino Oligomer Efficacy in Cardiac Muscles of Dystrophic Mice in an Age-Dependent Manner.,https://pubmed.ncbi.nlm.nih.gov/31629961/,Results > BMSP-PMO-GF Does Not Evoke Any Overt Toxicity in Adult mdx Mice; paragraph 14,985,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00187,P0,toxicity,toxicity_endpoint,723,yes,A,575,silk,CpG oligodeoxynucleotide,,,general safety: cell viability/cytotoxicity,12648,28694238,PMC5942204manuscript-id: NIHMS892590,10.1016/j.actbio.2017.07.014,Functionalized bioengineered spider silk spheres improve nuclease resistance and activity of oligonucleotide therapeutics providing a strategy for cancer treatment.,https://pubmed.ncbi.nlm.nih.gov/28694238/,2. Materials and Methods > 2.10. Cytotoxicity study; paragraph 19,1000,cell line,,cell line,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00188,P0,toxicity,toxicity_endpoint,727,yes,A,1053,"LNA-i-miR-221 (recovered B2 from PMID:27327137, curator:CM)",miRNA agomir,,,hepatic: hepatotoxicity,4239,27327137,PMC5022129,10.1038/mtna.2016.36,Pharmacokinetics and Pharmacodynamics of a 13-mer LNA-inhibitor-miR-221 in Mice and Non-human Primates.,https://pubmed.ncbi.nlm.nih.gov/27327137/,Results > P27 is upregulated in mouse tissues after LNA-i-miR-221 treatment; paragraph 8,1004,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00189,P0,toxicity,toxicity_endpoint,729,yes,A,1054,unspecified siRNA (PMID:27483025),siRNA,,,hepatic: hepatotoxicity,12672,27483025,PMC5023400,10.1038/mtna.2016.55,Silencing Myostatin Using Cholesterol-conjugated siRNAs Induces Muscle Growth.,https://pubmed.ncbi.nlm.nih.gov/27483025/,Results > Mstn siRNA cholesterol conjugate shows dose-dependent and sustained knockdown of Mstn mRNA in muscle and in circulating protein levels; paragraph 7,1006,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00190,P0,toxicity,toxicity_endpoint,731,yes,A,578,DUX4,PMO,,,hepatic: hepatotoxicity,15206,27378237,PMC5023379,10.1038/mt.2016.111,Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics.,https://pubmed.ncbi.nlm.nih.gov/27378237/,Results; paragraph 6,1008,,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00191,P0,toxicity,toxicity_endpoint,966,yes,A,1055,"siRMSTN (recovered B2 from PMID:41041909, curator:CM)",siRNA,,,hepatic: hepatotoxicity,1776,41041909,PMC12836460,10.1002/adhm.202502186,Silencing Myostatin Using In Vivo Self-Assembled siRNA Protects Against Cancer- and Dexamethasone-Induced Muscle Atrophy.,https://pubmed.ncbi.nlm.nih.gov/41041909/,Results > Evaluation of the Toxicity and Safety of IVSA‐siRNA Targeting MSTN; paragraph 28,1243,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00192,P0,toxicity,toxicity_endpoint,969,yes,A,1056,"APGS (recovered B2 from PMID:41732207, curator:CM)",ASO,,,hepatic: hepatotoxicity,3251,41732207,PMC12925542,10.1016/j.omtn.2026.102848,"Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer's disease.",https://pubmed.ncbi.nlm.nih.gov/41732207/,"Results > IC50, time course, and viability analysis; paragraph 10",1246,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00193,P0,toxicity,toxicity_endpoint,974,yes,A,994,"S9 (recovered B2 from PMID:41552385, curator:CM)",siRNA,,,hepatic: hepatotoxicity,3301,41552385,PMC12804371,10.1016/j.omtn.2025.102802,An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment.,https://pubmed.ncbi.nlm.nih.gov/41552385/,Results > S9-SNA ameliorated the phenotype of SBMA mice; paragraph 12,1251,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00194,P0,toxicity,toxicity_endpoint,1039,yes,A,1057,unspecified siRNA (PMID:40737088),siRNA,,,hepatic: hepatotoxicity,1497,40737088,PMC12309379,10.1093/nar/gkaf692,Single alkyl phosphonate modification of the siRNA backbone in the seed region enhances specificity and therapeutic profile.,https://pubmed.ncbi.nlm.nih.gov/40737088/,Results > Alkyl phosphonate at positions 6–7 enhances the therapeutic profile and reduces hepatotoxicity of Ttr and Marc1 siRNAs; paragraph 65,1316,rat,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00195,P0,toxicity,toxicity_endpoint,1043,yes,A,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hematological: thrombocytopenia,1828,40599554,PMC12208382,10.1097/CP9.0000000000000122,Rats subject to extracorporeal membrane oxygenation have improved cardiac function following anticoagulation and reversal with factor IXa aptamer-antidote oligonucleotide pair.,https://pubmed.ncbi.nlm.nih.gov/40599554/,Abstract > Results:; paragraph 3,1320,,,blood/serum,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00196,P0,toxicity,toxicity_endpoint,1057,yes,A,652,Single-dose,siRNA,,,general toxicity: toxicity,3701,40313754,PMC12045366,10.21203/rs.3.rs-6465542/v1,Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model.,https://pubmed.ncbi.nlm.nih.gov/40313754/,RESULTS > Lead MECP2-targeting siRNAs tune MECP2 levels in wildtype mice for up to four months; paragraph 16,1334,,,brain/CNS,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00197,P0,toxicity,toxicity_endpoint,1060,yes,A,652,Single-dose,siRNA,,,general safety: systemic safety/tolerability,3717,40196492,PMC11974818,10.1101/2025.03.26.645328,Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model.,https://pubmed.ncbi.nlm.nih.gov/40196492/,RESULTS > Single injection of non-isoform selective 1764_S1 but not E1 selective 112_S1 induces mixed behavioral effects in Tg1 mice; paragraph 36,1337,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00198,P0,toxicity,toxicity_endpoint,1083,yes,A,660,BHF7,siRNA,,,hepatic: hepatotoxicity,8415,40928446,PMC12790151,10.1016/j.jacbts.2025.101364,Small Interfering RNA Therapy Targeting the Long Noncoding RNA SMILR for Therapeutic Intervention in Coronary Artery Bypass Graft Failure.,https://pubmed.ncbi.nlm.nih.gov/40928446/,Methods > Lactate dehydrogenase cytotoxicity assay; paragraph 31,1360,cell line,,cell line,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00199,P0,toxicity,toxicity_endpoint,1117,yes,A,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic: hepatotoxicity,10025,40024534,PMC12022666,10.1016/j.jcmgh.2025.101485,Therapeutic Potential of STE20-Type Kinase STK25 Inhibition for the Prevention and Treatment of Metabolically Induced Hepatocellular Carcinoma.,https://pubmed.ncbi.nlm.nih.gov/40024534/,Results > Stk25-Targeting ASO Therapy Hinders the Development and Progression of MASH-Related HCC in a Mouse Model; paragraph 16,1394,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00200,P0,toxicity,toxicity_endpoint,1131,yes,A,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,renal: renal safety,12392,39940962,PMC11818693,10.3390/ijms26031196,"Cetuximab-Immunoliposomes Loaded with TGF-β1 siRNA for the Targeting Therapy of NSCLC: Design, and In Vitro and In Vivo Evaluation.",https://pubmed.ncbi.nlm.nih.gov/39940962/,2. Results > 2.7. In Vivo Anticancer Activity and Safety Evaluation; paragraph 16,1408,cell line,,kidney,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00201,P0,toxicity,toxicity_endpoint,1141,yes,A,1058,"siTMEM45A (recovered B2 from PMID:39910045, curator:CM)",siRNA,,,hepatic: hepatotoxicity,13436,39910045,PMC11799145,10.1038/s41420-025-02336-9,TMEM45A enhances palbociclib resistance and cellular glycolysis by activating AKT/mTOR signaling pathway in HR+ breast cancer.,https://pubmed.ncbi.nlm.nih.gov/39910045/,Results > Construction and characterization of the BRCA primary cell-derived engineered exosomes; paragraph 14,1418,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00202,P0,toxicity,toxicity_endpoint,1156,yes,A,1059,unspecified siRNA (PMID:38192611),siRNA,,,hepatic: hepatotoxicity,3958,38192611,PMC10772295,10.1016/j.omtn.2023.102088,Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors.,https://pubmed.ncbi.nlm.nih.gov/38192611/,Results > The structure and chemical configuration of the siRNA affects the distribution pattern in mouse retinas; paragraph 7,1433,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00203,P0,toxicity,toxicity_endpoint,1161,yes,A,231,givosiran,siRNA,,,hepatic: hepatotoxicity,10566,39597922,PMC11594983,10.3390/jcm13226779,German Real-World Experience of Patients with Diverse Features of Acute Intermittent Porphyria Treated with Givosiran.,https://pubmed.ncbi.nlm.nih.gov/39597922/,3. Results > 3.4. Safety > Adverse Events; paragraph 37,1438,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00204,P0,toxicity,toxicity_endpoint,1172,yes,A,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,10988,38979291,PMC11230209,10.1101/2024.06.20.599943,RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice.,https://pubmed.ncbi.nlm.nih.gov/38979291/,RESULTS > Extensive screening identifies a potent chemically stabilized SOD1-targeting siRNA; paragraph 11,1449,human,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00205,P0,toxicity,toxicity_endpoint,1203,yes,A,1060,unspecified ASO (PMID:37200862),ASO,,,hepatic: hepatotoxicity,1481,37200862,PMC10185704,10.1016/j.omtn.2023.04.010,Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1.,https://pubmed.ncbi.nlm.nih.gov/37200862/,Results > RNA-seq reveals activation of the interferon response by all ASOs and additional off-target effects on gene expression by the repeat gapmer; paragraph 19,1480,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00206,P0,toxicity,toxicity_endpoint,1214,yes,A,1061,"si3033 (recovered B2 from PMID:37925520, curator:CM)",siRNA,,,hepatic: hepatotoxicity,3979,37925520,PMC10625637,10.1038/s41467-023-42714-4,Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin.,https://pubmed.ncbi.nlm.nih.gov/37925520/,Results > si3033 has a good safety profile and enables JAK1 silencing in mouse skin over 5 weeks; paragraph 11,1491,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00207,P0,toxicity,toxicity_endpoint,1244,yes,A,1062,"SMN7D(-10-29) AO (recovered B2 from PMID:35464859, curator:CM)",ASO,,,hepatic: hepatotoxicity,2210,35464859,PMC9019733,10.3389/fgene.2022.791416,"Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In Vitro.",https://pubmed.ncbi.nlm.nih.gov/35464859/,"Results > PMOs, or Double-Stranded 2′ O-methyl Phosphorothioate AOs, Do Not Form Nuclear Inclusions, Protecting From Cellular Toxicity and Off-Target Transcript Effects; paragraph 53",1521,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00208,P0,toxicity,toxicity_endpoint,1247,yes,A,1063,"PC-DCA siRNA (recovered B2 from PMID:35847173, curator:CM)",siRNA,,,hepatic: hepatotoxicity,4069,35847173,PMC9263991,10.1016/j.omtn.2022.06.009,Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1.,https://pubmed.ncbi.nlm.nih.gov/35847173/,Results > Systematic chemical optimization of sFLT1-targeting siRNAs improves placental accumulation and degree of sFLT1 silencing with a better safety profile; paragraph 30,1524,,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00209,P0,toxicity,toxicity_endpoint,1257,yes,A,599,inotersen,ASO,,,hematological: thrombocytopenia,14599,35289020,PMC9314115,10.1111/ene.15325,Real-life experience with inotersen in hereditary transthyretin amyloidosis with late-onset phenotype: Data from an early-access program in Italy.,https://pubmed.ncbi.nlm.nih.gov/35289020/,RESULTS > Safety and tolerability; paragraph 21,1534,human,,blood/serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00210,P0,toxicity,toxicity_endpoint,1263,yes,A,695,Single-Stranded,ASO,,,hepatic: hepatotoxicity,1996,34037271,PMC9291828,10.1002/hep.31920,Clinical Study of Single-Stranded Oligonucleotide RO7062931 in Healthy Volunteers and Patients With Chronic Hepatitis B.,https://pubmed.ncbi.nlm.nih.gov/34037271/,Results > Safety and Tolerability; FIG. 3 caption,1540,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00211,P0,toxicity,toxicity_endpoint,1270,yes,A,435,siRNAs,siRNA,,,hepatic: hepatotoxicity,12553,34172725,PMC8233311,10.1038/s41467-021-24191-9,A smart multiantenna gene theranostic system based on the programmed assembly of hypoxia-related siRNAs.,https://pubmed.ncbi.nlm.nih.gov/34172725/,Results > In vitro cytotoxicity assay; paragraph 10,1547,cell line,,cell line,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00212,P0,toxicity,toxicity_endpoint,1293,yes,A,435,siRNAs,siRNA,,,hepatic: hepatotoxicity,12616,30535404,PMC6379714,10.1093/nar/gky1232,Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways.,https://pubmed.ncbi.nlm.nih.gov/30535404/,RESULTS > Lipid-hsiRNAs elicit sustained systemic in vivo gene silencing after a single injection > Liver; paragraph 26,1570,cell line,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00213,P0,offtarget,offtarget_evidence,3,yes,B,6,RGLS4326,ASO,,,off-target evidence,1410,41315228,PMC12663328,10.1038/s41467-025-65799-5,The nucleobase guanine at the 3'-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity.,https://pubmed.ncbi.nlm.nih.gov/41315228/,Results > RGLS4326 inhibits AMPAR ligand binding and function; paragraph 10,6,,molecular target binding panel and AMPAR assay,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00214,P0,offtarget,offtarget_evidence,12,yes,B,81,siRNA candidate from PMID 41020503,siRNA,,,seed-mediated off-target effect,2454,41020503,PMC12477598,10.1093/nar/gkaf937,Modified unlocked nucleic acid (MUNA) mitigates off-target effects of small interfering RNAs.,https://pubmed.ncbi.nlm.nih.gov/41020503/,Results > Modified UNAs mitigate off-target activity without reducing on-target activity in cell culture; paragraph 69,81,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00215,P0,offtarget,offtarget_evidence,13,yes,B,100,ASO candidate from PMID 36229039,ASO,,,hybridization/mismatch off-target effect,2306,36229039,PMC9825163,10.1093/nar/gkac838,INRI-seq enables global cell-free analysis of translation initiation and off-target effects of antisense inhibitors.,https://pubmed.ncbi.nlm.nih.gov/36229039/,RESULTS > INRI-seq identifies direct PNA off-targets; paragraph 50,100,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00216,P0,offtarget,offtarget_evidence,15,yes,B,102,siRNA candidate from PMID 29246303,siRNA,,,off-target evidence,4230,29246303,PMC5675723,10.1016/j.omtn.2017.10.003,Site-Specific Modification Using the 2'-Methoxyethyl Group Improves the Specificity and Activity of siRNAs.,https://pubmed.ncbi.nlm.nih.gov/29246303/,Results > Combination of MOE at Positions 9 and 10 with OMe at Position 14 Diminished or Eliminated Off-Target Effects; paragraph 17,102,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00217,P0,offtarget,offtarget_evidence,16,yes,B,126,siRNA candidate from PMID 41897327,siRNA,,,sequence-specificity/off-target evidence,5621,41897327,PMC13024069,10.3390/biom16030393,Selective Silencing of TDP-43 P. G376D Mutation Reverses Key Amyotrophic Lateral Sclerosis-Related Cellular Deficits.,https://pubmed.ncbi.nlm.nih.gov/41897327/,3. Results > 3.1. Allele-Specific Silencing Reduces TDP-43 Mislocalization in ALS Motor Neurons; paragraph 21,127,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00218,P0,offtarget,offtarget_evidence,144,yes,B,1064,unspecified siRNA (PMID:40142497),miRNA agomir,,,transcriptome-level off-target effect,9881,40142497,PMC11946339,10.3390/microorganisms13030604,In Vitro Transcribed Artificial Primary MicroRNA for the Inhibition of Hepatitis B Virus Gene Expression in Cultured Cells.,https://pubmed.ncbi.nlm.nih.gov/40142497/,Abstract; paragraph 1,621,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00219,P0,offtarget,offtarget_evidence,331,yes,B,778,F7-1 siRNA,siRNA,,,seed-mediated off-target effect,15845,22258146,PMC3342946,10.4161/rna.9.1.18121,Single base mismatches in the mRNA target site allow specific seed region-mediated off-target binding of siRNA targeting human coagulation factor 7.,https://pubmed.ncbi.nlm.nih.gov/22258146/,Results > Enrichment analysis of seed region matches in F7-1 off-target genes; paragraph 13,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00220,P0,offtarget,offtarget_evidence,333,yes,B,780,ATXN3-AON,ASO,,,hybridization/mismatch off-target effect,1442,41066632,PMC12581821,10.1093/hmg/ddaf153,Determining off-target effects of splice-switching antisense oligonucleotides using short read RNAseq in neuronally differentiated human induced pluripotent stem cells.,https://pubmed.ncbi.nlm.nih.gov/41066632/,Results > Hybridization-dependent off-target splicing events do not play a major role after transfection; paragraph 19,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00221,P0,offtarget,offtarget_evidence,334,yes,B,781,modified duplex,siRNA,,,seed-mediated off-target effect,1031,41994868,PMC13096799,10.1093/nar/gkag345,Acyclic serinol nucleic acid modification of siRNAs overcomes seed region mediated off-target effects while maintaining potency.,https://pubmed.ncbi.nlm.nih.gov/41994868/,Results > In vitro gene silencing of modified siRNA containing SNA modifications; paragraph 28,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00222,P0,offtarget,offtarget_evidence,337,yes,B,782,UNA-T siRNA,siRNA,,,seed-mediated off-target effect,15362,21047800,PMC3061082,10.1093/nar/gkq961,Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs.,https://pubmed.ncbi.nlm.nih.gov/21047800/,RESULTS > Global off-target analysis; paragraph 23,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00223,P0,offtarget,offtarget_evidence,338,yes,B,783,siCD46 siRNA,siRNA,,,hybridization/mismatch off-target effect,15336,18420656,PMC2396434,10.1093/nar/gkn190,Analysis of siRNA specificity on targets with double-nucleotide mismatches.,https://pubmed.ncbi.nlm.nih.gov/18420656/,RESULTS > Comparison of site-specific alterations in siRNA or target sequence; paragraph 15,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00224,P0,offtarget,offtarget_evidence,339,yes,B,784,BTK siRNA,siRNA,,,seed-mediated off-target effect,20894,16091630,PMC1184219,10.1093/nar/gki762,siRNA-mediated off-target gene silencing triggered by a 7 nt complementation.,https://pubmed.ncbi.nlm.nih.gov/16091630/,RESULTS > A 7 nt complementation between the original GRK4(O)/BTK(O) siRNAs and the hif-1α mRNA triggers the degradation of hif-1α mRNA; paragraph 15,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00225,P0,offtarget,offtarget_evidence,340,yes,B,785,GR18-3 ASO,ASO,,,hybridization/mismatch off-target effect,28684,34994962,PMC8766371,10.1007/s40291-021-00573-z,Reduction of Off-Target Effects of Gapmer Antisense Oligonucleotides by Oligonucleotide Extension.,https://pubmed.ncbi.nlm.nih.gov/34994962/,Results > Comparative Analysis of Off-Target Effects; paragraph 23,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00226,P0,offtarget,offtarget_evidence,343,yes,B,788,SOD-1,ASO,,,hybridization/mismatch off-target effect,31422,25072142,PMC4114480,10.1371/journal.pone.0101752,Defining the factors that contribute to on-target specificity of antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/25072142/,Results > Higher order structure of the SOD-1 minigene mRNA significantly influences ASO binding; paragraph 27,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00227,P0,offtarget,offtarget_evidence,344,yes,B,789,VIR-2218,siRNA,,,seed-mediated off-target effect,1411,35736224,PMC9262600,10.1093/nar/gkac539,From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization.,https://pubmed.ncbi.nlm.nih.gov/35736224/,RESULTS > Application of ESC+ design to improve the specificity and safety of ALN-HBV; paragraph 46,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00228,P0,offtarget,offtarget_evidence,345,yes,B,790,siRNA-5,siRNA,,,seed-mediated off-target effect,2058,29459660,PMC5818625,10.1038/s41467-018-02989-4,Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity.,https://pubmed.ncbi.nlm.nih.gov/29459660/,Results > siRNA off-targets are enriched for seed complementarity; paragraph 17,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00229,P0,offtarget,offtarget_evidence,346,yes,B,791,LNA-PS ASO,ASO,,,hybridization/mismatch off-target effect,2813,26338776,PMC4605310,10.1093/nar/gkv857,In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization.,https://pubmed.ncbi.nlm.nih.gov/26338776/,RESULTS > Intronic regions are markedly more susceptible to ASO-silencing than exonic sequence; paragraph 24,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00230,P0,offtarget,offtarget_evidence,348,yes,B,793,siRNA-604,siRNA,,,hybridization/mismatch off-target effect,15837,19815667,PMC2794185,10.1093/nar/gkp835,Profiling of mismatch discrimination in RNAi enabled rational design of allele-specific siRNAs.,https://pubmed.ncbi.nlm.nih.gov/19815667/,RESULTS > Rational design of allele-specific siRNA using the mismatch discrimination model as a guide; paragraph 20,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00231,P0,offtarget,offtarget_evidence,351,yes,B,796,siVIM-270,siRNA,,,seed-mediated off-target effect,1157,41467115,PMC12744844,10.1016/j.omtn.2025.102693,An siRMSD parameter of structural distortion induced by chemical modification is predictive of the off-target effect of siRNA.,https://pubmed.ncbi.nlm.nih.gov/41467115/,Results > RNAi and off-target activities of siRNAs with chemical modifications; paragraph 10,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00232,P0,offtarget,offtarget_evidence,355,yes,B,800,DM1,ASO,,,transcriptome-level off-target effect,1481,37200862,PMC10185704,10.1016/j.omtn.2023.04.010,Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1.,https://pubmed.ncbi.nlm.nih.gov/37200862/,Results > The repeat-blocking ASO and both gapmers lead to downregulation of DMPK RNA; paragraph 8,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00233,P0,offtarget,offtarget_evidence,364,yes,B,809,siCdc2,siRNA,,,off-target evidence,4230,29246303,PMC5675723,10.1016/j.omtn.2017.10.003,Site-Specific Modification Using the 2'-Methoxyethyl Group Improves the Specificity and Activity of siRNAs.,https://pubmed.ncbi.nlm.nih.gov/29246303/,Results > Combination of MOE at Positions 9 and 10 with OMe at Position 14 Diminished or Eliminated Off-Target Effects; paragraph 17,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00234,P0,offtarget,offtarget_evidence,370,yes,B,815,60 siRNA,siRNA,,,seed-mediated off-target effect,16582,24875475,PMC4081087,10.1093/nar/gku480,siPools: highly complex but accurately defined siRNA pools eliminate off-target effects.,https://pubmed.ncbi.nlm.nih.gov/24875475/,RESULTS > siPools affect global transcript levels less than single siRNAs; Figure 4. caption,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00235,P0,offtarget,offtarget_evidence,383,yes,B,826,siEGFP1,siRNA,,,seed-mediated off-target effect,15558,20453030,PMC2943616,10.1093/nar/gkq341,A screen of chemical modifications identifies position-specific modification by UNA to most potently reduce siRNA off-target effects.,https://pubmed.ncbi.nlm.nih.gov/20453030/,RESULTS > siRNA titrations reveal reduced off-targeting by siRNA UNA modification; paragraph 13,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00236,P0,offtarget,offtarget_evidence,384,yes,B,827,Ago2; Ago1,siRNA,,,seed-mediated off-target effect,15470,19767612,PMC2777430,10.1093/nar/gkp735,Off-target and a portion of target-specific siRNA mediated mRNA degradation is Ago2 'Slicer' independent and can be mediated by Ago1.,https://pubmed.ncbi.nlm.nih.gov/19767612/,RESULTS > Ago2 is not required for PIK3CB off-target and some on-target siRNA activity; paragraph 15,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00237,P0,offtarget,offtarget_evidence,386,yes,B,829,TNFR1,siRNA,,,seed-mediated off-target effect,21536,18096615,PMC2275081,10.1093/nar/gkm630,Knockdown of TNFR1 by the sense strand of an ICAM-1 siRNA: dissection of an off-target effect.,https://pubmed.ncbi.nlm.nih.gov/18096615/,RESULTS > 736 SENSE STRAND ACTIVITY IS INHIBITED BY CHEMICAL AND SEQUENCE MODIFICATION; paragraph 30,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00238,P0,offtarget,offtarget_evidence,390,yes,B,833,TS1-ASO,ASO,,,hybridization/mismatch off-target effect,1409,35801870,PMC9303313,10.1093/nar/gkac562,Identification of nucleobase chemical modifications that reduce the hepatotoxicity of gapmer antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/35801870/,RESULTS > Identification of potential nucleobase modifications that reduce LNA gapmer hepatotoxicity; Figure 2. caption,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00239,P0,offtarget,offtarget_evidence,391,yes,B,834,S10-1,ASO,,,hybridization/mismatch off-target effect,2253,36511872,PMC9825156,10.1093/nar/gkac1108,G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects.,https://pubmed.ncbi.nlm.nih.gov/36511872/,RESULTS > Indirect FXN activation is unlikely to be mediated by a mutual hybridization-dependent off-target transcript; paragraph 49,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00240,P0,offtarget,offtarget_evidence,392,yes,B,438,LNA ASO,ASO,,,hybridization/mismatch off-target effect,2062,26553810,PMC4797265,10.1093/nar/gkv1210,Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts.,https://pubmed.ncbi.nlm.nih.gov/26553810/,RESULTS > Hepatotoxicity of LNA ASOs is RNase H1 dependent; paragraph 35,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00241,P0,offtarget,offtarget_evidence,394,yes,B,836,TMEM16a,ASO,,,off-target evidence,1233,40926414,PMC12703169,10.1016/j.ymthe.2025.08.045,Restoring chloride efflux in cystic fibrosis with TMEM16a antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/40926414/,Results > TMEM16a ASO is specific and not toxic; paragraph 12,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00242,P0,offtarget,offtarget_evidence,411,yes,B,629,PS-ASO,ASO,,,hybridization/mismatch off-target effect,2060,29390093,PMC5861398,10.1093/nar/gky060,Acute hepatotoxicity of 2' fluoro-modified 5-10-5 gapmer phosphorothioate oligonucleotides in mice correlates with intracellular protein binding and the loss of DBHS proteins.,https://pubmed.ncbi.nlm.nih.gov/29390093/,RESULTS > Hybridization-dependent toxicity is not likely to account for the 2′ F PS-ASO-induced hepatotoxicity; Figure 5. caption,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00243,P0,offtarget,offtarget_evidence,414,yes,B,850,CD-1,ASO,,,off-target evidence,2063,24550163,PMC4005641,10.1093/nar/gku142,Sequence motifs associated with hepatotoxicity of locked nucleic acid--modified antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/24550163/,RESULTS > Evaluation of hepatotoxicity in mice; paragraph 14,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00244,P0,offtarget,offtarget_evidence,415,yes,B,851,WRN-108,ASO,,,hybridization/mismatch off-target effect,1055,42095135,PMC13140060,10.1016/j.omtn.2026.102930,Development of an exon 27-skipping antisense oligonucleotide as a targeted therapy for refractory skin ulcers in Werner syndrome.,https://pubmed.ncbi.nlm.nih.gov/42095135/,Results > Translational activity in non-human primates; paragraph 29,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00245,P0,offtarget,offtarget_evidence,416,yes,B,852,KCNT1,ASO,,,transcriptome-level off-target effect,1058,41981306,PMC13099374,10.1038/s41591-026-04314-9,Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy.,https://pubmed.ncbi.nlm.nih.gov/41981306/,Results > KCNT1-knockdown-ASO screening in cell lines and patient iPSC-derived neurons; paragraph 8,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00246,P0,offtarget,offtarget_evidence,417,yes,B,853,HIV-1,ASO/siRNA mixed context,,,hybridization/mismatch off-target effect,1210,41704705,PMC12907196,10.3389/fmed.2026.1719202,Investigation of the activity of phosphothioate and phosphothioate-LNA-modified oligonucleotides against HIV-1.,https://pubmed.ncbi.nlm.nih.gov/41704705/,Results > Study of antiretroviral activity; paragraph 38,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00247,P0,offtarget,offtarget_evidence,436,yes,B,869,ASO-001933,ASO,,,seed-mediated off-target effect,1615,36090761,PMC9424863,10.1016/j.omtn.2022.07.027,Identification and characterization of a MAPT-targeting locked nucleic acid antisense oligonucleotide therapeutic for tauopathies.,https://pubmed.ncbi.nlm.nih.gov/36090761/,Results > Initial tiling screen identified a parental ASO targeting the 3′ UTR of MAPT with high cross-species reactivity; paragraph 6,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00248,P0,offtarget,offtarget_evidence,441,yes,B,873,T39 ASO,ASO,,,off-target evidence,2770,32180554,PMC7036698,10.1016/j.molmet.2020.01.010,Antisense oligonucleotide treatment produces a type I interferon response that protects against diet-induced obesity.,https://pubmed.ncbi.nlm.nih.gov/32180554/,Results > ASOs targeting human mRNAs can also induce a type I IFN response in cultured THP1 cells; paragraph 31,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00249,P0,offtarget,offtarget_evidence,451,yes,B,882,IGF2BP2,siRNA,,,seed-mediated off-target effect,1732,41859644,PMC12996677,10.1016/j.mtbio.2026.102999,An exosome-biomimetic photothermal nanocarrier for IGF2BP2 siRNA delivery and enhanced ferroptosis in head and neck squamous cell carcinoma.,https://pubmed.ncbi.nlm.nih.gov/41859644/,Results > IGF2BP2 Knockdown promotes ferroptosis in HNSCC via reduced NRF2 mRNA stability; paragraph 10,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00250,P0,offtarget,offtarget_evidence,456,yes,B,886,modified siRNA,siRNA,,,hybridization/mismatch off-target effect,3301,41552385,PMC12804371,10.1016/j.omtn.2025.102802,An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment.,https://pubmed.ncbi.nlm.nih.gov/41552385/,Results > Screening of SNA-modified siRNAs targeting CAG repeats; paragraph 8,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00251,P0,offtarget,offtarget_evidence,462,yes,B,892,si098-DV29P,siRNA,,,seed-mediated off-target effect,1477,40385635,PMC12083919,10.1016/j.omtn.2025.102542,"Design, pharmacology, and toxicology of a novel chemically modified siRNA targeting hepatic angiotensinogen.",https://pubmed.ncbi.nlm.nih.gov/40385635/,Results > In vivo silencing of AGT by GalNAc-siRNAs; paragraph 13,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00252,P0,offtarget,offtarget_evidence,463,yes,B,893,YAP1; WWTR1,siRNA,,,seed-mediated off-target effect,1789,41341748,PMC12670904,10.1016/j.omtn.2025.102768,A novel bispecific siRNA concept: Efficient dual knockdown of YAP1 and WWTR1 with a single guide strand.,https://pubmed.ncbi.nlm.nih.gov/41341748/,Results > Characterization of off-target activities of bsYW-61; paragraph 14,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00253,P0,offtarget,offtarget_evidence,464,yes,B,894,ACTB ASO,ASO,,,transcriptome-level off-target effect,1824,40672232,PMC12265680,10.1101/2025.07.11.664364,Targeted RNA editing by direct delivery of an adenosine deaminase-antisense oligo conjugate.,https://pubmed.ncbi.nlm.nih.gov/40672232/,RESULTS > RNP delivery reduces transcriptome-wide off-target editing; paragraph 22,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00254,P0,offtarget,offtarget_evidence,473,yes,B,902,UU-3,siRNA,,,off-target evidence,3544,40861898,PMC12375242,10.1016/j.omtn.2025.102667,Development of conditional-siRNA programmable riboswitch for targeting adverse cardiac remodeling.,https://pubmed.ncbi.nlm.nih.gov/40861898/,Results > Synthesis of a Cond-siRNA construct based on RNA sensor Nppa and target gene calcineurin; paragraph 11,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00255,P0,offtarget,offtarget_evidence,483,yes,B,912,SMILR siRNA,siRNA,,,seed-mediated off-target effect,8415,40928446,PMC12790151,10.1016/j.jacbts.2025.101364,Small Interfering RNA Therapy Targeting the Long Noncoding RNA SMILR for Therapeutic Intervention in Coronary Artery Bypass Graft Failure.,https://pubmed.ncbi.nlm.nih.gov/40928446/,Results > Transcriptomic analyses of BHF7-transfected vSMCs demonstrates expected changes to known SMILR regulated genes without apparent toxic effect; paragraph 43,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00256,P0,offtarget,offtarget_evidence,493,yes,B,921,PH-762,siRNA,,,transcriptome-level off-target effect,10539,39697325,PMC11652358,10.3389/fimmu.2024.1501679,Self-delivering RNAi immunotherapeutic PH-762 silences PD-1 to generate local and abscopal antitumor efficacy.,https://pubmed.ncbi.nlm.nih.gov/39697325/,Results > PH-762 is a stabilized self-delivering siRNA compound designed to potently silence PDCD1 mRNA with high specificity; paragraph 10,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00257,P0,offtarget,offtarget_evidence,506,yes,B,932,siIGF1R-1,ASO,,,hybridization/mismatch off-target effect,14112,37251692,PMC10220282,10.1016/j.omtn.2023.04.028,Modified ASO conjugates encapsulated with cytidinyl/cationic lipids exhibit more potent and longer-lasting anti-HCC effects.,https://pubmed.ncbi.nlm.nih.gov/37251692/,Results > Transcriptomics and proteomics analysis of modified CT102s; paragraph 23,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00258,P0,offtarget,offtarget_evidence,510,yes,B,187,SLN360,siRNA,,,seed-mediated off-target effect,1486,35737426,PMC9516055,10.1093/toxsci/kfac067,"Preclinical Toxicological Assessment of A Novel siRNA, SLN360, Targeting Elevated Lipoprotein (a) in Cardiovascular Disease.",https://pubmed.ncbi.nlm.nih.gov/35737426/,RESULTS > SLN360 Specifically Downregulates LPA in Primary Hepatocytes; paragraph 28,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00259,P0,offtarget,offtarget_evidence,526,yes,B,881,HTT siRNA,siRNA,,,seed-mediated off-target effect,4213,31375812,PMC6879195manuscript-id: NIHMS1533065,10.1038/s41587-019-0205-0,A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.,https://pubmed.ncbi.nlm.nih.gov/31375812/,Results > Di-siRNA modulates HTT expression with minimal off-target events; paragraph 33,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00260,P0,offtarget,offtarget_evidence,530,yes,B,952,CEP290; LCA10,ASO/siRNA mixed context,,,hybridization/mismatch off-target effect,12621,30114557,PMC6092551,10.1016/j.omtn.2018.07.010,Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models.,https://pubmed.ncbi.nlm.nih.gov/30114557/,"Results > QR-110 Treatment Reduces Aberrant Splicing, Increases Wild-Type CEP290 in Homozygous Retinal Organoids, and Shows No Off-Target Pharmacology; paragraph 15",,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00261,P0,offtarget,offtarget_evidence,534,yes,B,956,89E-06,ASO,,,hybridization/mismatch off-target effect,2064,28918038,PMC5537172,10.1016/j.omtn.2017.07.003,Strategies for In Vivo Screening and Mitigation of Hepatotoxicity Associated with Antisense Drugs.,https://pubmed.ncbi.nlm.nih.gov/28918038/,Results > High Confirmation Rate of In Silico Predicted OTEs in Mouse Liver; paragraph 14,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00262,P0,toxicity,toxicity_endpoint,5,yes,B,18,BND6482,ASO,,,hepatic: hepatotoxicity,1780,41480638,PMC12754785,10.1093/narcan/zcaf053,IGF1R-targeted delivery of a bridged nucleic acid oligonucleotide-peptide conjugate for microRNA-21 inhibition in triple-negative breast cancer.,https://pubmed.ncbi.nlm.nih.gov/41480638/,Results > Liver and kidney toxicity assessment of BND6482; paragraph 61,18,mouse,EMT6 tumor-bearing female Balb/c mice treated with BND6482,serum; liver; kidney,5.0,mg/kg,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,FALSE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00263,P0,toxicity,toxicity_endpoint,6,yes,B,19,L2-Htt,siRNA,HTT,,general safety: safety/tolerability,1782,41446212,PMC12724171,10.64898/2025.12.17.693452,Evaluating the effects of aging on biodistribution and gene silencing activity of lipid-siRNA conjugates delivered into cerebrospinal fluid.,https://pubmed.ncbi.nlm.nih.gov/41446212/,Results > Safety assessments reveal L2-Htt is well tolerated in aged mice; paragraph 43,19,mouse,aged mice after CSF delivery of lipid-siRNA conjugate,cortex; serum; liver; kidney,15.0,nanomole,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,FALSE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00264,P0,toxicity,toxicity_endpoint,8,yes,B,21,anti-miR-21-SNA,ASO,MIR21,,hepatic: hepatotoxicity,1846,40178922,PMC12233840,10.34067/KID.0000000771,Ameliorative Effect of an Anti-MicroRNA-21 Oligonucleotide on Animal and Human Models of Cystic Kidney Disease.,https://pubmed.ncbi.nlm.nih.gov/40178922/,Results > Anti–miR-21-SNA Treatment Inhibits Kidney Cyst Growth; paragraph 32,21,mouse,pcy cystic kidney mouse model after systemic anti-miR-21-SNA administration,blood; kidney,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00265,P0,toxicity,toxicity_endpoint,16,yes,B,30,JNJ-3989/JNJ-73763989 GalNAc-siRNA regimen from PMID 40919077,siRNA,HBV,,general safety: safety/tolerability,3517,40919077,PMC12409438,10.1016/j.jhepr.2025.101516,Peginterferon-alpha-2a add-on to treatment with siRNA JNJ-73763989 in virologically suppressed chronic hepatitis B: The phase II PENGUIN study.,https://pubmed.ncbi.nlm.nih.gov/40919077/,Results > Safety; paragraph 22,30,human,,chronic hepatitis D participants,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00266,P0,toxicity,toxicity_endpoint,17,yes,B,31,scFv-MML@LPCAT1si from PMID 40898177,siRNA,LPCAT1,,general safety: systemic safety,3529,40898177,PMC12406594,10.1186/s12896-025-01000-5,Biomimetic nanoparticles coated with ScFv-modified macrophage membranes for siRNA delivery to relieve brain metastases of lung cancer.,https://pubmed.ncbi.nlm.nih.gov/40898177/,Results > In vivo safety access; paragraph 30,31,mouse,,heart; liver; spleen; lung; kidney; brain,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00267,P0,toxicity,toxicity_endpoint,19,yes,B,33,EM/siKEAP1@Fe-MOF@HA from PMID 40688667,siRNA,KEAP1,,general safety: systemic safety,3590,40688667,PMC12274865,10.1016/j.mtbio.2025.102074,Hyaluronic acid-anchored nanoparticles co-delivering emodin and siRNA confers protection against rheumatoid arthritis via macrophage polarization.,https://pubmed.ncbi.nlm.nih.gov/40688667/,Results > Assessment of biosafety in vivo; paragraph 42,33,mouse,,major organs,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00268,P0,toxicity,toxicity_endpoint,21,yes,B,35,hEV@FAP-siTGFbeta1 from PMID 40046817,siRNA,TGFB1,,general safety: systemic safety,3740,40046817,PMC11881639,10.2147/IJN.S497428,Injured Cardiac Tissue-Targeted Delivery of TGFβ1 siRNA by FAP Aptamer-Functionalized Extracellular Vesicles Promotes Cardiac Repair.,https://pubmed.ncbi.nlm.nih.gov/40046817/,Results > In vivo Safety of hEV@FAP-siTGFβ1; paragraph 42,35,mouse,,heart; liver; spleen; lung; kidney,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00269,P0,toxicity,toxicity_endpoint,22,yes,B,36,siAURKA-loaded milk extracellular vesicles from PMID 40896585,siRNA,AURKA,,immunotoxicity: immune activation,8490,40896585,PMC12396431,10.1016/j.omtn.2025.102644,RNAi delivery mediated by milk extracellular vesicles in colon cancer.,https://pubmed.ncbi.nlm.nih.gov/40896585/,Results > Safety and immune-response of siRNA-loaded mEVs; paragraph 15,36,human cell line; human PBMC,,CACO-2; NMC 460D; PBMC,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00270,P0,toxicity,toxicity_endpoint,23,yes,B,37,BSCgal gold-siRNA supracluster from PMID 39448881,siRNA,LGALS1,,general safety: systemic safety,12426,39448881,PMC12018592manuscript-id: NIHMS2046510,10.1038/s41587-024-02448-0,Gold-siRNA supraclusters enhance the anti-tumor immune response of stereotactic ablative radiotherapy at primary and metastatic tumors.,https://pubmed.ncbi.nlm.nih.gov/39448881/,Results > In vivo clearance and traceable delivery of supraclusters; paragraph 9,37,mouse,,major organs; serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00271,P0,toxicity,toxicity_endpoint,27,yes,B,41,LNP-siTGFbetaRI from PMID 39264964,siRNA,TGFBR1,,general safety: systemic safety,1886,39264964,PMC11421824,10.1371/journal.pntd.0012502,Development of a Lipid-encapsulated TGFβRI-siRNA Drug for Liver Fibrosis Induced by Schistosoma mansoni.,https://pubmed.ncbi.nlm.nih.gov/39264964/,3. Results > 3.7. In vivo toxicity of LNP-siTGFβRI; paragraph 36,41,mouse,,major organs,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00272,P0,toxicity,toxicity_endpoint,34,yes,B,48,OligoA-11/Skeen-11 phosphorothioate oligonucleotide panel from PMID 37185731,ASO,G6PD,,hepatic; renal: hepatic and renal toxicity,1138,37185731,PMC10137061,10.3390/cimb45040207,Inhibitory Effect of Phosphorothioate Oligonucleotide Complementary to G6PD mRNA on Murine Melanoma.,https://pubmed.ncbi.nlm.nih.gov/37185731/,2. Results; paragraph 20,48,mouse,,liver; kidney,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00273,P0,toxicity,toxicity_endpoint,35,yes,B,49,miR-125a-5p agomir from PMID 36632217,miRNA agomir,MIR125A,,general safety: cardiac and systemic safety,1960,36632217,PMC9830430,10.7150/thno.73568,Therapeutic delivery of microRNA-125a-5p oligonucleotides improves recovery from myocardial ischemia/reperfusion injury in mice and swine.,https://pubmed.ncbi.nlm.nih.gov/36632217/,Results > Myocardial I/R swine receiving miR-125a-5p agomir show no alterations in arrhythmic occurrences and serum chemistries; paragraph 23,49,swine,,heart; serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00274,P0,toxicity,toxicity_endpoint,36,yes,B,50,CLCN7G215R-siRNA-SiS12 formulation from PMID 37680985,siRNA,CLCN7,,general safety: systemic safety,2657,37680985,PMC10480457,10.1016/j.omtn.2023.08.020,Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. Implications for gene therapy in humans.,https://pubmed.ncbi.nlm.nih.gov/37680985/,Results > Safety analysis; paragraph 31,50,mouse,,major organs; serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00275,P0,toxicity,toxicity_endpoint,37,yes,B,51,RBCEV-delivered siMstn and siMlycd from PMID 37016578,siRNA,MSTN; MLYCD,,immunotoxicity: immune and systemic safety,4022,37016578,PMC10188904,10.1016/j.ymthe.2023.03.036,Red blood cell extracellular vesicles deliver therapeutic siRNAs to skeletal muscles for treatment of cancer cachexia.,https://pubmed.ncbi.nlm.nih.gov/37016578/,Results > Intramuscular delivery of siRNAs using RBCEVs is non-toxic and non-inflammatory; paragraph 22,51,mouse,,muscle; serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00276,P0,toxicity,toxicity_endpoint,41,yes,B,55,DLin-MC3-DMA-conjugated siRNA panel from PMID 35917865,siRNA,HTT; PPIB,,general safety: transcriptome toxicity,1427,35917865,PMC10281028manuscript-id: NIHMS1882446,10.1016/j.jconrel.2022.07.041,Engineered ionizable lipid siRNA conjugates enhance endosomal escape but induce toxicity in vivo.,https://pubmed.ncbi.nlm.nih.gov/35917865/,Results > DLin-MC3-DMA conjugate induces non-specific modulation of gene expression in several tissues; paragraph 35,55,mouse,,heart; fat; muscle; liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00277,P0,toxicity,toxicity_endpoint,47,yes,B,61,CYP3A5 PMO/PSO panel from PMID 33633318,ASO,CYP3A5,,renal: renal safety,1471,33633318,PMC7907328,10.1038/s41598-021-84194-w,Antisense oligonucleotide development for the selective modulation of CYP3A5 in renal disease.,https://pubmed.ncbi.nlm.nih.gov/33633318/,Results; paragraph 14,61,human cell model,,primary human proximal tubule epithelial cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00278,P0,toxicity,toxicity_endpoint,51,yes,B,65,LNPssPalmO-Phe-ASOap/ASOgr from PMID 33924589,ASO,APOB; NR3C1,,hepatic: hepatotoxicity,1999,33924589,PMC8070490,10.3390/pharmaceutics13040544,Delivery of Oligonucleotides Using a Self-Degradable Lipid-Like Material.,https://pubmed.ncbi.nlm.nih.gov/33924589/,3. Results > 3.3. Application for the Delivery of ASO > 3.3.2. Comparison of Hepatotoxicity between ASO and LNPssPalmO-Phe–ASO; paragraph 24,65,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00279,P0,toxicity,toxicity_endpoint,54,yes,B,68,PMO-P6 conjugate from PMID 34841414,ASO,DMD,,renal: renal safety,4112,34841414,PMC8611673,10.1021/jacsau.1c00327,Deep Learning Enables Discovery of a Short Nuclear Targeting Peptide for Efficient Delivery of Antisense Oligomers.,https://pubmed.ncbi.nlm.nih.gov/34841414/,Results > In Vivo Studies; paragraph 30,68,mouse,,kidney,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00280,P0,toxicity,toxicity_endpoint,55,yes,B,69,PM1(GT/siMMP-9) hydrogel from PMID 33952251,siRNA,MMP9,,general safety: local and systemic safety,4145,33952251,PMC8097905,10.1186/s12951-021-00869-6,Sustained delivery of MMP-9 siRNA via thermosensitive hydrogel accelerates diabetic wound healing.,https://pubmed.ncbi.nlm.nih.gov/33952251/,Results > In vivo biocompatibility of PM1(GT/siMMP-910) hydrogel; paragraph 60,69,rat; human cell line,,HaCaT; wound tissue,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00281,P0,toxicity,toxicity_endpoint,56,yes,B,70,stereocontrolled cEt gapmer ASO panel from PMID 31980820,ASO,MALAT1,,general safety: cytotoxicity,1423,31980820,PMC7038945,10.1093/nar/gkaa031,Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/31980820/,RESULTS > Biological evaluation of fully Rp and Sp gap modified ASOs; paragraph 18,70,mouse cell line; mouse,,liver; cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00282,P0,toxicity,toxicity_endpoint,65,yes,B,79,PMO-GF formulation from PMID 26964641,ASO,DMD,,general safety: systemic safety,4241,26964641,PMC4793046,10.1038/ncomms10981,Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice.,https://pubmed.ncbi.nlm.nih.gov/26964641/,Results > PMO-GF yields long-term efficacy and functional improvement; paragraph 8,79,mouse,,muscle; serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00283,P0,toxicity,toxicity_endpoint,69,yes,B,84,siRNA candidate from PMID 41716677,siRNA,,,immunotoxicity: immune activation,6238,41716677,PMC12914854,10.1016/j.bioactmat.2026.02.009,Spatiotemporal reprogramming of cardiac lipid metabolism by platelet-engineered RNA therapy epigenetically modulate heart repair and regeneration.,https://pubmed.ncbi.nlm.nih.gov/41716677/,Results > Platelet-mediated siRNA delivery exhibits a favorable safety profile; paragraph 32,84,swine,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00284,P0,toxicity,toxicity_endpoint,72,yes,B,87,siRNA candidate from PMID 40825982,siRNA,,,renal: renal safety,1815,40825982,PMC12361575,10.1038/s41467-025-63037-6,Targeting intestinal inflammation using locked nucleic acids delivered via lipid nanoparticles.,https://pubmed.ncbi.nlm.nih.gov/40825982/,Results > LNPs biodistribution to the inflamed colon; paragraph 17,87,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00285,P0,toxicity,toxicity_endpoint,76,yes,B,91,siRNA candidate from PMID 40503175,siRNA,,,hepatic: hepatotoxicity,9324,40503175,PMC12156227,10.1016/j.omtn.2025.102567,Targeting CIDEB alleviates liver steatosis and fibrosis in mouse MASH models.,https://pubmed.ncbi.nlm.nih.gov/40503175/,Results > Therapeutic potential of GalNAc-siCIDEB in MASH; paragraph 16,91,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00286,P0,toxicity,toxicity_endpoint,81,yes,B,96,ASO candidate from PMID 28853853,ASO,,,hepatic: hepatotoxicity,2231,28853853,PMC5694563manuscript-id: NIHMS917707,10.1021/acschembio.7b00416,BNA(NC) Gapmers Revert Splicing and Reduce RNA Foci with Low Toxicity in Myotonic Dystrophy Cells.,https://pubmed.ncbi.nlm.nih.gov/28853853/,article body; paragraph 5,96,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00287,P0,toxicity,toxicity_endpoint,85,yes,B,103,ASO candidate from PMID 41978261,ASO,,,immunotoxicity: immune activation,1043,41978261,PMC13076217,10.1093/nar/gkag311,Phosphorothioate antisense oligonucleotide induced innate immune activation is attenuated by tryptophan oxidation products.,https://pubmed.ncbi.nlm.nih.gov/41978261/,Results > Kinetics of cytokines released in response to PS ASOs vary according to sequence and chemistry; paragraph 30,103,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00288,P0,toxicity,toxicity_endpoint,86,yes,B,104,SRN001,siRNA,,,hepatic: hepatotoxicity,1149,42052104,PMC13112037,10.2147/DDDT.S589997,"Safety and Pharmacokinetics of SRN001, a Novel siRNA Drug Targeting Amphiregulin via the SAMiRNA Platform.",https://pubmed.ncbi.nlm.nih.gov/42052104/,Results > Safety Assessment; paragraph 23,104,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00289,P0,toxicity,toxicity_endpoint,88,yes,B,106,ASO candidate from PMID 39850319,ASO,,,renal: renal safety,1041,39850319,PMC11754010,10.1016/j.omtn.2024.102387,In vivo efficacy and safety of systemically administered serinol nucleic acid-modified antisense oligonucleotides in mouse kidney.,https://pubmed.ncbi.nlm.nih.gov/39850319/,Results > SGLT2-SNA2-ASO suppressed renal SGLT2 expression without severe liver and renal tubular damage; paragraph 12,106,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00290,P0,toxicity,toxicity_endpoint,89,yes,B,107,ASO candidate from PMID 40243849,ASO,,,renal: renal safety,1262,40243849,PMC11988723,10.3390/ijms26073098,Inhibiting Myostatin Expression by the Antisense Oligonucleotides Improves Muscle Wasting in a Chronic Kidney Disease Mouse Model.,https://pubmed.ncbi.nlm.nih.gov/40243849/,2. Results > 2.4. MSTN-ASO Treatment Does Not Affect Kidney Functions; paragraph 15,107,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00291,P0,toxicity,toxicity_endpoint,90,yes,B,108,TLR9-targeted; STAT3; PD1-targeting ASO from PMID 40214214,ASO,,,immunotoxicity: immune activation,1843,40214214,PMC12526143,10.1093/neuonc/noaf099,Multimodal glioma immunotherapy combining TLR9-targeted STAT3 antisense oligodeoxynucleotides with PD1 immune checkpoint blockade.,https://pubmed.ncbi.nlm.nih.gov/40214214/,Results > Reduced IFN-I-Related Neurotoxicities after Intracranial Administration of CpG-STAT3dsASO; paragraph 29,108,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00292,P0,toxicity,toxicity_endpoint,93,yes,B,111,siRNA candidate from PMID 41360814,siRNA,,,renal: renal safety,3423,41360814,PMC12804779,10.1038/s41467-025-67183-9,Inflammation-specific DNA origami nanodevice for delivery of siRNAs to treat ulcerative colitis.,https://pubmed.ncbi.nlm.nih.gov/41360814/,Results > Biodistribution of the nanodevice; paragraph 11,111,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00293,P0,toxicity,toxicity_endpoint,94,yes,B,112,CD11b-modified-targeting siRNA from PMID 40977834,siRNA,,,renal: renal safety,3501,40977834,PMC12446780,10.1016/j.mtbio.2025.102226,CD11b-modified ROS/pH-responsive nanoparticles co-deliver Dioscin and siRNA to improve cardiac repair after myocardial infarction by reducing neutrophil recruitment.,https://pubmed.ncbi.nlm.nih.gov/40977834/,Results > In vivo biosafety assessment; paragraph 50,112,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00294,P0,toxicity,toxicity_endpoint,95,yes,B,113,siRNA candidate from PMID 40380202,siRNA,,,immunotoxicity: immune activation,3685,40380202,PMC12082952,10.1186/s13046-025-03396-6,A novel pH-sensitive nanoparticles encapsulating anti-PD-1 antibody and MDK-siRNA overcome immune checkpoint blockade resistance in HCC via reshaping immunosuppressive TME.,https://pubmed.ncbi.nlm.nih.gov/40380202/,Methods > Biosafety of nanodrug; paragraph 27,113,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00295,P0,toxicity,toxicity_endpoint,97,yes,B,115,siRNA candidate from PMID 39606561,siRNA,,,renal: renal safety,12439,39606561,PMC11598607,10.2147/IJN.S492363,A Multifunctional Nanodrug Co-Delivering VEGF-siRNA and Dexamethasone for Synergistic Therapy in Ocular Neovascular Diseases.,https://pubmed.ncbi.nlm.nih.gov/39606561/,Results > Biocompatibility of MPDA Nanodrugs; paragraph 43,115,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00296,P0,toxicity,toxicity_endpoint,99,yes,B,117,siRNA candidate from PMID 38089931,siRNA,,,hematologic: platelet/bleeding safety,1117,38089931,PMC10711485,10.1016/j.omtn.2023.102080,"Dendritic amphiphilic siRNA: Selective albumin binding, in vivo efficacy, and low toxicity.",https://pubmed.ncbi.nlm.nih.gov/38089931/,Results > D-siRNA has a better safety profile; paragraph 15,117,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00297,P0,toxicity,toxicity_endpoint,101,yes,B,119,ASO candidate from PMID 37346977,ASO,,,neurological: neurotoxicity,1479,37346977,PMC10280097,10.1016/j.omtn.2023.05.005,Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection.,https://pubmed.ncbi.nlm.nih.gov/37346977/,Results > LNPs increase TA2 potency in vitro by decreasing lysosomal trafficking or increasing ASO uptake; paragraph 11,119,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00298,P0,toxicity,toxicity_endpoint,102,yes,B,120,siRNA candidate from PMID 38168301,siRNA,,,renal: renal safety,3970,38168301,PMC10760232,10.21203/rs.3.rs-3713164/v1,Evaluation of mAb 2C5-modified dendrimer-based micelles for the co-delivery of siRNA and chemotherapeutic drug in xenograft mice model.,https://pubmed.ncbi.nlm.nih.gov/38168301/,Results > Gender differences; paragraph 25,120,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00299,P0,toxicity,toxicity_endpoint,105,yes,B,122,ASO candidate from PMID 33567808,ASO,,,hematologic: platelet/bleeding safety,2730,33567808,PMC8804562,10.3324/haematol.2020.260059,"Sequence-specific 2'-O-methoxyethyl antisense oligonucleotides activate human platelets through glycoprotein VI, triggering formation of platelet-leukocyte aggregates.",https://pubmed.ncbi.nlm.nih.gov/33567808/,Results > 2’MOE ASO (104838 and 501861) and CpG ASO increase platelet P-selectin expression in platelet-rich plasma and whole blood; Figure 1. caption,123,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00300,P0,toxicity,toxicity_endpoint,106,yes,B,123,siRNA candidate from PMID 35992044,siRNA,,,hepatic: hepatotoxicity,12550,35992044,PMC9384066,10.1016/j.omtn.2022.08.011,A model system for antiviral siRNA therapeutics using exosome-based delivery.,https://pubmed.ncbi.nlm.nih.gov/35992044/,Results > Delivery of antiviral antigens simultaneously in vitro; paragraph 22,124,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00301,P0,toxicity,toxicity_endpoint,107,yes,B,124,siRNA candidate from PMID 30544191,siRNA,,,renal: renal safety,12615,30544191,PMC6379722,10.1093/nar/gky1239,Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo.,https://pubmed.ncbi.nlm.nih.gov/30544191/,RESULTS > The chemical nature of the lipid conjugate defines the liver/kidney distribution profile; paragraph 29,125,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00302,P0,toxicity,toxicity_endpoint,112,yes,B,131,DNA/RNA heteroduplex oligonucleotide candidate from PMID 39252874,DNA/RNA heteroduplex oligonucleotide,,,immunotoxicity: immune activation,10778,39252874,PMC11382116,10.1016/j.omtn.2024.102289,DNA/RNA heteroduplex technology with cationic oligopeptide reduces class-related adverse effects of nucleic acid drugs.,https://pubmed.ncbi.nlm.nih.gov/39252874/,Results > The effect of HDO formation on class-related toxicities; paragraph 7,133,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00303,P0,toxicity,toxicity_endpoint,117,yes,B,144,siRNA candidate from PMID 41949334,siRNA,,,hepatic: hepatotoxicity,1059,41949334,PMC13059674,10.1111/cts.70536,A Long-Term Human Liver Spheroid Model for Assessing Silencing and Durability of GalNAc-Conjugated siRNAs.,https://pubmed.ncbi.nlm.nih.gov/41949334/,Results > Treatment of Hepatic Spheroids With Chemically Modified GalNAc‐Conjugated siRNA; FIGURE 2 caption,148,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00304,P0,toxicity,toxicity_endpoint,136,yes,B,163,Fitusiran,ASO,,,hepatic: hepatotoxicity,2538,39642315,PMC11914172,10.1182/bloodadvances.2024013900,"Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B.",https://pubmed.ncbi.nlm.nih.gov/39642315/,Results > Safety; paragraph 20,167,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00305,P0,toxicity,toxicity_endpoint,159,yes,B,182,ASO candidate from PMID 37920238,ASO,,,neurological: neurotoxicity,1164,37920238,PMC10618110,10.1016/j.omtm.2023.101122,Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model.,https://pubmed.ncbi.nlm.nih.gov/37920238/,Materials and methods > Lateral i.c.v. catheter and reservoir implantation through new anchoring system; paragraph 37,190,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00306,P0,toxicity,toxicity_endpoint,176,yes,B,197,Foxp3,ASO,,,hepatic: hepatotoxicity,2257,33801683,PMC8037512,10.3390/ijms22073470,Foxp3 Silencing with Antisense Oligonucleotide Improves Immunogenicity of an Adjuvanted Recombinant Vaccine against Sporothrix schenckii.,https://pubmed.ncbi.nlm.nih.gov/33801683/,Abstract; paragraph 1,207,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00307,P0,toxicity,toxicity_endpoint,177,yes,B,198,Revusiran,siRNA,,,hepatic: hepatotoxicity,1430,31821125,PMC6987735,10.1089/nat.2019.0796,"Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis.",https://pubmed.ncbi.nlm.nih.gov/31821125/,Abstract; paragraph 1,208,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00308,P0,toxicity,toxicity_endpoint,184,yes,B,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,4234,28633548,PMC8241187,10.1080/10717544.2017.1337827,Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice.,https://pubmed.ncbi.nlm.nih.gov/28633548/,Abstract; paragraph 1,215,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00309,P0,toxicity,toxicity_endpoint,188,yes,B,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,4259,24405395,PMC4028087,10.1089/hum.2013.156,Evaluation of Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked polyethylenimine as antisense morpholino oligomer delivery vehicle in cell culture and dystrophic mdx mice.,https://pubmed.ncbi.nlm.nih.gov/24405395/,Abstract; paragraph 1,219,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00310,P0,toxicity,toxicity_endpoint,209,yes,B,231,givosiran,siRNA,,,hepatic: hepatotoxicity,2503,40312531,PMC12045942,10.1038/s41598-025-99526-3,Efficacy and safety of givosiran in Japanese patients with acute hepatic porphyria: clinical findings from an expanded access study.,https://pubmed.ncbi.nlm.nih.gov/40312531/,Abstract; paragraph 1,252,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00311,P0,toxicity,toxicity_endpoint,230,yes,B,245,PF4,ASO,,,renal: renal safety,2680,37111598,PMC10143489,10.3390/pharmaceutics15041112,"Platelet Activation by Antisense Oligonucleotides (ASOs) in the Göttingen Minipig, including an Evaluation of Glycoprotein VI (GPVI) and Platelet Factor 4 (PF4) Ontogeny.",https://pubmed.ncbi.nlm.nih.gov/37111598/,Abstract; paragraph 1,273,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00312,P0,toxicity,toxicity_endpoint,243,yes,B,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,2329,23952551,PMC3760025,10.1089/nat.2013.0436,Hepatotoxic potential of therapeutic oligonucleotides can be predicted from their sequence and modification pattern.,https://pubmed.ncbi.nlm.nih.gov/23952551/,Abstract; paragraph 1,286,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00313,P0,toxicity,toxicity_endpoint,418,yes,B,374,AVI-6002/AVI-6003 PMOplus,PMOplus,,,general safety: clinical safety and pharmacokinetics,2822,25155593,PMC4249403,10.1128/AAC.03442-14,Safety and pharmacokinetic profiles of phosphorodiamidate morpholino oligomers with activity against ebola virus and marburg virus: results of two single-ascending-dose studies.,https://pubmed.ncbi.nlm.nih.gov/25155593/,Abstract; paragraph 1,477,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00314,P0,toxicity,toxicity_endpoint,437,yes,B,393,LNP-SNORA49 RNA/SOX9 ASO,ASO/RNA mixed context,SOX9,,general safety: organ morphology/body weight/cytokine safety,6668,41565615,PMC12824223,10.1038/s41467-025-66486-1,SNORA49 negatively regulates self-renewal of liver cancer stem cells and hepatocarcinogenesis via suppressing SOX9 transcription.,https://pubmed.ncbi.nlm.nih.gov/41565615/,Results > Lipid nanoparticle delivery of SNORA49 and ASOs against SOX9 exerts synergistic antitumor effect on HCC tumors; paragraph 25,496,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00315,P0,toxicity,toxicity_endpoint,442,yes,B,398,ISIS 405879,ASO,,,hematological: thrombocytopenia,1131,40101742,PMC12043408manuscript-id: NIHMS2042395,10.1093/jimmun/vkae055,Cellular immune changes during severe antisense oligonucleotide-associated thrombocytopenia in a nonhuman primate model.,https://pubmed.ncbi.nlm.nih.gov/40101742/,Abstract; paragraph 1,501,cynomolgus monkey,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00316,P0,toxicity,toxicity_endpoint,448,yes,B,404,P-HyOMe-WRAP5:siF3 nanoparticles,siRNA,FADD,,general safety: cell viability/cytotoxicity,9935,40084851,PMC12132915,10.1002/cmdc.202400885,Enhancing WRAP-Based Nanoparticles for Small Interfering Ribonucleic Acid Delivery in pH-Sensitive Environments.,https://pubmed.ncbi.nlm.nih.gov/40084851/,Results and Discussion > FADD Knockdown in Human Cardiomyocytes Using PEGylated pH-Sensitive WRAP5 Nanoparticles at pH 5; paragraph 53,507,human cell line,,hiPSC-derived cardiomyocytes; EA.hy926 endothelial cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00317,P0,toxicity,toxicity_endpoint,460,yes,B,416,r/si-ORF7 flexible nano-liposomes,siRNA,ORF7,,general safety: cell viability/cytotoxicity,12511,37700336,PMC10496174,10.1186/s13578-023-01108-1,A novel recombinant ORF7-siRNA delivered by flexible nano-liposomes inhibits varicella zoster virus infection.,https://pubmed.ncbi.nlm.nih.gov/37700336/,Results > Preparation and characterization of flexible nano-liposomes; paragraph 18,519,human cell line,,ARPE-19,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00318,P0,toxicity,toxicity_endpoint,512,yes,B,1066,"elsunersen (recovered B2 from PMID:40263630, curator:CM)",ASO,,,hepatic: hepatotoxicity,1013,40263630,PMC12283366,10.1038/s41591-025-03656-0,Antisense oligonucleotide treatment in a preterm infant with early-onset SCN2A developmental and epileptic encephalopathy.,https://pubmed.ncbi.nlm.nih.gov/40263630/,Methods > Patient eligibility; paragraph 16,789,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00319,P0,toxicity,toxicity_endpoint,516,yes,B,521,single-patient,ASO,,,hepatic: hepatotoxicity,1254,40414239,PMC12407188manuscript-id: NIHMS2086788embargo-date: 2026/06/07,10.1016/S0140-6736(25)00513-6,"Antisense oligonucleotide jacifusen for FUS-ALS: an investigator-initiated, multicentre, open-label case series.",https://pubmed.ncbi.nlm.nih.gov/40414239/,Abstract > Methods; paragraph 2,793,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00320,P0,toxicity,toxicity_endpoint,527,yes,B,1067,"STP705 (recovered B2 from PMID:39692702, curator:CM)",siRNA,,,hepatic: hepatotoxicity,2535,39692702,PMC11845920,10.1111/jocd.16722,A Novel Injectable Polypeptide Nanoparticle Encapsulated siRNA Targeting TGF-β1 and COX-2 for Localized Fat Reduction II: Phase I Clinical Trial.,https://pubmed.ncbi.nlm.nih.gov/39692702/,Abstract > Methods; paragraph 3,804,,,,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00321,P0,toxicity,toxicity_endpoint,538,yes,B,1068,unspecified ASO (PMID:40736236),ASO,,,general safety: cell viability/cytotoxicity,3573,40736236,PMC12403870,10.1128/spectrum.02622-24,An antisense peptide-conjugated peptide nucleic acid (PPNA) for peptidoglycan recycling inhibition reduces AmpC hyperproduction and β-lactam resistance in Pseudomonas aeruginosa.,https://pubmed.ncbi.nlm.nih.gov/40736236/,OBSERVATION; Fig 1 caption,815,cell line,,lung,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00322,P0,toxicity,toxicity_endpoint,565,yes,B,1069,"PUMCH-E13 (recovered B2 from PMID:40970667, curator:CM)",ASO,,,hepatic: hepatotoxicity,8330,40970667,PMC12453064,10.1167/iovs.66.12.46,Exon Skipping Therapy Restores Ciliary Function in USH2A-Related Retinal Degeneration.,https://pubmed.ncbi.nlm.nih.gov/40970667/,article body; paragraph 6,842,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00323,P0,toxicity,toxicity_endpoint,568,yes,B,1070,unspecified siRNA (PMID:40848064),siRNA,,,general safety: cell viability/cytotoxicity,8603,40848064,PMC12374930,10.1007/s00262-025-04148-3,"Heat up, silence on: IDO1 gene silencing in THP-1-derived dendritic cells triggered by magnetic hyperthermia.",https://pubmed.ncbi.nlm.nih.gov/40848064/,Materials and methods > Cell viability assay; paragraph 25,845,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00324,P0,toxicity,toxicity_endpoint,574,yes,B,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic: hepatotoxicity,9180,40574101,PMC12196915,10.3390/pharmaceutics17060789,Intravenous Administration of sRNA Nanoparticles for Treatment of Osteoporosis in Mice.,https://pubmed.ncbi.nlm.nih.gov/40574101/,Abstract; paragraph 1,851,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00325,P0,toxicity,toxicity_endpoint,586,yes,B,1071,unspecified siRNA (PMID:40207309),siRNA,,,general toxicity: toxicity,9792,40207309,PMC11980806,10.2147/IJN.S501561,Si-ITGA6-Loaded Liposomes Inhibit Capsule Fibrosis via the FAK/PI3K/Akt Signaling Pathway in Adhesive Capsulitis of Shoulder.,https://pubmed.ncbi.nlm.nih.gov/40207309/,Abstract > Methods; paragraph 2,863,human,,,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00326,P0,toxicity,toxicity_endpoint,603,yes,B,1072,unspecified ASO (PMID:38276948),ASO,,,hepatic: hepatotoxicity,2618,38276948,PMC11221813,10.1152/ajpheart.00745.2023,An antisense oligonucleotide efficiently suppresses splicing of an alternative exon in vascular smooth muscle in vivo.,https://pubmed.ncbi.nlm.nih.gov/38276948/,Abstract; paragraph 1,880,,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00327,P0,toxicity,toxicity_endpoint,605,yes,B,1073,unspecified siRNA (PMID:39296267),siRNA,,,hepatic: hepatotoxicity,3848,39296267,PMC11406681,10.1021/acsptsci.4c00336,Lipopolymer/siRNA Nanoparticles Targeting the Signal Transducer and Activator of Transcription 5A Disrupts Proliferation of Acute Lymphoblastic Leukemia.,https://pubmed.ncbi.nlm.nih.gov/39296267/,Abstract; paragraph 1,882,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00328,P0,toxicity,toxicity_endpoint,609,yes,B,1074,unspecified siRNA (PMID:38613388),ASO,,,renal: renal safety,3913,38613388,PMC11109979,10.1093/nar/gkae260,Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo.,https://pubmed.ncbi.nlm.nih.gov/38613388/,Materials and methods > Animal experiments; paragraph 12,886,mouse,,kidney,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00329,P0,toxicity,toxicity_endpoint,634,yes,B,1075,"siIRF1 (recovered B2 from PMID:39629104, curator:CM)",siRNA,,,general safety: cell viability/cytotoxicity,13678,39629104,PMC11614587,10.2147/IJN.S487598,T Lymphocyte-Macrophage Hybrid Membrane-Coated Biomimetic Nanoparticles Alleviate Myocarditis via Suppressing Pyroptosis by Targeting Gene Silencing.,https://pubmed.ncbi.nlm.nih.gov/39629104/,Abstract > Methods; paragraph 2,911,mouse,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00330,P0,toxicity,toxicity_endpoint,641,yes,B,494,unspecified ASO,ASO,,,hepatic: hepatotoxicity,1955,36798116,PMC9927222,10.3389/fimmu.2023.1006741,"The exon-skipping oligonucleotide, KitStop, depletes tissue-resident mast cells in vivo to ameliorate anaphylaxis.",https://pubmed.ncbi.nlm.nih.gov/36798116/,Abstract > Methods; paragraph 2,918,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00331,P0,toxicity,toxicity_endpoint,651,yes,B,557,single-chain,ASO,,,renal: renal safety,13975,37692001,PMC10483903,10.4103/JAPTR.JAPTR_117_23,Preventive treatment of coronavirus disease-2019 virus using coronavirus disease-2019-receptor-binding domain 1C aptamer by suppress the expression of angiotensin-converting enzyme 2 receptor.,https://pubmed.ncbi.nlm.nih.gov/37692001/,Abstract; paragraph 1,928,cell line,,kidney,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00332,P0,toxicity,toxicity_endpoint,657,yes,B,559,single-ascending,ASO,,,hepatic: hepatotoxicity,14202,37002654,PMC10175964,10.1111/cts.13498,"A multi-purpose Japanese phase I study in the global development of vupanorsen: Randomized, placebo-controlled, single-ascending dose study in adults.",https://pubmed.ncbi.nlm.nih.gov/37002654/,Abstract; paragraph 1,934,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00333,P0,toxicity,toxicity_endpoint,678,yes,B,563,Site-specific,ASO,,,hepatic: hepatotoxicity,1638,34141070,PMC8201499,10.1021/acsmedchemlett.1c00072,"Site-specific Incorporation of 2',5'-Linked Nucleic Acids Enhances Therapeutic Profile of Antisense Oligonucleotides.",https://pubmed.ncbi.nlm.nih.gov/34141070/,Abstract; paragraph 1,955,,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00334,P0,toxicity,toxicity_endpoint,687,yes,B,1076,"MRCV-19 (recovered B2 from PMID:34440222, curator:CM)",PMO,,,hepatic: hepatotoxicity,14753,34440222,PMC8394971,10.3390/biomedicines9081018,Vivo-Morpholino-Based Antiviral for SARS-CoV-2: Implications for Novel Therapies in the Treatment of Acute COVID-19 Disease.,https://pubmed.ncbi.nlm.nih.gov/34440222/,2. Materials and Methods > 2.4. Method for Measuring Cytotoxic Effect of Compounds; paragraph 9,964,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00335,P0,toxicity,toxicity_endpoint,690,yes,B,569,sided,ASO,,,general toxicity: toxicity,14789,34251414,PMC8288671,10.1182/bloodadvances.2021004233,Phase 3 randomized trial of chemotherapy with or without oblimersen in older AML patients: CALGB 10201 (Alliance).,https://pubmed.ncbi.nlm.nih.gov/34251414/,Abstract; paragraph 2,967,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00336,P0,toxicity,toxicity_endpoint,717,yes,B,1077,"revusiran (recovered B2 from PMID:28129130, curator:CM)",siRNA,,,hepatic: hepatotoxicity,2323,28129130,PMC5363199,10.1016/j.ymthe.2016.10.019,Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate.,https://pubmed.ncbi.nlm.nih.gov/28129130/,Abstract; paragraph 1,994,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00337,P0,toxicity,toxicity_endpoint,736,yes,B,494,unspecified ASO,ASO,,,general toxicity: toxicity,4256,24586944,PMC3931823,10.1371/journal.pone.0089659,In vitro and in vivo activity of a novel locked nucleic acid (LNA)-inhibitor-miR-221 against multiple myeloma cells.,https://pubmed.ncbi.nlm.nih.gov/24586944/,Abstract > Methods; paragraph 2,1013,mouse,,cell line,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00338,P0,toxicity,toxicity_endpoint,742,yes,B,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic: hepatotoxicity,4288,20886816,PMC2993159manuscript-id: NIHMS242039,10.1021/ja105875e,An exocyclic methylene group acts as a bioisostere of the 2'-oxygen atom in LNA.,https://pubmed.ncbi.nlm.nih.gov/20886816/,Abstract; paragraph 1,1019,,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00339,P0,toxicity,toxicity_endpoint,958,yes,B,633,SQY51,ASO,,,general safety: cell viability/cytotoxicity,1213,41583579,PMC12828233,10.1093/narmme/ugag001,Systemic distribution of tricyclo-DNA antisense oligonucleotide following intratracheal instillation in the mouse.,https://pubmed.ncbi.nlm.nih.gov/41583579/,Materials and methods > Cell viability assay; paragraph 31,1235,cell line,,cell line,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00340,P0,toxicity,toxicity_endpoint,1179,yes,B,1078,"OA2-siRAPSYN (recovered B2 from PMID:38741123, curator:CM)",siRNA,,,hepatic: hepatotoxicity,11191,38741123,PMC11089668,10.1186/s12951-024-02505-5,Targeted degradation of oncogenic BCR-ABL by silencing the gene of NEDD8 E3 ligase RAPSYN.,https://pubmed.ncbi.nlm.nih.gov/38741123/,Materials and methods > Cell viability assay; paragraph 15,1456,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00341,P0,toxicity,toxicity_endpoint,1180,yes,B,299,nusinersen,ASO,,,general safety: systemic safety/tolerability,11199,38722537,PMC11213752,10.1007/s12325-024-02852-7,A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results.,https://pubmed.ncbi.nlm.nih.gov/38722537/,Abstract > Methods; paragraph 2,1457,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00342,P0,toxicity,toxicity_endpoint,1240,yes,B,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic: hepatotoxicity,1619,35667416,PMC9270256,10.1016/j.jlr.2022.100237,ANGPTL4 silencing via antisense oligonucleotides reduces plasma triglycerides and glucose in mice without causing lymphadenopathy.,https://pubmed.ncbi.nlm.nih.gov/35667416/,Materials and methods > Animal studies > Study 1; paragraph 15,1517,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00343,P0,toxicity,toxicity_endpoint,1292,yes,B,1079,"RGD-PEG-ECO/siβ3 (recovered B2 from PMID:31140918, curator:CM)",siRNA,,,hepatic: hepatotoxicity,12604,31140918,PMC6686697,10.1089/nat.2019.0784,Formulation of Biocompatible Targeted ECO/siRNA Nanoparticles with Long-Term Stability for Clinical Translation of RNAi.,https://pubmed.ncbi.nlm.nih.gov/31140918/,Abstract; paragraph 1,1569,,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00344,P0,toxicity,toxicity_endpoint,1306,yes,B,1080,"MORF1-MORF2 (recovered B2 from PMID:24308267, curator:CM)",PMO,,,hepatic: hepatotoxicity,4261,24308267,PMC3908873manuscript-id: NIHMS548399,10.1021/nn4053827,Cell surface self-assembly of hybrid nanoconjugates via oligonucleotide hybridization induces apoptosis.,https://pubmed.ncbi.nlm.nih.gov/24308267/,MATERIALS AND METHODS > In vivo anticancer efficacy; paragraph 31,1583,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00345,P1,offtarget,offtarget_evidence,40,no,A,218,SPC2996; PC3; CaCo2,ASO,,,off-target evidence,2845,19854938,PMC2800216,10.1093/nar/gkp841,"Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents.",https://pubmed.ncbi.nlm.nih.gov/19854938/,RESULTS AND DISCUSSION > Gene silencing by gymnotic delivery is a general phenomenon; paragraph 15,235,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00346,P1,toxicity,toxicity_endpoint,166,no,A,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,1613,36191773,PMC10695009manuscript-id: NIHMS1944143,10.1016/j.actbio.2022.09.065,Development of amino acid-modified biodegradable lipid nanoparticles for siRNA delivery.,https://pubmed.ncbi.nlm.nih.gov/36191773/,"Results and discussion > Characterization of LHHK LNPs for morphology, toxicity, serum stability, and hemolytic assay; paragraph 33",197,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00347,P1,toxicity,toxicity_endpoint,169,no,A,190,RAW264; cck8,siRNA,,,hepatic: hepatotoxicity,4065,36064365,PMC9446571,10.1186/s12951-022-01603-6,Hydrogel-metal-organic-framework hybrids mediated efficient oral delivery of siRNA for the treatment of ulcerative colitis.,https://pubmed.ncbi.nlm.nih.gov/36064365/,Results and discussion > In vitro inhibition of siTNFα; paragraph 21,200,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00348,P1,toxicity,toxicity_endpoint,182,no,A,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,2787,29858054,PMC5992344,10.1016/j.omtn.2018.02.004,Saponins as Natural Adjuvant for Antisense Morpholino Oligonucleotides Delivery In Vitro and in mdx Mice.,https://pubmed.ncbi.nlm.nih.gov/29858054/,Results and Discussion > Cytotoxicity; paragraph 5,213,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00349,P1,toxicity,toxicity_endpoint,186,no,A,204,hDysE50,ASO,,,hepatic: hepatotoxicity,2812,26366082,PMC4562748,10.2147/IJN.S89910,Cationic polyelectrolyte-mediated delivery of antisense morpholino oligonucleotides for exon-skipping in vitro and in mdx mice.,https://pubmed.ncbi.nlm.nih.gov/26366082/,Results and discussion > PMO delivery in C2C12 myoblast cell lines expressing GFP/hDysE50; paragraph 17,217,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00350,P1,toxicity,toxicity_endpoint,196,no,A,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,1511,41810474,PMC12925264,10.1016/j.ajps.2026.101128,Freezing shock monocytes deliver antisense oligonucleotides via liposomes for the treatment of idiopathic pulmonary fibrosis.,https://pubmed.ncbi.nlm.nih.gov/41810474/,Results and discussion > In vitro anti-fibrotic effect validation of ASO and characterization of ZMO-E5-NPs-ASO properties; paragraph 38,239,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00351,P1,toxicity,toxicity_endpoint,203,no,A,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,1551,39861693,PMC11769357,10.3390/pharmaceutics17010045,Innovative Lipid Nanoparticles Co-Delivering Hydroxychloroquine and siRNA for Enhanced Rheumatoid Arthritis Therapy.,https://pubmed.ncbi.nlm.nih.gov/39861693/,3. Results and Discussion > 3.2. Cytotoxicity of LNP-siN.C./siTNF-α and LNP-siN.C./siTNF-α-HCQ; paragraph 26,246,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00352,P1,toxicity,toxicity_endpoint,205,no,A,228,RAW264; HepG2,siRNA,,,hepatic: hepatotoxicity,1825,40654340,PMC12254858,10.1016/j.apsb.2025.04.003,"Self-degradable ""gemini-like"" ionizable lipid-mediated delivery of siRNA for subcellular-specific gene therapy of hepatic diseases.",https://pubmed.ncbi.nlm.nih.gov/40654340/,Results and discussion > Cell viability; paragraph 39,248,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00353,P1,toxicity,toxicity_endpoint,213,no,A,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,3614,40616527,PMC12269356,10.1021/acsnano.5c05395,Reprogramming Immunodeficiency in Lung Metastases via PD-L1 siRNA Delivery and Antigen Capture of Nanosponge-Mediated Dendritic Cell Modulation.,https://pubmed.ncbi.nlm.nih.gov/40616527/,Results and Discussion > In Vivo GFP-pDNA Delivery by TP-SL@PB; paragraph 46,256,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00354,P1,toxicity,toxicity_endpoint,214,no,A,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,3626,40582054,PMC12268195,10.1016/j.ultsonch.2025.107446,A novel ultrasound-responsive cluster bomb system for efficient siRNA delivery in brain.,https://pubmed.ncbi.nlm.nih.gov/40582054/,Results and Discussion > Cell uptake and toxicity of siRNA@NP@ND in vitro; paragraph 12,257,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00355,P1,toxicity,toxicity_endpoint,215,no,A,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,3640,40524159,PMC12168320,10.1186/s12951-025-03521-9,An immune activator encapsulating PD-L1 siRNA for augmented immune checkpoint blockade immunotherapy through Zn(2+) overload triggered pyroptosis.,https://pubmed.ncbi.nlm.nih.gov/40524159/,Results and discussion > Anti-metastasis and in vivo biosafety evaluation; paragraph 28,258,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00356,P1,toxicity,toxicity_endpoint,216,no,A,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,3690,40364633,PMC12147987,10.1002/adhm.202500107,Engineered Chitosan-Derived Nanocarrier for Efficient siRNA Delivery to Peripheral and Central Neurons.,https://pubmed.ncbi.nlm.nih.gov/40364633/,Results and Discussion > Enhanced Cellular Uptake And Neurospecificity of Targeted Polyplexes; paragraph 14,259,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00357,P1,toxicity,toxicity_endpoint,237,no,A,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,2768,32260534,PMC7226018,10.3390/biom10040553,Chitosan Nanocomplexes for the Delivery of ENaC Antisense Oligonucleotides to Airway Epithelial Cells.,https://pubmed.ncbi.nlm.nih.gov/32260534/,4. Discussion > 4.2. Supplemented Transfection Medium Stabilizes Nanocomplexes but Harms Human Respiratory Epithelial Cells; paragraph 34,280,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00358,P1,toxicity,toxicity_endpoint,423,no,A,379,CaP-siSlc31a1,siRNA,SLC31A1,,general safety: cell viability/cytotoxicity,1735,41809386,PMC12969650,10.1016/j.mtbio.2026.102991,Transdermal siRNA delivery via biomineralized nanoparticle-incorporated microneedles modulates cuproptosis-ferroptosis interaction for psoriasis therapy.,https://pubmed.ncbi.nlm.nih.gov/41809386/,Results and discussion > Fabrication of CaP-siSlc31a1 for efficient cellular uptake and gene silencing; paragraph 13,482,cell line,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00359,P1,toxicity,toxicity_endpoint,429,no,A,385,TGF-beta siRNA PMNP,siRNA,TGFB1,,hepatic/renal: hepatotoxicity and kidney function,3262,41704233,PMC12907503,10.1016/j.bioactmat.2026.02.006,Polyplex of peptide-mannan and RNA for intranasal delivery of TGF-β siRNA in treatment of pulmonary fibrosis.,https://pubmed.ncbi.nlm.nih.gov/41704233/,Results and discussion > Assessment of hepatotoxicity and kidney function; paragraph 15,488,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00360,P1,toxicity,toxicity_endpoint,434,no,A,390,siB/QU@L-OL,siRNA,BACE1,,general safety: in vivo biosafety/hepatic renal and local nasal toxicity,3297,41560836,PMC12814076,10.1016/j.mtbio.2026.102764,Intranasal delivery of Odorranalectin-modified lipid nanoparticles for multi-targeted therapy of Alzheimer's disease.,https://pubmed.ncbi.nlm.nih.gov/41560836/,Results and discussion > In vivo biosafety assessment; paragraph 52,493,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00361,P1,toxicity,toxicity_endpoint,435,no,A,391,curcumin coacervate siRNA formulation,siRNA,,,general safety: hemolysis/blood compatibility,3316,41510156,PMC12775651,10.7150/thno.121865,Curcumin coacervates for supramolecular-interaction-responsive cytosolic siRNA delivery to enhance pyroptosis.,https://pubmed.ncbi.nlm.nih.gov/41510156/,Results and Discussion > Curcumin coacervates enrich in the tumor site through tail vein injection; paragraph 31,494,ex vivo blood/RBC,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00362,P1,toxicity,toxicity_endpoint,438,no,A,394,pPB-siRNATRAF6/NMm-pPB-siRNATRAF6,siRNA,TRAF6,,general safety: cell viability/cytotoxicity,6682,41560791,PMC12813177,10.1016/j.mtbio.2025.102734,Spatiotemporal delivery of multifunctional nanozymes for neuroinflammation alleviation via autophagy modulation in spinal cord injury.,https://pubmed.ncbi.nlm.nih.gov/41560791/,Results and discussion > In vitro targeting of inflammatory macrophages and nerve cells; paragraph 11,497,cell line,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00363,P1,toxicity,toxicity_endpoint,443,no,A,399,LNP TNF-alpha siRNA,siRNA,TNF,,general safety: cell viability/cytotoxicity,1538,40776451,PMC12616584,10.1002/adhm.202500695,Pulmonary Delivery of siRNA Anti-TNFα-loaded Lipid Nanoparticles for Rapid Recovery in Murine Acute Lung Injury.,https://pubmed.ncbi.nlm.nih.gov/40776451/,Results and Discussion > Characterizing the In Vitro Anti‐Inflammatory Effect of LNP TNF‐α siRNA; paragraph 11,502,cell line,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00364,P1,toxicity,toxicity_endpoint,446,no,A,402,CDs-pDEAEMA/BIRC5 siRNA,siRNA,BIRC5,,general safety: cytocompatibility/erythrocompatibility,3695,40347142,PMC12152932,10.1021/acs.biomac.5c00267,Poly(2-(diethylamino)ethyl methacrylate)-Functionalized Carbon Nanodots as Theranostic Platforms for siRNA Delivery and Survivin Silencing in Triple-Negative Breast Cancer.,https://pubmed.ncbi.nlm.nih.gov/40347142/,Results and Discussion > Cell Compatibility; paragraph 50,505,human dermal fibroblasts/RBC,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00365,P1,toxicity,toxicity_endpoint,1297,no,A,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic: hepatotoxicity,12621,30114557,PMC6092551,10.1016/j.omtn.2018.07.010,Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models.,https://pubmed.ncbi.nlm.nih.gov/30114557/,Results > Identification of Lead Oligonucleotide Targeting the CEP290 c.2991+1655A>G Mutation; paragraph 6,1574,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00366,P1,offtarget,offtarget_evidence,33,no,B,212,ADO2,siRNA,,,seed-mediated off-target effect,3759,39934917,PMC11816505,10.1186/s13287-025-04151-6,Development and evaluation of siRNA-mediated gene silencing strategies for ADO2 therapy utilizing iPSCs model and DMPC-SPIONs delivery system.,https://pubmed.ncbi.nlm.nih.gov/39934917/,Discussion; paragraph 15,228,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00367,P1,offtarget,offtarget_evidence,36,no,B,215,CCND1; Drisapersen; Mipomersen,ASO,,,transcriptome-level off-target effect,2210,35464859,PMC9019733,10.3389/fgene.2022.791416,"Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In Vitro.",https://pubmed.ncbi.nlm.nih.gov/35464859/,Discussion; paragraph 62,231,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00368,P1,offtarget,offtarget_evidence,42,no,B,253,TNRC6; GW182; AGO2,siRNA,,,seed-mediated off-target effect,1011,40134378,,10.1089/nat.2024.0090,Mechanistic Insights into Hybridization-Based Off-Target Activity of GalNAc-siRNA Conjugates.,https://pubmed.ncbi.nlm.nih.gov/40134378/,PubMed abstract,289,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00369,P1,offtarget,offtarget_evidence,44,no,B,186,unspecified antisense oligonucleotide,ASO,,,transcriptome-level off-target effect,2193,37310350,,10.1039/d3ob00574g,Alteration of target cleavage patterns and off-target reduction of antisense oligonucleotides incorporating 2-N-carbamoyl- or (2-pyridyl)guanine.,https://pubmed.ncbi.nlm.nih.gov/37310350/,PubMed abstract,291,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00370,P1,offtarget,offtarget_evidence,47,no,B,186,unspecified antisense oligonucleotide,ASO,,,transcriptome-level off-target effect,2216,33314011,,10.1007/s40291-020-00504-4,Using RNA-seq to Assess Off-Target Effects of Antisense Oligonucleotides in Human Cell Lines.,https://pubmed.ncbi.nlm.nih.gov/33314011/,PubMed abstract,294,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00371,P1,offtarget,offtarget_evidence,49,no,B,256,siF12; siF12-A; siF12-AC9,siRNA,,,hybridization/mismatch off-target effect,12115,31063435,,10.1089/nat.2018.0767,"Use of ""C9/11 Mismatch"" Control siRNA Reveals Sequence-Related Off-Target Effect on Coagulation of an siRNA Targeting Mouse Coagulation Factor XII.",https://pubmed.ncbi.nlm.nih.gov/31063435/,PubMed abstract,296,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00372,P1,offtarget,offtarget_evidence,50,no,B,257,Malat1-targeting,ASO,,,transcriptome-level off-target effect,12059,30095329,,10.1089/nat.2018.0724,Role of Computationally Evaluated Target Specificity in the Hepatotoxicity of Gapmer Antisense Oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/30095329/,PubMed abstract,297,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00373,P1,offtarget,offtarget_evidence,51,no,B,328,ARL4C; ARL4C-targeting; CP8,ASO,,,transcriptome-level off-target effect,1037,41792408,,10.1007/s11626-026-01154-2,Development of ARL4C antisense oligonucleotide with reduced off-target effects and enhanced efficacy as an anti-cancer drug.,https://pubmed.ncbi.nlm.nih.gov/41792408/,PubMed abstract,410,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00374,P1,offtarget,offtarget_evidence,56,no,B,370,PDIA3; oblimersen,siRNA,,,off-target evidence,2270,20544786,,10.1002/cmdc.201000156,Off-target effects related to the phosphorothioate modification of nucleic acids.,https://pubmed.ncbi.nlm.nih.gov/20544786/,PubMed abstract,472,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00375,P1,offtarget,offtarget_evidence,73,no,B,441,designated ASO,ASO,,,hybridization/mismatch off-target effect,1453,39192097,,10.1007/s10120-024-01548-9,Preclinical toxicological assessment of amido-bridged nucleic acid-modified antisense oligonucleotides targeting synaptotagmin XIII for intra-abdominal treatment of peritoneal metastasis of gastric cancer.,https://pubmed.ncbi.nlm.nih.gov/39192097/,PubMed abstract,550,human,,blood/serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00376,P1,offtarget,offtarget_evidence,229,no,B,1065,"MC3 LNP-siRNA (recovered B2 from PMID:41708903, curator:CM)",siRNA,,,off-target evidence,1209,41708903,,10.1007/s13346-026-02076-y,Comparative analysis of clinically approved lipid nanoparticles for intranasal siRNA delivery against SARS-CoV-2.,https://pubmed.ncbi.nlm.nih.gov/41708903/,PubMed abstract,706,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00377,P1,offtarget,offtarget_evidence,275,no,B,299,nusinersen,ASO,,,transcriptome-level off-target effect,28702,34445083,PMC8395096,10.3390/ijms22168378,High Concentration of an ISS-N1-Targeting Antisense Oligonucleotide Causes Massive Perturbation of the Transcriptome.,https://pubmed.ncbi.nlm.nih.gov/34445083/,PubMed abstract,1716,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00378,P1,offtarget,offtarget_evidence,535,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,seed-based off-target suppression,12077,39231537,PMC11472056,10.1093/nar/gkae741,Synthesis of 2'-formamidonucleoside phosphoramidites for suppressing the seed-based off-target effects of siRNAs.,https://pubmed.ncbi.nlm.nih.gov/39231537/,PMC full text high-precision preselection,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00379,P1,offtarget,offtarget_evidence,536,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,unexpected cGAS signaling suppression,2281,40337520,PMC12055788,10.3389/fphar.2025.1576151,An off-target effect of class A CpG-oligonucleotides on suppressing the cyclic GMP-AMP synthase signaling in fibroblastic reticular cells.,https://pubmed.ncbi.nlm.nih.gov/40337520/,PMC full text high-precision preselection,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00380,P1,offtarget,offtarget_evidence,537,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,seed-mediated off-target,2308,35205363,PMC8872465,10.3390/genes13020319,The siRNA Off-Target Effect Is Determined by Base-Pairing Stabilities of Two Different Regions with Opposite Effects.,https://pubmed.ncbi.nlm.nih.gov/35205363/,PMC full text high-precision preselection,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00381,P1,offtarget,offtarget_evidence,538,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,minimal off-target activity,4173,33068777,PMC7854280,10.1016/j.ymthe.2020.10.010,DUX4 Transcript Knockdown with Antisense 2'-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy.,https://pubmed.ncbi.nlm.nih.gov/33068777/,PMC full text high-precision preselection,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00382,P1,toxicity,toxicity_endpoint,165,no,B,187,SLN360,siRNA,,,hepatic: hepatotoxicity,1486,35737426,PMC9516055,10.1093/toxsci/kfac067,"Preclinical Toxicological Assessment of A Novel siRNA, SLN360, Targeting Elevated Lipoprotein (a) in Cardiovascular Disease.",https://pubmed.ncbi.nlm.nih.gov/35737426/,DISCUSSION; paragraph 52,196,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00383,P1,toxicity,toxicity_endpoint,170,no,B,191,SKOV3,siRNA,,,hepatic: hepatotoxicity,4105,34382360,PMC11468224,10.1002/adhm.202101202,Functional siRNA Delivery by Extracellular Vesicle-Liposome Hybrid Nanoparticles.,https://pubmed.ncbi.nlm.nih.gov/34382360/,Discussion; paragraph 36,201,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00384,P1,toxicity,toxicity_endpoint,172,no,B,193,RTR5001,ASO,,,hepatic: hepatotoxicity,1463,34575518,PMC8470776,10.3390/pharmaceutics13091442,"Safety Testing of an Antisense Oligonucleotide Intended for Pediatric Indications in the Juvenile Göttingen Minipig, including an Evaluation of the Ontogeny of Key Nucleases.",https://pubmed.ncbi.nlm.nih.gov/34575518/,4. Discussion; paragraph 49,203,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00385,P1,toxicity,toxicity_endpoint,173,no,B,194,casimersen,ASO,,,hepatic: hepatotoxicity,1639,34105177,PMC9290993,10.1002/mus.27347,"Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial.",https://pubmed.ncbi.nlm.nih.gov/34105177/,DISCUSSION; paragraph 40,204,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00386,P1,toxicity,toxicity_endpoint,175,no,B,196,Nusinersen,ASO,,,hepatic: hepatotoxicity,1643,33899154,PMC8563549,10.1007/s00415-021-10569-8,Nusinersen treatment in adult patients with spinal muscular atrophy: a safety analysis of laboratory parameters.,https://pubmed.ncbi.nlm.nih.gov/33899154/,Discussion; paragraph 36,206,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00387,P1,toxicity,toxicity_endpoint,178,no,B,199,Stk25,ASO,,,hepatic: hepatotoxicity,2784,30576769,PMC6411916,10.1016/j.jcmgh.2018.12.004,Targeted Delivery of Stk25 Antisense Oligonucleotides to Hepatocytes Protects Mice Against Nonalcoholic Fatty Liver Disease.,https://pubmed.ncbi.nlm.nih.gov/30576769/,Discussion; paragraph 23,209,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00388,P1,toxicity,toxicity_endpoint,179,no,B,200,Huh7; MCF7; nanoparticles43,siRNA,,,hepatic: hepatotoxicity,4207,31676815,PMC6825139,10.1038/s41598-019-52142-4,Therapeutic potential of functionalized siRNA nanoparticles on regression of liver cancer in experimental mice.,https://pubmed.ncbi.nlm.nih.gov/31676815/,Discussion; paragraph 27,210,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00389,P1,toxicity,toxicity_endpoint,180,no,B,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,2060,29390093,PMC5861398,10.1093/nar/gky060,Acute hepatotoxicity of 2' fluoro-modified 5-10-5 gapmer phosphorothioate oligonucleotides in mice correlates with intracellular protein binding and the loss of DBHS proteins.,https://pubmed.ncbi.nlm.nih.gov/29390093/,DISCUSSION; paragraph 39,211,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00390,P1,toxicity,toxicity_endpoint,183,no,B,202,RTR3649; RTR5001; RTR4955,ASO,,,hepatic: hepatotoxicity,2324,28123102,PMC5414856,10.1093/toxsci/kfx025,From the Cover: The Minipig is a Suitable Non-Rodent Model in the Safety Assessment of Single Stranded Oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/28123102/,DISCUSSION; paragraph 65,214,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00391,P1,toxicity,toxicity_endpoint,185,no,B,203,TLR3; TLR7; TLR8,siRNA,,,hepatic: hepatotoxicity,2327,26444021,PMC5058343manuscript-id: NIHMS820714,10.3233/JHD-150163,Safety of Striatal Infusion of siRNA in a Transgenic Huntington's Disease Mouse Model.,https://pubmed.ncbi.nlm.nih.gov/26444021/,Discussion; paragraph 33,216,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00392,P1,toxicity,toxicity_endpoint,190,no,B,206,Inclisiran,siRNA,,,hepatic: hepatotoxicity,10925,39064553,PMC11278919,10.3390/medicina60071124,"Efficacy of Alirocumab, Evolocumab, and Inclisiran in Patients with Hypercholesterolemia at Increased Cardiovascular Risk.",https://pubmed.ncbi.nlm.nih.gov/39064553/,4. Discussion; paragraph 28,221,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00393,P1,toxicity,toxicity_endpoint,193,no,B,219,MKK4,siRNA,,,hepatic: hepatotoxicity,1152,41970250,PMC13067123,10.1016/j.mtbio.2026.103059,Dual targeted gene delivery strategy mediated by GalNAc-modified lipid nanoparticles enhances liver regeneration through specific knockdown of MKK4.,https://pubmed.ncbi.nlm.nih.gov/41970250/,Discussion; paragraph 52,236,cell line,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00394,P1,toxicity,toxicity_endpoint,197,no,B,221,Gb3S; MC3-containing; Gb3,siRNA,,,hepatic: hepatotoxicity,4532,42170556,PMC13187621,10.1016/j.ijpx.2026.100555,Nanomedicines for DNA and interference RNA co-delivery: Combined gene therapy for Fabry disease.,https://pubmed.ncbi.nlm.nih.gov/42170556/,Conclusions; paragraph 92,240,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00395,P1,toxicity,toxicity_endpoint,199,no,B,223,FN1,siRNA,,,hepatic: hepatotoxicity,6205,41732388,PMC12925212,10.1016/j.mtbio.2026.102877,Targeting FN1 to overcome gemcitabine resistance in gallbladder cancer: Mechanistic insights and an iRGD-modified PEG-PLGA nanoparticle delivery strategy.,https://pubmed.ncbi.nlm.nih.gov/41732388/,Discussion; paragraph 44,242,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00396,P1,toxicity,toxicity_endpoint,202,no,B,226,si097-DV29PG5; si061-DV27PG5,siRNA,,,hepatic: hepatotoxicity,1477,40385635,PMC12083919,10.1016/j.omtn.2025.102542,"Design, pharmacology, and toxicology of a novel chemically modified siRNA targeting hepatic angiotensinogen.",https://pubmed.ncbi.nlm.nih.gov/40385635/,Discussion; paragraph 30,245,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00397,P1,toxicity,toxicity_endpoint,204,no,B,227,AS456; IC50; TRL7,siRNA,,,hepatic: hepatotoxicity,1788,41373822,PMC12692593,10.3390/ijms262311675,Respiratory Delivery of Highly Conserved Antiviral siRNAs Suppress SARS-CoV-2 Infection.,https://pubmed.ncbi.nlm.nih.gov/41373822/,3. Discussion; paragraph 24,247,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00398,P1,toxicity,toxicity_endpoint,208,no,B,230,Tofersen,ASO,,,neurological: neurotoxicity,2347,40017137,PMC12060635,10.1002/mus.28372,Serious Neurologic Adverse Events in Tofersen Clinical Trials for Amyotrophic Lateral Sclerosis.,https://pubmed.ncbi.nlm.nih.gov/40017137/,Discussion; paragraph 30,251,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00399,P1,toxicity,toxicity_endpoint,212,no,B,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,3595,40681573,PMC12274417,10.1038/s41598-025-10960-9,Peptide dendrimer and hyaluronic acid modified nanovesicles for ocular delivery of timolol maleate and siRNA.,https://pubmed.ncbi.nlm.nih.gov/40681573/,Conclusion; paragraph 65,255,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00400,P1,toxicity,toxicity_endpoint,218,no,B,235,APP36; Iba1,siRNA,,,hepatic: hepatotoxicity,7646,41323796,PMC12663631,10.1016/j.omtn.2025.102757,SCAD: A modular platform for efficient delivery of duplex RNA to the CNS and beyond.,https://pubmed.ncbi.nlm.nih.gov/41323796/,Discussion; paragraph 46,261,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00401,P1,toxicity,toxicity_endpoint,224,no,B,93,Inclisiran,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,2348,39459005,PMC11511047,10.3390/ph17101365,Safety of Inclisiran: A Disproportionality Analysis from the EudraVigilance Database.,https://pubmed.ncbi.nlm.nih.gov/39459005/,3. Discussion > 3.1. Descriptive Analysis; paragraph 18,267,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00402,P1,toxicity,toxicity_endpoint,228,no,B,243,HSP47,siRNA,,,hepatic: hepatotoxicity,1458,37654022,PMC10582666,10.1111/cts.13581,"Pharmacokinetics, safety, and tolerability of BMS-986263, a lipid nanoparticle containing HSP47 siRNA, in participants with hepatic impairment.",https://pubmed.ncbi.nlm.nih.gov/37654022/,DISCUSSION; paragraph 37,271,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00403,P1,toxicity,toxicity_endpoint,231,no,B,246,lumasiran,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,2701,35913563,PMC9925547,10.1007/s00467-022-05684-1,Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial.,https://pubmed.ncbi.nlm.nih.gov/35913563/,Discussion; paragraph 35,274,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00404,P1,toxicity,toxicity_endpoint,238,no,B,251,revusiran,siRNA,,,hepatic: hepatotoxicity,2105,30820542,PMC6468299,10.1093/nar/gkz140,Safety evaluation of 2'-deoxy-2'-fluoro nucleotides in GalNAc-siRNA conjugates.,https://pubmed.ncbi.nlm.nih.gov/30820542/,DISCUSSION; paragraph 57,281,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00405,P1,toxicity,toxicity_endpoint,240,no,B,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,2322,28145801,PMC5467133,10.1089/nat.2016.0650,The Effects of 2'-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical Trials.,https://pubmed.ncbi.nlm.nih.gov/28145801/,Discussion; paragraph 36,283,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00406,P1,toxicity,toxicity_endpoint,241,no,B,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,2237,27357629,PMC5112040,10.1038/mt.2016.136,Integrated Safety Assessment of 2'-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human Volunteers.,https://pubmed.ncbi.nlm.nih.gov/27357629/,Discussion; paragraph 26,284,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00407,P1,toxicity,toxicity_endpoint,242,no,B,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,12119,27504598,PMC5023396,10.1038/mtna.2016.50,Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain.,https://pubmed.ncbi.nlm.nih.gov/27504598/,Discussion; paragraph 21,285,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00408,P1,toxicity,toxicity_endpoint,247,no,B,260,SMN2; Nusinersen,ASO,,,hepatic: hepatotoxicity,1064,41813449,,10.1016/j.nmd.2025.106256,"A prospective, multi-center, observational study of the safety, tolerability and effectiveness of Nusinersen in adult patients with spinal muscular atrophy.",https://pubmed.ncbi.nlm.nih.gov/41813449/,PubMed abstract,300,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00409,P1,toxicity,toxicity_endpoint,248,no,B,261,LRP4; NCT05070858; cemdisiran,siRNA,,,hepatic: hepatotoxicity,1150,42030965,,10.1016/S0140-6736(26)00690-2,"Efficacy and safety of cemdisiran siRNA in myasthenia gravis (NIMBLE): a double-blind, randomised, placebo-controlled, phase 3 trial.",https://pubmed.ncbi.nlm.nih.gov/42030965/,PubMed abstract,301,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00410,P1,toxicity,toxicity_endpoint,250,no,B,263,PCSK9; Inclisiran,siRNA,,,general safety: safety/tolerability,1217,41289434,,10.1097/FJC.0000000000001778,Muscle Adverse Events Associated With Inclisiran: Data Mining of FAERS Database and Mendelian Randomization Analysis.,https://pubmed.ncbi.nlm.nih.gov/41289434/,PubMed abstract,303,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00411,P1,toxicity,toxicity_endpoint,251,no,B,264,patisiran; givosiran; vutrisiran,siRNA,,,hepatic: hepatotoxicity,1218,41274662,,10.1177/21593337251391029,Class-Specific Adverse Events of Patients Treated with Small Interfering RNA Therapeutics: A Disproportionality Analysis of the United States Food and Drug Administration Adverse Event Reporting System Database Based on the MY FAERS Platform.,https://pubmed.ncbi.nlm.nih.gov/41274662/,PubMed abstract,304,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00412,P1,toxicity,toxicity_endpoint,264,no,B,252,nusinersen,ASO/siRNA mixed context,,,general safety: safety/tolerability,12078,39690144,,10.1080/14740338.2024.2443796,Adverse events of nusinersen: a real-world drug safety surveillance study based on the FDA adverse event reporting system (FAERS) database.,https://pubmed.ncbi.nlm.nih.gov/39690144/,PubMed abstract,317,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00413,P1,toxicity,toxicity_endpoint,266,no,B,274,PNPLA3; rs738409; PNPLA3I148M,siRNA,,,hepatic: hepatotoxicity,1048,40899091,,10.1177/21593337251375804,Use of a Transgenic Human PNPLA3(I148M) Knock-in Mouse for Translational Safety Evaluations of siRNA Therapeutics.,https://pubmed.ncbi.nlm.nih.gov/40899091/,PubMed abstract,319,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00414,P1,toxicity,toxicity_endpoint,270,no,B,276,SPL84,ASO,,,hepatic: hepatotoxicity,1133,39500647,,10.1016/j.jcf.2024.10.004,"A phase I study assessing the safety and tolerability of SPL84, an inhaled antisense oligonucleotide for treatment of cystic fibrosis patients with the 3849 +10kb C->T.",https://pubmed.ncbi.nlm.nih.gov/39500647/,PubMed abstract,323,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00415,P1,toxicity,toxicity_endpoint,273,no,B,279,TfR1,ASO,,,hepatic: hepatotoxicity,1468,39325927,,10.1002/anie.202415272,Self-Assembled Antibody-Oligonucleotide Conjugates for Targeted Delivery of Complementary Antisense Oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/39325927/,PubMed abstract,326,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00416,P1,toxicity,toxicity_endpoint,293,no,B,289,NCT05089084; Plozasiran,siRNA,,,hepatic: hepatotoxicity,10466,39225259,,10.1056/NEJMoa2409368,Plozasiran for Managing Persistent Chylomicronemia and Pancreatitis Risk.,https://pubmed.ncbi.nlm.nih.gov/39225259/,PubMed abstract,346,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00417,P1,toxicity,toxicity_endpoint,296,no,B,291,GalNAc3; GalNAc3-conjugated,ASO,,,hepatic: hepatotoxicity,1112,38227794,,10.1089/nat.2023.0026,Safety and Tolerability of GalNAc(3)-Conjugated Antisense Drugs Compared to the Same-Sequence 2'-O-Methoxyethyl-Modified Antisense Drugs: Results from an Integrated Assessment of Phase 1 Clinical Trial Data.,https://pubmed.ncbi.nlm.nih.gov/38227794/,PubMed abstract,349,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00418,P1,toxicity,toxicity_endpoint,297,no,B,292,SYT13; Syt13-deficient; Syt13-deficiency,ASO,,,hepatic: hepatotoxicity,1453,39192097,,10.1007/s10120-024-01548-9,Preclinical toxicological assessment of amido-bridged nucleic acid-modified antisense oligonucleotides targeting synaptotagmin XIII for intra-abdominal treatment of peritoneal metastasis of gastric cancer.,https://pubmed.ncbi.nlm.nih.gov/39192097/,PubMed abstract,350,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00419,P1,toxicity,toxicity_endpoint,298,no,B,293,BIIB078; NCT03626012; BIIB078-treated,ASO,,,hepatic: hepatotoxicity,1454,39059407,,10.1016/S1474-4422(24)00216-3,"Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study.",https://pubmed.ncbi.nlm.nih.gov/39059407/,PubMed abstract,351,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00420,P1,toxicity,toxicity_endpoint,304,no,B,299,nusinersen,ASO,,,hepatic: hepatotoxicity,2630,37649429,,10.1080/00207454.2023.2251662,"Safety and effectiveness of nusinersen, a treatment for spinal muscular atrophy, in 524 patients: results from an interim analysis of post-marketing surveillance in Japan.",https://pubmed.ncbi.nlm.nih.gov/37649429/,PubMed abstract,357,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00421,P1,toxicity,toxicity_endpoint,305,no,B,299,nusinersen,ASO,,,hepatic: hepatotoxicity,2634,35787224,,10.1080/00207454.2022.2095270,"Real-world safety and effectiveness of nusinersen, a treatment for spinal muscular atrophy, in 401 Japanese patients: results from an interim analysis of post-marketing surveillance.",https://pubmed.ncbi.nlm.nih.gov/35787224/,PubMed abstract,358,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00422,P1,toxicity,toxicity_endpoint,318,no,B,305,NCT03218995,ASO/siRNA mixed context,,,renal: renal safety,1600,37207382,,10.1016/j.nmd.2023.03.008,"Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping.",https://pubmed.ncbi.nlm.nih.gov/37207382/,PubMed abstract,371,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00423,P1,toxicity,toxicity_endpoint,320,no,B,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,2304,36415122,,10.1002/cpdd.1197,"Pharmacokinetics, Safety, and Tolerability of the siRNA JNJ-73763989 in Healthy Chinese Adult Participants.",https://pubmed.ncbi.nlm.nih.gov/36415122/,PubMed abstract,373,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00424,P1,toxicity,toxicity_endpoint,321,no,B,307,NCT03338816; givosiran,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,2666,37479139,,10.1016/j.jhep.2023.06.013,Efficacy and safety of givosiran for acute hepatic porphyria: Final results of the randomized phase III ENVISION trial.,https://pubmed.ncbi.nlm.nih.gov/37479139/,PubMed abstract,374,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00425,P1,toxicity,toxicity_endpoint,323,no,B,309,NCT03417102; fitusiran,siRNA,,,hepatic: hepatotoxicity,2685,37003287,,10.1016/S0140-6736(23)00284-2,"Efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial.",https://pubmed.ncbi.nlm.nih.gov/37003287/,PubMed abstract,376,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00426,P1,toxicity,toxicity_endpoint,325,no,B,311,pelacarsen,ASO,,,hepatic: hepatotoxicity,12201,36529659,,10.1016/j.jacl.2022.12.001,Efficacy and safety of pelacarsen in lowering Lp(a) in healthy Japanese subjects.,https://pubmed.ncbi.nlm.nih.gov/36529659/,PubMed abstract,378,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00427,P1,toxicity,toxicity_endpoint,327,no,B,312,CL1H6; CL1H6-LNP,siRNA,,,hepatic: hepatotoxicity,1428,34592399,,10.1016/j.ijpharm.2021.121140,The hydrophobic tail of a pH-sensitive cationic lipid influences siRNA transfection activity and toxicity in human NK cell lines.,https://pubmed.ncbi.nlm.nih.gov/34592399/,PubMed abstract,380,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00428,P1,toxicity,toxicity_endpoint,329,no,B,313,GalNAc3; GalNAc3-MOE; GalNAc3-conjugated,ASO,,,hepatic: hepatotoxicity,1465,33431610,,10.1124/jpet.120.000222,"Safety, Pharmacokinetic, and Pharmacodynamic Evaluation of a 2'-(2-Methoxyethyl)-D-ribose Antisense Oligonucleotide-Triantenarry N-Acetyl-galactosamine Conjugate that Targets the Human Transmembrane Protease Serine 6.",https://pubmed.ncbi.nlm.nih.gov/33431610/,PubMed abstract,382,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00429,P1,toxicity,toxicity_endpoint,330,no,B,314,NCT02300233; volanesorsen,ASO,,,hepatic: hepatotoxicity,2754,33798466,,10.1016/S2213-8587(21)00046-2,"Efficacy and safety of volanesorsen in patients with multifactorial chylomicronaemia (COMPASS): a multicentre, double-blind, randomised, placebo-controlled, phase 3 trial.",https://pubmed.ncbi.nlm.nih.gov/33798466/,PubMed abstract,383,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00430,P1,toxicity,toxicity_endpoint,334,no,B,317,NCT01737398; Inotersen,ASO,,,hepatic: hepatotoxicity,1422,32833564,,10.1089/nat.2020.0867,"Immunogenicity Assessment of Inotersen, a 2'-O-(2-Methoxyethyl) Antisense Oligonucleotide in Animals and Humans: Effect on Pharmacokinetics, Pharmacodynamics, and Safety.",https://pubmed.ncbi.nlm.nih.gov/32833564/,PubMed abstract,387,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00431,P1,toxicity,toxicity_endpoint,345,no,B,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,2801,27854228,,10.3233/JND-160157,Toxicological Characterization of Exon Skipping Phosphorodiamidate Morpholino Oligomers (PMOs) in Non-human Primates.,https://pubmed.ncbi.nlm.nih.gov/27854228/,PubMed abstract,398,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00432,P1,toxicity,toxicity_endpoint,347,no,B,323,NCT01153932; drisapersen,ASO,,,hepatic: hepatotoxicity,2821,25209738,,10.1016/S1474-4422(14)70195-4,"Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study.",https://pubmed.ncbi.nlm.nih.gov/25209738/,PubMed abstract,400,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00433,P1,toxicity,toxicity_endpoint,348,no,B,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,2823,24932560,,10.3109/08958378.2014.907587,Local and systemic tolerability of a 2'O-methoxyethyl antisense oligonucleotide targeting interleukin-4 receptor-α delivery by inhalation in mouse and monkey.,https://pubmed.ncbi.nlm.nih.gov/24932560/,PubMed abstract,401,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00434,P1,toxicity,toxicity_endpoint,352,no,B,327,Myd88-deficient; MDA5; LGP2,ASO,,,hepatic: hepatotoxicity,4277,22505629,,10.1124/jpet.112.193789,Unique O-methoxyethyl ribose-DNA chimeric oligonucleotide induces an atypical melanoma differentiation-associated gene 5-dependent induction of type I interferon response.,https://pubmed.ncbi.nlm.nih.gov/22505629/,PubMed abstract,405,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00435,P1,toxicity,toxicity_endpoint,354,no,B,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,2066,20886212,,10.1007/s00280-010-1473-z,"Preclinical toxicity and toxicokinetics of GTI-2040, a phosphorothioate oligonucleotide targeting ribonucleotide reductase R2.",https://pubmed.ncbi.nlm.nih.gov/20886212/,PubMed abstract,407,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00436,P1,toxicity,toxicity_endpoint,355,no,B,186,unspecified antisense oligonucleotide,ASO,,,renal: renal safety,2839,21540336,,10.1177/1091581811403505,"Repeat-dose toxicology evaluation in cynomolgus monkeys of AVI-4658, a phosphorodiamidate morpholino oligomer (PMO) drug for the treatment of duchenne muscular dystrophy.",https://pubmed.ncbi.nlm.nih.gov/21540336/,PubMed abstract,408,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00437,P1,toxicity,toxicity_endpoint,356,no,B,186,unspecified antisense oligonucleotide,ASO,,,renal: renal safety,2840,21540335,,10.1177/1091581811403504,Chemical and mechanistic toxicology evaluation of exon skipping phosphorodiamidate morpholino oligomers in mdx mice.,https://pubmed.ncbi.nlm.nih.gov/21540335/,PubMed abstract,409,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00438,P1,toxicity,toxicity_endpoint,358,no,B,329,log10; NCT06115993; CTR20232098,ASO,,,hepatic: hepatotoxicity,1051,42176239,,10.1007/s12072-026-11102-7,"Safety, pharmacokinetics and antiviral activity of AHB‑137 in healthy volunteers and chronic hepatitis B patients: a phase 1a/1b study.",https://pubmed.ncbi.nlm.nih.gov/42176239/,PubMed abstract,412,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00439,P1,toxicity,toxicity_endpoint,359,no,B,330,PCSK9; NCT04652726; inclisiran,siRNA,,,hepatic: hepatotoxicity,1155,41616799,,10.1016/S2213-8587(25)00351-1,"Efficacy and safety of inclisiran in adolescents with heterozygous familial hypercholesterolaemia (ORION-16): a two-part, randomised, multicentre clinical trial.",https://pubmed.ncbi.nlm.nih.gov/41616799/,PubMed abstract,413,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00440,P1,toxicity,toxicity_endpoint,361,no,B,332,PCSK9; inclisiran,siRNA,,,hepatic: hepatotoxicity,1179,42141407,,10.1186/s12872-026-05931-5,Comparative effectiveness and safety of inclisiran versus evolocumab and alirocumab: a 180-day real-world study.,https://pubmed.ncbi.nlm.nih.gov/42141407/,PubMed abstract,415,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00441,P1,toxicity,toxicity_endpoint,362,no,B,333,AUC0-inf; Zerlasiran,siRNA,,,hepatic: hepatotoxicity,1182,42108101,,10.5551/jat.66214,"A Phase 1 Study Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Zerlasiran in Japanese Participants.",https://pubmed.ncbi.nlm.nih.gov/42108101/,PubMed abstract,416,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00442,P1,toxicity,toxicity_endpoint,363,no,B,93,Inclisiran,ASO/siRNA mixed context,,,general safety: safety/tolerability,1201,41860775,,10.2459/JCM.0000000000001852,Inclisiran safety and efficacy in real-world clinical practice: results of a retrospective observational investigation.,https://pubmed.ncbi.nlm.nih.gov/41860775/,PubMed abstract,417,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00443,P1,toxicity,toxicity_endpoint,364,no,B,334,MC3; MC3-based,siRNA,,,hepatic: hepatotoxicity,1209,41708903,,10.1007/s13346-026-02076-y,Comparative analysis of clinically approved lipid nanoparticles for intranasal siRNA delivery against SARS-CoV-2.,https://pubmed.ncbi.nlm.nih.gov/41708903/,PubMed abstract,418,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00444,P1,toxicity,toxicity_endpoint,365,no,B,335,NCT04014335; RO7434656; sefaxersen,ASO,,,hepatic: hepatotoxicity,1308,41443406,,10.1016/j.kint.2025.11.017,A single-arm phase 2 trial of an investigational RNA therapeutic to complement factor B sefaxersen for treatment of IgA nephropathy.,https://pubmed.ncbi.nlm.nih.gov/41443406/,PubMed abstract,419,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00445,P1,toxicity,toxicity_endpoint,371,no,B,341,NCT04439539; FUW48; log10IU,siRNA,,,hepatic: hepatotoxicity,1775,41193172,,10.1136/gutjnl-2025-336592,Peginterferon alpha-2a add-on to siRNA JNJ-73763989 in untreated patients with HBeAg-positive chronic hepatitis B virus (HBV) infection: the phase 2 REEF-IT study.,https://pubmed.ncbi.nlm.nih.gov/41193172/,PubMed abstract,425,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00446,P1,toxicity,toxicity_endpoint,372,no,B,342,NCT05618379; Nusinersen,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,2417,41461996,,10.1007/s12325-025-03475-2,Effectiveness and Safety of Nusinersen Among Adults with 5q-Spinal Muscular Atrophy: A Multicenter Disease Registry in China.,https://pubmed.ncbi.nlm.nih.gov/41461996/,PubMed abstract,426,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00447,P1,toxicity,toxicity_endpoint,376,no,B,345,CORE2-TIMI; NCT05079919; NCT05552326,ASO,,,hepatic: hepatotoxicity,7173,41211918,,10.1056/NEJMoa2512761,Olezarsen for Managing Severe Hypertriglyceridemia and Pancreatitis Risk.,https://pubmed.ncbi.nlm.nih.gov/41211918/,PubMed abstract,430,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00448,P1,toxicity,toxicity_endpoint,378,no,B,346,viltolarsen,ASO,,,hepatic: hepatotoxicity,1274,39549635,,10.1016/j.braindev.2024.10.005,Safety and efficacy of viltolarsen treatment in patients with Duchenne muscular dystrophy: A retrospective study with 3-year follow-up.,https://pubmed.ncbi.nlm.nih.gov/39549635/,PubMed abstract,432,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00449,P1,toxicity,toxicity_endpoint,379,no,B,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,1541,40607962,,10.1002/cpdd.1569,"A Double-Blind, Placebo-Controlled, Single-Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-101, an RNAi Therapeutic Targeting HBV Infection.",https://pubmed.ncbi.nlm.nih.gov/40607962/,PubMed abstract,433,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00450,P1,toxicity,toxicity_endpoint,382,no,B,349,PCSK9; NCT05763875; Inclisiran,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,2280,40392667,,10.1016/j.jacc.2025.04.049,Safety and Lipid-Lowering Efficacy of Inclisiran Monotherapy in Patients Without ASCVD: The VICTORION-Mono Randomized Clinical Trial.,https://pubmed.ncbi.nlm.nih.gov/40392667/,PubMed abstract,436,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00451,P1,toxicity,toxicity_endpoint,384,no,B,244,inclisiran,ASO/siRNA mixed context,,,general safety: safety/tolerability,2510,40242982,,10.1093/eurjpc/zwaf214,Efficacy and safety of inclisiran based on background lipid-lowering treatment.,https://pubmed.ncbi.nlm.nih.gov/40242982/,PubMed abstract,438,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00452,P1,toxicity,toxicity_endpoint,386,no,B,196,Nusinersen,ASO,,,hepatic: hepatotoxicity,7632,41337773,,,Real-Time Ultrasound-Guided Intrathecal Delivery of Nusinersen in Adult Patients With Spinal Muscular Atrophy and Complex Spinal Anatomy.,https://pubmed.ncbi.nlm.nih.gov/41337773/,PubMed abstract,440,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00453,P1,toxicity,toxicity_endpoint,389,no,B,352,NCT04971928; AUC0; Bepirovirsen,ASO,,,hepatic: hepatotoxicity,1564,39268699,,10.1002/cpdd.1454,"A Phase 1, Single-Dose Study to Evaluate the Pharmacokinetics and Safety of Bepirovirsen in Adults with Hepatic Impairment and Healthy Participants (B-Assured).",https://pubmed.ncbi.nlm.nih.gov/39268699/,PubMed abstract,443,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00454,P1,toxicity,toxicity_endpoint,390,no,B,353,NCT05139810,ASO,,,hepatic: hepatotoxicity,1580,38819395,,10.1056/NEJMoa2402478,Efficacy and Safety of Donidalorsen for Hereditary Angioedema.,https://pubmed.ncbi.nlm.nih.gov/38819395/,PubMed abstract,444,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00455,P1,toxicity,toxicity_endpoint,392,no,B,354,CDR132L,ASO,,,general safety: safety/tolerability,2619,38269451,,10.1002/ejhf.3139,Efficacy and safety of CDR132L in patients with reduced left ventricular ejection fraction after myocardial infarction: Rationale and design of the HF-REVERT trial.,https://pubmed.ncbi.nlm.nih.gov/38269451/,PubMed abstract,446,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00456,P1,toxicity,toxicity_endpoint,393,no,B,355,caspase3,siRNA,,,hepatic: hepatotoxicity,3866,39146787,,10.1016/j.intimp.2024.112892,Delivery of SiRNA-PD-L1 by attenuated Salmonella in combination with oxaliplatin in a hepatocellular carcinoma mouse model.,https://pubmed.ncbi.nlm.nih.gov/39146787/,PubMed abstract,447,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00457,P1,toxicity,toxicity_endpoint,394,no,B,356,NCT03672188; log10; elebsiran,siRNA,,,hepatic: hepatotoxicity,10687,39389081,,10.1016/S2468-1253(24)00237-1,VIR-2218 (elebsiran) plus pegylated interferon-alfa-2a in participants with chronic hepatitis B virus infection: a phase 2 study.,https://pubmed.ncbi.nlm.nih.gov/39389081/,PubMed abstract,448,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00458,P1,toxicity,toxicity_endpoint,398,no,B,206,Inclisiran,siRNA,,,hepatic: hepatotoxicity,2301,38057066,,10.1016/j.jacc.2023.10.007,Safety and Tolerability of Inclisiran for Treatment of Hypercholesterolemia in 7 Clinical Trials.,https://pubmed.ncbi.nlm.nih.gov/38057066/,PubMed abstract,452,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00459,P1,toxicity,toxicity_endpoint,399,no,B,359,NCT03982186,siRNA,,,hepatic: hepatotoxicity,2303,37442152,,10.1016/S2468-1253(23)00148-6,"Efficacy and safety of the siRNA JNJ-73763989 and the capsid assembly modulator JNJ-56136379 (bersacapavir) with nucleos(t)ide analogues for the treatment of chronic hepatitis B virus infection (REEF-1): a multicentre, double-blind, active-controlled, randomised, phase 2b trial.",https://pubmed.ncbi.nlm.nih.gov/37442152/,PubMed abstract,453,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00460,P1,toxicity,toxicity_endpoint,400,no,B,360,Volanesorsen,ASO,,,hepatic: hepatotoxicity,2681,37100699,,10.1016/j.jacl.2023.03.007,Volanesorsen and triglyceride levels in familial chylomicronemia syndrome: Long-term efficacy and safety data from patients in an open-label extension trial.,https://pubmed.ncbi.nlm.nih.gov/37100699/,PubMed abstract,454,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00461,P1,toxicity,toxicity_endpoint,401,no,B,361,PCSK9; NCT03060577; inclisiran,siRNA,,,hepatic: hepatotoxicity,2697,36620965,,10.1016/S2213-8587(22)00353-9,Long-term efficacy and safety of inclisiran in patients with high cardiovascular risk and elevated LDL cholesterol (ORION-3): results from the 4-year open-label extension of the ORION-1 trial.,https://pubmed.ncbi.nlm.nih.gov/36620965/,PubMed abstract,455,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00462,P1,toxicity,toxicity_endpoint,402,no,B,276,SPL84,ASO,,,hepatic: hepatotoxicity,12058,37799089,,10.1080/17425255.2023.2266361,"The safety and toxicity profile of SPL84, an inhaled antisense oligonucleotide for treatment of cystic fibrosis patients with the 3849 +10kb C->T mutation, supports a Phase 1/2 clinical study.",https://pubmed.ncbi.nlm.nih.gov/37799089/,PubMed abstract,456,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00463,P1,toxicity,toxicity_endpoint,411,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,2109,22704926,,10.1016/j.vaccine.2012.05.074,Safety and immunogenicity of different two-dose regimens of an investigational hepatitis B vaccine (hepatitis B surface antigen co-administered with an immunostimulatory phosphorothioate oligodeoxyribonucleotide) in healthy young adults.,https://pubmed.ncbi.nlm.nih.gov/22704926/,PubMed abstract,465,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00464,P1,toxicity,toxicity_endpoint,412,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,2110,22326642,,10.1016/j.vaccine.2012.01.087,Comparison of safety and immunogenicity of two doses of investigational hepatitis B virus surface antigen co-administered with an immunostimulatory phosphorothioate oligodeoxyribonucleotide and three doses of a licensed hepatitis B vaccine in healthy adults 18-55 years of age.,https://pubmed.ncbi.nlm.nih.gov/22326642/,PubMed abstract,466,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00465,P1,toxicity,toxicity_endpoint,444,no,B,400,siSTAT3 silk sphere formulation,siRNA,STAT3,,general safety: cell viability/cytotoxicity,1829,40584785,PMC12204100,10.2147/IJN.S519906,"Developing the Strategy to Use Silk Spheres for Efficient, Targeted Delivery of Oligonucleotide Therapeutics to Cancer Cells.",https://pubmed.ncbi.nlm.nih.gov/40584785/,Results and Discussion > Analysis of the Gene Silencing Effect Exerted of siRNA Delivered Using H2.1MS1KN and H2.1MS1:MS2KN Spheres; paragraph 47,503,cell line,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00466,P1,toxicity,toxicity_endpoint,469,no,B,425,fully chemically modified hsiRNAs,siRNA,,,general safety: systemic safety/tolerability,12636,29432571,PMC5861422,10.1093/nar/gky037,Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo.,https://pubmed.ncbi.nlm.nih.gov/29432571/,RESULTS > Full chemical stabilization enables productive silencing in vivo.; paragraph 35,528,mouse,,blood chemistry; liver; kidney,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00467,P1,toxicity,toxicity_endpoint,474,no,B,430,ISIS 405879 2'-MOE ASO,ASO,,,hematological: thrombocytopenia,2090,37093125,,10.1089/nat.2022.0042,Complement C3d/C4d Deposition on Platelets Correlates with 2'-O-Methoxyethyl Antisense Oligonucleotide-Induced Thrombocytopenia in Monkeys.,https://pubmed.ncbi.nlm.nih.gov/37093125/,PubMed abstract,533,cynomolgus monkey,,platelets; serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00468,P1,toxicity,toxicity_endpoint,743,no,B,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,general safety: systemic safety/tolerability,1063,41876647,,10.1038/s41591-026-04262-4,LRRK2-targeting antisense oligonucleotide in Parkinson's disease: a phase 1 randomized controlled trial.,https://pubmed.ncbi.nlm.nih.gov/41876647/,PubMed abstract,1020,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00469,P1,toxicity,toxicity_endpoint,754,no,B,583,siRNA-micelles,siRNA,,,hepatic: hepatotoxicity,3333,41443351,,10.1016/j.jconrel.2025.114568,Structurally-defined non-cationic docosahexaenoic acid based siRNA-micelles for safe and effective combinatorial glioblastoma therapy.,https://pubmed.ncbi.nlm.nih.gov/41443351/,PubMed abstract,1031,mouse,,blood/serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00470,P1,toxicity,toxicity_endpoint,759,no,B,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic: hepatotoxicity,4787,42108271,,10.1038/s41591-026-04408-4,The microRNA inhibitor CDR132L in patients with reduced left ventricular ejection fraction after myocardial infarction: a randomized phase 2 trial.,https://pubmed.ncbi.nlm.nih.gov/42108271/,PubMed abstract,1036,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00471,P1,toxicity,toxicity_endpoint,812,no,B,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic: hepatotoxicity,1014,39798707,,10.1016/j.jhep.2024.12.046,"AZD2693, a PNPLA3 antisense oligonucleotide, for the treatment of MASH in 148M homozygous participants: Two randomized phase I trials.",https://pubmed.ncbi.nlm.nih.gov/39798707/,PubMed abstract,1089,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00472,P1,toxicity,toxicity_endpoint,819,no,B,986,"unspecified ASO (v1 extraction artefact, pending source re-verification)",ASO,,,hepatic: hepatotoxicity,1842,40280496,,10.1016/j.spinee.2025.04.021,NF-kappa B Oligo DNA decoy provides 12 months of pain relief and disc height restoration for patients with chronic discogenic low back pain-a randomized clinical trial.,https://pubmed.ncbi.nlm.nih.gov/40280496/,PubMed abstract,1096,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00473,P1,toxicity,toxicity_endpoint,830,no,B,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,3790,39723844,,10.1021/acsabm.4c01319,"Application of Strontium Chloride Hexahydrate to Synthesize Strontium-Substituted Carbonate Apatite as a pH-Sensitive, Biologically Safe, and Highly Efficient siRNA Nanocarrier.",https://pubmed.ncbi.nlm.nih.gov/39723844/,PubMed abstract,1107,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00474,P1,toxicity,toxicity_endpoint,903,no,B,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,1166,37497888,,10.1002/ajh.27015,"SLN124, a GalNAc conjugated 19-mer siRNA targeting tmprss6, reduces plasma iron and increases hepcidin levels of healthy volunteers.",https://pubmed.ncbi.nlm.nih.gov/37497888/,PubMed abstract,1180,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00475,P1,toxicity,toxicity_endpoint,905,no,B,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,4042,36513186,,10.1016/j.jhep.2022.11.025,"A phase I/II study of ARO-HSD, an RNA interference therapeutic, for the treatment of non-alcoholic steatohepatitis.",https://pubmed.ncbi.nlm.nih.gov/36513186/,PubMed abstract,1182,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00476,P1,toxicity,toxicity_endpoint,909,no,B,619,single-dose,siRNA,,,hepatic: hepatotoxicity,12017,37524230,,10.1016/j.jhep.2023.07.026,First-in-human randomized study of RNAi therapeutic RG6346 for chronic hepatitis B virus infection.,https://pubmed.ncbi.nlm.nih.gov/37524230/,PubMed abstract,1186,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00477,P1,toxicity,toxicity_endpoint,950,no,B,299,nusinersen,ASO,,,hepatic: hepatotoxicity,15203,27939059,,10.1016/S0140-6736(16)31408-8,"Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.",https://pubmed.ncbi.nlm.nih.gov/27939059/,PubMed abstract,1227,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00478,P1,toxicity,toxicity_endpoint,1220,no,B,599,inotersen,ASO,,,general safety: systemic safety/tolerability,11924,37768671,PMC10540057,10.1001/jama.2023.18688,Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy.,https://pubmed.ncbi.nlm.nih.gov/37768671/,PubMed abstract,1497,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00479,P1,toxicity,toxicity_endpoint,1317,no,B,1081,unspecified siRNA (PMID:41797405),siRNA,,,hepatic: hepatotoxicity,1737,41797405,,10.1021/acsnano.5c16693,Lung-Accumulating Mucin 5AC Silencing by Noninflammatory Lipid Nanoparticles for Asthma Treatment.,https://pubmed.ncbi.nlm.nih.gov/41797405/,PubMed abstract,1594,human,,lung,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00480,P1,toxicity,toxicity_endpoint,1329,no,B,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,4516,42173110,,10.1016/S2468-1253(26)00058-0,"The small interfering RNA imdusiran as single and multiple doses in healthy, randomised individuals and non-randomised individuals with chronic hepatitis B (AB-729-001): a phase 1a/b trial.",https://pubmed.ncbi.nlm.nih.gov/42173110/,PubMed abstract,1606,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00481,P1,toxicity,toxicity_endpoint,1387,no,B,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,1478,40179932,,10.1016/S0140-6736(25)00507-0,"Durability and efficacy of solbinsiran, a GalNAc-conjugated siRNA targeting ANGPTL3, in adults with mixed dyslipidaemia (PROLONG-ANG3): a double-blind, randomised, placebo-controlled, phase 2 trial.",https://pubmed.ncbi.nlm.nih.gov/40179932/,PubMed abstract,1664,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00482,P1,toxicity,toxicity_endpoint,1388,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,toxicity: intraocular pressure spike / ocular safety,3960,38157582,,10.1016/j.bbrc.2023.149397,"Silencing of ocular transthyretin, a gene responsible for hereditary transthyretin amyloidosis, by intravitreal injection of an siRNA conjugate into rabbit eyes.",https://pubmed.ncbi.nlm.nih.gov/38157582/,PubMed abstract sentence 3,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00483,P1,toxicity,toxicity_endpoint,1397,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: GalNAc3 conjugation potency increase,1465,33431610,,10.1124/jpet.120.000222,"Safety, Pharmacokinetic, and Pharmacodynamic Evaluation of a 2'-(2-Methoxyethyl)-D-ribose Antisense Oligonucleotide-Triantenarry N-Acetyl-galactosamine Conjugate that Targets the Human Transmembrane Protease Serine 6.",https://pubmed.ncbi.nlm.nih.gov/33431610/,PubMed abstract sentence 4,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00484,P1,toxicity,toxicity_endpoint,1401,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: VHP peptide modification targeted siRNA-exosome delivery,3325,41491215,PMC12781530,10.1186/s12951-025-03954-2,Endothelial-targeted modification of ginseng-derived exosomes for IL-6 SiRNA delivery ameliorates hepatic ischemia-reperfusion injury.,https://pubmed.ncbi.nlm.nih.gov/41491215/,PubMed abstract sentence 4,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00485,P1,toxicity,toxicity_endpoint,1402,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: PDA/CD11b modification improves siRNA nanoparticle release and uptake,3501,40977834,PMC12446780,10.1016/j.mtbio.2025.102226,CD11b-modified ROS/pH-responsive nanoparticles co-deliver Dioscin and siRNA to improve cardiac repair after myocardial infarction by reducing neutrophil recruitment.,https://pubmed.ncbi.nlm.nih.gov/40977834/,PubMed title,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00486,P1,toxicity,toxicity_endpoint,1403,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: fluorocarbon/aptamer comodification enhances siRNA delivery,3571,40747059,PMC12308423,10.1021/jacsau.5c00474,Programmable Comodification of DNA Soccer Framework with Fluorocarbon and Aptamer for Efficient siRNA Delivery.,https://pubmed.ncbi.nlm.nih.gov/40747059/,PubMed abstract sentence 4,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00487,P1,toxicity,toxicity_endpoint,1404,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: cholesterol-siRNA conjugate liver macrophage delivery,4047,36318487,,10.1089/nat.2022.0038,Cholesterol-Conjugated siRNA Silencing Tnf for the Treatment of Liver Macrophage-Mediated Acute Inflammation in Nonalcoholic Fatty Liver Disease.,https://pubmed.ncbi.nlm.nih.gov/36318487/,PubMed abstract sentence 7,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00488,P1,toxicity,toxicity_endpoint,1405,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: chemically modified siRNA allele-specific silencing,4061,36192390,PMC9530163,10.1038/s41467-022-33061-x,Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models.,https://pubmed.ncbi.nlm.nih.gov/36192390/,PubMed abstract sentence 2,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00489,P1,toxicity,toxicity_endpoint,1406,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: 2'-O-methyl/5' stabilization improves placental siRNA,4069,35847173,PMC9263991,10.1016/j.omtn.2022.06.009,Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1.,https://pubmed.ncbi.nlm.nih.gov/35847173/,PubMed abstract sentence 4,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00490,P1,toxicity,toxicity_endpoint,1407,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: Pip6a conjugation enhances PMO muscle delivery,4212,31479430,PMC6819114,10.1172/JCI128205,Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice.,https://pubmed.ncbi.nlm.nih.gov/31479430/,PubMed abstract sentence 4,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00491,P1,toxicity,toxicity_endpoint,1408,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: sgc8 aptamer conjugation improves siRNA nanoparticle targeting,12442,39583320,PMC11585994,10.2147/IJN.S477597,Aptamer sgc8-Modified PAMAM Nanoparticles for Targeted siRNA Delivery to Inhibit BCL11B in T-Cell Acute Lymphoblastic Leukemia.,https://pubmed.ncbi.nlm.nih.gov/39583320/,PubMed abstract sentence 5,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00492,P1,toxicity,toxicity_endpoint,1409,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: fully modified siRNA improves tissue retention,12636,29432571,PMC5861422,10.1093/nar/gky037,Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo.,https://pubmed.ncbi.nlm.nih.gov/29432571/,PubMed abstract sentence 4,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00493,P1,toxicity,toxicity_endpoint,1410,no,B,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: 5'-vinylphosphonate improves siRNA accumulation/silencing,12652,28591791,PMC5570069,10.1093/nar/gkx507,5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo.,https://pubmed.ncbi.nlm.nih.gov/28591791/,PubMed abstract sentence 4,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00494,P2,offtarget,offtarget_evidence,19,no,C,134,siRNA candidate from PMID 40737088,siRNA,,,seed-mediated off-target effect,1497,40737088,PMC12309379,10.1093/nar/gkaf692,Single alkyl phosphonate modification of the siRNA backbone in the seed region enhances specificity and therapeutic profile.,https://pubmed.ncbi.nlm.nih.gov/40737088/,Introduction; paragraph 5,137,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00495,P2,offtarget,offtarget_evidence,20,no,C,135,siRNA candidate from PMID 41646886,siRNA,,,seed-mediated off-target effect,1154,41646886,PMC12870468,10.1016/j.omtn.2026.102835,2'-Ribose-modified nucleotides: A strategy for reducing off-target effects of oligonucleotide drugs.,https://pubmed.ncbi.nlm.nih.gov/41646886/,Discussion; paragraph 27,139,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00496,P2,offtarget,offtarget_evidence,21,no,C,136,siRNA candidate from PMID 39813044,siRNA,,,sequence-specificity/off-target evidence,12147,39813044,PMC12522049manuscript-id: NIHMS2116935,10.1021/acschembio.4c00824,Amide Internucleoside Linkages Suppress the MicroRNA-like Off-Target Activity of Short Interfering RNA.,https://pubmed.ncbi.nlm.nih.gov/39813044/,Results and Discussion; paragraph 13,140,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00497,P2,offtarget,offtarget_evidence,22,no,C,137,siRNA candidate from PMID 39231537,siRNA,,,seed-mediated off-target effect,12077,39231537,PMC11472056,10.1093/nar/gkae741,Synthesis of 2'-formamidonucleoside phosphoramidites for suppressing the seed-based off-target effects of siRNAs.,https://pubmed.ncbi.nlm.nih.gov/39231537/,Results and discussion > Genome-wide evaluation of off-target activities; paragraph 77,141,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00498,P2,offtarget,offtarget_evidence,23,no,C,138,siRNA candidate from PMID 35921401,siRNA,,,seed-mediated off-target effect,1484,35921401,PMC9389587,10.1021/jacs.2c01679,"Role of a ""Magic"" Methyl: 2'-Deoxy-2'-α-F-2'-β-C-methyl Pyrimidine Nucleotides Modulate RNA Interference Activity through Synergy with 5'-Phosphate Mimics and Mitigation of Off-Target Effects.",https://pubmed.ncbi.nlm.nih.gov/35921401/,Results and Discussion > Off-Target Effects of siRNAs Modified with 2′-F/Me; Figure 5 caption,142,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00499,P2,offtarget,offtarget_evidence,24,no,C,139,siRNA candidate from PMID 35071927,siRNA,,,seed-mediated off-target effect,2255,35071927,PMC8771963,10.1021/acsomega.1c06455,siRNA Seed Region Is Divided into Two Functionally Different Domains in RNA Interference in Response to 2'-OMe Modifications.,https://pubmed.ncbi.nlm.nih.gov/35071927/,Introduction; paragraph 4,143,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00500,P2,offtarget,offtarget_evidence,25,no,C,140,siRNA candidate from PMID 35205363,siRNA,,,seed-mediated off-target effect,2308,35205363,PMC8872465,10.3390/genes13020319,The siRNA Off-Target Effect Is Determined by Base-Pairing Stabilities of Two Different Regions with Opposite Effects.,https://pubmed.ncbi.nlm.nih.gov/35205363/,1. Introduction; paragraph 4,144,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00501,P2,offtarget,offtarget_evidence,26,no,C,141,ASO candidate from PMID 34388351,ASO,,,hybridization/mismatch off-target effect,2113,34388351,PMC8713556,10.1089/nat.2020.0921,Transcriptome-Wide Off-Target Effects of Steric-Blocking Oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/34388351/,Introduction; paragraph 4,145,experimental model,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00502,P2,offtarget,offtarget_evidence,27,no,C,142,siRNA candidate from PMID 29450406,siRNA,,,seed-mediated off-target effect,2117,29450406,PMC5808362,10.1021/acsomega.7b00291,Chemical Modification of the siRNA Seed Region Suppresses Off-Target Effects by Steric Hindrance to Base-Pairing with Targets.,https://pubmed.ncbi.nlm.nih.gov/29450406/,Results and Discussion; paragraph 20,146,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00503,P2,offtarget,offtarget_evidence,28,no,C,143,siRNA candidate from PMID 21687525,siRNA,,,seed-mediated off-target effect,2246,21687525,PMC3109444,10.4161/adna.1.1.12204,In vivo efficacy and off-target effects of locked nucleic acid (LNA) and unlocked nucleic acid (UNA) modified siRNA and small internally segmented interfering RNA (sisiRNA) in mice bearing human tumor xenografts.,https://pubmed.ncbi.nlm.nih.gov/21687525/,Abstract; paragraph 1,147,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00504,P2,offtarget,offtarget_evidence,201,no,C,435,siRNAs,siRNA,,,seed-mediated off-target effect,4163,33577685,PMC7969009,10.1093/nar/gkab050,siRNAs containing 2'-fluorinated Northern-methanocarbacyclic (2'-F-NMC) nucleotides: in vitro and in vivo RNAi activity and inability of mitochondrial polymerases to incorporate 2'-F-NMC NTPs.,https://pubmed.ncbi.nlm.nih.gov/33577685/,RESULTS AND DISCUSSION > On-target and off-target activities of siRNAs modified with 2′-F-NMC; paragraph 42,678,rat,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00505,P2,offtarget,offtarget_evidence,281,no,C,733,bulge-siRNA,siRNA,,,seed-mediated off-target effect,15373,21673662,PMC3182346,10.1038/mt.2011.109,Modified siRNA structure with a single nucleotide bulge overcomes conventional siRNA-mediated off-target silencing.,https://pubmed.ncbi.nlm.nih.gov/21673662/,PubMed abstract,1722,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00506,P2,offtarget,offtarget_evidence,539,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,TLR3-mediated off-target immune activation,4056,36365241,PMC9692777,10.3390/pharmaceutics14112424,A β-Cyclodextrin-Based Nanoparticle with Very High Transfection Efficiency Unveils siRNA-Activated TLR3 Responses in Human Prostate Cancer Cells.,https://pubmed.ncbi.nlm.nih.gov/36365241/,PMC full text high-precision preselection,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00507,P2,toxicity,toxicity_endpoint,7,no,C,20,TP-siRC@tHyNPs (CYP3A4-siRNA),siRNA,CYP3A4,,general safety: safety/tolerability,1822,40700483,PMC12285696,10.1126/sciadv.adv6990,Engineering hybrid nanoparticles for targeted codelivery of triptolide and CYP3A4-siRNA against pulmonary metastatic melanoma.,https://pubmed.ncbi.nlm.nih.gov/40700483/,RESULTS > Anti-PMM effect of TP-siRC@tHyNPs in vivo; paragraph 30,20,mouse,pulmonary metastatic melanoma mouse model treated with TP-siRC@tHyNPs,serum; lung; hematology,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00508,P2,toxicity,toxicity_endpoint,11,no,C,25,nebulized siIL-1beta.2 from PMID 41898774,siRNA,IL1B,,general safety: cytotoxicity,3211,41898774,PMC13026121,10.3390/ijms27062915,Functional siRNA Delivery via Jet Nebulization: Proof-of-Concept IL-1ß Silencing in Macrophage-like THP-1 Cells.,https://pubmed.ncbi.nlm.nih.gov/41898774/,2. Results > 2.6. Knockdown of Endogenously Expressed IL-1β by Nebulized siRNA; paragraph 27,25,human cell line,,THP-1 macrophage-like cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00509,P2,toxicity,toxicity_endpoint,15,no,C,29,siSNCA PEI/PPI nanoparticle panel from PMID 40978531,siRNA,SNCA,,general safety: cytotoxicity,3500,40978531,PMC12447568,10.1016/j.omtn.2025.102671,Nose-to-brain siRNA delivery by PEI/PPI-based nanoparticles reduces α-synuclein expression in a Parkinson's disease mouse model.,https://pubmed.ncbi.nlm.nih.gov/40978531/,Results > PEI/PPI-based nanoparticles reveal low cytotoxic potential and polymer-dependently knock down SNCA mRNA in vitro; paragraph 6,29,human cell line,,differentiated SH-SY5Y,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00510,P2,toxicity,toxicity_endpoint,18,no,C,32,Cet/siFAK@ZIF-8@TCM from PMID 40735032,siRNA,PTK2,,general safety: cytotoxicity,3574,40735032,PMC12305587,10.1016/j.jpha.2025.101203,pH-responsive biomimetic zeolitic imidazolate framework-based nanoparticles for co-delivery of cetuximab and siRNA in synergistic therapy of laryngeal squamous cell carcinoma.,https://pubmed.ncbi.nlm.nih.gov/40735032/,"Results > In vitro evaluation of cytotoxicity, homologous targeting, and immune escape; paragraph 32",32,human cell line,,TU177; NHBEC,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00511,P2,toxicity,toxicity_endpoint,20,no,C,34,DCA-siRNADUX4/DU01 from PMID 40673060,siRNA,DUX4,,general safety: transcriptome toxicity,3598,40673060,PMC12264612,10.1016/j.omtm.2025.101513,A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic.,https://pubmed.ncbi.nlm.nih.gov/40673060/,Results > Transcriptomic analysis of FSHD myotubes after siRNADUX4 treatment; paragraph 9,34,human cell model,,FSHD patient-derived myotubes,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00512,P2,toxicity,toxicity_endpoint,26,no,C,40,pVEC-ASO panel from PMID 38391508,ASO,,,general safety: cytotoxicity,1417,38391508,PMC10885958,10.3390/antibiotics13020122,General and Specific Cytotoxicity of Chimeric Antisense Oligonucleotides in Bacterial Cells and Human Cell Lines.,https://pubmed.ncbi.nlm.nih.gov/38391508/,3. Results > 3.3. Cell Growth Inhibition by Antisense Oligonucleotides; paragraph 21,40,human cell line,,Lep3 fibroblasts; MDA-MB-231; HT29,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00513,P2,toxicity,toxicity_endpoint,30,no,C,44,GxS5/siRNA polyplexes from PMID 39263399,siRNA,tdTomato,,general safety: cytotoxicity,3851,39263399,PMC11386170,10.1039/d4na00363b,Efficient siRNA delivery to murine melanoma cells via a novel genipin-based nano-polymer.,https://pubmed.ncbi.nlm.nih.gov/39263399/,Results > Efficacy as siRNA carrier for RNA interference; paragraph 52,44,mouse cell line,,B16F10,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00514,P2,toxicity,toxicity_endpoint,31,no,C,45,GBE-PLL/H2O-PLL solid lipid nanoparticle siRNA carriers from PMID 39684010,siRNA,,,general safety: apoptosis/cytotoxicity,12064,39684010,PMC11644305,10.3390/polym16233265,Bio-Inspired Polymeric Solid Lipid Nanoparticles for siRNA Delivery: Cytotoxicity and Cellular Uptake In Vitro.,https://pubmed.ncbi.nlm.nih.gov/39684010/,3. Results > 3.6. Caspase 3/7 Activity; paragraph 35,45,human cell lines,,HEK293; SH-SY5Y,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00515,P2,toxicity,toxicity_endpoint,32,no,C,46,P-E/S Lip PD-L1 siRNA nanovesicle from PMID 39068444,siRNA,CD274,,general safety: cytotoxicity,12122,39068444,PMC11282766,10.1186/s12951-024-02700-4,PD-L1 siRNA hitched polyethyleneimine-elastase constituting nanovesicle induces tumor immunogenicity and PD-L1 silencing for synergistic antitumor immunotherapy.,https://pubmed.ncbi.nlm.nih.gov/39068444/,Results > Synthesis and functional validation of P-E/S lip in vitro; paragraph 26,46,mouse and human cell lines,,B16-F10; MDA-MB-231; MCF-10A; lymphocytes,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00516,P2,toxicity,toxicity_endpoint,33,no,C,47,SWNT-PL-PEG-S-S-siLUC conjugate from PMID 39627280,siRNA,LUC,,general safety: acute systemic safety,12435,39627280,PMC11615393,10.1038/s41598-024-80646-1,Polyethylene glycol-phospholipid functionalized single-walled carbon nanotubes for enhanced siRNA systemic delivery.,https://pubmed.ncbi.nlm.nih.gov/39627280/,Results > Significant knock-down efficacy using SWNT-PL-PEG-S-S-siRNA conjugate in a mouse xenograft model; paragraph 19,47,mouse,,xenograft model,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00517,P2,toxicity,toxicity_endpoint,43,no,C,57,PEG-SSN/siRNA nanoparticle formulation from PMID 36412852,siRNA,EGFR,,general safety: cytotoxicity and hemolysis,2305,36412852,PMC9680366,10.3390/jfb13040211,PEGylated Strontium Sulfite Nanoparticles with Spontaneously Formed Surface-Embedded Protein Corona Restrict Off-Target Distribution and Accelerate Breast Tumour-Selective Delivery of siRNA.,https://pubmed.ncbi.nlm.nih.gov/36412852/,3. Results > 3.8. Biocompatibility Assay; paragraph 41,57,human and mouse cell lines,,MCF-7; 4T1; HEK293,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00518,P2,toxicity,toxicity_endpoint,45,no,C,59,GalNAc-G-quadruplex oligonucleotide panel from PMID 35745067,ASO,,,general safety: cytotoxicity,4073,35745067,PMC9228010,10.3390/molecules27123944,Properties of Parallel Tetramolecular G-Quadruplex Carrying N-Acetylgalactosamine as Potential Enhancer for Oligonucleotide Delivery to Hepatocytes.,https://pubmed.ncbi.nlm.nih.gov/35745067/,2. Results > 2.4. Toxicity Assays; paragraph 17,59,human cell lines,,HeLa; HepG2,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00519,P2,toxicity,toxicity_endpoint,46,no,C,60,A10-3.2/siCAT-1/3WJ nanodroplets from PMID 35037525,siRNA,SLC7A1,,general safety: cytotoxicity,4096,35037525,PMC8765274,10.1080/10717544.2022.2026532,Utilizing RNA nanotechnology to construct negatively charged and ultrasound-responsive nanodroplets for targeted delivery of siRNA.,https://pubmed.ncbi.nlm.nih.gov/35037525/,Results > In vitro bio-safety of NDs; paragraph 33,60,human cell lines,,22RV1; 16HBE,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00520,P2,toxicity,toxicity_endpoint,49,no,C,63,LNA-ASO renal histopathology panel from PMID 34629736,ASO,,,renal: renal histopathology,1636,34629736,PMC8484925,10.1293/tox.2021-0007,Karnovsky's fixative prevents artifacts appearing as vacuolation derived from tissue processing in kidneys treated with antisense oligonucleotide.,https://pubmed.ncbi.nlm.nih.gov/34629736/,article body; paragraph 10,63,rat,,kidney; serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00521,P2,toxicity,toxicity_endpoint,50,no,C,64,LUNAR-REPU910 siRNA from PMID 33738134,siRNA,AR,,general safety: neurodevelopmental safety,1646,33738134,PMC7937577,10.1016/j.omtn.2021.02.007,Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS.,https://pubmed.ncbi.nlm.nih.gov/33738134/,Results > Selective suppression of polyQ-expanded AR by i.c.v.-injected LUNAR-REPU910 siRNA; paragraph 12,64,mouse,,CNS,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00522,P2,toxicity,toxicity_endpoint,52,no,C,66,2'-O-methoxyethyl chimeric ASO tolerability dataset from PMID 34242101,ASO,,,general safety: tolerability events,2741,34242101,PMC8713270,10.1089/nat.2020.0917,"Improvements in the Tolerability Profile of 2'-O-Methoxyethyl Chimeric Antisense Oligonucleotides in Parallel with Advances in Design, Screening, and Other Methods.",https://pubmed.ncbi.nlm.nih.gov/34242101/,Results > Incidence and percentage of doses leading to adverse events; paragraph 22,66,human clinical aggregate,,injection-site and flu-like tolerability events,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00523,P2,toxicity,toxicity_endpoint,53,no,C,67,dual-RGD/EGFR siRNA protein nanocarrier from PMID 34959463,siRNA,EGFR,,general safety: systemic safety,4106,34959463,PMC8703291,10.3390/pharmaceutics13122182,Non-Cationic RGD-Containing Protein Nanocarrier for Tumor-Targeted siRNA Delivery.,https://pubmed.ncbi.nlm.nih.gov/34959463/,3. Results > 3.9. Toxicity Assessment; paragraph 31,67,mouse; human cell lines,,brain; heart; kidney; liver; lung; intestine; muscle; spleen; HEK293T,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00524,P2,toxicity,toxicity_endpoint,57,no,C,71,306-O12B-3 ASO/LNP formulation from PMID 32160706,ASO,GFP,,general safety: cytotoxicity,1493,32160706,PMC7036716,10.1016/j.omtn.2020.01.018,Efficient Delivery of Antisense Oligonucleotides Using Bioreducible Lipid Nanoparticles In Vitro and In Vivo.,https://pubmed.ncbi.nlm.nih.gov/32160706/,Results > Screening the Bioreducible Lipids for ASO Delivery In Vitro; paragraph 16,71,human cell line,,GFP-HEK,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00525,P2,toxicity,toxicity_endpoint,67,no,C,82,Bepirovirsen,ASO,,,hepatic: hepatotoxicity,1075,41085094,PMC12856967,10.1002/cpdd.1615,"A Phase 1, Randomized, Open-Label, Parallel Group Study to Evaluate the Relative Bioavailability and Safety of Subcutaneous Bepirovirsen when Delivered from a Vial or Prefilled Syringe Fitted with a Safety Syringe Device in Healthy Adult Participants.",https://pubmed.ncbi.nlm.nih.gov/41085094/,Abstract; paragraph 1,82,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00526,P2,toxicity,toxicity_endpoint,74,no,C,89,siRNA candidate from PMID 40314193,siRNA,,,immunotoxicity: immune activation,3700,40314193,PMC12051533,10.1080/13880209.2025.2498169,Delivery of small interfering RNA through lyophilized natural lipid nanoparticles: effects of natural lipid selection.,https://pubmed.ncbi.nlm.nih.gov/40314193/,Introduction; paragraph 7,89,cell line,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00527,P2,toxicity,toxicity_endpoint,75,no,C,90,siRNA candidate from PMID 41214041,siRNA,,,hepatic: hepatotoxicity,7859,41214041,PMC12603185,10.1038/s41598-025-22922-2,LINC02178 drives lung adenocarcinoma progression and serves as a therapeutic target for nanodelivery-based intervention.,https://pubmed.ncbi.nlm.nih.gov/41214041/,Results > Evaluation of the cytotoxicity and siRNA transfection efficiency of LaN; paragraph 27,90,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00528,P2,toxicity,toxicity_endpoint,77,no,C,92,siRNA candidate from PMID 39896757,siRNA,,,renal: renal safety,12278,39896757,PMC11782793,10.34133/bmr.0136,Isoprenaline-Modified Polyethyleneimine as an Efficient Gene Delivery System for Targeted Asthma Therapy and Airway Remodeling Inhibition.,https://pubmed.ncbi.nlm.nih.gov/39896757/,Discussion; paragraph 45,92,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00529,P2,toxicity,toxicity_endpoint,83,no,C,98,Imetelstat,ASO/siRNA mixed context,,,hematologic: platelet/bleeding safety,8005,41135032,PMC12614338manuscript-id: NIHMS2109501,10.1200/JCO-25-01369,US Food and Drug Administration Approval Summary: Imetelstat for Selected Patients With Low- to Intermediate-1 Risk Myelodysplastic Syndromes With Transfusion-Dependent Anemia.,https://pubmed.ncbi.nlm.nih.gov/41135032/,Abstract; paragraph 1,98,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00530,P2,toxicity,toxicity_endpoint,87,no,C,105,Alox15-targeting siRNA from PMID 41847300,siRNA,,,renal: renal safety,1202,41847300,PMC12990253,10.1016/j.bbrep.2026.102534,Dual knockdown of Alox15 and TGF-β1 by lipid nanoparticle-delivered siRNA in bleomycin-induced pulmonary fibrosis.,https://pubmed.ncbi.nlm.nih.gov/41847300/,Results > Assessing in vivo LNP safety: biochemical indicators and vital organ health; paragraph 29,105,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00531,P2,toxicity,toxicity_endpoint,91,no,C,109,Imetelstat,ASO,,,immunotoxicity: immune activation,2111,41390988,PMC12702135,10.1111/cts.70431,"No Effect of Immunogenicity on Pharmacokinetics, Efficacy, and Safety of the Oligonucleotide Telomerase Inhibitor Imetelstat in Lower-Risk Myelodysplastic Syndromes.",https://pubmed.ncbi.nlm.nih.gov/41390988/,Abstract; paragraph 1,109,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00532,P2,toxicity,toxicity_endpoint,92,no,C,110,siRNA candidate from PMID 41625362,siRNA,,,immunotoxicity: immune activation,3396,41625362,PMC12859616,10.1016/j.mtbio.2025.102267,Tumor-homing exosomes enable targeted delivery of siRNA and isoimperatorin for overcoming BTK inhibitor resistance in DLBCL.,https://pubmed.ncbi.nlm.nih.gov/41625362/,Discussion; paragraph 29,110,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00533,P2,toxicity,toxicity_endpoint,104,no,C,82,Bepirovirsen,ASO,,,hepatic: hepatotoxicity,1460,35971951,PMC9804925,10.1002/cpdd.1154,"Preclinical and Phase 1 Assessment of Antisense Oligonucleotide Bepirovirsen in Hepatitis B Virus-Transgenic Mice and Healthy Human Volunteers: Support for Clinical Dose Selection and Evaluation of Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses.",https://pubmed.ncbi.nlm.nih.gov/35971951/,Discussion; paragraph 52,122,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00534,P2,toxicity,toxicity_endpoint,108,no,C,125,ASO candidate from PMID 29107969,ASO,,,immunotoxicity: immune activation,2321,29107969,PMC5673186,10.1371/journal.pone.0187574,Assessing single-stranded oligonucleotide drug-induced effects in vitro reveals key risk factors for thrombocytopenia.,https://pubmed.ncbi.nlm.nih.gov/29107969/,Results > Immunostimulatory effect of ONs; paragraph 44,126,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00535,P2,toxicity,toxicity_endpoint,109,no,C,129,siRNA candidate from PMID 40654369,siRNA,,,hepatic: hepatotoxicity,9006,40654369,PMC12254698,10.1016/j.apsb.2025.03.015,Silencing PTPN2 with nanoparticle-delivered small interfering RNA remodels tumor microenvironment to sensitize immunotherapy in hepatocellular carcinoma.,https://pubmed.ncbi.nlm.nih.gov/40654369/,Results > HPssPT delivers siRNA into HCC cells with potent transfection efficiency and favorable safety performance; paragraph 47,130,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00536,P2,toxicity,toxicity_endpoint,115,no,C,133,ASO candidate from PMID 32530964,ASO,,,hepatic: hepatotoxicity,1431,32530964,PMC7292364,10.1371/journal.pone.0232603,Refining LNA safety profile by controlling phosphorothioate stereochemistry.,https://pubmed.ncbi.nlm.nih.gov/32530964/,Conclusion; paragraph 41,136,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00537,P2,toxicity,toxicity_endpoint,118,no,C,145,ASO candidate from PMID 41754970,ASO,,,hepatic: hepatotoxicity,1065,41754970,PMC12944301,10.3390/pharmaceutics18020228,Unconventional Lysine-Type Lipid Assemblies Enable Efficient Antisense Oligonucleotide Delivery with Distinct Structural Features.,https://pubmed.ncbi.nlm.nih.gov/41754970/,3. Results and Discussions > 3.3. In Vitro Delivery of Splice-Switch ASO; paragraph 26,149,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00538,P2,toxicity,toxicity_endpoint,119,no,C,146,Vutrisiran,ASO/siRNA mixed context,,,neurological: neurotoxicity,1173,41984862,PMC13082648,10.1371/journal.pone.0347417,Assessing the real-world safety of vutrisiran for transthyretin-mediated amyloidosis with polyneuropathy: Based on WHO-VigiAccess and FAERS databases.,https://pubmed.ncbi.nlm.nih.gov/41984862/,Discussion; paragraph 43,150,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00539,P2,toxicity,toxicity_endpoint,120,no,C,147,ASO candidate from PMID 41399312,ASO,,,renal: renal safety,1215,41399312,PMC12877987,10.1002/smll.202509094,Delivery of Antisense Oligonucleotides Using the Nano-Cell Vesicle Technology System (nCVTs) for Targeted Cancer Therapy.,https://pubmed.ncbi.nlm.nih.gov/41399312/,Results and Discussion; paragraph 25,151,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00540,P2,toxicity,toxicity_endpoint,121,no,C,148,siRNA candidate from PMID 41755009,siRNA,,,renal: renal safety,1744,41755009,PMC12944025,10.3390/pharmaceutics18020268,Calcium/Aluminum-Cored Asymmetric Bilayer Nanoparticles for Codelivery of Ziyuglycoside II and PD-L1 siRNA Exert Anti-Breast Tumor Effects.,https://pubmed.ncbi.nlm.nih.gov/41755009/,3. Results and Discussion > 3.12. Assessment of Biochemical Parameters; paragraph 53,152,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00541,P2,toxicity,toxicity_endpoint,123,no,C,150,siRNA candidate from PMID 41477663,siRNA,,,immunotoxicity: immune activation,3330,41477663,PMC12752751,10.1016/j.bioactmat.2025.11.049,Chiral metal nanozyme-enabled SOD2 activation and siRNA delivery cooperatively mitigating chronic heart failure.,https://pubmed.ncbi.nlm.nih.gov/41477663/,Results and discussion > Biosafety of MnO2 nanozymes; paragraph 22,154,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00542,P2,toxicity,toxicity_endpoint,124,no,C,151,siRNA candidate from PMID 41896932,siRNA,,,renal: renal safety,5624,41896932,PMC13151111,10.1186/s12951-026-04311-7,Overcoming lysosomal barrier via V-ATPase: an exosome-based co-delivery platform for combined chemo/RNAi therapy against breast cancer.,https://pubmed.ncbi.nlm.nih.gov/41896932/,Result and discussion > In vivo safety assessment; paragraph 61,155,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00543,P2,toxicity,toxicity_endpoint,125,no,C,152,Nusinersen,ASO/siRNA mixed context,,,renal: renal safety,1015,39829133,PMC11743982,10.1002/brb3.70221,Safety and Efficacy of Nusinersen Focusing on Renal and Hematological Parameters in Spinal Muscular Atrophy.,https://pubmed.ncbi.nlm.nih.gov/39829133/,Abstract; paragraph 6,156,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00544,P2,toxicity,toxicity_endpoint,126,no,C,153,Pelacarsen,ASO,,,renal: renal safety,1030,41021771,PMC12478598,10.1111/cts.70344,"Pharmacokinetics and Safety of Pelacarsen, a GalNAc(3)-Conjugated Antisense Oligonucleotide Targeting Apo(a), in Participants With Mild Hepatic Impairment.",https://pubmed.ncbi.nlm.nih.gov/41021771/,Introduction; paragraph 4,157,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00545,P2,toxicity,toxicity_endpoint,127,no,C,154,RO7239958,ASO,,,neurological: neurotoxicity,1040,41186228,PMC12691668,10.1128/aac.00679-25,"Safety, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide RO7239958 in healthy volunteers and adults with chronic hepatitis B infection.",https://pubmed.ncbi.nlm.nih.gov/41186228/,INTRODUCTION; paragraph 4,158,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00546,P2,toxicity,toxicity_endpoint,128,no,C,155,ASO candidate from PMID 40654713,ASO,,,neurological: neurotoxicity,1160,40654713,PMC12247940,10.1101/2025.05.01.651722,"Anti-Sense Oligonucleotide as a Therapeutic for Synucleinopathies: Pharmacokinetic, Safety and Efficacy Evaluation.",https://pubmed.ncbi.nlm.nih.gov/40654713/,Abstract; paragraph 1,159,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00547,P2,toxicity,toxicity_endpoint,129,no,C,156,ASO candidate from PMID 41169576,ASO,,,renal: renal safety,1408,41169576,PMC12569656,10.1021/jacsau.5c01005,"Cycloalkane Incorporation Into the 2',4'-Bridge of Locked Nucleic Acid: Enhancing Nuclease Stability, Reducing Phosphorothioate Modifications, and Lowering Hepatotoxicity in Antisense Oligonucleotides.",https://pubmed.ncbi.nlm.nih.gov/41169576/,Results and Discussion > Reduction of Hepatotoxicity by scpBNA Series; paragraph 23,160,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00548,P2,toxicity,toxicity_endpoint,130,no,C,157,ASO/siRNA mixed context candidate from PMID 41268339,ASO/siRNA mixed context,,,immunotoxicity: immune activation,1791,41268339,PMC12628137,10.1016/j.bioactmat.2025.09.007,A naturally derived lipopeptide lipid nanoparticle platform enabling multiple nucleic acids delivery.,https://pubmed.ncbi.nlm.nih.gov/41268339/,Results and discussion > Discussion; paragraph 49,161,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00549,P2,toxicity,toxicity_endpoint,131,no,C,158,siRNA candidate from PMID 41439881,siRNA,,,hepatic: hepatotoxicity,2096,41439881,PMC12730731,10.3390/biomimetics10120812,"Biocompatible Copolymerized Gold Nanoclusters: Anti-TNF-α siRNA Binding, Cellular Uptake, Cytotoxicity, Oxidative Stress and Cell Cycle Effects In Vitro.",https://pubmed.ncbi.nlm.nih.gov/41439881/,3. Results and Discussion > 3.3. Cytotoxicity Studies > 3.3.1. MTT Cytotoxicity Assay; paragraph 40,162,cell line,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00550,P2,toxicity,toxicity_endpoint,132,no,C,159,siRNA candidate from PMID 40911569,siRNA,,,renal: renal safety,2098,40911569,PMC12412931,10.1371/journal.pone.0327375,"Upscaling, toxicity and efficacy of multifaceted dressing embedded with dsirna-loaded gold nanoparticles for enhancing diabetic wound treatment.",https://pubmed.ncbi.nlm.nih.gov/40911569/,3 Results and discussion > 3.7. Sub-acute skin toxicity of PF127 gel > 3.7.5. Assessment of renal function and potential toxicity.; paragraph 102,163,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00551,P2,toxicity,toxicity_endpoint,134,no,C,161,ASO candidate from PMID 40604820,ASO,,,hepatic: hepatotoxicity,2483,40604820,PMC12225093,10.1186/s12951-025-03529-1,Ultrasmall nanoparticles for co-delivery of antisense oligonucleotides targeting miR-21 and miR-210 to treat glioblastoma.,https://pubmed.ncbi.nlm.nih.gov/40604820/,Result and discussion > In-vitro cytotoxicity evaluation of surface-modified USMP; paragraph 45,165,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00552,P2,toxicity,toxicity_endpoint,135,no,C,162,siRNA candidate from PMID 40006524,siRNA,,,renal: renal safety,2526,40006524,PMC11859093,10.3390/pharmaceutics17020157,Engineering Lipid Nanoparticles to Enhance Intracellular Delivery of Transforming Growth Factor-Beta siRNA (siTGF-β1) via Inhalation for Improving Pulmonary Fibrosis Post-Bleomycin Challenge.,https://pubmed.ncbi.nlm.nih.gov/40006524/,"3. Results and Discussion > 3.7. Toxicity Evaluation of Heart, Kidney, Liver, and Spleen; paragraph 55",166,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00553,P2,toxicity,toxicity_endpoint,137,no,C,164,Ce6-targeting siRNA from PMID 41299672,siRNA,,,hepatic: hepatotoxicity,3428,41299672,PMC12837076,10.1186/s12951-025-03872-3,Peptide-modified phase-transition nanoparticles co-deliver FTO siRNA and Ce6 for sonodynamic metabolism-immunotherapy of melanoma.,https://pubmed.ncbi.nlm.nih.gov/41299672/,Results and discussion > Preparation and characterization of si-Ce6@tLyP-1-NPs; paragraph 13,168,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00554,P2,toxicity,toxicity_endpoint,138,no,C,165,siRNA candidate from PMID 41007671,siRNA,,,hepatic: hepatotoxicity,3493,41007671,PMC12467891,10.3390/biomedicines13092108,Crossing Barriers: PEGylated Gold Nanoparticles as Promising Delivery Vehicles for siRNA Delivery in Alzheimer's Disease.,https://pubmed.ncbi.nlm.nih.gov/41007671/,4. Discussion; paragraph 29,169,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00555,P2,toxicity,toxicity_endpoint,140,no,C,167,siRNA candidate from PMID 40433119,siRNA,,,renal: renal safety,3666,40433119,PMC12106909,10.2147/IJN.S510514,Lipid-Coated Ag@MnO(2) Core-Shell Nanoparticles for Co-Delivery of Survivin siRNA in Breast Tumor Therapy.,https://pubmed.ncbi.nlm.nih.gov/40433119/,Results and Discussion > Biochemical Parameter Determination; paragraph 50,171,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00556,P2,toxicity,toxicity_endpoint,141,no,C,168,siRNA candidate from PMID 39990947,siRNA,,,hepatic: hepatotoxicity,3749,39990947,PMC11843345,10.1021/acsbiomedchemau.4c00061,Development of Small Interfering RNA Loaded Cationic Lipid Nanoparticles for the Treatment of Liver Cancer with Elevated α-Fetoprotein Expression.,https://pubmed.ncbi.nlm.nih.gov/39990947/,Results and Discussion > Safety Profile of DDAB-cLNPs on Liver Cancer Cells; paragraph 28,172,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00557,P2,toxicity,toxicity_endpoint,142,no,C,109,Imetelstat,ASO,,,hepatic: hepatotoxicity,3751,39980062,PMC11842220,10.1111/cts.70169,"Low Proarrhythmic Risk of Imetelstat, a Novel Oligonucleotide Telomerase Inhibitor: A Translational Analysis.",https://pubmed.ncbi.nlm.nih.gov/39980062/,Introduction; paragraph 4,173,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00558,P2,toxicity,toxicity_endpoint,143,no,C,169,siRNA candidate from PMID 39934917,siRNA,,,immunotoxicity: immune activation,3759,39934917,PMC11816505,10.1186/s13287-025-04151-6,Development and evaluation of siRNA-mediated gene silencing strategies for ADO2 therapy utilizing iPSCs model and DMPC-SPIONs delivery system.,https://pubmed.ncbi.nlm.nih.gov/39934917/,Discussion; paragraph 15,174,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00559,P2,toxicity,toxicity_endpoint,144,no,C,93,Inclisiran,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,8812,40735483,PMC12303805,10.3389/fphar.2025.1554631,Systemic evaluation of inclisiran on the risk of new-onset diabetes and hyperglycemia compared to evolocumab and atorvastatin.,https://pubmed.ncbi.nlm.nih.gov/40735483/,Abstract > Methods and results; paragraph 2,175,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00560,P2,toxicity,toxicity_endpoint,145,no,C,170,siRNA candidate from PMID 40444266,siRNA,,,renal: renal safety,9437,40444266,PMC12120247,10.34133/bmr.0209,Reactive Oxygen Species-Responsive Ferrocene Nanoparticles Delivering Small Interfering RNA Targeting NOP2/Sun RNA Methyltransferase Family Member 2 for Gastric Cancer Therapy.,https://pubmed.ncbi.nlm.nih.gov/40444266/,Results and Discussion > In vivo biosafety evaluation; paragraph 42,176,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00561,P2,toxicity,toxicity_endpoint,148,no,C,172,siRNA candidate from PMID 41304812,siRNA,,,neurological: neurotoxicity,12342,41304812,PMC12655120,10.3390/pharmaceutics17111476,Heterofunctional Cationic Polyester Dendrimers as Potent Nonviral Vectors for siRNA Delivery.,https://pubmed.ncbi.nlm.nih.gov/41304812/,3. Results and Discussion; paragraph 21,179,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00562,P2,toxicity,toxicity_endpoint,149,no,C,173,PMO candidate from PMID 39119918,PMO,,,neurological: neurotoxicity,1135,39119918,PMC11417397,10.1093/nar/gkae686,"Efficacy, biodistribution and safety comparison of chemically modified antisense oligonucleotides in the retina.",https://pubmed.ncbi.nlm.nih.gov/39119918/,Discussion; paragraph 46,180,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00563,P2,toxicity,toxicity_endpoint,152,no,C,176,siRNA candidate from PMID 39539730,siRNA,,,renal: renal safety,2104,39539730,PMC11558258,10.1016/j.bioactmat.2024.06.008,Gelatin and lipidoid integrate to create gelasomes to enhance siRNA delivery with low toxicity.,https://pubmed.ncbi.nlm.nih.gov/39539730/,Results and discussion > Design of gelasomes; paragraph 27,183,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00564,P2,toxicity,toxicity_endpoint,153,no,C,177,Fesomersen,ASO,,,hematologic: platelet/bleeding safety,2297,38563414,PMC10985948,10.1111/cts.13784,"Pharmacokinetics, pharmacodynamics, and safety of fesomersen, a novel antisense inhibitor of factor XI, in healthy Chinese, Japanese, and Caucasian volunteers.",https://pubmed.ncbi.nlm.nih.gov/38563414/,Abstract; paragraph 1,184,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00565,P2,toxicity,toxicity_endpoint,155,no,C,178,siRNA candidate from PMID 38126895,siRNA,,,immunotoxicity: immune activation,3961,38126895,PMC7616748manuscript-id: EMS199502,10.1002/smll.202308775,Pulmonary siRNA Delivery with Sophisticated Amphiphilic Poly(Spermine Acrylamides) for the Treatment of Lung Fibrosis.,https://pubmed.ncbi.nlm.nih.gov/38126895/,Introduction; paragraph 2,186,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00566,P2,toxicity,toxicity_endpoint,156,no,C,179,siRNA candidate from PMID 39274180,siRNA,,,immunotoxicity: immune activation,12106,39274180,PMC11398103,10.3390/polym16172547,Chitosan siRNA Nanoparticles Produce Significant Non-Toxic Functional Gene Silencing in Kidney Cortices.,https://pubmed.ncbi.nlm.nih.gov/39274180/,4. Discussion; paragraph 46,187,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00567,P2,toxicity,toxicity_endpoint,157,no,C,180,siRNA candidate from PMID 39684325,siRNA,,,hepatic: hepatotoxicity,12433,39684325,PMC11641217,10.3390/ijms252312614,Non-Viral Systems Based on PAMAM-Calix-Dendrimers for Regulatory siRNA Delivery into Cancer Cells.,https://pubmed.ncbi.nlm.nih.gov/39684325/,2. Results and Discussion > 2.3. Biological Activity of Dendrimer/siRNA Complexes > 2.3.3. Cytotoxic Effect of Dendrimers and Their Complexes with siRNAs on HeLa Cells; paragraph 30,188,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00568,P2,toxicity,toxicity_endpoint,158,no,C,181,ASO candidate from PMID 39329032,ASO,,,immunotoxicity: immune activation,12464,39329032,PMC11424689,10.2147/IJN.S461167,Ion Doped Hollow Silica Nanoparticles as Promising Oligonucleotide Delivery Systems to Mesenchymal Stem Cells.,https://pubmed.ncbi.nlm.nih.gov/39329032/,Introduction; paragraph 5,189,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00569,P2,toxicity,toxicity_endpoint,161,no,C,184,wireframe DNA origami NANP,DNA nanostructure,,,hepatic: hepatotoxicity,2195,37040258,PMC10189729manuscript-id: NIHMS1894368,10.1021/acsabm.3c00155,Evaluation of Nonmodified Wireframe DNA Origami for Acute Toxicity and Biodistribution in Mice.,https://pubmed.ncbi.nlm.nih.gov/37040258/,Abstract; paragraph 1,192,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00570,P2,toxicity,toxicity_endpoint,162,no,C,184,wireframe DNA origami NANP,DNA nanostructure,,,hepatic: hepatotoxicity,2198,36909507,PMC10002694,10.1101/2023.02.25.530026,Evaluation of non-modified wireframe DNA origami for acute toxicity and biodistribution in mice.,https://pubmed.ncbi.nlm.nih.gov/36909507/,Abstract; paragraph 1,193,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00571,P2,toxicity,toxicity_endpoint,189,no,C,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,4268,23876758,PMC3810404manuscript-id: NIHMS502785,10.1016/j.biomaterials.2013.06.066,Albumin-based nanoconjugates for targeted delivery of therapeutic oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/23876758/,Discussion; paragraph 38,220,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00572,P2,toxicity,toxicity_endpoint,207,no,C,229,STAT3-targeted,ASO,,,immunotoxicity: immune activation,2155,40882634,PMC12404672manuscript-id: NIHMS2102076embargo-date: 2026/08/28,10.1016/j.ccell.2025.07.015,Safety and efficacy of a STAT3-targeted cyclic oligonucleotide: From murine models to a phase 1 clinical trial in pet cats with oral cancer.,https://pubmed.ncbi.nlm.nih.gov/40882634/,Abstract; paragraph 2,250,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00573,P2,toxicity,toxicity_endpoint,219,no,C,236,Loop3,aptamer,,,renal: renal safety,9397,40464222,PMC12134771,10.1002/jcsm.13831,Novel Aptamers Targeting Sclerostin Loop3 Improve Skeletal and Muscle Properties Without Adverse Cardiovascular Effects in Orchiectomized Mice.,https://pubmed.ncbi.nlm.nih.gov/40464222/,Discussion; paragraph 48,262,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00574,P2,toxicity,toxicity_endpoint,245,no,C,258,SPC5001,ASO,,,hepatic: hepatotoxicity,1029,41500580,,10.2131/jts.51.75,Predicting nucleic acid drug-induced nephrotoxicity using a 3D human renal proximal tubule spheroid model.,https://pubmed.ncbi.nlm.nih.gov/41500580/,PubMed abstract,298,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00575,P2,toxicity,toxicity_endpoint,246,no,C,259,ApoB11; ApoB11-conjugated,ASO,,,hepatic: hepatotoxicity,1033,42172871,,10.1016/j.dmd.2026.100304,"Peptide-mediated delivery of an α-synuclein-targeting antisense oligonucleotide: Pharmacokinetics, safety, and central nervous system efficacy in a synucleinopathy model.",https://pubmed.ncbi.nlm.nih.gov/42172871/,PubMed abstract,299,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00576,P2,toxicity,toxicity_endpoint,249,no,C,262,RyR2; Cy5-labeled; RyR2-targeting,siRNA,,,hepatic: hepatotoxicity,1151,41984094,,10.1007/s00204-026-04370-7,Imaging and safety profiling of inhaled siRNA RyR2 in human respiratory models.,https://pubmed.ncbi.nlm.nih.gov/41984094/,PubMed abstract,302,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00577,P2,toxicity,toxicity_endpoint,253,no,C,6,RGLS4326,ASO,,,hepatic: hepatotoxicity,1472,42132413,,10.1002/jcph.70207,"Pharmacokinetics, Safety, and Tolerability of Anti-miR-17 Oligonucleotide RGLS4326 in Healthy Adult Subjects.",https://pubmed.ncbi.nlm.nih.gov/42132413/,PubMed abstract,306,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00578,P2,toxicity,toxicity_endpoint,254,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,1726,41956189,,10.1016/j.ijbiomac.2026.151876,Facilitating siRNA delivery into mammalian cells via the LDL receptor.,https://pubmed.ncbi.nlm.nih.gov/41956189/,PubMed abstract,307,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00579,P2,toxicity,toxicity_endpoint,258,no,C,268,PEG6000,siRNA,,,hepatic: hepatotoxicity,3272,41664462,,10.1021/acsami.5c12952,Hemocompatibility of Carbosilane Dendrimers as a Therapeutic siRNA Delivery System across Blood-Brain Barrier.,https://pubmed.ncbi.nlm.nih.gov/41664462/,PubMed abstract,311,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00580,P2,toxicity,toxicity_endpoint,259,no,C,269,Cy5-siRNA; HepG2,siRNA,,,hepatic: hepatotoxicity,3312,41518301,,10.1021/acs.bioconjchem.5c00554,"""Zip-to-Cytosol"": Glutathione-Cleavable Fluorinated Polyplexes Deliver siRNA at Single-Digit Nanomolar Dose with >90% Gene Silencing.",https://pubmed.ncbi.nlm.nih.gov/41518301/,PubMed abstract,312,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00581,P2,toxicity,toxicity_endpoint,260,no,C,270,PEG2000,siRNA,,,hepatic: hepatotoxicity,3376,41076799,,10.1016/j.bioadv.2025.214537,Novel vector for efficient siRNA delivery to lymphoblasts and melanoma based on genipin-spermine nanocarriers protected with hybrid erythrocyte membrane coating.,https://pubmed.ncbi.nlm.nih.gov/41076799/,PubMed abstract,313,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00582,P2,toxicity,toxicity_endpoint,262,no,C,272,CCL2; MMP9,siRNA,,,hepatic: hepatotoxicity,5189,42007960,,10.1002/smll.202512276,Smart Nanodelivery for Eye-Brain Disorders: Synergistic CCL2 Neutralization and MMP9 Silencing Reverse Anxiety in High Myopia.,https://pubmed.ncbi.nlm.nih.gov/42007960/,PubMed abstract,315,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00583,P2,toxicity,toxicity_endpoint,265,no,C,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,8,41078095,,10.1002/cbic.202500584,Toxicity of Antisense Oligonucleotides is Determined by the Synergistic Interplay of Chemical Modifications and Nucleotide Sequences,https://pubmed.ncbi.nlm.nih.gov/41078095/,PubMed abstract,318,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00584,P2,toxicity,toxicity_endpoint,267,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,1077,41457341,,10.1177/01926233251404016,Proceedings of the 2024 Classic Examples in Toxicologic Pathology XXXI.,https://pubmed.ncbi.nlm.nih.gov/41457341/,PubMed abstract sentence 5,320,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00585,P2,toxicity,toxicity_endpoint,268,no,C,275,Atp5o,ASO,,,hepatic: hepatotoxicity,1108,40134366,,10.1089/nat.2024.0047,"Hepatotoxicity Reduction Profiles of Antisense Oligonucleotides Containing Amido-Bridged Nucleic Acid and 2'-O,4'-C-Spirocyclopropylene Bridged Nucleic Acid.",https://pubmed.ncbi.nlm.nih.gov/40134366/,PubMed abstract,321,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00586,P2,toxicity,toxicity_endpoint,271,no,C,277,NS102; ED50,siRNA,,,hepatic: hepatotoxicity,1425,41167339,,10.1016/j.jconrel.2025.114366,Incorporation of a polyamine into lipid nanoparticles increases the endosomal release and transfection of nucleic acids without toxicity.,https://pubmed.ncbi.nlm.nih.gov/41167339/,PubMed abstract,324,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00587,P2,toxicity,toxicity_endpoint,278,no,C,282,LP5-SeNP; LP1-SeNP,siRNA,,,hepatic: hepatotoxicity,3484,41037472,,10.1021/acsabm.5c01486,Peptide-Functionalized Selenium Nanoparticle-Based Effective Delivery System for Src-Targeting siRNA in Triple-Negative Breast Cancer Cells.,https://pubmed.ncbi.nlm.nih.gov/41037472/,PubMed abstract,331,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00588,P2,toxicity,toxicity_endpoint,283,no,C,284,TUBB3; TUBB3-siRNA; TUBB3-LNPs,siRNA,,,hepatic: hepatotoxicity,3572,40736337,,10.1021/acsami.5c07808,TUBB3/DTX LNPs Dry Powder Inhaler and an Efficient Delivery Device for Targeted Therapy of Lung Cancer.,https://pubmed.ncbi.nlm.nih.gov/40736337/,PubMed abstract,336,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00589,P2,toxicity,toxicity_endpoint,288,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,3764,39907973,,10.1007/s13346-025-01800-4,Topical delivery of siRNA to psoriatic skin model using high molecular weight chitosan derivatives: In vitro and in vivo studies.,https://pubmed.ncbi.nlm.nih.gov/39907973/,PubMed abstract,341,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00590,P2,toxicity,toxicity_endpoint,290,no,C,286,miRNA34a; LNA34a; NOTCH1,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,9647,40306578,,10.1016/j.jconrel.2025.113791,Gemcitabine and miRNA34a mimic codelivery with magnetic nanoparticles enhanced anti-tumor effect against pancreatic cancer.,https://pubmed.ncbi.nlm.nih.gov/40306578/,PubMed abstract,343,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00591,P2,toxicity,toxicity_endpoint,291,no,C,287,siG12Ss; siG12S; KRASG12S,siRNA,,,hepatic: hepatotoxicity,9951,40073943,,10.1016/j.jconrel.2025.113624,A Mannosylated peptidyl lipid CManDA doped into cytidinyl/cationic lipids efficiently delivers siG12Ss to lung cancer in vivo.,https://pubmed.ncbi.nlm.nih.gov/40073943/,PubMed abstract,344,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00592,P2,toxicity,toxicity_endpoint,300,no,C,295,SMN2; BPP8-PMO; Cy7,ASO,,,hepatic: hepatotoxicity,1582,38749176,,10.1016/j.biopha.2024.116737,Efficient systemic CNS delivery of a therapeutic antisense oligonucleotide with a blood-brain barrier-penetrating ApoE-derived peptide.,https://pubmed.ncbi.nlm.nih.gov/38749176/,PubMed abstract,353,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00593,P2,toxicity,toxicity_endpoint,301,no,C,296,Ki67,siRNA,,,hepatic: hepatotoxicity,1878,39447842,,10.1016/j.jconrel.2024.10.047,"Optimizing lipid nanoparticles for fetal gene delivery in vitro, ex vivo, and aided with machine learning.",https://pubmed.ncbi.nlm.nih.gov/39447842/,PubMed abstract,354,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00594,P2,toxicity,toxicity_endpoint,303,no,C,298,PNMVA24,siRNA,,,hepatic: hepatotoxicity,1926,37994483,,10.1002/adhm.202302712,Poly(N-methyl-N-vinylacetamide): A Strong Alternative to PEG for Lipid-Based Nanocarriers Delivering siRNA.,https://pubmed.ncbi.nlm.nih.gov/37994483/,PubMed abstract,356,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00595,P2,toxicity,toxicity_endpoint,308,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,3957,38197438,,10.1039/d3nr05738k,Cellular uptake and fate of cationic polymer-coated nanodiamonds delivering siRNA: a mechanistic study.,https://pubmed.ncbi.nlm.nih.gov/38197438/,PubMed abstract,361,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00596,P2,toxicity,toxicity_endpoint,309,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,3959,38191947,,10.1208/s12249-023-02728-z,Development and Characterization of Modified Chitosan Lipopolyplex for an Effective siRNA Delivery.,https://pubmed.ncbi.nlm.nih.gov/38191947/,PubMed abstract,362,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00597,P2,toxicity,toxicity_endpoint,311,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,10888,39106627,,10.1016/j.ijbiomac.2024.134515,Construction of charge-reversible coordination-crosslinked spherical nucleic acids to deliver dual anti-cancer genes and ferroptosis payloads.,https://pubmed.ncbi.nlm.nih.gov/39106627/,PubMed abstract,364,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00598,P2,toxicity,toxicity_endpoint,313,no,C,302,PCSK9; GN3; MC3-LNP,siRNA,,,hepatic: hepatotoxicity,11712,37951519,,10.1016/j.actbio.2023.10.038,Engineering a biomimetic system for hepatocyte-specific RNAi treatment of non-alcoholic fatty liver disease.,https://pubmed.ncbi.nlm.nih.gov/37951519/,PubMed abstract,366,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00599,P2,toxicity,toxicity_endpoint,315,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,13711,39521158,,10.1016/j.ijpharm.2024.124928,Cysteamine functionalized gold nanoparticles exhibit high efficiency delivery of genetic materials in embryonic stem cells majorly via clathrin mediated endocytosis.,https://pubmed.ncbi.nlm.nih.gov/39521158/,PubMed abstract,368,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00600,P2,toxicity,toxicity_endpoint,316,no,C,185,unspecified siRNA,siRNA,,,general toxicity: toxicity,13881,39031853,,10.1002/smll.202401776,A Metal-Phenolic Network-Enabled Nanoadjuvant to Modulate Immune Responses.,https://pubmed.ncbi.nlm.nih.gov/39031853/,PubMed abstract sentence 4,369,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00601,P2,toxicity,toxicity_endpoint,319,no,C,306,HepG2,siRNA,,,hepatic: hepatotoxicity,1933,37871289,,10.1021/acs.biomac.3c00813,Bioresponsive Chimaeric Polymersomes Mediate Sustained and Liver-Specific siRNA Transfection In Vivo.,https://pubmed.ncbi.nlm.nih.gov/37871289/,PubMed abstract,372,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00602,P2,toxicity,toxicity_endpoint,322,no,C,308,BldA50M50; MixA50; MixD50,ASO,,,hepatic: hepatotoxicity,2669,37437196,,10.1021/acs.bioconjchem.3c00186,Blended Block Polycation Micelles Enhance Antisense Oligonucleotide Delivery.,https://pubmed.ncbi.nlm.nih.gov/37437196/,PubMed abstract,375,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00603,P2,toxicity,toxicity_endpoint,324,no,C,310,CpG55; rH7HA,ASO/siRNA mixed context,,,hepatic: hepatotoxicity,12177,37659896,,10.1016/j.vaccine.2023.08.053,Developmental and reproductive safety of Advax-CpG55.2™ adjuvanted COVID-19 and influenza vaccines in mice.,https://pubmed.ncbi.nlm.nih.gov/37659896/,PubMed abstract,377,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00604,P2,toxicity,toxicity_endpoint,328,no,C,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,1429,34060347,,10.1177/01926233211011615,"Safety, Tissue Distribution, and Metabolism of LNA-Containing Antisense Oligonucleotides in Rats.",https://pubmed.ncbi.nlm.nih.gov/34060347/,PubMed abstract,381,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00605,P2,toxicity,toxicity_endpoint,331,no,C,315,TPPS2a; TPPS2a-,siRNA,,,hepatic: hepatotoxicity,4126,34437914,,10.1016/j.jconrel.2021.08.039,TNFα siRNA delivery by nanoparticles and photochemical internalization for psoriasis topical therapy.,https://pubmed.ncbi.nlm.nih.gov/34437914/,PubMed abstract,384,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00606,P2,toxicity,toxicity_endpoint,333,no,C,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,1421,33084520,,10.1177/0192623320964391,Urinary Kidney Biomarker Panel Detects Preclinical Antisense Oligonucleotide-Induced Tubular Toxicity.,https://pubmed.ncbi.nlm.nih.gov/33084520/,PubMed abstract,386,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00607,P2,toxicity,toxicity_endpoint,335,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,12219,31877238,,10.1002/jat.3938,Safety of bovine milk derived extracellular vesicles used for delivery of RNA therapeutics in zebrafish and mice.,https://pubmed.ncbi.nlm.nih.gov/31877238/,PubMed abstract,388,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00608,P2,toxicity,toxicity_endpoint,338,no,C,186,unspecified antisense oligonucleotide,ASO,,,general safety: safety/tolerability,2082,29708844,,10.1089/nat.2017.0706,Chronic Toxicity Assessment of 2'-O-Methoxyethyl Antisense Oligonucleotides in Mice.,https://pubmed.ncbi.nlm.nih.gov/29708844/,PubMed abstract,391,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00609,P2,toxicity,toxicity_endpoint,339,no,C,257,Malat1-targeting,ASO,,,hepatic: hepatotoxicity,12059,30095329,,10.1089/nat.2018.0724,Role of Computationally Evaluated Target Specificity in the Hepatotoxicity of Gapmer Antisense Oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/30095329/,PubMed abstract,392,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00610,P2,toxicity,toxicity_endpoint,341,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,2083,28986168,,10.1016/j.jconrel.2017.09.044,Highly specific delivery of siRNA to hepatocytes circumvents endothelial cell-mediated lipid nanoparticle-associated toxicity leading to the safe and efficacious decrease in the hepatitis B virus.,https://pubmed.ncbi.nlm.nih.gov/28986168/,PubMed abstract,394,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00611,P2,toxicity,toxicity_endpoint,342,no,C,320,HepG2,ASO,,,hepatic: hepatotoxicity,2107,27923110,,10.1089/nat.2016.0639,"Impact of Oligonucleotide Structure, Chemistry, and Delivery Method on In Vitro Cytotoxicity.",https://pubmed.ncbi.nlm.nih.gov/27923110/,PubMed abstract,395,cell line,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00612,P2,toxicity,toxicity_endpoint,343,no,C,186,unspecified antisense oligonucleotide,ASO,,,genotoxicity: DNA damage response,2232,28295562,,10.1002/em.22076,Locked nucleic acid (LNA): Based single-stranded oligonucleotides are not genotoxic.,https://pubmed.ncbi.nlm.nih.gov/28295562/,PubMed abstract sentence 2,396,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00613,P2,toxicity,toxicity_endpoint,344,no,C,321,KL4,siRNA,,,hepatic: hepatotoxicity,12641,29121767,,10.1021/acs.molpharmaceut.7b00725,"From Pulmonary Surfactant, Synthetic KL4 Peptide as Effective siRNA Delivery Vector for Pulmonary Delivery.",https://pubmed.ncbi.nlm.nih.gov/29121767/,PubMed abstract,397,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00614,P2,toxicity,toxicity_endpoint,346,no,C,322,Myo1E,ASO,,,hepatic: hepatotoxicity,2084,24336348,,10.1093/toxsci/kft278,Comparison of hepatic transcription profiles of locked ribonucleic acid antisense oligonucleotides: evidence of distinct pathways contributing to non-target mediated toxicity in mice.,https://pubmed.ncbi.nlm.nih.gov/24336348/,PubMed abstract,399,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00615,P2,toxicity,toxicity_endpoint,350,no,C,325,STAT3,ASO,,,hepatic: hepatotoxicity,2331,23692080,,10.1089/nat.2013.0422,Preclinical evaluation of the toxicological effects of a novel constrained ethyl modified antisense compound targeting signal transducer and activator of transcription 3 in mice and cynomolgus monkeys.,https://pubmed.ncbi.nlm.nih.gov/23692080/,PubMed abstract,403,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00616,P2,toxicity,toxicity_endpoint,353,no,C,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,4279,22246038,,10.1182/blood-2011-10-387134,Antisense inhibition of coagulation factor XI prolongs APTT without increased bleeding risk in cynomolgus monkeys.,https://pubmed.ncbi.nlm.nih.gov/22246038/,PubMed abstract,406,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00617,P2,toxicity,toxicity_endpoint,360,no,C,331,DUX4; iDUX4pA,ASO,,,hepatic: hepatotoxicity,1176,42198843,,10.1016/j.ymthe.2026.05.010,Lipid nanoparticle delivery of antisense gapmers attenuates pathology in a mouse model of facioscapulohumeral muscular dystrophy.,https://pubmed.ncbi.nlm.nih.gov/42198843/,PubMed abstract,414,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00618,P2,toxicity,toxicity_endpoint,368,no,C,338,MC3-LNP,siRNA,,,hepatic: hepatotoxicity,1375,41985596,,10.1016/j.ijpharm.2026.126878,Mucus-penetrating lipid nanoparticles overcome lung barriers for pulmonary co-delivery of siGSDMD and pDNA-SAP in idiopathic pulmonary fibrosis.,https://pubmed.ncbi.nlm.nih.gov/41985596/,PubMed abstract,422,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00619,P2,toxicity,toxicity_endpoint,369,no,C,339,ABCG2; HCC1937; Ki67,siRNA,,,hepatic: hepatotoxicity,1392,41865608,,10.1016/j.colsurfb.2026.115616,"cRGD-modified, pH-sensitive liposomes for co-delivery of docetaxel and ABCG2 siRNA enhance therapeutic efficacy in triple-negative breast cancer.",https://pubmed.ncbi.nlm.nih.gov/41865608/,PubMed abstract,423,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00620,P2,toxicity,toxicity_endpoint,370,no,C,340,GATHER2; NCT04435366; mm2,aptamer,,,hepatic: hepatotoxicity,1520,41407269,,10.1016/j.ophtha.2025.12.011,Avacincaptad Pegol for Geographic Atrophy Secondary to Age-Related Macular Degeneration: Two-Year Efficacy and Safety Results from the GATHER2 Phase 3 Trial.,https://pubmed.ncbi.nlm.nih.gov/41407269/,PubMed abstract,424,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00621,P2,toxicity,toxicity_endpoint,377,no,C,186,unspecified antisense oligonucleotide,ASO,,,hepatic: hepatotoxicity,1127,40967408,,10.1016/j.jconrel.2025.114239,Safety and efficacy of KRAS antisense oligonucleotides and RIG-I agonists delivered by extracellular vesicles for pancreatic cancer peritoneal metastasis treatment.,https://pubmed.ncbi.nlm.nih.gov/40967408/,PubMed abstract,431,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00622,P2,toxicity,toxicity_endpoint,380,no,C,347,GPC3-LPPEI; GPC3-targeting; GPC3,siRNA,,,hepatic: hepatotoxicity,2097,41121471,,10.2174/0113816128395585250923122829,Research on Anti-tumor Pharmacodynamics of Multi-functional Magnetic Lipid Polymer with Specific Targeted Transmission of siRNA and its Toxicity Evaluation.,https://pubmed.ncbi.nlm.nih.gov/41121471/,PubMed abstract,434,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00623,P2,toxicity,toxicity_endpoint,383,no,C,350,SOX9; MC3-DMA; siSOX9,siRNA,,,hepatic: hepatotoxicity,2475,40717437,,10.1021/acs.molpharmaceut.5c00272,"SOX9 siRNA Loaded Lipid Nanoparticles Actively Targeted: Formulation, Delivery, and Antitumor Effect on Colorectal Cancer In Vitro and In Vivo.",https://pubmed.ncbi.nlm.nih.gov/40717437/,PubMed abstract,437,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00624,P2,toxicity,toxicity_endpoint,385,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,3747,40010411,,10.1016/j.jconrel.2025.02.067,Surfactant protein B-derived peptides as endosomal escape enhancers for pulmonary delivery of siRNA.,https://pubmed.ncbi.nlm.nih.gov/40010411/,PubMed abstract,439,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00625,P2,toxicity,toxicity_endpoint,388,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,12427,39447239,,10.1016/j.bioadv.2024.214078,Bone-targeted ultrasound-responsive nanobubbles for siRNA delivery to treat osteoporosis in mice.,https://pubmed.ncbi.nlm.nih.gov/39447239/,PubMed abstract,442,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00626,P2,toxicity,toxicity_endpoint,391,no,C,185,unspecified siRNA,siRNA,,,hepatic: hepatotoxicity,1585,38636882,,10.1016/j.ejpb.2024.114296,A potent bioreducible ionizable lipid nanoparticle enables siRNA delivery for retinal neovascularization inhibition.,https://pubmed.ncbi.nlm.nih.gov/38636882/,PubMed abstract,445,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00627,P2,toxicity,toxicity_endpoint,396,no,C,358,Ago2,siRNA,,,general safety: safety/tolerability,1137,37638886,,10.1021/jacs.3c04744,"Shorter Is Better: The α-(l)-Threofuranosyl Nucleic Acid Modification Improves Stability, Potency, Safety, and Ago2 Binding and Mitigates Off-Target Effects of Small Interfering RNAs.",https://pubmed.ncbi.nlm.nih.gov/37638886/,PubMed abstract,450,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00628,P2,toxicity,toxicity_endpoint,397,no,C,186,unspecified antisense oligonucleotide,ASO,,,hematological: thrombocytopenia,2199,36801361,,10.1016/j.tiv.2023.105569,The mechanism of thrombocytopenia caused by cholesterol-conjugated antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/36801361/,PubMed abstract,451,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00629,P2,toxicity,toxicity_endpoint,407,no,C,186,unspecified antisense oligonucleotide,ASO,,,hematological: thrombocytopenia,2314,31687889,,10.1089/nat.2019.0780,Impurity Qualification Toxicology Study for a 2'-O-Methoxyethyl-Modified Antisense Inhibitor in Mice.,https://pubmed.ncbi.nlm.nih.gov/31687889/,PubMed abstract,461,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00630,P2,toxicity,toxicity_endpoint,408,no,C,365,antiPF4,ASO,,,hepatic: hepatotoxicity,2318,29846725,,10.1093/toxsci/kfy119,"Investigation into the Mechanism(s) That Leads to Platelet Decreases in Cynomolgus Monkeys During Administration of ISIS 104838, a 2'-MOE-Modified Antisense Oligonucleotide.",https://pubmed.ncbi.nlm.nih.gov/29846725/,PubMed abstract,462,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00631,P2,toxicity,toxicity_endpoint,410,no,C,366,SGLT2,ASO,,,renal: renal safety,2086,22915769,,10.1124/jpet.112.197426,"Pharmacodynamics and subchronic toxicity in mice and monkeys of ISIS 388626, a second-generation antisense oligonucleotide that targets human sodium glucose cotransporter 2.",https://pubmed.ncbi.nlm.nih.gov/22915769/,PubMed abstract,464,human,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00632,P2,toxicity,toxicity_endpoint,413,no,C,186,unspecified antisense oligonucleotide,ASO,,,renal: renal safety,2269,22709826,,10.1016/j.tox.2012.06.005,Renal uptake and tolerability of a 2'-O-methoxyethyl modified antisense oligonucleotide (ISIS 113715) in monkey.,https://pubmed.ncbi.nlm.nih.gov/22709826/,PubMed abstract,467,rat,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00633,P2,toxicity,toxicity_endpoint,415,no,C,372,IL-4Ralpha ASO,ASO,IL4RA,,general safety: body weight/general tolerability,9646,40308203,PMC12303760manuscript-id: NIHMS2075744,10.1161/CIRCULATIONAHA.124.072411,CD206(+)IL-4Rα(+) Macrophages Are Drivers of Adverse Cardiac Remodeling in Ischemic Cardiomyopathy.,https://pubmed.ncbi.nlm.nih.gov/40308203/,"RESULTS > IL-4Rα silencing in vivo reverses LV remodeling, reduces cardiac fibrosis, and improves neovascularization in HF.; paragraph 25",474,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00634,P2,toxicity,toxicity_endpoint,420,no,C,376,G5-GalNAc siRNA platform,siRNA,PCSK9/AGT,,general safety: nonclinical toxicology and biodistribution safety,1069,41541269,PMC12800395,10.1016/j.omtn.2025.102801,Ribofuranose-based GalNAc-conjugated siRNA enhances the liver-targeted delivery and elicits robust RNAi-mediated gene silencing.,https://pubmed.ncbi.nlm.nih.gov/41541269/,Discussion; paragraph 17,479,cynomolgus monkey/mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00635,P2,toxicity,toxicity_endpoint,430,no,C,386,siRNA polyplexes,siRNA,Raf-1/PD-L1,,general safety: systemic toxicity,3263,41691982,PMC13183465manuscript-id: NIHMS2163813,10.1016/j.bbrc.2026.153430,An effective tumor-inhibiting siRNA delivery platform.,https://pubmed.ncbi.nlm.nih.gov/41691982/,Conclusion.; paragraph 24,489,mouse,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00636,P2,toxicity,toxicity_endpoint,447,no,C,403,NLP-exosome complex STAT3 siRNA,siRNA,STAT3,,general safety: cell viability/cytotoxicity,9471,40427146,PMC12109797,10.3390/cancers17101648,Exosome Enveloped by Nano Lipid Particle a New Model for Signal Transducer and Activator of Transcription 3 Silencer Ribonucleic Acid Delivery System to a Glioblastoma Mice Model.,https://pubmed.ncbi.nlm.nih.gov/40427146/,3. Results > 3.1. The Use of NLP-EXOSOME COMPLEX Nanoparticles Results in the Production of Reliable and Efficient Formulations for Delivering siRNA; paragraph 29,506,human cell line,,U87MG glioblastoma cells,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00637,P2,toxicity,toxicity_endpoint,482,no,C,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,1773,41220296,PMC12908211,10.1002/adhm.202503748,Photosensitizing Lipid Nanoparticles for Ferroptosis-Enhanced Photodynamic Cancer Therapy via GPX4 Silencing.,https://pubmed.ncbi.nlm.nih.gov/41220296/,Results and Discussion > Synergistic Effect of Ferroptosis Induction via GPX4 Inhibition and PDT; paragraph 15,759,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00638,P2,toxicity,toxicity_endpoint,509,no,C,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,7065,41334811,PMC12801319,10.1021/acs.biomac.5c01202,Hydrolytically Stable Cationic Bis-MPA Dendrimers as Efficient Transfectants for Glioblastoma Cells and Primary Astrocytes.,https://pubmed.ncbi.nlm.nih.gov/41334811/,Results and Discussion > Cytotoxicity Assessment of Dendrimers; paragraph 43,786,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00639,P2,toxicity,toxicity_endpoint,510,no,C,988,"unspecified CpG oligodeoxynucleotide (v1 extraction artefact, pending source re-verification)",CpG oligodeoxynucleotide,,,hepatic: hepatotoxicity,7129,41270206,PMC12866702,10.1002/advs.202515052,Spatiotemporally Controlled Tumor Photodynamic/Immunotherapy Therapy Based on Upconversion Hybrid Nanosystem.,https://pubmed.ncbi.nlm.nih.gov/41270206/,Results and Discussion > In Vitro Photo‐Controlled Activation and Cytotoxicity of PURH; paragraph 13,787,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00640,P2,toxicity,toxicity_endpoint,528,no,C,1082,"PDA@Mn-siSur-c-NPs (recovered B2 from PMID:41599118, curator:CM)",siRNA,,,hepatic: hepatotoxicity,3399,41599118,PMC12844724,10.3390/pharmaceutics18010010,Construction and Characterization of PDA@MnO(2)-Cored Multifunctional Targeting Nanoparticles Loaded with Survivin siRNA for Breast Tumor Therapy.,https://pubmed.ncbi.nlm.nih.gov/41599118/,3. Results and Discussion > 3.12. Safety Evaluation; paragraph 56,805,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00641,P2,toxicity,toxicity_endpoint,539,no,C,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,general safety: cell viability/cytotoxicity,3596,40679089,PMC12344721,10.1021/acs.biomac.5c00344,Cleavable Cationic Carbosilane Dendrimers with pH-Tunable Charge as siRNA Carriers.,https://pubmed.ncbi.nlm.nih.gov/40679089/,Results and Discussion > Biocompatibility of Cleavable Dendrimers; paragraph 51,816,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00642,P2,toxicity,toxicity_endpoint,545,no,C,1083,unspecified siRNA (PMID:39906612),siRNA,,,hepatic: hepatotoxicity,3765,39906612,PMC11790197,10.22038/ijbms.2024.81998.17744,Nanomolecular silencing of TSC22D4 mRNA via a DNAsome-siRNA for enhancing insulin sensitization in hepatocytes.,https://pubmed.ncbi.nlm.nih.gov/39906612/,Conclusion; paragraph 41,822,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00643,P2,toxicity,toxicity_endpoint,551,no,C,528,Silica,siRNA,,,hepatic: hepatotoxicity,7559,41392621,PMC12754758manuscript-id: NIHMS2132376,10.1021/acsami.5c15253,"Modular Design of Mesoporous Silica Nanoparticles Enables Bioimaging, Dual Chemotherapy, and Combinatorial Gene Silencing in Triple-Negative Breast Cancer.",https://pubmed.ncbi.nlm.nih.gov/41392621/,Results and Discussion > Specific Gene Silencing Studies; paragraph 21,828,cell line,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00644,P2,toxicity,toxicity_endpoint,571,no,C,443,inclisiran,siRNA,,,hepatic: hepatotoxicity,9021,40645721,PMC12441398,10.1016/j.jaccas.2025.103897,Treatment With Inclisiran During Peritoneal Dialysis.,https://pubmed.ncbi.nlm.nih.gov/40645721/,Discussion; paragraph 11,848,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00645,P2,toxicity,toxicity_endpoint,572,no,C,1084,unspecified PMO (PMID:40608957),PMO,,,hepatic: hepatotoxicity,9102,40608957,PMC12677909,10.1093/brain/awaf249,Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy.,https://pubmed.ncbi.nlm.nih.gov/40608957/,Discussion; paragraph 48,849,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00646,P2,toxicity,toxicity_endpoint,576,no,C,1085,"si-Cx43 (recovered B2 from PMID:40528841, curator:CM)",siRNA,,,hepatic: hepatotoxicity,9274,40528841,PMC12173670,10.1016/j.mtbio.2025.101917,Thermosensitive hydrogel composite with si-Cx43 nanoparticles and anti-VEGF agent for synergistic treatment of diabetic retinopathy.,https://pubmed.ncbi.nlm.nih.gov/40528841/,Discussion; paragraph 47,853,rat,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00647,P2,toxicity,toxicity_endpoint,583,no,C,1086,"D-siRNA-Plk1 (recovered B2 from PMID:40236812, curator:CM)",siRNA,,,hepatic: hepatotoxicity,9750,40236812,PMC11999372,10.1016/j.mtbio.2025.101718,Bundling gold nanorods with RCA-produced DNA tape into an intelligently reconfigurable nanocluster bomb for multimodal precision cancer therapy.,https://pubmed.ncbi.nlm.nih.gov/40236812/,Results and discussion > In vivo anti-tumor activity of DS-ARGN; paragraph 25,860,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00648,P2,toxicity,toxicity_endpoint,588,no,C,1087,"AS-T9/U4_MH (recovered B2 from PMID:39946362, curator:CM)",ASO,,,hepatic: hepatotoxicity,10132,39946362,PMC11825018,10.1371/journal.pone.0317559,"Multifunctional molecular hybrid for targeted colorectal cancer cells: Integrating doxorubicin, AS1411 aptamer, and T9/U4 ASO.",https://pubmed.ncbi.nlm.nih.gov/39946362/,Results and discussion > Effect of Dox-loaded AS-T9/U4_MH on cell proliferation; paragraph 31,865,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00649,P2,toxicity,toxicity_endpoint,601,no,C,545,Site-blocking,ASO,,,hepatic: hepatotoxicity,2556,39379106,PMC11571808,10.1261/rna.080220.124,Site-blocking antisense oligonucleotides as a mechanism to fine-tune MeCP2 expression.,https://pubmed.ncbi.nlm.nih.gov/39379106/,DISCUSSION > Risk of adverse effects; paragraph 53,878,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00650,P2,toxicity,toxicity_endpoint,604,no,C,1088,"NANP-siRhoA (recovered B2 from PMID:40308740, curator:CM)",siRNA,,,hepatic: hepatotoxicity,3813,40308740,PMC12043328manuscript-id: NIHMS2068822,10.59566/isrnn.2024.0101079,The Reduction of Traumatic Spinal Cord Secondary Injury by Anti-RhoA siRNA Functionalized Nucleic Acid Nanoparticles (NANPs).,https://pubmed.ncbi.nlm.nih.gov/40308740/,RESULTS AND DISCUSSION; paragraph 20,881,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00651,P2,toxicity,toxicity_endpoint,611,no,C,1089,"vivo-3′UTR (recovered B2 from PMID:39676860, curator:CM)",PMO,,,hepatic: hepatotoxicity,10547,39676860,PMC11638040,10.3389/fimmu.2024.1491230,Effective inhibition of dengue virus replication using 3'UTR-targeted Vivo-Morpholinos.,https://pubmed.ncbi.nlm.nih.gov/39676860/,Discussion; paragraph 31,888,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00652,P2,toxicity,toxicity_endpoint,615,no,C,1090,"HNP-siHOXB7 (recovered B2 from PMID:39458966, curator:CM)",siRNA,,,hepatic: hepatotoxicity,10648,39458966,PMC11509954,10.3390/ph17101325,In Vivo HOXB7 Gene Silencing and Cotreatment with Tamoxifen for Luminal A Breast Cancer Therapy.,https://pubmed.ncbi.nlm.nih.gov/39458966/,5. Conclusions; paragraph 51,892,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00653,P2,toxicity,toxicity_endpoint,622,no,C,493,patisiran,siRNA,,,immunotoxicity: immune activation,11357,38532802,PMC10964446,10.1177/17562864241239755,Patisiran exposure in early pregnancy: a case report.,https://pubmed.ncbi.nlm.nih.gov/38532802/,Discussion; paragraph 9,899,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00654,P2,toxicity,toxicity_endpoint,625,no,C,435,siRNAs,siRNA,,,hepatic: hepatotoxicity,11479,38383439,PMC10882889,10.1186/s12943-024-01955-7,The therapeutically actionable long non-coding RNA 'T-RECS' is essential to cancer cells' survival in NRAS/MAPK-driven melanoma.,https://pubmed.ncbi.nlm.nih.gov/38383439/,Discussion; paragraph 62,902,cell line,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00655,P2,toxicity,toxicity_endpoint,627,no,C,472,siRNA-mediated,siRNA,,,hepatic: hepatotoxicity,12444,39553720,PMC11566511,10.7717/peerj.18214,Investigation of the efficacy of siRNA-mediated KRAS gene silencing in pancreatic cancer therapy.,https://pubmed.ncbi.nlm.nih.gov/39553720/,RESULTS AND DISCUSSION > Evaluation of the efficacy of gene silencing with in vitro RT-PCR; paragraph 45,904,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00656,P2,toxicity,toxicity_endpoint,629,no,C,1091,"cRGD-PEG-siRNA (recovered B2 from PMID:39431993, curator:CM)",siRNA,,,hepatic: hepatotoxicity,12453,39431993,PMC11583972,10.1021/acs.bioconjchem.4c00255,A pH-Sensitive cRGD-PEG-siRNA Conjugated Compound Targeting Glioblastoma.,https://pubmed.ncbi.nlm.nih.gov/39431993/,Results and Discussion > Toxicity of cRGD-PEG-siRNA to Renal Tubular Epithelial Cells; paragraph 11,906,human,,kidney,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00657,P2,toxicity,toxicity_endpoint,631,no,C,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,12500,39006767,PMC11240087,10.1039/d4ra01965b,"Carboxymethylcellulose encapsulated fingolimod, siRNA@ZnO hybrid nanocomposite as a new anti-Alzheimer's material.",https://pubmed.ncbi.nlm.nih.gov/39006767/,Results and discussion > Inflammatory inhibition effect of the hybrid nanocomposite; paragraph 40,908,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00658,P2,toxicity,toxicity_endpoint,643,no,C,435,siRNAs,siRNA,,,hepatic: hepatotoxicity,11811,38077055,PMC10705697,10.21203/rs.3.rs-1297358/v3,The therapeutically actionable long non-coding RNA 'T-RECS' is essential to cancer cells' survival in NRAS/MAPK-driven melanoma.,https://pubmed.ncbi.nlm.nih.gov/38077055/,Discussion; paragraph 24,920,mouse,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00659,P2,toxicity,toxicity_endpoint,652,no,C,443,inclisiran,siRNA,,,hepatic: hepatotoxicity,14031,37424909,PMC10326624,10.3389/fcvm.2023.1181720,Painful and recurring injection site reaction to alirocumab and evolocumab in a young woman with familial hypercholesterolemia and effective therapeutic alternative based on inclisiran: a case report.,https://pubmed.ncbi.nlm.nih.gov/37424909/,Discussion; paragraph 14,929,human,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00660,P2,toxicity,toxicity_endpoint,656,no,C,443,inclisiran,siRNA,,,hepatic: hepatotoxicity,14195,37021909,PMC10077952,10.1002/prp2.1080,The N-Acetylgalactosamine-conjugated small interfering RNA inclisiran can be coadministered safely with atorvastatin in cynomolgus monkeys resulting in additive low-density lipoprotein cholesterol reductions.,https://pubmed.ncbi.nlm.nih.gov/37021909/,DISCUSSION; paragraph 60,933,non-human primate,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00661,P2,toxicity,toxicity_endpoint,665,no,C,1092,"PNA4986 (recovered B2 from PMID:35436109, curator:CM)",ASO,,,hepatic: hepatotoxicity,1980,35436109,PMC9112330,10.1021/acsinfecdis.2c00089,Antisense Peptide Nucleic Acid-Diaminobutanoic Acid Dendron Conjugates with SbmA-Independent Antimicrobial Activity against Gram-Negative Bacteria.,https://pubmed.ncbi.nlm.nih.gov/35436109/,Results and Discussion > Cell Toxicity of Dendron–PNAs; paragraph 13,942,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00662,P2,toxicity,toxicity_endpoint,671,no,C,1093,"HA-SeSe-COOH/siR-93C@PAMAM (recovered B2 from PMID:35156491, curator:CM)",siRNA,,,hepatic: hepatotoxicity,4091,35156491,PMC8856077,10.1080/10717544.2022.2032874,Hyaluronic acid-modified redox-sensitive hybrid nanocomplex loading with siRNA for non-small-cell lung carcinoma therapy.,https://pubmed.ncbi.nlm.nih.gov/35156491/,Results and discussion > In vivo anticancer activity and safety evaluation; paragraph 49,948,mouse,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00663,P2,toxicity,toxicity_endpoint,672,no,C,562,siRNA-Based,siRNA,,,hepatic: hepatotoxicity,4092,35143189,PMC8938942manuscript-id: NIHMS1785508,10.1021/jacs.1c12750,Hairpin-like siRNA-Based Spherical Nucleic Acids.,https://pubmed.ncbi.nlm.nih.gov/35143189/,RESULTS AND DISCUSSION > Hairpin-like Design Increases Serum Nuclease Resistance and Decreases SNA Cytotoxicity.; paragraph 12,949,cell line,,blood/serum,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00664,P2,toxicity,toxicity_endpoint,677,no,C,485,siRNA-Loaded,siRNA,,,hepatic: hepatotoxicity,1635,34683974,PMC8539450,10.3390/pharmaceutics13101681,Development of siRNA-Loaded Lipid Nanoparticles Targeting Long Non-Coding RNA LINC01257 as a Novel and Safe Therapeutic Approach for t(8;21) Pediatric Acute Myeloid Leukemia.,https://pubmed.ncbi.nlm.nih.gov/34683974/,4. Discussion; paragraph 36,954,cell line,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00665,P2,toxicity,toxicity_endpoint,680,no,C,1094,"IONIS-AGT-LRx (recovered B2 from PMID:34222719, curator:CM)",ASO,,,hepatic: hepatotoxicity,1992,34222719,PMC8246029,10.1016/j.jacbts.2021.04.004,Antisense Inhibition of Angiotensinogen With IONIS-AGT-L(Rx): Results of Phase 1 and Phase 2 Studies.,https://pubmed.ncbi.nlm.nih.gov/34222719/,Discussion; paragraph 25,957,human,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00666,P2,toxicity,toxicity_endpoint,684,no,C,1095,"peptide-siRNA-DOX (recovered B2 from PMID:34948105, curator:CM)",siRNA,,,hepatic: hepatotoxicity,14708,34948105,PMC8703959,10.3390/ijms222413314,Cell Penetrating Peptide-Based Self-Assembly for PD-L1 Targeted Tumor Regression.,https://pubmed.ncbi.nlm.nih.gov/34948105/,5. Conclusions; paragraph 30,961,mouse,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00667,P2,toxicity,toxicity_endpoint,813,no,C,599,inotersen,ASO,,,hepatic: hepatotoxicity,1161,39580013,,10.1016/j.yrtph.2024.105743,Carcinogenicity assessment of inotersen in Tg.rasH2 mice and Sprague-Dawley rats: Implications for 2'-MOE antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/39580013/,PubMed abstract,1090,rat,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00668,P2,toxicity,toxicity_endpoint,829,no,C,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,hepatic: hepatotoxicity,3789,39733890,,10.1016/j.ijbiomac.2024.139219,Shielding siRNA by peptide-based nanofibers: An efficient approach for turning off EGFR gene in breast cancer.,https://pubmed.ncbi.nlm.nih.gov/39733890/,PubMed abstract,1106,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00669,P2,toxicity,toxicity_endpoint,947,no,C,1096,unspecified ASO (PMID:26855051),ASO,,,hepatic: hepatotoxicity,2807,26855051,,10.1016/j.jconrel.2016.02.007,"Evaluation of the effects of chemically different linkers on hepatic accumulations, cell tropism and gene silencing ability of cholesterol-conjugated antisense oligonucleotides.",https://pubmed.ncbi.nlm.nih.gov/26855051/,PubMed abstract,1224,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00670,P2,toxicity,toxicity_endpoint,952,no,C,631,AMO122,ASO,,,general safety: cell viability/cytotoxicity,4257,24511611,,,The systemic administration of an anti-miRNA oligonucleotide encapsulated pH-sensitive liposome results in reduced level of hepatic microRNA-122 in mice.,https://pubmed.ncbi.nlm.nih.gov/24511611/,PubMed abstract sentence 5,1229,cell line,,cell line,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00671,P2,toxicity,toxicity_endpoint,1087,no,C,985,"unspecified siRNA (v1 extraction artefact, pending source re-verification)",siRNA,,,general safety: cell viability/cytotoxicity,8595,40852577,PMC12369976manuscript-id: NIHMS2101912,10.1002/adfm.202508122,Reconfigurable Nucleic Acid Nanoparticles with Therapeutic RNAi Responses to Intracellular Disease Markers.,https://pubmed.ncbi.nlm.nih.gov/40852577/,Results and Discussion > Uptake and Conditional Gene Silencing in 3D Spheroids; paragraph 19,1364,cell line,,cell line,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00672,P2,toxicity,toxicity_endpoint,1124,no,C,600,the ASO,ASO,,,hepatic: hepatotoxicity,10126,39950874,PMC11967816,10.1002/advs.202415169,Nanodot-Inspired Precise Bacterial Gene Suppression in a Smart Hydrogel Bandage for Underwater Wound Healing.,https://pubmed.ncbi.nlm.nih.gov/39950874/,Results and Discussion > Gene Silencing Effects of the ASO/CDs; paragraph 39,1401,cell line,,liver,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00673,P2,toxicity,toxicity_endpoint,1198,no,C,1097,"siRNA‐CaP@PD1‐NVs (recovered B2 from PMID:38973319, curator:CM)",siRNA,,,hepatic: hepatotoxicity,13888,38973319,PMC11425963,10.1002/advs.202400951,Multifunctional Biomimetic Nanocarriers for Dual-Targeted Immuno-Gene Therapy Against Hepatocellular Carcinoma.,https://pubmed.ncbi.nlm.nih.gov/38973319/,Results and Discussion > Multifunctional Validation of siRNA‐CaP@PD1‐NVs In Vitro; paragraph 15,1475,cell line,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00674,P2,toxicity,toxicity_endpoint,1289,no,C,1098,"Pip6a-PMO (recovered B2 from PMID:31479430, curator:CM)",PMO,,,hepatic: hepatotoxicity,4212,31479430,PMC6819114,10.1172/JCI128205,Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice.,https://pubmed.ncbi.nlm.nih.gov/31479430/,Results and Discussion; paragraph 10,1566,,,liver,,,,,,,,,,,,,,,unspecified,unspecified,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,FALSE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00675,P2,toxicity,toxicity_endpoint,1389,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,toxicity: no adverse effects in chronic rodent dosing,4253,25434769,,10.1021/ja505986a,Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing.,https://pubmed.ncbi.nlm.nih.gov/25434769/,PubMed abstract sentence 6,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00676,P2,toxicity,toxicity_endpoint,1390,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: chemical-modification off-target reduction,1003,40877521,,10.1007/978-1-0716-4742-4_24,siRMSD: A Structural Parameter to Reduce Sequence-Dependent Off-Target Effects for siRNA Design with Chemical Modifications.,https://pubmed.ncbi.nlm.nih.gov/40877521/,PubMed abstract sentence 7,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00677,P2,toxicity,toxicity_endpoint,1391,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: PANAC GalNAc-siRNA conjugation improves gene silencing,1049,40151235,PMC11938030,10.1021/jacsau.5c00012,General Platform for Efficient and Modular Assembly of GalNAc-siRNA Conjugates via Primary Amines and o-Nitrobenzyl Alcohol Cyclization Photoclick Chemistry Enabling Rapid Access to Therapeutic Oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/40151235/,PubMed abstract sentence 3,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00678,P2,toxicity,toxicity_endpoint,1392,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: 2-prime ribose modification improves silencing and reduces off-targets,1154,41646886,PMC12870468,10.1016/j.omtn.2026.102835,2'-Ribose-modified nucleotides: A strategy for reducing off-target effects of oligonucleotide drugs.,https://pubmed.ncbi.nlm.nih.gov/41646886/,PubMed abstract sentence 1,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00679,P2,toxicity,toxicity_endpoint,1393,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: seed-region modification reduces off-target binding,1157,41467115,PMC12744844,10.1016/j.omtn.2025.102693,An siRMSD parameter of structural distortion induced by chemical modification is predictive of the off-target effect of siRNA.,https://pubmed.ncbi.nlm.nih.gov/41467115/,PubMed abstract sentence 1,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00680,P2,toxicity,toxicity_endpoint,1394,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: LNA modification alters antiviral oligonucleotide activity,1210,41704705,PMC12907196,10.3389/fmed.2026.1719202,Investigation of the activity of phosphothioate and phosphothioate-LNA-modified oligonucleotides against HIV-1.,https://pubmed.ncbi.nlm.nih.gov/41704705/,PubMed abstract sentence 10,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00681,P2,toxicity,toxicity_endpoint,1395,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: scpBNA/scpBNA2 nuclease-stability and activity profile,1408,41169576,PMC12569656,10.1021/jacsau.5c01005,"Cycloalkane Incorporation Into the 2',4'-Bridge of Locked Nucleic Acid: Enhancing Nuclease Stability, Reducing Phosphorothioate Modifications, and Lowering Hepatotoxicity in Antisense Oligonucleotides.",https://pubmed.ncbi.nlm.nih.gov/41169576/,PubMed abstract sentence 2,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00682,P2,toxicity,toxicity_endpoint,1396,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: nucleobase modification reduces ASO hepatotoxicity,1409,35801870,PMC9303313,10.1093/nar/gkac562,Identification of nucleobase chemical modifications that reduce the hepatotoxicity of gapmer antisense oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/35801870/,PubMed abstract sentence 2,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00683,P2,toxicity,toxicity_endpoint,1398,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: alkyl phosphonate seed modification improves specificity,1497,40737088,PMC12309379,10.1093/nar/gkaf692,Single alkyl phosphonate modification of the siRNA backbone in the seed region enhances specificity and therapeutic profile.,https://pubmed.ncbi.nlm.nih.gov/40737088/,PubMed abstract sentence 2,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00684,P2,toxicity,toxicity_endpoint,1399,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: GNA isonucleotides improve activity/off-target profile,2309,34648028,PMC8565336,10.1093/nar/gkab916,Overcoming GNA/RNA base-pairing limitations using isonucleotides improves the pharmacodynamic activity of ESC+ GalNAc-siRNAs.,https://pubmed.ncbi.nlm.nih.gov/34648028/,PubMed abstract sentence 1,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00685,P2,toxicity,toxicity_endpoint,1400,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,chemistry: 3' terminal 2'-O-methyl activity reduction,2762,32610253,PMC7327867,10.1016/j.omtn.2020.05.010,2'-O-Methyl at 20-mer Guide Strand 3' Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA.,https://pubmed.ncbi.nlm.nih.gov/32610253/,PubMed abstract sentence 3,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00686,P2,toxicity,toxicity_endpoint,1411,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: TfR antibody-ASO brain transcytosis delivery,1080,41166443,PMC12574672,10.1093/nar/gkaf487,Improved targeted delivery of antisense oligonucleotide with an antibody mask.,https://pubmed.ncbi.nlm.nih.gov/41166443/,PubMed abstract sentence 2,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00687,P2,toxicity,toxicity_endpoint,1412,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: PEG-(GK)10 ASO polyplex sentinel lymph node delivery,1246,40540297,,10.1021/jacs.5c04234,Structurally Dynamic Polyplexes Enhance Sentinel Lymph Node Delivery of Antisense Oligonucleotides to Inhibit Breast Cancer Recurrence and Metastasis.,https://pubmed.ncbi.nlm.nih.gov/40540297/,PubMed abstract sentence 3,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00688,P2,toxicity,toxicity_endpoint,1413,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: PNMVA lipid carrier siRNA uptake/endosomal escape,1926,37994483,,10.1002/adhm.202302712,Poly(N-methyl-N-vinylacetamide): A Strong Alternative to PEG for Lipid-Based Nanocarriers Delivering siRNA.,https://pubmed.ncbi.nlm.nih.gov/37994483/,PubMed abstract sentence 2,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00689,P2,toxicity,toxicity_endpoint,1414,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: aptamer-mediated tumor accumulation of cisplatin conjugate,2204,35921069,,10.1089/nat.2022.0024,Nucleic Acid Aptamers Increase the Anticancer Efficiency and Reduce the Toxicity of Cisplatin-Arabinogalactan Conjugates In Vivo.,https://pubmed.ncbi.nlm.nih.gov/35921069/,PubMed abstract sentence 4,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00690,P2,toxicity,toxicity_endpoint,1415,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: PLGA/GelMA hydrogel siRNA cytoplasmic delivery,3276,41647289,PMC12869795,10.1093/rb/rbaf115,A smart nanocomposite bioactive ink for controlled siRNA delivery in calvarial mesenchymal stromal cells as a minimally invasive treatment for craniosynostosis.,https://pubmed.ncbi.nlm.nih.gov/41647289/,PubMed abstract sentence 4,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00691,P2,toxicity,toxicity_endpoint,1416,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: scFv macrophage-membrane liposome siRNA uptake,3529,40898177,PMC12406594,10.1186/s12896-025-01000-5,Biomimetic nanoparticles coated with ScFv-modified macrophage membranes for siRNA delivery to relieve brain metastases of lung cancer.,https://pubmed.ncbi.nlm.nih.gov/40898177/,PubMed title,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00692,P2,toxicity,toxicity_endpoint,1417,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: hydrophobically modified peptide lung siRNA delivery,3950,38311577,PMC11005685,10.1002/advs.202308345,Identification of Potent siRNA Delivery Peptides Using Computer Modeling.,https://pubmed.ncbi.nlm.nih.gov/38311577/,PubMed abstract sentence 1,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00693,P2,toxicity,toxicity_endpoint,1418,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: PAMAM dendrimer morpholino cellular delivery,4246,26134311,PMC4598944manuscript-id: NIHMS704679,10.1039/c5nr01665g,Dendritic nanoconjugates for intracellular delivery of neutral oligonucleotides.,https://pubmed.ncbi.nlm.nih.gov/26134311/,PubMed abstract sentence 1,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00694,P2,toxicity,toxicity_endpoint,1419,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: RGD/KA26 nanoparticle NSUN2 siRNA uptake,4844,42093739,PMC13141635,10.1016/j.mtbio.2026.103116,Nanocarriers engineering for efficient NSUN2 silencing and immune responsive therapy in metastatic colorectal cancer.,https://pubmed.ncbi.nlm.nih.gov/42093739/,PubMed abstract sentence 1,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00695,P2,toxicity,toxicity_endpoint,1420,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: SANG tumor RNAi payload delivery,6833,41500988,PMC12780282,10.1038/s41467-025-66788-4,Tumor agnostic drug delivery with dynamic nanohydrogels.,https://pubmed.ncbi.nlm.nih.gov/41500988/,PubMed abstract sentence 2,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00696,P2,toxicity,toxicity_endpoint,1421,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: recNANP conditional RNAi inducer release,8595,40852577,PMC12369976manuscript-id: NIHMS2101912,10.1002/adfm.202508122,Reconfigurable Nucleic Acid Nanoparticles with Therapeutic RNAi Responses to Intracellular Disease Markers.,https://pubmed.ncbi.nlm.nih.gov/40852577/,PubMed abstract sentence 5,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00697,P2,toxicity,toxicity_endpoint,1422,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: DNA nanohydrogel ASO release after uptake,9651,40305283,PMC12024947,10.3390/biom15040537,A pH-Responsive Dendritic-DNA-Based Nanohydrogel for Dual Drug Delivery.,https://pubmed.ncbi.nlm.nih.gov/40305283/,PubMed abstract sentence 1,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00698,P2,toxicity,toxicity_endpoint,1423,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: hyaluronic acid nanocarrier siGLUT1 tumor delivery,9875,40147647,,10.1016/j.ijbiomac.2025.142492,Overcoming chemoresistance in acute myeloid leukemia via co-delivery of siGLUT1 and hydroxycamptothecin using hyaluronic acid-conjugated nanocarriers.,https://pubmed.ncbi.nlm.nih.gov/40147647/,PubMed title,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00699,P2,toxicity,toxicity_endpoint,1424,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: mannosylated peptidyl lipid siG12S tumor delivery,9951,40073943,,10.1016/j.jconrel.2025.113624,A Mannosylated peptidyl lipid CManDA doped into cytidinyl/cationic lipids efficiently delivers siG12Ss to lung cancer in vivo.,https://pubmed.ncbi.nlm.nih.gov/40073943/,PubMed abstract sentence 1,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00700,P2,toxicity,toxicity_endpoint,1425,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: lysine-modified chitosan siRNA uptake,12104,39348656,,,"A novel, non-toxic chitosan modified with lysine to enhance buffer capacity for siRNA delivery.",https://pubmed.ncbi.nlm.nih.gov/39348656/,PubMed abstract sentence 1,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00701,P2,toxicity,toxicity_endpoint,1426,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: modified PEI nanoparticle siRNA transfection efficacy,12538,36541426,,10.1002/biot.202200415,Analysis of polymeric nanoparticle properties for siRNA/DNA delivery in a tumor xenograft tissue slice air-liquid interface model.,https://pubmed.ncbi.nlm.nih.gov/36541426/,PubMed title,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00702,P2,toxicity,toxicity_endpoint,1427,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: ZNF416 siRNA liposome fibrotic lung targeting,12545,36371191,PMC9652794,10.1186/s12967-022-03740-w,Targeted delivery of ZNF416 siRNA-loaded liposomes attenuates experimental pulmonary fibrosis.,https://pubmed.ncbi.nlm.nih.gov/36371191/,PubMed abstract sentence 8,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00703,P2,toxicity,toxicity_endpoint,1428,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: pH-responsive NP siRNA-prodrug tumor release,12601,31381852,,10.1021/acs.nanolett.9b01660,Stimuli-Responsive Polymer-Prodrug Hybrid Nanoplatform for Multistage siRNA Delivery and Combination Cancer Therapy.,https://pubmed.ncbi.nlm.nih.gov/31381852/,PubMed abstract sentence 1,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00704,P2,toxicity,toxicity_endpoint,1429,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: cysteamine gold nanoparticle siRNA delivery,13711,39521158,,10.1016/j.ijpharm.2024.124928,Cysteamine functionalized gold nanoparticles exhibit high efficiency delivery of genetic materials in embryonic stem cells majorly via clathrin mediated endocytosis.,https://pubmed.ncbi.nlm.nih.gov/39521158/,PubMed abstract sentence 7,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00
core-field-00705,P2,toxicity,toxicity_endpoint,1430,no,C,208,unspecified oligonucleotide,ASO/siRNA mixed context,,,delivery: magnetic hyperthermia siRNA transfection,13877,39052238,,10.1039/d4nr01955e,Membrane-localized magnetic hyperthermia promotes intracellular delivery of cell-impermeant probes.,https://pubmed.ncbi.nlm.nih.gov/39052238/,PubMed abstract sentence 2,,,,,,,,,,,,,,,,,,,needs_curator_sequence_curation,needs_curator_modification_curation,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,TRUE,,,,pending,,,needs_core_field_curation,,TRUE,2026-06-07T21:54:18+00:00