{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T15:14:19Z","timestamp":1788880459687,"version":"build-2803163510"},"reference-count":41,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T00:00:00Z","timestamp":1788220800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T00:00:00Z","timestamp":1788220800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,7,21]],"date-time":"2026-07-21T00:00:00Z","timestamp":1784592000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Nucleic Acids"],"published-print":{"date-parts":[[2026,9]]},"DOI":"10.1016\/j.omtn.2026.103035","type":"journal-article","created":{"date-parts":[[2026,7,24]],"date-time":"2026-07-24T01:19:17Z","timestamp":1784855957000},"page":"103035","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["SafeSense: An open-access safety atlas for antisense oligonucleotides adverse events in human"],"prefix":"10.1016","volume":"37","author":[{"given":"Noam","family":"Vaknin","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aviv","family":"Ziv","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tom","family":"Chernobylsky","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sharon","family":"Avkin-Nachum","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gideon","family":"Rechavi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dan","family":"Dominissini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5826-4533","authenticated-orcid":false,"given":"Nofar","family":"Mor","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtn.2026.103035_bib1","doi-asserted-by":"crossref","first-page":"643681","DOI":"10.3389\/fmolb.2021.643681","article-title":"Nucleic Acid\u2013Based Therapeutics in Orphan Neurological Disorders: Recent Developments","volume":"8","author":"Khorkova","year":"2021","journal-title":"Front. Mol. Biosci."},{"key":"10.1016\/j.omtn.2026.103035_bib2","doi-asserted-by":"crossref","DOI":"10.1016\/j.jbc.2021.100416","article-title":"Antisense technology: A review","volume":"296","author":"Crooke","year":"2021","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.omtn.2026.103035_bib3","doi-asserted-by":"crossref","DOI":"10.3389\/fphar.2023.1304342","article-title":"Antisense oligonucleotides: a novel Frontier in pharmacological strategy","volume":"14","author":"Collotta","year":"2023","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.omtn.2026.103035_bib4","doi-asserted-by":"crossref","first-page":"714","DOI":"10.1016\/j.cmet.2018.03.004","article-title":"RNA-Targeted Therapeutics","volume":"27","author":"Crooke","year":"2018","journal-title":"Cell Metab."},{"key":"10.1016\/j.omtn.2026.103035_bib5","doi-asserted-by":"crossref","first-page":"2529","DOI":"10.1093\/nar\/gkad067","article-title":"Chemistry, structure and function of approved oligonucleotide therapeutics","volume":"51","author":"Egli","year":"2023","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2026.103035_bib6","first-page":"347","article-title":"Antisense Oligonucleotides: Basic Concepts and Mechanisms","volume":"1","author":"Dias","year":"2002","journal-title":"Mol. Cancer Therapeut."},{"key":"10.1016\/j.omtn.2026.103035_bib7","doi-asserted-by":"crossref","first-page":"2075","DOI":"10.1016\/j.ymthe.2017.06.002","article-title":"RNase H1-Dependent Antisense Oligonucleotides Are Robustly Active in Directing RNA Cleavage in Both the Cytoplasm and the Nucleus","volume":"25","author":"Liang","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2026.103035_bib8","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1038\/nn.4508","article-title":"Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy","volume":"20","author":"Corey","year":"2017","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.omtn.2026.103035_bib9","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aaz6100","article-title":"Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome","volume":"12","author":"Han","year":"2020","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtn.2026.103035_bib10","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1089\/nat.2017.0693","article-title":"The Effects of 2\u2019-O-Methoxyethyl Oligonucleotides on Renal Function in Humans","volume":"28","author":"Crooke","year":"2018","journal-title":"Nucleic Acid Therapeut."},{"key":"10.1016\/j.omtn.2026.103035_bib11","doi-asserted-by":"crossref","first-page":"297","DOI":"10.1089\/nat.2018.0723","article-title":"Characterization of the Activity and Distribution of a 2\u2032-O-Methoxyethyl-Modified Antisense Oligonucleotide in Models of Acute and Chronic Kidney Disease","volume":"28","author":"Donner","year":"2018","journal-title":"Nucleic Acid Therapeut."},{"key":"10.1016\/j.omtn.2026.103035_bib12","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1089\/nat.2021.0009","article-title":"Allele-Selective LNA Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1R206H Transcript and Inhibit Osteogenic Differentiation","volume":"32","author":"Maruyama","year":"2022","journal-title":"Nucleic Acid Therapeut."},{"key":"10.1016\/j.omtn.2026.103035_bib13","doi-asserted-by":"crossref","first-page":"640","DOI":"10.1038\/s41587-019-0106-2","article-title":"Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index","volume":"37","author":"Shen","year":"2019","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2026.103035_bib14","doi-asserted-by":"crossref","first-page":"569","DOI":"10.1517\/14740338.2013.793670","article-title":"Efficacy and safety of mipomersen sodium (Kynamro)","volume":"12","author":"Hovingh","year":"2013","journal-title":"Expert Opin. Drug Saf."},{"key":"10.1016\/j.omtn.2026.103035_bib15","doi-asserted-by":"crossref","first-page":"473","DOI":"10.1089\/nat.2022.0034","article-title":"Inflammatory Non-CpG Antisense Oligonucleotides Are Signaling Through TLR9 in Human Burkitt Lymphoma B Bjab Cells","volume":"32","author":"Pollak","year":"2022","journal-title":"Nucleic Acid Therapeut."},{"key":"10.1016\/j.omtn.2026.103035_bib16","doi-asserted-by":"crossref","first-page":"4882","DOI":"10.1093\/nar\/gku142","article-title":"Sequence motifs associated with hepatotoxicity of locked nucleic acid\u2014modified antisense oligonucleotides","volume":"42","author":"Burdick","year":"2014","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2026.103035_bib17","doi-asserted-by":"crossref","DOI":"10.1016\/j.eclinm.2024.102495","article-title":"Effects of tofersen treatment in patients with <em>SOD1<\/em>-ALS in a \u201creal-world\u201d setting \u2013 a 12-month multicenter cohort study from the German early access program","volume":"69","author":"Wiesenfarth","year":"2024","journal-title":"eClinicalMedicine"},{"key":"10.1016\/j.omtn.2026.103035_bib18","doi-asserted-by":"crossref","first-page":"1584","DOI":"10.1093\/nar\/gkx1239","article-title":"Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs","volume":"46","author":"Shen","year":"2018","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2026.103035_bib19","doi-asserted-by":"crossref","first-page":"567","DOI":"10.1002\/cpt.3701","article-title":"FDA Adverse Event Reporting System (FAERS) Essentials: A Guide to Understanding, Applying, and Interpreting Adverse Event Data Reported to FAERS","volume":"118","author":"Potter","year":"2025","journal-title":"Clin. Pharmacol. Ther."},{"key":"10.1016\/j.omtn.2026.103035_bib20","doi-asserted-by":"crossref","first-page":"1065","DOI":"10.1002\/jcph.70034","article-title":"Trends in First-in-Human Studies for Intrathecal Antisense Oligonucleotides: Insights From 2010 to 2024","volume":"65","author":"Schmitz","year":"2025","journal-title":"J. Clin. Pharmacol."},{"key":"10.1016\/j.omtn.2026.103035_bib21","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1089\/nat.2016.0650","article-title":"The Effects of 2\u2019-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical Trials","volume":"27","author":"Crooke","year":"2017","journal-title":"Nucleic Acid Therapeut."},{"key":"10.1016\/j.omtn.2026.103035_bib22","doi-asserted-by":"crossref","first-page":"1644","DOI":"10.1056\/NEJMoa1813279","article-title":"Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease","volume":"381","author":"Kim","year":"2019","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2026.103035_bib23","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtn.2024.102432","article-title":"Personalized allele-specific antisense oligonucleotides for GNAO1-neurodevelopmental disorder","volume":"36","author":"Shomer","year":"2025","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtn.2026.103035_bib24","doi-asserted-by":"crossref","first-page":"5235","DOI":"10.1093\/nar\/gkaa299","article-title":"Phosphorothioate modified oligonucleotide\u2013protein interactions","volume":"48","author":"Crooke","year":"2020","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2026.103035_bib25","doi-asserted-by":"crossref","first-page":"687","DOI":"10.1093\/nar\/gkl1071","article-title":"Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals","volume":"35","author":"Swayze","year":"2007","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2026.103035_bib26","doi-asserted-by":"crossref","first-page":"1911","DOI":"10.1093\/nar\/30.9.1911","article-title":"Design of antisense oligonucleotides stabilized by locked nucleic acids","volume":"30","author":"Kurreck","year":"2002","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2026.103035_bib27","doi-asserted-by":"crossref","first-page":"9634","DOI":"10.1093\/nar\/gkt725","article-title":"Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS","volume":"41","author":"\u00d8stergaard","year":"2013","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2026.103035_bib28","doi-asserted-by":"crossref","first-page":"2093","DOI":"10.1038\/mt.2014.153","article-title":"In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides","volume":"22","author":"Southwell","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2026.103035_bib29","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1038\/s41591-021-01557-6","article-title":"Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide","volume":"28","author":"Tran","year":"2022","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtn.2026.103035_bib30","doi-asserted-by":"crossref","DOI":"10.1093\/nar\/gkaf1333","article-title":"Acute neuronal inhibition response caused by phosphorothioate antisense oligonucleotides following local delivery to the central nervous system","volume":"54","author":"O\u2019Rourke","year":"2026","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2026.103035_bib31","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1038\/nrneurol.2017.148","article-title":"Antisense oligonucleotides: the next frontier for treatment of neurological disorders","volume":"14","author":"Rinaldi","year":"2018","journal-title":"Nat. Rev. Neurol."},{"key":"10.1016\/j.omtn.2026.103035_bib32","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1146\/annurev-pharmtox-010919-023738","article-title":"Antisense Drugs Make Sense for Neurological Diseases","volume":"61","author":"Bennett","year":"2021","journal-title":"Annu. Rev. Pharmacol. Toxicol."},{"key":"10.1016\/j.omtn.2026.103035_bib33","doi-asserted-by":"crossref","first-page":"8107","DOI":"10.1093\/nar\/gkac618","article-title":"Insights into innate immune activation via PS-ASO\u2013protein\u2013TLR9 interactions","volume":"50","author":"Pollak","year":"2022","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2026.103035_bib34","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.161246","article-title":"FXYD2 antisense oligonucleotide provides an efficient approach for long-lasting relief of chronic peripheral pain","volume":"8","author":"Derre","year":"2023","journal-title":"JCI Insight"},{"key":"10.1016\/j.omtn.2026.103035_bib35","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-025-65799-5","article-title":"The nucleobase guanine at the 3\u2019-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity","volume":"16","author":"Valencia","year":"2025","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtn.2026.103035_bib36","unstructured":"European Medicines Agency. (2017). Assessment report: Spinraza (EMA\/289068\/2017). https:\/\/www.ema.europa.eu\/en\/documents\/assessment-report\/spinraza-epar-public-assessment-report_en.pdf."},{"key":"10.1016\/j.omtn.2026.103035_bib37","doi-asserted-by":"crossref","first-page":"1031","DOI":"10.1002\/jcph.884","article-title":"Population Pharmacokinetics of Nusinersen in the Cerebral Spinal Fluid and Plasma of Pediatric Patients With Spinal Muscular Atrophy Following Intrathecal Administrations","volume":"57","author":"Luu","year":"2017","journal-title":"J. Clin. Pharmacol."},{"key":"10.1016\/j.omtn.2026.103035_bib38","unstructured":"No, P. (2024). Assessment Report. 31."},{"key":"10.1016\/j.omtn.2026.103035_bib39","first-page":"2167","article-title":"Intrathecal Therapies for Neurodegenerative Diseases: A Review of Current Approaches and the Urgent Need for Advanced Delivery Systems","volume":"13","author":"Schreiner","year":"2025","journal-title":"omedicines"},{"key":"10.1016\/j.omtn.2026.103035_bib40","doi-asserted-by":"crossref","first-page":"385","DOI":"10.2165\/00002018-200629050-00003","article-title":"Under-Reporting of Adverse Drug Reactions","volume":"29","author":"Hazell","year":"2006","journal-title":"Drug Saf."},{"key":"10.1016\/j.omtn.2026.103035_bib41","doi-asserted-by":"crossref","first-page":"443","DOI":"10.3904\/kjim.2012.27.4.443","article-title":"A Comparison of Active Surveillance Programs Including a Spontaneous Reporting Model for Phamacovigilance of Adverse Drug Events in a Hospital FAU - Yun, Il Seon FAU - Koo","volume":"27","author":"Jin FAU - Park","year":"2012","journal-title":"Korean J Intern Med"}],"container-title":["Molecular Therapy Nucleic Acids"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253126002064?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253126002064?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T15:04:30Z","timestamp":1788879870000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2162253126002064"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,9]]},"references-count":41,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2026,9]]}},"alternative-id":["S2162253126002064"],"URL":"https:\/\/doi.org\/10.1016\/j.omtn.2026.103035","relation":{},"ISSN":["2162-2531"],"issn-type":[{"value":"2162-2531","type":"print"}],"subject":[],"published":{"date-parts":[[2026,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"SafeSense: An open-access safety atlas for antisense oligonucleotides adverse events in human","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Nucleic Acids","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtn.2026.103035","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Author(s). Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"103035"}}