{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T00:58:52Z","timestamp":1791939532333,"version":"4.3.4"},"reference-count":33,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2023,12,1]],"date-time":"2023-12-01T00:00:00Z","timestamp":1701388800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,12,1]],"date-time":"2023-12-01T00:00:00Z","timestamp":1701388800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,9,15]],"date-time":"2023-09-15T00:00:00Z","timestamp":1694736000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100018036","name":"Nippon Shinyaku","doi-asserted-by":"publisher","award":["28-6"],"award-info":[{"award-number":["28-6"]}],"id":[{"id":"10.13039\/100018036","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100009438","name":"National Center of Neurology and Psychiatry","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100009438","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100009619","name":"Japan Agency for Medical Research and Development","doi-asserted-by":"publisher","award":["18lm0203066"],"award-info":[{"award-number":["18lm0203066"]}],"id":[{"id":"10.13039\/100009619","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Nucleic Acids"],"published-print":{"date-parts":[[2023,12]]},"DOI":"10.1016\/j.omtn.2023.102034","type":"journal-article","created":{"date-parts":[[2023,9,20]],"date-time":"2023-09-20T15:43:59Z","timestamp":1695224639000},"page":"102034","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":14,"special_numbering":"C","title":["Exon 44 skipping in Duchenne muscular dystrophy: NS-089\/NCNP-02, a dual-targeting antisense oligonucleotide"],"prefix":"10.1016","volume":"34","author":[{"given":"Naoki","family":"Watanabe","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuichiro","family":"Tone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tetsuya","family":"Nagata","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Satoru","family":"Masuda","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Takashi","family":"Saito","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Norio","family":"Motohashi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kazuchika","family":"Takagaki","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshitsugu","family":"Aoki","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shin\u2019ichi","family":"Takeda","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtn.2023.102034_bib1","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1016\/0960-8966(91)90039-U","article-title":"Population frequencies of inherited neuromuscular diseases--a world survey","volume":"1","author":"Emery","year":"1991","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.omtn.2023.102034_bib2","first-page":"498","article-title":"The Molecular Basis for Duchenne versus Becker Muscular Dystrophy: Correlation of Severity with Type of Deletion","volume":"45","author":"Koenig","year":"1989","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.omtn.2023.102034_bib3","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1007\/978-1-4939-8651-4_2","article-title":"An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases","volume":"1828","author":"Rodrigues","year":"2018","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.omtn.2023.102034_bib4","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1007\/s40265-020-01267-2","article-title":"Golodirsen: First Approval","volume":"80","author":"Heo","year":"2020","journal-title":"Drugs"},{"key":"10.1016\/j.omtn.2023.102034_bib5","doi-asserted-by":"crossref","first-page":"1027","DOI":"10.1007\/s40265-020-01339-3","article-title":"Viltolarsen: First Approval","volume":"80","author":"Dhillon","year":"2020","journal-title":"Drugs"},{"key":"10.1016\/j.omtn.2023.102034_bib6","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1007\/s40265-021-01512-2","article-title":"Casimersen: First Approval","volume":"81","author":"Shirley","year":"2021","journal-title":"Drugs"},{"key":"10.1016\/j.omtn.2023.102034_bib7","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1002\/humu.20918","article-title":"Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations","volume":"30","author":"Aartsma-Rus","year":"2009","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.omtn.2023.102034_bib9","doi-asserted-by":"crossref","first-page":"2393","DOI":"10.1002\/acn3.51235","article-title":"Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1\/2 study","volume":"7","author":"Komaki","year":"2020","journal-title":"Ann. Clin. Transl. Neurol."},{"key":"10.1016\/j.omtn.2023.102034_bib10","doi-asserted-by":"crossref","first-page":"S71","DOI":"10.1016\/S0960-8966(02)00086-X","article-title":"Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy","volume":"12","author":"Aartsma-Rus","year":"2002","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.omtn.2023.102034_bib11","series-title":"ENA nucleic acid drugs modifying splicing in mRNA precursor","author":"Matsuo","year":"2004"},{"key":"10.1016\/j.omtn.2023.102034_bib12","series-title":"Antisense oligonucleotides for inducing exon skipping and methods of use thereof","author":"Wilton","year":"2006"},{"key":"10.1016\/j.omtn.2023.102034_bib13","series-title":"Method for efficient exon (44) skipping in Duchenne muscular dystrophy and associated means","author":"Platenburg","year":"2009"},{"key":"10.1016\/j.omtn.2023.102034_bib14","series-title":"Multiple exon skipping compositions for DMD","author":"Sazani","year":"2010"},{"key":"10.1016\/j.omtn.2023.102034_bib15","doi-asserted-by":"crossref","first-page":"554","DOI":"10.1038\/mt.2008.287","article-title":"Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene","volume":"17","author":"Popplewell","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2023.102034_bib16","series-title":"Antisense molecules and methods for treating pathologies","author":"Wilton","year":"2011"},{"key":"10.1016\/j.omtn.2023.102034_bib17","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1007\/978-1-0716-2772-3_7","article-title":"Restoring Dystrophin Expression with Exon 44 and 53 Skipping in the DMD Gene in Immortalized Myotubes","volume":"2587","author":"Echigoya","year":"2023","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.omtn.2023.102034_bib18","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0120058","article-title":"In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy","volume":"10","author":"Echigoya","year":"2015","journal-title":"PLoS One"},{"key":"10.1016\/j.omtn.2023.102034_bib19","doi-asserted-by":"crossref","first-page":"1288","DOI":"10.1038\/sj.mt.6300095","article-title":"Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript","volume":"15","author":"Wilton","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2023.102034_bib20","doi-asserted-by":"crossref","first-page":"1995","DOI":"10.1038\/mt.2010.186","article-title":"In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse","volume":"18","author":"Aoki","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2023.102034_bib21","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1186\/1471-2350-9-105","article-title":"Assessment of the feasibility of exon 45-55 multiexon skipping for duchenne muscular dystrophy","volume":"9","author":"van Vliet","year":"2008","journal-title":"BMC Med. Genet."},{"key":"10.1016\/j.omtn.2023.102034_bib22","doi-asserted-by":"crossref","first-page":"9456","DOI":"10.1073\/pnas.142302299","article-title":"Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells","volume":"99","author":"De Angelis","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2023.102034_bib23","doi-asserted-by":"crossref","first-page":"557","DOI":"10.1007\/s000180300047","article-title":"U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping","volume":"60","author":"Brun","year":"2003","journal-title":"Cell. Mol. Life Sci."},{"key":"10.1016\/j.omtn.2023.102034_bib24","doi-asserted-by":"crossref","first-page":"982","DOI":"10.1001\/jamaneurol.2020.1264","article-title":"Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial","volume":"77","author":"Clemens","year":"2020","journal-title":"JAMA Neurol."},{"key":"10.1016\/j.omtn.2023.102034_bib25","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1016\/j.omtn.2018.09.017","article-title":"NS-065\/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy","volume":"13","author":"Watanabe","year":"2018","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtn.2023.102034_bib26","doi-asserted-by":"crossref","first-page":"1547","DOI":"10.1093\/hmg\/10.15.1547","article-title":"Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells","volume":"10","author":"van Deutekom","year":"2001","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2023.102034_bib27","doi-asserted-by":"crossref","first-page":"668","DOI":"10.1002\/ana.24365","article-title":"Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45","volume":"77","author":"Findlay","year":"2015","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omtn.2023.102034_bib28","first-page":"54","article-title":"Exploring the Molecular Basis for Variability among Patients with Becker Muscular Dystrophy: Dystrophin Gene and Protein Studies","volume":"49","author":"Beggs","year":"1991","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.omtn.2023.102034_bib29","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1007\/s12017-012-8186-x","article-title":"Assessing pathogenicity for novel mutation\/sequence variants: the value of healthy older individuals","volume":"14","author":"Zatz","year":"2012","journal-title":"NeuroMolecular Med."},{"issue":"Pt 1","key":"10.1016\/j.omtn.2023.102034_bib30","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1093\/brain\/117.1.1-a","article-title":"Clinical variability in Becker muscular dystrophy. Genetic, biochemical and immunohistochemical correlates","volume":"117","author":"Comi","year":"1994","journal-title":"Brain"},{"key":"10.1016\/j.omtn.2023.102034_bib31","doi-asserted-by":"crossref","first-page":"4914","DOI":"10.1093\/hmg\/ddt341","article-title":"Highly efficient in\u00a0vivo delivery of PMO into regenerating myotubes and rescue in laminin-\u03b12 chain-null congenital muscular dystrophy mice","volume":"22","author":"Aoki","year":"2013","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2023.102034_bib32","doi-asserted-by":"crossref","first-page":"175","DOI":"10.1038\/nm1345","article-title":"Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology","volume":"12","author":"Alter","year":"2006","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtn.2023.102034_bib33","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1038\/gt.2009.120","article-title":"Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino","volume":"17","author":"Wu","year":"2010","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtn.2023.102034_bib34","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1186\/s13395-015-0070-6","article-title":"Elusive sources of variability of dystrophin rescue by exon skipping","volume":"5","author":"Vila","year":"2015","journal-title":"Skeletal Muscle"}],"container-title":["Molecular Therapy Nucleic Acids"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253123002524?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253123002524?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,4,13]],"date-time":"2025-04-13T01:36:32Z","timestamp":1744508192000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2162253123002524"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,12]]},"references-count":33,"alternative-id":["S2162253123002524"],"URL":"https:\/\/doi.org\/10.1016\/j.omtn.2023.102034","relation":{},"ISSN":["2162-2531"],"issn-type":[{"value":"2162-2531","type":"print"}],"subject":[],"published":{"date-parts":[[2023,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Exon 44 skipping in Duchenne muscular dystrophy: NS-089\/NCNP-02, a dual-targeting antisense oligonucleotide","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Nucleic Acids","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtn.2023.102034","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 The Author(s).","name":"copyright","label":"Copyright"}],"article-number":"102034"}}