{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,4]],"date-time":"2026-09-04T21:13:37Z","timestamp":1788556417060,"version":"build-2803163510"},"reference-count":28,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T00:00:00Z","timestamp":1777507200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000065","name":"National Institute of Neurological Disorders and Stroke","doi-asserted-by":"publisher","award":["R01NS126352"],"award-info":[{"award-number":["R01NS126352"]}],"id":[{"id":"10.13039\/100000065","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100009633","name":"National Institute of Child Health and Human Development","doi-asserted-by":"publisher","award":["P50HD117373"],"award-info":[{"award-number":["P50HD117373"]}],"id":[{"id":"10.13039\/100009633","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Advances"],"published-print":{"date-parts":[[2026,6]]},"DOI":"10.1016\/j.omta.2026.201750","type":"journal-article","created":{"date-parts":[[2026,5,6]],"date-time":"2026-05-06T15:35:21Z","timestamp":1778081721000},"page":"201750","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["U7snRNA-mediated skipping of intron-derived pseudoexons restores full-length DMD expression in patient-derived cell lines"],"prefix":"10.1016","volume":"34","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7428-2563","authenticated-orcid":false,"given":"Joseph C.","family":"Beljan","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefan","family":"Nicolau","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hui","family":"Lin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacob W.","family":"Lay","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma C.","family":"Frair","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liubov V.","family":"Gushchina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert B.","family":"Weiss","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6440-3376","authenticated-orcid":false,"given":"Kevin M.","family":"Flanigan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omta.2026.201750_bib1","doi-asserted-by":"crossref","first-page":"412","DOI":"10.1016\/0968-0004(89)90290-9","article-title":"the protein product of the Duchenne\/Becker muscular dystrophy gene","volume":"14","author":"Monaco","year":"1989","journal-title":"Trends Biochem. Sci."},{"key":"10.1016\/j.omta.2026.201750_bib2","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1083\/jcb.201212142","article-title":"The cell biology of disease: cellular and molecular mechanisms underlying muscular dystrophy","volume":"201","author":"Rahimov","year":"2013","journal-title":"J. Cell Biol."},{"key":"10.1016\/j.omta.2026.201750_bib3","doi-asserted-by":"crossref","first-page":"e2304","DOI":"10.1212\/WNL.0000000000012910","article-title":"Life Expectancy in Duchenne Muscular Dystrophy: Reproduced Individual Patient Data Meta-analysis","volume":"97","author":"Broomfield","year":"2021","journal-title":"Neurology"},{"key":"10.1016\/j.omta.2026.201750_bib4","doi-asserted-by":"crossref","first-page":"679","DOI":"10.3233\/JND-230107","article-title":"Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study","volume":"11","author":"Nicolau","year":"2024","journal-title":"J. Neuromuscul. Dis."},{"key":"10.1016\/j.omta.2026.201750_bib5","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1002\/ana.24555","article-title":"Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy","volume":"79","author":"Mendell","year":"2016","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omta.2026.201750_bib6","doi-asserted-by":"crossref","first-page":"2689","DOI":"10.1016\/j.ymthe.2025.04.038","article-title":"Clinical applications of exon-skipping antisense oligonucleotides in neuromuscular diseases","volume":"33","author":"Torres-Masjoan","year":"2025","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omta.2026.201750_bib7","series-title":"FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy","year":"2023"},{"key":"10.1016\/j.omta.2026.201750_bib8","doi-asserted-by":"crossref","first-page":"332","DOI":"10.1038\/s41591-024-03304-z","article-title":"AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial","volume":"31","author":"Mendell","year":"2025","journal-title":"Nat. Med."},{"key":"10.1016\/j.omta.2026.201750_bib9","doi-asserted-by":"crossref","DOI":"10.1212\/WNL.0000000000214085","article-title":"Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy: The Ethics and Financial Toxicity of Exuberant Hope","volume":"105","author":"Smith","year":"2025","journal-title":"Neurology"},{"key":"10.1016\/j.omta.2026.201750_bib10","doi-asserted-by":"crossref","first-page":"3141","DOI":"10.1093\/nar\/23.16.3141","article-title":"Assembly, nuclear import and function of U7 snRNPs studied by microinjection of synthetic U7 RNA into Xenopus oocytes","volume":"23","author":"Stefanovic","year":"1995","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omta.2026.201750_bib11","doi-asserted-by":"crossref","first-page":"4929","DOI":"10.1073\/pnas.95.9.4929","article-title":"Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs","volume":"95","author":"Gorman","year":"1998","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omta.2026.201750_bib12","first-page":"1317","article-title":"U7 snRNA, a Small RNA with a Big Impact in Gene Therapy","volume":"32","author":"Lesman","year":"2021","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omta.2026.201750_bib13","doi-asserted-by":"crossref","first-page":"1796","DOI":"10.1126\/science.1104297","article-title":"Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping","volume":"306","author":"Goyenvalle","year":"2004","journal-title":"Science"},{"key":"10.1016\/j.omta.2026.201750_bib14","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.omta.2026.201750_bib15","doi-asserted-by":"crossref","DOI":"10.1002\/jgm.3321","article-title":"U7 snRNA: A tool for gene therapy","volume":"23","author":"Gadgil","year":"2021","journal-title":"J. Gene Med."},{"key":"10.1016\/j.omta.2026.201750_bib16","doi-asserted-by":"crossref","first-page":"77","DOI":"10.3233\/JND-190431","article-title":"Pseudoexons of the DMD Gene","volume":"7","author":"Keegan","year":"2020","journal-title":"J. Neuromuscul. Dis."},{"key":"10.1016\/j.omta.2026.201750_bib17","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1002\/ajmg.a.30617","article-title":"Improved molecular diagnosis of dystrophinopathies in an unselected clinical cohort","volume":"134","author":"Dent","year":"2005","journal-title":"Am. J. Med. Genet."},{"key":"10.1016\/j.omta.2026.201750_bib18","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1002\/humu.24343","article-title":"Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy","volume":"43","author":"Waldrop","year":"2022","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.omta.2026.201750_bib19","article-title":"Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders","volume":"170","author":"Almeida","year":"2021","journal-title":"J. Vis. Exp."},{"key":"10.1016\/j.omta.2026.201750_bib20","doi-asserted-by":"crossref","first-page":"637","DOI":"10.1002\/ana.23982","article-title":"Eteplirsen for the treatment of Duchenne muscular dystrophy","volume":"74","author":"Mendell","year":"2013","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omta.2026.201750_bib21","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.omta.2026.201750_bib22","doi-asserted-by":"crossref","first-page":"3038","DOI":"10.1182\/blood-2011-09-382317","article-title":"Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer","volume":"119","author":"Buchlis","year":"2012","journal-title":"Blood"},{"key":"10.1016\/j.omta.2026.201750_bib23","doi-asserted-by":"crossref","first-page":"1364","DOI":"10.1016\/j.ymthe.2022.03.004","article-title":"Durability of transgene expression after rAAV gene therapy","volume":"30","author":"Muhuri","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omta.2026.201750_bib24","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2023.101144","article-title":"Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model","volume":"31","author":"Gushchina","year":"2023","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omta.2026.201750_bib25","doi-asserted-by":"crossref","first-page":"882","DOI":"10.1089\/hum.2020.286","article-title":"Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping","volume":"32","author":"Gushchina","year":"2021","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omta.2026.201750_bib26","doi-asserted-by":"crossref","first-page":"325","DOI":"10.1016\/j.omtm.2021.03.014","article-title":"Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping","volume":"21","author":"Simmons","year":"2021","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omta.2026.201750_bib27","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1186\/1743-422X-10-74","article-title":"A survey of ex vivo\/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype","volume":"10","author":"Ellis","year":"2013","journal-title":"Virol. J."},{"key":"10.1016\/j.omta.2026.201750_bib28","doi-asserted-by":"crossref","DOI":"10.1093\/nar\/gkaf241","article-title":"AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy","volume":"53","author":"Etxaniz","year":"2025","journal-title":"Nucleic Acids Res."}],"container-title":["Molecular Therapy Advances"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S3117387X26000856?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S3117387X26000856?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,9,4]],"date-time":"2026-09-04T20:30:17Z","timestamp":1788553817000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S3117387X26000856"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,6]]},"references-count":28,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2026,6]]}},"alternative-id":["S3117387X26000856"],"URL":"https:\/\/doi.org\/10.1016\/j.omta.2026.201750","relation":{},"ISSN":["3117-387X"],"issn-type":[{"value":"3117-387X","type":"print"}],"subject":[],"published":{"date-parts":[[2026,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"U7snRNA-mediated skipping of intron-derived pseudoexons restores full-length DMD expression in patient-derived cell lines","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Advances","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omta.2026.201750","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Authors. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"201750"}}