{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T08:07:05Z","timestamp":1790842025694,"version":"4.1.0"},"reference-count":36,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2020,12,1]],"date-time":"2020-12-01T00:00:00Z","timestamp":1606780800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2020,12,1]],"date-time":"2020-12-01T00:00:00Z","timestamp":1606780800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2020,9,24]],"date-time":"2020-09-24T00:00:00Z","timestamp":1600905600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["DP2-OD008586"],"award-info":[{"award-number":["DP2-OD008586"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["R01AR069085"],"award-info":[{"award-number":["R01AR069085"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["R21AR072265"],"award-info":[{"award-number":["R21AR072265"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["U01EB028901"],"award-info":[{"award-number":["U01EB028901"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["UG3AR075336"],"award-info":[{"award-number":["UG3AR075336"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["UH3TR002142"],"award-info":[{"award-number":["UH3TR002142"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005202","name":"Muscular Dystrophy Association","doi-asserted-by":"publisher","award":["RO1AR18860"],"award-info":[{"award-number":["RO1AR18860"]}],"id":[{"id":"10.13039\/100005202","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005202","name":"Muscular Dystrophy Association","doi-asserted-by":"publisher","award":["U54AR065139"],"award-info":[{"award-number":["U54AR065139"]}],"id":[{"id":"10.13039\/100005202","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100014943","name":"Sarepta Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014943","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"published-print":{"date-parts":[[2020,12]]},"DOI":"10.1016\/j.omtm.2020.09.016","type":"journal-article","created":{"date-parts":[[2020,9,28]],"date-time":"2020-09-28T22:28:30Z","timestamp":1601332110000},"page":"320-329","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":61,"special_numbering":"C","title":["In\u00a0Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy"],"prefix":"10.1016","volume":"19","author":[{"given":"Jennifer B.","family":"Kwon","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adarsh R.","family":"Ettyreddy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashish","family":"Vankara","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel D.","family":"Bohning","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Garth","family":"Devlin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephen D.","family":"Hauschka","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aravind","family":"Asokan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1478-4013","authenticated-orcid":false,"given":"Charles A.","family":"Gersbach","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2020.09.016_bib1","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1038\/nrg3460","article-title":"Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches","volume":"14","author":"Fairclough","year":"2013","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.omtm.2020.09.016_bib2","doi-asserted-by":"crossref","first-page":"1059","DOI":"10.1016\/j.cell.2010.11.039","article-title":"Short telomeres and stem cell exhaustion model Duchenne muscular dystrophy in mdx\/mTR mice","volume":"143","author":"Sacco","year":"2010","journal-title":"Cell"},{"key":"10.1016\/j.omtm.2020.09.016_bib3","doi-asserted-by":"crossref","first-page":"722","DOI":"10.1097\/WCO.0000000000000739","article-title":"Update in Duchenne and Becker muscular dystrophy","volume":"32","author":"Waldrop","year":"2019","journal-title":"Curr. Opin. Neurol."},{"key":"10.1016\/j.omtm.2020.09.016_bib4","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1038\/nrneurol.2017.126","article-title":"Genome engineering: a new approach to gene therapy for neuromuscular disorders","volume":"13","author":"Nelson","year":"2017","journal-title":"Nat. Rev. Neurol."},{"key":"10.1016\/j.omtm.2020.09.016_bib5","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1126\/science.aad5143","article-title":"In\u00a0vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy","volume":"351","author":"Nelson","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.omtm.2020.09.016_bib6","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1126\/science.aad5177","article-title":"In\u00a0vivo gene editing in dystrophic mouse muscle and muscle stem cells","volume":"351","author":"Tabebordbar","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.omtm.2020.09.016_bib7","doi-asserted-by":"crossref","first-page":"400","DOI":"10.1126\/science.aad5725","article-title":"Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy","volume":"351","author":"Long","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.omtm.2020.09.016_bib8","doi-asserted-by":"crossref","first-page":"564","DOI":"10.1038\/mt.2015.192","article-title":"CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice","volume":"24","author":"Xu","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.09.016_bib9","doi-asserted-by":"crossref","first-page":"14454","DOI":"10.1038\/ncomms14454","article-title":"Muscle-specific CRISPR\/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy","volume":"8","author":"Bengtsson","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2020.09.016_bib10","doi-asserted-by":"crossref","first-page":"eaan8081","DOI":"10.1126\/scitranslmed.aan8081","article-title":"Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy","volume":"9","author":"Amoasii","year":"2017","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2020.09.016_bib11","doi-asserted-by":"crossref","first-page":"86","DOI":"10.1126\/science.aau1549","article-title":"Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy","volume":"362","author":"Amoasii","year":"2018","journal-title":"Science"},{"key":"10.1016\/j.omtm.2020.09.016_bib12","doi-asserted-by":"crossref","first-page":"eaan4672","DOI":"10.1126\/science.aan4672","article-title":"Gene therapy comes of age","volume":"359","author":"Dunbar","year":"2018","journal-title":"Science"},{"key":"10.1016\/j.omtm.2020.09.016_bib13","doi-asserted-by":"crossref","first-page":"321","DOI":"10.1038\/nbt1073","article-title":"Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart","volume":"23","author":"Wang","year":"2005","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.09.016_bib14","doi-asserted-by":"crossref","first-page":"14038","DOI":"10.1038\/mtm.2014.38","article-title":"Adeno-associated viral (AAV) vectors do not efficiently target muscle satellite cells","volume":"1","author":"Arnett","year":"2014","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2020.09.016_bib15","doi-asserted-by":"crossref","first-page":"1254","DOI":"10.1016\/j.celrep.2019.03.105","article-title":"In Situ Modification of Tissue Stem and Progenitor Cell Genomes","volume":"27","author":"Goldstein","year":"2019","journal-title":"Cell Rep."},{"key":"10.1016\/j.omtm.2020.09.016_bib16","doi-asserted-by":"crossref","first-page":"1568","DOI":"10.1016\/j.ymthe.2019.06.012","article-title":"AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice","volume":"27","author":"Nance","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.09.016_bib17","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1038\/nn.2467","article-title":"A robust and high-throughput Cre reporting and characterization system for the whole mouse brain","volume":"13","author":"Madisen","year":"2010","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.omtm.2020.09.016_bib18","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1016\/j.ydbio.2013.04.018","article-title":"Embryonic founders of adult muscle stem cells are primed by the determination gene Mrf4","volume":"381","author":"Sambasivan","year":"2013","journal-title":"Dev. Biol."},{"key":"10.1016\/j.omtm.2020.09.016_bib19","doi-asserted-by":"crossref","first-page":"294","DOI":"10.1038\/mt.2008.261","article-title":"Transduction efficiencies of novel AAV vectors in mouse airway epithelium in\u00a0vivo and human ciliated airway epithelium in\u00a0vitro","volume":"17","author":"Limberis","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.09.016_bib20","first-page":"997","article-title":"Notch signaling deficiency underlies age-dependent depletion of satellite cells in muscular dystrophy","volume":"7","author":"Jiang","year":"2014","journal-title":"Dis. Model. Mech."},{"key":"10.1016\/j.omtm.2020.09.016_bib21","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1038\/s41591-019-0344-3","article-title":"Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy","volume":"25","author":"Nelson","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2020.09.016_bib22","doi-asserted-by":"crossref","first-page":"525","DOI":"10.1006\/mthe.2001.0496","article-title":"Immune evasion by muscle-specific gene expression in dystrophic muscle","volume":"4","author":"Hartigan-O\u2019Connor","year":"2001","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.09.016_bib23","series-title":"Muscle Gene Therapy: Methods and Protocols","first-page":"3","article-title":"Design and testing of regulatory cassettes for optimal activity in skeletal and cardiac muscles","author":"Himeda","year":"2011"},{"key":"10.1016\/j.omtm.2020.09.016_bib24","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1038\/6981","article-title":"Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences","volume":"17","author":"Li","year":"1999","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2020.09.016_bib25","doi-asserted-by":"crossref","first-page":"320","DOI":"10.1038\/sj.mt.6300027","article-title":"Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle","volume":"15","author":"Salva","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.09.016_bib26","doi-asserted-by":"crossref","first-page":"e0147198","DOI":"10.1371\/journal.pone.0147198","article-title":"Comparative Study of Injury Models for Studying Muscle Regeneration in Mice","volume":"11","author":"Hardy","year":"2016","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2020.09.016_bib27","doi-asserted-by":"crossref","first-page":"1455","DOI":"10.1038\/nm.3990","article-title":"Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division","volume":"21","author":"Dumont","year":"2015","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2020.09.016_bib28","doi-asserted-by":"crossref","first-page":"1971","DOI":"10.1002\/stem.1158","article-title":"Donor satellite cell engraftment is significantly augmented when the host niche is preserved and endogenous satellite cells are incapacitated","volume":"30","author":"Boldrin","year":"2012","journal-title":"Stem Cells"},{"key":"10.1016\/j.omtm.2020.09.016_bib29","doi-asserted-by":"crossref","first-page":"e72147","DOI":"10.1371\/journal.pone.0072147","article-title":"Revertant fibers in the mdx murine model of Duchenne muscular dystrophy: an age- and muscle-related reappraisal","volume":"8","author":"Pigozzo","year":"2013","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2020.09.016_bib30","doi-asserted-by":"crossref","first-page":"11842","DOI":"10.1038\/s41598-019-48156-7","article-title":"Muscle satellite cells and impaired late stage regeneration in different murine models for muscular dystrophies","volume":"9","author":"Ribeiro","year":"2019","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2020.09.016_bib31","doi-asserted-by":"crossref","first-page":"1572","DOI":"10.1242\/dev.114223","article-title":"Intrinsic and extrinsic mechanisms regulating satellite cell function","volume":"142","author":"Dumont","year":"2015","journal-title":"Development"},{"key":"10.1016\/j.omtm.2020.09.016_bib32","doi-asserted-by":"crossref","first-page":"7087","DOI":"10.1038\/ncomms8087","article-title":"Muscle stem cells contribute to myofibres in sedentary adult mice","volume":"6","author":"Keefe","year":"2015","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2020.09.016_bib33","doi-asserted-by":"crossref","first-page":"638","DOI":"10.1369\/0022155412452417","article-title":"A contemporary atlas of the mouse diaphragm: myogenicity, vascularity, and the Pax3 connection","volume":"60","author":"Stuelsatz","year":"2012","journal-title":"J.\u00a0Histochem. Cytochem."},{"key":"10.1016\/j.omtm.2020.09.016_bib34","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1016\/j.coviro.2016.05.001","article-title":"Engineering AAV receptor footprints for gene therapy","volume":"18","author":"Madigan","year":"2016","journal-title":"Curr. Opin. Virol."},{"key":"10.1016\/j.omtm.2020.09.016_bib35","doi-asserted-by":"crossref","first-page":"889","DOI":"10.1038\/s41551-017-0137-2","article-title":"Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in\u00a0vivo induces homology-directed DNA repair","volume":"1","author":"Lee","year":"2017","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.omtm.2020.09.016_bib36","doi-asserted-by":"crossref","first-page":"637","DOI":"10.1146\/annurev-chembioeng-080615-034711","article-title":"Engineering Delivery Vehicles for Genome Editing","volume":"7","author":"Nelson","year":"2016","journal-title":"Annu. Rev. Chem. Biomol. Eng."}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050120302011?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050120302011?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:25:22Z","timestamp":1762421122000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050120302011"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,12]]},"references-count":36,"alternative-id":["S2329050120302011"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2020.09.016","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2020,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"In\u00a0Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2020.09.016","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2020 The Author(s).","name":"copyright","label":"Copyright"}]}}