{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T08:08:03Z","timestamp":1790842083989,"version":"4.1.0"},"reference-count":24,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2023,3,1]],"date-time":"2023-03-01T00:00:00Z","timestamp":1677628800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,3,1]],"date-time":"2023-03-01T00:00:00Z","timestamp":1677628800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,2,4]],"date-time":"2023-02-04T00:00:00Z","timestamp":1675468800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["F30 HL145955"],"award-info":[{"award-number":["F30 HL145955"]}],"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":["P50 AR065139"],"award-info":[{"award-number":["P50 AR065139"]}],"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":["P50 AR070604"],"award-info":[{"award-number":["P50 AR070604"]}],"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":["R01 AG060542"],"award-info":[{"award-number":["R01 AG060542"]}],"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":["R01 NS124681"],"award-info":[{"award-number":["R01 NS124681"]}],"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":["T32 HL134616"],"award-info":[{"award-number":["T32 HL134616"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods & Clinical Development"],"published-print":{"date-parts":[[2023,3]]},"DOI":"10.1016\/j.omtm.2023.02.001","type":"journal-article","created":{"date-parts":[[2023,2,9]],"date-time":"2023-02-09T01:25:35Z","timestamp":1675905935000},"page":"344-354","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":19,"special_numbering":"C","title":["Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model"],"prefix":"10.1016","volume":"28","author":[{"given":"Arden B.","family":"Piepho","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeovanna","family":"Lowe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurel R.","family":"Cumby","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lisa E.","family":"Dorn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dana M.","family":"Lake","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neha","family":"Rastogi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Megan D.","family":"Gertzen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah L.","family":"Sturgill","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guy L.","family":"Odom","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark T.","family":"Ziolo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federica","family":"Accornero","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey S.","family":"Chamberlain","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jill A.","family":"Rafael-Fortney","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2023.02.001_bib1","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1146\/annurev-genom-090314-025003","article-title":"The pathogenesis and therapy of muscular dystrophies","volume":"16","author":"Guiraud","year":"2015","journal-title":"Annu. Rev. Genom. Hum. Genet."},{"key":"10.1016\/j.omtm.2023.02.001_bib2","doi-asserted-by":"crossref","DOI":"10.3390\/ijms20174098","article-title":"Cardiac pathophysiology and the future of cardiac therapies in Duchenne muscular dystrophy","volume":"20","author":"Meyers","year":"2019","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.omtm.2023.02.001_bib3","doi-asserted-by":"crossref","first-page":"3106","DOI":"10.1096\/fj.10-178913","article-title":"Distinct pathophysiological mechanisms of cardiomyopathy in hearts lacking dystrophin or the sarcoglycan complex","volume":"25","author":"Townsend","year":"2011","journal-title":"Faseb. J."},{"key":"10.1016\/j.omtm.2023.02.001_bib4","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1016\/j.ijcard.2020.05.052","article-title":"Duchenne and Becker muscular dystrophy carriers: evidence of cardiomyopathy by exercise and cardiac MRI testing","volume":"316","author":"Mah","year":"2020","journal-title":"Int. J. Cardiol."},{"key":"10.1016\/j.omtm.2023.02.001_bib5","doi-asserted-by":"crossref","first-page":"1125","DOI":"10.1016\/j.ymthe.2017.02.019","article-title":"Progress toward gene therapy for Duchenne muscular dystrophy","volume":"25","author":"Chamberlain","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2023.02.001_bib6","doi-asserted-by":"crossref","first-page":"2337","DOI":"10.1016\/j.ymthe.2018.07.011","article-title":"Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy","volume":"26","author":"Duan","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2023.02.001_bib7","doi-asserted-by":"crossref","first-page":"1590","DOI":"10.1161\/CIRCULATIONAHA.114.015151","article-title":"Contemporary cardiac issues in Duchenne muscular dystrophy working group of the national heart, lung, and blood institute in collaboration with parent Project muscular dystrophy","volume":"131","author":"McNally","year":"2015","journal-title":"Circulation"},{"key":"10.1016\/j.omtm.2023.02.001_bib8","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.146511","article-title":"Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model","volume":"6","author":"Howard","year":"2021","journal-title":"JCI Insight"},{"key":"10.1016\/j.omtm.2023.02.001_bib9","doi-asserted-by":"crossref","first-page":"623","DOI":"10.1016\/j.ymthe.2019.01.002","article-title":"Development of novel micro-dystrophins with enhanced functionality","volume":"27","author":"Ramos","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2023.02.001_bib10","doi-asserted-by":"crossref","DOI":"10.1093\/hmg\/ddz128","article-title":"AAV-based gene therapies for the muscular dystrophies","volume":"28","author":"Crudele","year":"2019","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2023.02.001_bib11","doi-asserted-by":"crossref","first-page":"1159","DOI":"10.1007\/s00246-012-0622-z","article-title":"Abnormal circumferential strain is present in young Duchenne muscular dystrophy patients","volume":"34","author":"Ryan","year":"2013","journal-title":"Pediatr. Cardiol."},{"key":"10.1016\/j.omtm.2023.02.001_bib12","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1161\/CIRCIMAGING.110.960740","article-title":"Cardiac involvement in patients with muscular dystrophies: magnetic resonance imaging phenotype and genotypic considerations","volume":"4","author":"Verhaert","year":"2011","journal-title":"Circ. Cardiovasc. Imaging"},{"key":"10.1016\/j.omtm.2023.02.001_bib13","doi-asserted-by":"crossref","first-page":"1378","DOI":"10.1038\/mt.2012.81","article-title":"Sustaining cardiac claudin-5 levels prevents functional hallmarks of cardiomyopathy in a muscular dystrophy mouse model","volume":"20","author":"Delf\u00edn","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2023.02.001_bib14","doi-asserted-by":"crossref","first-page":"H2373","DOI":"10.1152\/ajpheart.00448.2005","article-title":"Utrophin deficiency worsens cardiac contractile dysfunction present in dystrophin-deficient mdx mice","volume":"289","author":"Janssen","year":"2005","journal-title":"Am. J. Physiol. Heart Circ. Physiol."},{"key":"10.1016\/j.omtm.2023.02.001_bib15","doi-asserted-by":"crossref","first-page":"766","DOI":"10.1002\/ppul.25227","article-title":"The role of imaging in characterizing the cardiac natural history of Duchenne muscular dystrophy","volume":"56","author":"Lee","year":"2021","journal-title":"Pediatr. Pulmonol."},{"key":"10.1016\/j.omtm.2023.02.001_bib16","doi-asserted-by":"crossref","first-page":"395","DOI":"10.3233\/JND-160173","article-title":"Similar efficacy from specific and non-specific mineralocorticoid receptor antagonist treatment of muscular dystrophy mice","volume":"3","author":"Lowe","year":"2016","journal-title":"J.\u00a0Neuromuscul. Dis."},{"key":"10.1016\/j.omtm.2023.02.001_bib17","doi-asserted-by":"crossref","first-page":"3983","DOI":"10.1002\/ehf2.12996","article-title":"Mineralocorticoid receptor antagonism by finerenone is sufficient to improve function in preclinical muscular dystrophy","volume":"7","author":"Lowe","year":"2020","journal-title":"ESC Heart Fail."},{"key":"10.1016\/j.omtm.2023.02.001_bib18","doi-asserted-by":"crossref","first-page":"582","DOI":"10.1161\/CIRCULATIONAHA.111.031716","article-title":"Early treatment with lisinopril and spironolactone preserves cardiac and skeletal muscle in Duchenne muscular dystrophy mice","volume":"124","author":"Rafael-Fortney","year":"2011","journal-title":"Circulation"},{"key":"10.1016\/j.omtm.2023.02.001_bib24","doi-asserted-by":"crossref","first-page":"1450","DOI":"10.1093\/cvr\/cvaa324","article-title":"Cardiac fibrosis","volume":"117","author":"Frangogiannis","year":"2021","journal-title":"Cardiovasc. Res."},{"key":"10.1016\/j.omtm.2023.02.001_bib19","doi-asserted-by":"crossref","first-page":"1441","DOI":"10.1038\/4033","article-title":"Expression of full-length utrophin prevents muscular dystrophy in mdx mice","volume":"4","author":"Tinsley","year":"1998","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2023.02.001_bib20","doi-asserted-by":"crossref","first-page":"717","DOI":"10.1016\/S0092-8674(00)80532-2","article-title":"Utrophin-dystrophin-deficient\u00a0mice as a model for Duchenne muscular dystrophy","volume":"90","author":"Deconinck","year":"1997","journal-title":"Cell"},{"key":"10.1016\/j.omtm.2023.02.001_bib21","doi-asserted-by":"crossref","first-page":"294","DOI":"10.1016\/S0960-8966(02)00286-9","article-title":"Cardiomyopathic features associated with muscular dystrophy are independent of dystrophin absence in cardiovasculature","volume":"13","author":"Hainsey","year":"2003","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.omtm.2023.02.001_bib22","first-page":"2030","article-title":"Mineralocorticoid receptor antagonists improve membrane integrity independent of muscle force in muscular dystrophy","volume":"28","author":"Hauck","year":"2019","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2023.02.001_bib23","doi-asserted-by":"crossref","first-page":"295","DOI":"10.3233\/JND-180323","article-title":"Mineralocorticoid receptor antagonists in muscular dystrophy mice during aging and exercise","volume":"5","author":"Lowe","year":"2018","journal-title":"J.\u00a0Neuromuscul. Dis."}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050123000116?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050123000116?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:28:43Z","timestamp":1762421323000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050123000116"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,3]]},"references-count":24,"alternative-id":["S2329050123000116"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2023.02.001","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2023,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2023.02.001","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"}]}}