{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T16:21:23Z","timestamp":1791908483083,"version":"4.3.4"},"reference-count":140,"publisher":"MDPI AG","issue":"8","license":[{"start":{"date-parts":[[2020,7,23]],"date-time":"2020-07-23T00:00:00Z","timestamp":1595462400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Zhejiang Province Natural Science Foundation","award":["LZ19H160001"],"award-info":[{"award-number":["LZ19H160001"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Genes"],"abstract":"<jats:p>Neuromuscular disorders encompass a heterogeneous group of conditions that impair the function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being the most common and most severe type of muscular dystrophy, Duchenne muscular dystrophy (DMD), is caused by mutations in the X-linked dystrophin gene. Loss of dystrophin protein leads to recurrent myofiber damage, chronic inflammation, progressive fibrosis, and dysfunction of muscle stem cells. Over the last few years, there has been considerable development of diagnosis and therapeutics for DMD, but current treatments do not cure the disease. Here, we review the current status of DMD pathogenesis and therapy, focusing on mutational spectrum, diagnosis tools, clinical trials, and therapeutic approaches including dystrophin restoration, gene therapy, and myogenic cell transplantation. Furthermore, we present the clinical potential of advanced strategies combining gene editing, cell-based therapy with tissue engineering for the treatment of muscular dystrophy.<\/jats:p>","DOI":"10.3390\/genes11080837","type":"journal-article","created":{"date-parts":[[2020,7,23]],"date-time":"2020-07-23T11:26:01Z","timestamp":1595503561000},"page":"837","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":155,"title":["Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update"],"prefix":"10.3390","volume":"11","author":[{"given":"Chengmei","family":"Sun","sequence":"first","affiliation":[{"name":"Zhejiang University-University of Edinburgh Institute, School of Medicine, Zhejiang University, Haining 314400, China"},{"name":"Department of Medical Oncology, the Second Affiliated Hospital of Zhejiang University School of Medicine, Zhejiang University, Hangzhou 310009, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luoan","family":"Shen","sequence":"additional","affiliation":[{"name":"Zhejiang University-University of Edinburgh Institute, School of Medicine, Zhejiang University, Haining 314400, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zheng","family":"Zhang","sequence":"additional","affiliation":[{"name":"Zhejiang University-University of Edinburgh Institute, School of Medicine, Zhejiang University, Haining 314400, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xin","family":"Xie","sequence":"additional","affiliation":[{"name":"Zhejiang University-University of Edinburgh Institute, School of Medicine, Zhejiang University, Haining 314400, China"},{"name":"Department of Medical Oncology, the Second Affiliated Hospital of Zhejiang University School of Medicine, Zhejiang University, Hangzhou 310009, China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2020,7,23]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"778","DOI":"10.1016\/j.jacbts.2019.06.006","article-title":"Gene Therapy Rescues Cardiac Dysfunction in Duchenne Muscular Dystrophy Mice by Elevating Cardiomyocyte Deoxy-Adenosine Triphosphate","volume":"4","author":"Kolwicz","year":"2019","journal-title":"JACC Basic Transl. 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