{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T01:01:52Z","timestamp":1790816512579,"version":"4.1.0"},"reference-count":47,"publisher":"SAGE Publications","issue":"9-10","license":[{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2023,5]]},"abstract":"<jats:p>\n                    Duchenne muscular dystrophy (DMD) was named more than 150 years ago. About four decades ago, the\n                    <jats:italic toggle=\"yes\">DMD<\/jats:italic>\n                    gene was discovered, and the reading frame shift was determined as the genetic underpinning. These pivotal findings significantly changed the landscape of DMD therapy development. Restoration of dystrophin expression with gene therapy became a primary focus. Investment in gene therapy has led to the approval of exon skipping by regulatory agencies, multiple clinical trials of systemic microdystrophin therapy using adeno-associated virus vectors, and revolutionary genome editing therapy using the CRISPR technology. However, many important issues surfaced during the clinical translation of DMD gene therapy (such as low efficiency of exon skipping, immune toxicity-induced serious adverse events, and patient death). In this issue of\n                    <jats:italic toggle=\"yes\">Human Gene Therapy<\/jats:italic>\n                    , several research articles highlighted some of the latest developments in DMD gene therapy. Importantly, a collection of articles from experts in the field reviewed the progress, major challenges, and future directions of DMD gene therapy. These insightful discussions have significant implications for gene therapy of other neuromuscular diseases.\n                  <\/jats:p>","DOI":"10.1089\/hum.2023.29242.ddu","type":"journal-article","created":{"date-parts":[[2023,5,23]],"date-time":"2023-05-23T12:31:32Z","timestamp":1684845092000},"page":"345-349","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":41,"title":["Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond"],"prefix":"10.1177","volume":"34","author":[{"given":"Dongsheng","family":"Duan","sequence":"first","affiliation":[{"name":"Department of Molecular Microbiology and Immunology, University of Missouri, Columbia, Missouri, USA."},{"name":"Department of Neurology, School of Medicine, University of Missouri, Columbia, Missouri, USA."},{"name":"Department of Biomedical Sciences, College of Veterinary Medicine, University of Missouri, Columbia, Missouri, USA."},{"name":"Department of Chemical and Biomedical Engineering, College of Engineering, University of Missouri, Columbia, Missouri, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2023,5,17]]},"reference":[{"key":"e_1_3_3_2_2","doi-asserted-by":"publisher","DOI":"10.1017\/S0317167100004315"},{"key":"e_1_3_3_3_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-005-0874-0"},{"key":"e_1_3_3_4_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-021-00248-3"},{"key":"e_1_3_3_5_2","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(87)90504-6"},{"key":"e_1_3_3_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(87)90579-4"},{"key":"e_1_3_3_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/0888-7543(88)90113-9"},{"issue":"1","key":"e_1_3_3_8_2","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 AH","year":"1991","unstructured":"Beggs AH, , Hoffman EP, , Snyder JR, et al. Exploring the molecular basis for variability among patients with Becker muscular dystrophy: Dystrophin gene and protein studies. Am J Hum Genet, 1991; 49(1):54\u201367.","journal-title":"Am J Hum Genet"},{"key":"e_1_3_3_9_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199509283331303"},{"issue":"1","key":"e_1_3_3_10_2","first-page":"86","article-title":"Myodys, a full-length dystrophin plasmid vector for Duchenne and Becker muscular dystrophy gene therapy","volume":"10","author":"Duan D","year":"2008","unstructured":"Duan D. Myodys, a full-length dystrophin plasmid vector for Duchenne and Becker muscular dystrophy gene therapy. Curr Opin Mol Ther, 2008; 10(1):86\u201394.","journal-title":"Curr Opin Mol Ther"},{"key":"e_1_3_3_11_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2023.026"},{"key":"e_1_3_3_12_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2022.190"},{"key":"e_1_3_3_13_2","unstructured":"Dreghici RD Gonzale P Brown K et al. 3-Year Outcomes Update in the IGNITE DMD Phase 1\/2 Study of SGT-001 Microdystrophin Gene Therapy. 2023 MDA Clinical & Scientific Conference 2023 March 19\u201322 2023 Dallas TX. 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