{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,26]],"date-time":"2026-04-26T07:25:01Z","timestamp":1777188301959,"version":"3.51.4"},"reference-count":47,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,1,6]],"date-time":"2024-01-06T00:00:00Z","timestamp":1704499200000},"content-version":"vor","delay-in-days":5,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81602699"],"award-info":[{"award-number":["81602699"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["MedComm"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Duchenne muscular dystrophy (DMD) is an incurable X\u2010linked recessive genetic disease caused by mutations in the dystrophin gene. Many researchers aim to restore truncated dystrophin via viral vectors. However, the low packaging capacity and immunogenicity of vectors have hampered their clinical application. Herein, we constructed four lentiviral vectors with truncated and sequence\u2010optimized dystrophin genes driven by muscle\u2010specific promoters. The four lentiviral vectors stably expressed mini\u2010dystrophin in C2C12 muscle cells in vitro. To estimate the treatment effect in vivo, we transferred the lentiviral vectors into neonatal C57BL\/10ScSn\u2010<jats:italic>Dmd<jats:sup>mdx<\/jats:sup><\/jats:italic> mice through local injection. The levels of modified dystrophin expression increased, and their distribution was also restored in treated mice. At the same time, they exhibited the restoration of pull force and a decrease in the number of mononuclear cells. The remissions lasted 3\u20136 months in vivo. Moreover, no integration sites of vectors were distributed into the oncogenes. In summary, this study preliminarily demonstrated the feasibility and safety of lentiviral vectors with mini\u2010dystrophin for DMD gene therapy and provided a new strategy to restore truncated dystrophin.<\/jats:p>","DOI":"10.1002\/mco2.423","type":"journal-article","created":{"date-parts":[[2024,1,6]],"date-time":"2024-01-06T13:15:25Z","timestamp":1704546925000},"source":"Crossref","is-referenced-by-count":5,"title":["Duchenne muscular dystrophy treatment with lentiviral vector containing mini\u2010dystrophin gene in vivo"],"prefix":"10.1002","volume":"5","author":[{"given":"Xiaoyu","family":"Wang","sequence":"first","affiliation":[{"name":"Department of Pharmacy Cancer Center and State Key Laboratory of Biotherapy West China Hospital Sichuan University  Chengdu Sichuan China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yanghui","family":"Zhu","sequence":"additional","affiliation":[{"name":"Department of Pharmacy Cancer Center and State Key Laboratory of 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