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Gene therapy has been proven to ameliorate dystrophic pathology. To investigate therapeutic benefits from long\u2010term effect of human mini\u2010dystrophin and functional outcomes, transgenic\n                    <jats:italic>mdx<\/jats:italic>\n                    mice (Tg\u2010\n                    <jats:italic>mdx<\/jats:italic>\n                    ) containing a single copy of human mini\u2010\n                    <jats:italic>dystrophin<\/jats:italic>\n                    (\u2206hDys3849) gene, five rods (Rods1\u20102, Rods22\u201024), and two hinges (H1 and H4) driven by a truncated creatine\u2010kinase promoter (dMCK) in a recombinant adeno\u2010associated viral vector (rAAV) backbone, were generated and used to determine gene expression and improvement of muscle function. Human mini\u2010\n                    <jats:italic>dystrophin<\/jats:italic>\n                    gene expression was found in a majority of the skeletal muscles, but no expression in cardiac muscle. Dystrophin\u2010associated glycoproteins (DAGs) such as sarcoglycans and nNOS were restored at the sarcolemma and coincided with human mini\u2010\n                    <jats:italic>dystrophin<\/jats:italic>\n                    gene expression at the ages of 6, 10, and 20 months; Morphology of dystrophic muscle expressing the human mini\u2010\n                    <jats:italic>dystrophin<\/jats:italic>\n                    gene was improved and central nuclei were reduced. Myofiber membrane integrity was improved by Evans blue dye test. Improvement in treadmill running and grip force was observed in transgenic mice at 6 months. Tetanic force and specific force of tibialis anterior (TA) muscle were significantly increased at the ages of 6, 10, and 20 months. Pseudohypertrophy was not found in TA muscle at 10 and 20 months when compared with wild\u2010type C57 (WT) group. This study demonstrated that the long\u2010term effects of human mini\u2010dystrophin effectively ameliorated pathology and improved the functions of the dystrophic muscles in the transgenic DMD mouse model.\n                  <\/jats:p>","DOI":"10.1096\/fj.202100057rr","type":"journal-article","created":{"date-parts":[[2021,5,13]],"date-time":"2021-05-13T03:46:41Z","timestamp":1620877601000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Long\u2010term effect of human mini\u2010dystrophin in transgenic\n                    <i>mdx<\/i>\n                    mice improves muscle physiological function"],"prefix":"10.1096","volume":"35","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3320-9359","authenticated-orcid":false,"given":"Xiangyu","family":"Chu","sequence":"first","affiliation":[{"name":"Department of Orthopaedic Surgery University of Pittsburgh Pittsburgh PA USA"},{"name":"Department of Orthopedics, Tongji Hospital Huazhong University of Science and Technology Wuhan China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Juan","family":"Li","sequence":"additional","affiliation":[{"name":"Division of Molecular Pharmaceutics, School of Pharmacy University of North Carolina Chapel Hill NC USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chunping","family":"Qiao","sequence":"additional","affiliation":[{"name":"Division of Molecular Pharmaceutics, School of Pharmacy University of North Carolina Chapel Hill NC USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jing","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Orthopaedic Surgery University of Pittsburgh Pittsburgh PA USA"},{"name":"Department of Neurology, Tongji Hospital Huazhong University of Science and Technology Wuhan 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