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To evaluate the clinical translatability of an adeno-associated virus serotype 9 vector (AAV9)\u2013microdystrophin (\u03bcDys5) construct, we performed a blinded, placebo-controlled study in which 12 GRMD dogs were divided among four dose groups [control, 1 \u00d7 10<jats:sup>13<\/jats:sup>vector genomes per kilogram (vg\/kg), 1 \u00d7 10<jats:sup>14<\/jats:sup>vg\/kg, and 2 \u00d7 10<jats:sup>14<\/jats:sup>vg\/kg;<jats:italic>n<\/jats:italic>= 3 each], treated intravenously at 3 months of age with a canine codon-optimized microdystrophin construct, rAAV9-CK8e-c-\u03bcDys5, and followed for 90 days after dosing. All dogs received prednisone (1 milligram\/kilogram) for a total of 5 weeks from day \u22127 through day 28. We observed dose-dependent increases in tissue vector genome copy numbers; \u03bcDys5 protein in multiple appendicular muscles, the diaphragm, and heart; limb and respiratory muscle functional improvement; and reduction of histopathologic lesions. As expected, given that a truncated dystrophin protein was generated, phenotypic test results and histopathologic lesions did not fully normalize. All administrations were well tolerated, and adverse events were not seen. These data suggest that systemically administered AAV-microdystrophin may be dosed safely and could provide therapeutic benefit for patients with DMD.<\/jats:p>","DOI":"10.1126\/scitranslmed.abo1815","type":"journal-article","created":{"date-parts":[[2023,1,4]],"date-time":"2023-01-04T18:58:14Z","timestamp":1672858694000},"update-policy":"https:\/\/doi.org\/10.34133\/aaas_crossmark","source":"Crossref","is-referenced-by-count":66,"title":["Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy"],"prefix":"10.1126","volume":"15","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8088-8104","authenticated-orcid":true,"given":"Sharla M.","family":"Birch","sequence":"first","affiliation":[{"name":"Texas A&amp;M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, 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N.\u00a0Kornegay, Mutation segregation and rapid carrier detection of X-linked muscular dystrophy in dogs. Am.\u00a0J.\u00a0Vet. Res. 57, 650\u2013654 (1996).","journal-title":"Am.\u00a0J.\u00a0Vet. Res."},{"key":"e_1_3_3_74_2","doi-asserted-by":"publisher","DOI":"10.1093\/gerona\/52A.3.B171"},{"key":"e_1_3_3_75_2","doi-asserted-by":"publisher","DOI":"10.1097\/00002060-199509001-00003"},{"key":"e_1_3_3_76_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2010.73"},{"key":"e_1_3_3_77_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2010.107"},{"key":"e_1_3_3_78_2","first-page":"51586","article-title":"Tissue triage and freezing for models of skeletal muscle disease","author":"Meng H.","year":"2014","unstructured":"H.\u00a0Meng, P. M.\u00a0Janssen, R. W.\u00a0Grange, L.\u00a0Yang, A. H.\u00a0Beggs, L. C.\u00a0Swanson, S. A.\u00a0Cossette, A.\u00a0Frase, M. K.\u00a0Childers, H.\u00a0Granzier, E.\u00a0Gussoni, M. W.\u00a0Lawlor, Tissue triage and freezing for models of skeletal muscle disease. J. Vis. Exp. , 51586 (2014).","journal-title":"J. Vis. 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