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Because several cassettes are being tested in clinical trials, this study compared the efficacies of four shortened dystrophin-promoter combinations with implications for outcomes in clinical trials: MHCK7 or MCK promoter with a shortened dystrophin transgene containing the N-terminus and spectrin repeats R1, R2, R3 and R24 (rAAVrh74.MHCK7.micro-dystrophin and rAAVrh74.MCK.micro-dystrophin, respectively); shortened dystrophin construct containing the neuronal nitric oxide (nNOS) binding site (rAAVrh74.MHCK7.DV.mini-dystrophin); and shortened dystrophin containing the C-terminus (rAAVrh74.MHCK7.micro-dystrophin.Cterm). Functional and histological benefit were examined at 4\u2005weeks following intramuscular delivery in mdx mice. rAAVrh74.MHCK7.micro-dystrophin provided the most robust transgene expression and significantly increased specific force output in the tibialis anterior muscle. Muscle environment was normalized (i.e. reductions in central nucleation), indicating functional and histological advantages of rAAVrh74.MHCK7.micro-dystrophin. Thus, promoter choice and transgene design are critical for optimal dystrophin expression\/distribution for maximal functional improvement.<\/jats:p>","DOI":"10.1242\/bio.059797","type":"journal-article","created":{"date-parts":[[2023,9,6]],"date-time":"2023-09-06T07:15:01Z","timestamp":1693984501000},"source":"Crossref","is-referenced-by-count":4,"title":["Expression and function of four AAV-based constructs for dystrophin restoration in the <i>mdx<\/i> mouse model of Duchenne muscular dystrophy"],"prefix":"10.1242","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5564-8993","authenticated-orcid":false,"given":"Rachael A.","family":"Potter","sequence":"first","affiliation":[{"name":"Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital 1 , Columbus, OH 43205 , USA"},{"name":"Sarepta Therapeutics, Inc. 2 , Cambridge, MA 02142 , USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Danielle A.","family":"Griffin","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital 1 , Columbus, OH 43205 , USA"},{"name":"Sarepta Therapeutics, Inc. 2 , Cambridge, MA 02142 , USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kristin N.","family":"Heller","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital 1 , Columbus, OH 43205 , USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jerry R.","family":"Mendell","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital 1 , Columbus, OH 43205 , USA"},{"name":"The Ohio State University 3 Department of Pediatrics and Neurology , , Columbus, OH 43210 , USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Louise R.","family":"Rodino-Klapac","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital 1 , Columbus, OH 43205 , USA"},{"name":"Sarepta Therapeutics, Inc. 2 , Cambridge, MA 02142 , USA"},{"name":"The Ohio State University 3 Department of Pediatrics and Neurology , , Columbus, OH 43210 , USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"237","published-online":{"date-parts":[[2023,9,18]]},"reference":[{"key":"2023091909353711800_BIO059797C1","doi-asserted-by":"publisher","first-page":"e15858","DOI":"10.1097\/MD.0000000000015858","article-title":"Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy","volume":"98","author":"Alfano","year":"2019","journal-title":"Medicine"},{"key":"2023091909353711800_BIO059797C2","doi-asserted-by":"publisher","first-page":"e1000958","DOI":"10.1371\/journal.pgen.1000958","article-title":"The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins","volume":"6","author":"Banks","year":"2010","journal-title":"PLoS Genet."},{"key":"2023091909353711800_BIO059797C3","doi-asserted-by":"publisher","first-page":"445","DOI":"10.1016\/S1474-4422(18)30026-7","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan","volume":"17","author":"Birnkrant","year":"2018","journal-title":"Lancet Neurol."},{"key":"2023091909353711800_BIO059797C4","doi-asserted-by":"publisher","first-page":"91","DOI":"10.1002\/mus.880060204","article-title":"Clinical investigation in Duchenne dystrophy: 2. 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