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Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model.
Piepho AB, Lowe J, Cumby LR, Dorn LE, Lake DM, Rastogi N, Gertzen MD, Sturgill SL, Odom GL, Ziolo MT, Accornero F, Chamberlain JS, Rafael-Fortney JA. Piepho AB, et al. Among authors: cumby lr. Mol Ther Methods Clin Dev. 2023 Feb 9;28:344-354. doi: 10.1016/j.omtm.2023.02.001. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2023. PMID: 36874243 Free PMC article.