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Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model.
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.
Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model.
Howard ZM, Dorn LE, Lowe J, Gertzen MD, Ciccone P, Rastogi N, Odom GL, Accornero F, Chamberlain JS, Rafael-Fortney JA.
Howard ZM, et al. Among authors: gertzen md.
JCI Insight. 2021 Apr 8;6(7):e146511. doi: 10.1172/jci.insight.146511.
JCI Insight. 2021.
PMID: 33651713
Free PMC article.
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