Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model

Gene replacement for Duchenne muscular dystrophy (DMD) with micro-dystrophins has entered clinical trials, but efficacy in preventing heart failure is unknown. Although most patients with DMD die from heart failure, cardiomyopathy is undetectable until the teens, so efficacy from trials in young boy...

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Detalles Bibliográficos
Autores principales: Howard, Zachary M., Dorn, Lisa E., Lowe, Jeovanna, Gertzen, Megan D., Ciccone, Pierce, Rastogi, Neha, Odom, Guy L., Accornero, Federica, Chamberlain, Jeffrey S., Rafael-Fortney, Jill A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2021
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8119181/
https://www.ncbi.nlm.nih.gov/pubmed/33651713
http://dx.doi.org/10.1172/jci.insight.146511

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