Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model

Micro-dystrophin gene replacement therapies for Duchenne muscular dystrophy (DMD) are currently in clinical trials, but have not been thoroughly investigated for their efficacy on cardiomyopathy progression to heart failure. We previously validated Fiona/dystrophin-utrophin-deficient (dko) mice as a...

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Detalles Bibliográficos
Autores principales: Piepho, Arden B., Lowe, Jeovanna, Cumby, Laurel R., Dorn, Lisa E., Lake, Dana M., Rastogi, Neha, Gertzen, Megan D., Sturgill, Sarah L., Odom, Guy L., Ziolo, Mark T., Accornero, Federica, Chamberlain, Jeffrey S., Rafael-Fortney, Jill A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9981810/
https://www.ncbi.nlm.nih.gov/pubmed/36874243
http://dx.doi.org/10.1016/j.omtm.2023.02.001

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