MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) cardiomyopathy patients currently have no therapeutic options. We evaluated catheter-based transendocardial delivery of a recombinant adeno-associated virus (rAAV) expressing a small nuclear U7 RNA (U7smOPT) complementary to specific cis-acting splicing signals. Eli...

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Main Authors: Barbash, Israel M., Cecchini, Sylvain, Faranesh, Anthony Z., Virag, Tamas, Li, Lina, Yang, Yu, Hoyt, Robert F., Kornegay, Joe N, Bogan, Janet R., Garcia, Luis, Lederman, Robert J., Kotin, Robert M.
Formato: Artigo
Idioma:Inglês
Publicado: 2012
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3424392/
https://ncbi.nlm.nih.gov/pubmed/22551778
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2012.38
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