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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: 2012
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3424392/
https://www.ncbi.nlm.nih.gov/pubmed/22551778
http://dx.doi.org/10.1038/gt.2012.38

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3424392/
https://www.ncbi.nlm.nih.gov/pubmed/22551778
http://dx.doi.org/10.1038/gt.2012.38

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