Long-term Restoration of Cardiac Dystrophin Expression in Golden Retriever Muscular Dystrophy Following rAAV6-mediated Exon Skipping

Although restoration of dystrophin expression via exon skipping in both cardiac and skeletal muscle has been successfully demonstrated in the mdx mouse, restoration of cardiac dystrophin expression in large animal models of Duchenne muscular dystrophy (DMD) has proven to be a challenge. In large ani...

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Detalhes bibliográficos
Main Authors: Bish, Lawrence T, Sleeper, Meg M, Forbes, Sean C, Wang, Bingjing, Reynolds, Caryn, Singletary, Gretchen E, Trafny, Dennis, Morine, Kevin J, Sanmiguel, Julio, Cecchini, Sylvain, Virag, Tamas, Vulin, Adeline, Beley, Cyriaque, Bogan, Janet, Wilson, James M, Vandenborne, Krista, Kornegay, Joe N, Walter, Glenn A, Kotin, Robert M, Garcia, Luis, Sweeney, H Lee
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2012
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3293605/
https://ncbi.nlm.nih.gov/pubmed/22146342
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.264
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