Long-Term Systemic Myostatin Inhibition via Liver-Targeted Gene Transfer in Golden Retriever Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a lethal, X-linked recessive disease affecting 1 in 3,500 newborn boys for which there is no effective treatment or cure. One novel strategy that has therapeutic potential for DMD is inhibition of myostatin, a negative regulator of skeletal muscle mass that may a...

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Main Authors: Bish, Lawrence T., Sleeper, Meg M., Forbes, Sean C., Morine, Kevin J., Reynolds, Caryn, Singletary, Gretchen E., Trafny, Dennis, Pham, Jennifer, Bogan, Janet, Kornegay, Joe N., Vandenborne, Krista, Walter, Glenn A., Sweeney, H. Lee
Formato: Artigo
Idioma:Inglês
Publicado: Mary Ann Liebert, Inc. 2011
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Research Articles
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3237695/
https://ncbi.nlm.nih.gov/pubmed/21787232
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.102
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