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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Bibliografske podrobnosti
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
Format: Artigo
Jezik:Inglês
Izdano: Mary Ann Liebert, Inc. 2011
Teme:
Research Articles
Online dostop: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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