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Plasmid-Mediated Gene Therapy in Mouse Models of Limb Girdle Muscular Dystrophy

We delivered plasmid DNA encoding therapeutic genes to the muscles of mouse models of limb girdle muscular dystrophy (LGMD) 2A, 2B, and 2D, deficient in calpain3, dysferlin, and alpha-sarcoglycan, respectively. We also delivered the human follistatin gene, which has the potential to increase therape...

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Detalles Bibliográficos
Publicado en:Mol Ther Methods Clin Dev
Main Authors: Guha, Tuhin K., Pichavant, Christophe, Calos, Michele P.
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Gene & Cell Therapy 2019
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6923511/
https://ncbi.nlm.nih.gov/pubmed/31890729
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.10.002
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