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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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Détails bibliographiques
Publié dans:Mol Ther Methods Clin Dev
Auteurs principaux: Guha, Tuhin K., Pichavant, Christophe, Calos, Michele P.
Format: Artigo
Langue:Inglês
Publié: American Society of Gene & Cell Therapy 2019
Sujets:
Accès en ligne: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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