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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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| Publié dans: | Mol Ther Methods Clin Dev |
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| Auteurs principaux: | , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
American Society of Gene & Cell Therapy
2019
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| 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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