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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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| Publicado no: | Mol Ther Methods Clin Dev |
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| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Gene & Cell Therapy
2019
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| Assuntos: | |
| Acesso em linha: | 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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