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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...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2019
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6923511/ https://www.ncbi.nlm.nih.gov/pubmed/31890729 http://dx.doi.org/10.1016/j.omtm.2019.10.002 |