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
Autores principales: Guha, Tuhin K., Pichavant, Christophe, Calos, Michele P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
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

Internet

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

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