Dose-Dependent Effects of FKRP Gene-Replacement Therapy on Functional Rescue and Longevity in Dystrophic Mice

Muscular dystrophy-dystroglycanopathies (MDDGs) resulting from fukutin-related protein (FKRP) gene mutations are rare disorders that result in a wide spectrum of clinical severity based on the age of onset, the degree of myogenic atrophy, and/or neurologic involvement. There is no cure for any of th...

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Detalles Bibliográficos
Autores principales: Vannoy, Charles Harvey, Leroy, Victoria, Lu, Qi Long
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6222079/
https://www.ncbi.nlm.nih.gov/pubmed/30417025
http://dx.doi.org/10.1016/j.omtm.2018.10.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6222079/
https://www.ncbi.nlm.nih.gov/pubmed/30417025
http://dx.doi.org/10.1016/j.omtm.2018.10.004

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