Efficacy of Gene Therapy Is Dependent on Disease Progression in Dystrophic Mice with Mutations in the FKRP Gene

Loss-of-function mutations in the Fukutin-related protein (FKRP) gene cause limb-girdle muscular dystrophy type 2I (LGMD2I) and other forms of congenital muscular dystrophy-dystroglycanopathy that are associated with glycosylation defects in the α-dystroglycan (α-DG) protein. Systemic administration...

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Detalles Bibliográficos
Autores principales: Vannoy, Charles Harvey, Xiao, Will, Lu, Peijuan, Xiao, Xiao, Lu, Qi Long
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5415313/
https://www.ncbi.nlm.nih.gov/pubmed/28480302
http://dx.doi.org/10.1016/j.omtm.2017.02.002

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