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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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Vannoy, Charles Harvey, Xiao, Will, Lu, Peijuan, Xiao, Xiao, Lu, Qi Long
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2017
Fag:
Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5415313/
https://ncbi.nlm.nih.gov/pubmed/28480302
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2017.02.002
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