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

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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Bibliographic Details
Main Authors: Charles Harvey Vannoy, Will Xiao, Peijuan Lu, Xiao Xiao, Qi Long Lu
Format: Article
Language:English
Published: Elsevier 2017-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
adeno-associated virus
dystroglycanopathy
fukutin-related protein
gene therapy
muscular dystrophy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050117300426

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http://www.sciencedirect.com/science/article/pii/S2329050117300426

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