Adeno-associated Virus 9 Mediated FKRP Gene Therapy Restores Functional Glycosylation of α-dystroglycan and Improves Muscle Functions

Mutations in the FKRP gene are associated with a wide range of muscular dystrophies from mild limb-girdle muscular dystrophy (LGMD) 2I to severe Walker–Warburg syndrome and muscle-eye-brain disease. The characteristic biochemical feature of these diseases is the hypoglycosylation of α-dystroglycan (...

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Autores principales: Xu, Lei, Lu, Pei Juan, Wang, Chi-Hsien, Keramaris, Elizabeth, Qiao, Chunping, Xiao, Bin, Blake, Derek J, Xiao, Xiao, Lu, Qi Long
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2013
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3808132/
https://ncbi.nlm.nih.gov/pubmed/23817215
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.156
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