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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Dettagli Bibliografici
Pubblicato in:Mol Ther Methods Clin Dev
Autori principali: Vannoy, Charles Harvey, Xiao, Will, Lu, Peijuan, Xiao, Xiao, Lu, Qi Long
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2017
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Original Article
Accesso online: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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