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ISPD Overexpression Enhances Ribitol-Induced Glycosylation of α-Dystroglycan in Dystrophic FKRP Mutant Mice

Dystroglycanopathy, a subgroup of muscular dystrophies, is characterized by hypoglycosylation of α-dystroglycan (α-DG), which reduces its laminin-binding activity to extracellular matrix proteins, causing progressive loss of muscle integrity and function. Mutations in the fukutin-related protein (FK...

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Dettagli Bibliografici
Pubblicato in:Mol Ther Methods Clin Dev
Autori principali: Cataldi, Marcela P., Blaeser, Anthony, Lu, Peijuan, Leroy, Victoria, Lu, Qi Long
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6970132/
https://ncbi.nlm.nih.gov/pubmed/31988979
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.12.005
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