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Metabolomics Analysis of Skeletal Muscles from FKRP-Deficient Mice Indicates Improvement After Gene Replacement Therapy

Muscular dystrophy-dystroglycanopathies comprise a heterogeneous and complex group of disorders caused by loss-of-function mutations in a multitude of genes that disrupt the glycobiology of α-dystroglycan, thereby affecting its ability to function as a receptor for extracellular matrix proteins. Of...

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Detalles Bibliográficos
Autores principales:	Vannoy, Charles Harvey, Leroy, Victoria, Broniowska, Katarzyna, Lu, Qi Long
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6624266/ https://www.ncbi.nlm.nih.gov/pubmed/31296900 http://dx.doi.org/10.1038/s41598-019-46431-1

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