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CDP-ribitol prodrug treatment ameliorates ISPD-deficient muscular dystrophy mouse model

Ribitol-phosphate modification is crucial for the functional maturation of α-dystroglycan. Its dysfunction is associated with muscular dystrophy, cardiomyopathy, and central nervous system abnormalities; however, no effective treatments are currently available for diseases caused by ribitol-phosphat...

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Detalles Bibliográficos
Autores principales: Tokuoka, Hideki, Imae, Rieko, Nakashima, Hitomi, Manya, Hiroshi, Masuda, Chiaki, Hoshino, Shunsuke, Kobayashi, Kazuhiro, Lefeber, Dirk J., Matsumoto, Riki, Okada, Takashi, Endo, Tamao, Kanagawa, Motoi, Toda, Tatsushi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9010444/
https://www.ncbi.nlm.nih.gov/pubmed/35422047
http://dx.doi.org/10.1038/s41467-022-29473-4