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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...
Autores principales: | , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2022
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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 |