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Azasugar inhibitors as pharmacological chaperones for Krabbe disease

Krabbe disease is a devastating neurodegenerative disorder characterized by rapid demyelination of nerve fibers. This disease is caused by defects in the lysosomal enzyme β-galactocerebrosidase (GALC), which hydrolyzes the terminal galactose from glycosphingolipids. These lipids are essential compon...

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Detalles Bibliográficos
Autores principales:	Hill, Chris H., Viuff, Agnete H., Spratley, Samantha J., Salamone, Stéphane, Christensen, Stig H., Read, Randy J., Moriarty, Nigel W., Jensen, Henrik H., Deane, Janet E.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Royal Society of Chemistry 2015
Materias:	Chemistry
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4445328/ https://www.ncbi.nlm.nih.gov/pubmed/26029356 http://dx.doi.org/10.1039/c5sc00754b

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