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Lipid-mimicking phosphorus-based glycosidase inactivators as pharmacological chaperones for the treatment of Gaucher's disease

Gaucher's disease, the most prevalent lysosomal storage disorder, is caused by missense mutation of the GBA gene, ultimately resulting in deficient GCase activity, hence the excessive build-up of cellular glucosylceramide. Among different therapeutic strategies, pharmacological chaperoning of m...

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Detalles Bibliográficos
Autores principales:	Scherer, Manuel, Santana, Andrés G., Robinson, Kyle, Zhou, Steven, Overkleeft, Hermen S., Clarke, Lorne, Withers, Stephen G.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	The Royal Society of Chemistry 2021
Materias:	Chemistry
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8549773/ https://www.ncbi.nlm.nih.gov/pubmed/34760177 http://dx.doi.org/10.1039/d1sc03831a

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