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Binding of 3,4,5,6-tetrahydroxyazepanes to the acid-β-glucosidase active site: implications for pharmacological chaperone design for Gaucher disease
Pharmacological chaperoning is a therapeutic strategy being developed to restore cellular folding and trafficking defects associated with Gaucher disease, a lysosomal storage disorder caused by point mutations in the gene encoding for acid- β-glucosidase (GCase). In this approach, small molecules bi...
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| Main Authors: | , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2011
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3235649/ https://ncbi.nlm.nih.gov/pubmed/22047104 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/bi201619z |
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