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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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Podrobná bibliografie
Hlavní autoři: Orwig, Susan. D., Tan, Yun Lei, Grimster, Neil P., Yu, Zhanqian, Powers, Evan, Kelly, Jeffery W., Lieberman, Raquel L.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2011
Témata:
On-line přístup: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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