Abstract
New human β-glucocerebrosidase (GCase) ligands with rigid 1,6-anhydro-β-L-idonojirimycin cores have been designed with the aid of molecular modeling. Efficient pharmacological chaperones for the L444P (trafficking-incompetent) mutant GCase enzyme associated with type 2 and 3 Gaucher disease (GD) were identified.
Copyright © 2013 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim.
Publication types
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Research Support, Non-U.S. Gov't
MeSH terms
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Animals
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COS Cells
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Cell Line
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Chlorocebus aethiops
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Gaucher Disease / enzymology*
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Gaucher Disease / genetics
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Glucosylceramidase / genetics
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Glucosylceramidase / metabolism*
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Humans
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Imino Pyranoses / chemistry*
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Imino Pyranoses / pharmacology*
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Ligands
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Molecular Docking Simulation
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Mutation
Substances
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Imino Pyranoses
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Ligands
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Glucosylceramidase