Bicyclic Derivatives of L-Idonojirimycin as Pharmacological Chaperones for Neuronopathic Forms of Gaucher Disease

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) wer...

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Published in:Chembiochem : a European journal of chemical biology 2013-05, Vol.14 (8), p.943-949
Main Authors: Alfonso, Pilar, Andreu, Vanesa, Pino-Angeles, Almudena, Moya-García, Aurelio A., García-Moreno, M. Isabel, Rodríguez-Rey, José C., Sánchez-Jiménez, Francisca, Pocoví, Miguel, Ortiz Mellet, Carmen, García Fernández, Jose M., Giraldo, Pilar
Format: Article
Language:English
Subjects:
Animals
carbohydrates
Cell Line
Cercopithecus aethiops
COS Cells
Gaucher disease
Gaucher Disease - enzymology
Gaucher Disease - genetics
Glucosylceramidase - genetics
Glucosylceramidase - metabolism
Humans
Imino Pyranoses - chemistry
Imino Pyranoses - pharmacology
L-idonojirimycin
Ligands
Molecular Docking Simulation
molecular modeling
Mutation
pharmacological chaperones
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Summary: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.
ISSN:1439-4227
1439-7633
DOI:10.1002/cbic.201200708