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Bicyclic derivatives of L-idonojirimycin as pharmacological chaperones for neuronopathic forms of Gaucher disease.

Alfonso, P; Andreu, V; Pino-Angeles, A; Moya-García, AA; García-Moreno, MI; Rodríguez-Rey, JC; Sánchez-Jiménez, F; ... Giraldo, P; + view all (2013) Bicyclic derivatives of L-idonojirimycin as pharmacological chaperones for neuronopathic forms of Gaucher disease. Chembiochem , 14 (8) pp. 943-949. 10.1002/cbic.201200708.

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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.

Type: Article
Title: Bicyclic derivatives of L-idonojirimycin as pharmacological chaperones for neuronopathic forms of Gaucher disease.
Location: Germany
DOI: 10.1002/cbic.201200708
Keywords: Animals, COS Cells, Cell Line, Cercopithecus aethiops, Gaucher Disease, Glucosylceramidase, Humans, Imino Pyranoses, Ligands, Molecular Docking Simulation, Mutation
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > Div of Biosciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > Div of Biosciences > Structural and Molecular Biology
URI: http://discovery.ucl.ac.uk/id/eprint/1432055
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