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. 2013 May 27;14(8):943-9.
doi: 10.1002/cbic.201200708. Epub 2013 Apr 18.

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

Pilar Alfonso  1 , Vanesa Andreu, Almudena Pino-Angeles, Aurelio A Moya-García, M Isabel García-Moreno, José C Rodríguez-Rey, Francisca Sánchez-Jiménez, Miguel Pocoví, Carmen Ortiz Mellet, Jose M García Fernández, Pilar Giraldo
Affiliations

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

Pilar Alfonso et al. Chembiochem. .

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.

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