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

AuthorsAlfonso Palacin, Pilar; Andreu, Vanesa; Pino-Ángeles, Almudena; Moya García, Aurelio; García-Moreno, M. Isabel; Rodríguez, Rey, José C.; Sánchez-Jiménez, Francisca; Pocovi Mieras, Miguel; García Fernández, José Manuel ; Giraldo, Pilar
Molecular modeling
Pharmacological chaperones
Gaucher disease
Issue Date2013
PublisherJohn Wiley & Sons
CitationChemistry: a European Journal 14: 943- 949 (2013)
AbstractNew 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.
Identifiersdoi: 10.1002/cbic.201200708
issn: 1439-4227
Appears in Collections:(IIQ) Artículos
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