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Elsevier, Molecular Genetics and Metabolism, 2(111), p. S18-S19, 2014

DOI: 10.1016/j.ymgme.2013.12.022

Wiley, ChemBioChem, 8(14), p. 943-949, 2013

DOI: 10.1002/cbic.201200708

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

Journal article published in 2013 by Pilar Alfonso, Vanesa Andreu, Almudena Pino-Angeles, Aurelio A. Moya-García, María Isabel García-Moreno ORCID, José C. Rodríguez-Rey, Francisca Sánchez-Jiménez, Miguel Pocoví, Carmen Ortiz Mellet, Carmen Ortiz Mellet, Jose Manuel García Fernández ORCID, Pilar Giraldo
This paper is available in a repository.
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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.