Bicyclic Derivatives of L‐Idonojirimycin as Pharmacological Chaperones for Neuronopathic Forms of Gaucher Disease. Issue 8 (18th April 2013)

• Record Type: Journal Article
• 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
• Is Part Of: Chembiochem. Volume 14:Issue 8(2013)
• Page Start: 943
• Page End: 949
• Publication Date: 2013-04-18
• Subjects: Biochemistry -- Periodicals; Molecular biology -- Periodicals; Pharmaceutical chemistry -- Periodicals; 572
• DOI: 10.1002/cbic.201200708
• Languages: English
• ISSNs: 1439-4227

Abstract

<abstract abstract-type="graphical" xml:lang="en"> <title> <x xml:space="preserve">Abstract</x> </title> <p> <bold>New human β‐glucocerebrosidase (GCase) ligands</bold> with rigid 1, 6‐anhydro‐β‐<sc>L</sc>‐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.<boxed-text content-type="graphic" position="anchor" orientation="portrait"><graphic position="anchor" mimetype="image" xlink:href="ark:/27927/pgg20p7463s" orientation="portrait" xlink:type="simple" xmlns:xlink="http://www.w3.org/1999/xlink" /></boxed-text></p> </abstract>