Literature DB >> 23606264

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

Pilar Alfonso1, 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.   

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.
Copyright © 2013 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim.

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Year:  2013        PMID： 23606264     DOI： 10.1002/cbic.201200708

Source DB:  PubMed          Journal:  Chembiochem        ISSN： 1439-4227            Impact factor:   3.164


  5 in total

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Journal:  PLoS One       Date:  2013-10-04       Impact factor: 3.240

2.  Pharmacological Chaperones and Coenzyme Q10 Treatment Improves Mutant β-Glucocerebrosidase Activity and Mitochondrial Function in Neuronopathic Forms of Gaucher Disease.

Authors:  Mario de la Mata; David Cotán; Manuel Oropesa-Ávila; Juan Garrido-Maraver; Mario D Cordero; Marina Villanueva Paz; Ana Delgado Pavón; Elizabet Alcocer-Gómez; Isabel de Lavera; Patricia Ybot-González; Ana Paula Zaderenko; Carmen Ortiz Mellet; José M García Fernández; José A Sánchez-Alcázar
Journal:  Sci Rep       Date:  2015-06-05       Impact factor: 4.379

3.  Probing the Inhibitor versus Chaperone Properties of sp²-Iminosugars towards Human β-Glucocerebrosidase: A Picomolar Chaperone for Gaucher Disease.

Authors:  Teresa Mena-Barragán; M Isabel García-Moreno; Alen Sevšek; Tetsuya Okazaki; Eiji Nanba; Katsumi Higaki; Nathaniel I Martin; Roland J Pieters; José M García Fernández; Carmen Ortiz Mellet
Journal:  Molecules       Date:  2018-04-17       Impact factor: 4.411

4.  Thiol-ene "Click" Synthesis and Pharmacological Evaluation of C-Glycoside sp2-Iminosugar Glycolipids.

Authors:  Elena M Sánchez-Fernández; M Isabel García-Moreno; Raquel García-Hernández; José M Padrón; José M García Fernández; Francisco Gamarro; Carmen Ortiz Mellet
Journal:  Molecules       Date:  2019-08-08       Impact factor: 4.411

5.  Mechanistic Insights into the Chaperoning of Human Lysosomal-Galactosidase Activity: Highly Functionalized Aminocyclopentanes and C-5a-Substituted Derivatives of 4-epi-Isofagomine.

Authors:  Patrick Weber; Martin Thonhofer; Summer Averill; Gideon J Davies; Andres Gonzalez Santana; Philipp Müller; Seyed A Nasseri; Wendy A Offen; Bettina M Pabst; Eduard Paschke; Michael Schalli; Ana Torvisco; Marion Tschernutter; Christina Tysoe; Werner Windischhofer; Stephen G Withers; Andreas Wolfsgruber; Tanja M Wrodnigg; Arnold E Stütz
Journal:  Molecules       Date:  2020-09-03       Impact factor: 4.411
  5 in total

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