Identification of an Allosteric Binding Site on Human Lysosomal Alpha-Galactosidase Opens the Way to New Pharmacological Chaperones for Fabry Disease

Personalized therapies are required for Fabry disease due to its large phenotypic spectrum and numerous different genotypes. In principle, missense mutations that do not affect the active site could be rescued with pharmacological chaperones. At present pharmacological chaperones for Fabry disease b...

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Shranjeno v:
Bibliografske podrobnosti
izdano v:PLoS One
Main Authors: Citro, Valentina, Peña-García, Jorge, den-Haan, Helena, Pérez-Sánchez, Horacio, Del Prete, Rosita, Liguori, Ludovica, Cimmaruta, Chiara, Lukas, Jan, Cubellis, Maria Vittoria, Andreotti, Giuseppina
Format: Artigo
Jezik:Inglês
Izdano: Public Library of Science 2016
Teme:
Research Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC5082870/
https://ncbi.nlm.nih.gov/pubmed/27788225
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0165463
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