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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Publicado en:PLoS One
Autores principales: 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
Formato: Artigo
Lenguaje:Inglês
Publicado: Public Library of Science 2016
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Research Article
Acceso en línea: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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