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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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發表在: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
格式: Artigo
語言:Inglês
出版: Public Library of Science 2016
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在線閱讀: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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