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Article

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

PloS one - 1 Jan 2016

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

Abstract excerpt

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 bind the active site and couple a stabilizing effect, which is required, to an inhibitory effect, which is...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Chlorocebus aethiops

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Source

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Identifiers

PMID: 27788225
DOI: 10.1371/journal.pone.0165463
PMCID: PMC5082870

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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