Structure of human lysosomal acid α-glucosidase–a guide for the treatment of Pompe disease

Structure of human lysosomal acid α-glucosidase–a guide for the treatment of Pompe disease

Pompe disease is caused by mutations in lysosomal acid α-glucosidase (GAA) and patients are being treated with recombinant human α-glucosidase (rhGAA). Here the authors present the crystal structures of rhGAA and its complexes with inhibitors and a pharmacological chaperone, which is important for d...

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Bibliographic Details
Main Authors: Véronique Roig-Zamboni, Beatrice Cobucci-Ponzano, Roberta Iacono, Maria Carmina Ferrara, Stanley Germany, Yves Bourne, Giancarlo Parenti, Marco Moracci, Gerlind Sulzenbacher
Format: Article
Language:English
Published: Nature Publishing Group 2017-10-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-017-01263-3

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https://doi.org/10.1038/s41467-017-01263-3

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