Pharmacological Enhancement of α-Glucosidase by the Allosteric Chaperone N-acetylcysteine

Pompe disease (PD) is a metabolic myopathy due to the deficiency of the lysosomal enzyme α-glucosidase (GAA). The only approved treatment for this disorder, enzyme replacement with recombinant human GAA (rhGAA), has shown limited therapeutic efficacy in some PD patients. Pharmacological chaperone th...

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Bibliographische Detailangaben
Hauptverfasser: Porto, Caterina, Ferrara, Maria C, Meli, Massimiliano, Acampora, Emma, Avolio, Valeria, Rosa, Margherita, Cobucci-Ponzano, Beatrice, Colombo, Giorgio, Moracci, Marco, Andria, Generoso, Parenti, Giancarlo
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2012
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3519985/
https://ncbi.nlm.nih.gov/pubmed/22990675
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.152
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