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

Autor: Porto, Caterina, Ferrara, Maria C, Meli, Massimiliano, Acampora, Emma, Avolio, Valeria, Rosa, Margherita, Cobucci-Ponzano, Beatrice, Colombo, Giorgio, Moracci, Marco, Andria, Generoso, Parenti, Giancarlo

Publikováno v: In Molecular Therapy December 2012 20(12):2201-2211

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

Autor: Giorgio Colombo, Valeria Avolio, Maria Carmina Ferrara, Giancarlo Parenti, Caterina Porto, Beatrice Cobucci-Ponzano, Margherita Rosa, Massimiliano Meli, Emma Acampora, Generoso Andria, Marco Moracci

Publikováno v: Molecular therapy
20 (2012): 2201–2211. doi:10.1038/mt.2012.152
info:cnr-pdr/source/autori:Porto, Caterina; Ferrara, Maria C.; Meli, Massimiliano; Acampora, Emma; Avolio, Valeria; Rosa, Margherita; Cobucci-Ponzano, Beatrice; Colombo, Giorgio; Moracci, Marco; Andria, Generoso; Parenti, Giancarlo/titolo:Pharmacological Enhancement of alpha-Glucosidase by the Allosteric Chaperone N-acetylcysteine/doi:10.1038%2Fmt.2012.152/rivista:Molecular therapy (Print)/anno:2012/pagina_da:2201/pagina_a:2211/intervallo_pagine:2201–2211/volume:20
Molecular therapy (Online) 20 (2012): 2201–2211. doi:10.1038/mt.2012.152
info:cnr-pdr/source/autori:Porto C., Ferrara M.C., Meli M., Acampora E., Avolio V., Rosa M., Cobucci-Ponzano B./titolo:Pharmacological enhancement of alpha-glucosidase by the allosteric chaperone N-acetylcysteine/doi:10.1038%2Fmt.2012.152/rivista:Molecular therapy (Online)/anno:2012/pagina_da:2201/pagina_a:2211/intervallo_pagine:2201–2211/volume:20

Pompe disease (PD) is a metabolic myopathy due to the deficiency of the lysosomal enzyme alpha-glucosidase (GAA). The only approved treatment for this disorder, enzyme replacement with recombinant human GAA (rhGAA), has shown limited therapeutic effi

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1e21bfe39b6d90526e4c5b9b8eaf13b7
https://europepmc.org/articles/PMC3519985/