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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Autores principales: | Véronique Roig-Zamboni, Beatrice Cobucci-Ponzano, Roberta Iacono, Maria Carmina Ferrara, Stanley Germany, Yves Bourne, Giancarlo Parenti, Marco Moracci, Gerlind Sulzenbacher |
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Formato: | article |
Lenguaje: | EN |
Publicado: |
Nature Portfolio
2017
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Materias: | |
Acceso en línea: | https://doaj.org/article/1092edf30ccd4d65964b3a4b8b5fdd66 |
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