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Advancements in AAV-mediated Gene Therapy for Pompe Disease

Pompe disease (glycogen storage disease type II) is caused by mutations in acid α-glucosidase (GAA) resulting in lysosomal pathology and impairment of the muscular and cardio-pulmonary systems. Enzyme replacement therapy (ERT), the only approved therapy for Pompe disease, improves muscle function by...

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Detalles Bibliográficos
Autores principales:	Salabarria, S.M., Nair, J., Clement, N., Smith, B.K., Raben, N., Fuller, D.D., Byrne, B.J., Corti, M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	IOS Press 2020
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7029369/ https://www.ncbi.nlm.nih.gov/pubmed/31796685 http://dx.doi.org/10.3233/JND-190426

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