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AAV-mediated delivery of secreted acid α-glucosidase with enhanced uptake corrects neuromuscular pathology in Pompe mice

Gene therapy is under advanced clinical development for several lysosomal storage disorders. Pompe disease, a debilitating neuromuscular illness affecting infants, children, and adults with different severity, is caused by a deficiency of lysosomal glycogen-degrading enzyme acid α-glucosidase (GAA)....

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Detalles Bibliográficos
Autores principales:	Meena, Naresh K., Randazzo, Davide, Raben, Nina, Puertollano, Rosa
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society for Clinical Investigation 2023
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10543735/ https://www.ncbi.nlm.nih.gov/pubmed/37463048 http://dx.doi.org/10.1172/jci.insight.170199

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