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AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease

Pompe disease (PD) is a metabolic myopathy due to acid alpha-glucosidase deficiency and characterized by extensive glycogen storage and impaired autophagy. We previously showed that modulation of autophagy and lysosomal exocytosis by overexpression of the transcription factor EB (TFEB) gene was effe...

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Detalles Bibliográficos
Autores principales:	Gatto, Francesca, Rossi, Barbara, Tarallo, Antonietta, Polishchuk, Elena, Polishchuk, Roman, Carrella, Alessandra, Nusco, Edoardo, Alvino, Filomena Grazia, Iacobellis, Francesca, De Leonibus, Elvira, Auricchio, Alberto, Diez-Roux, Graciana, Ballabio, Andrea, Parenti, Giancarlo
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2017
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5678083/ https://www.ncbi.nlm.nih.gov/pubmed/29118420 http://dx.doi.org/10.1038/s41598-017-15352-2

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