AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease
Abstract 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...
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Autores principales: | , , , , , , , , , , , , , |
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Formato: | article |
Lenguaje: | EN |
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Nature Portfolio
2017
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Materias: | |
Acceso en línea: | https://doaj.org/article/678d39e7a19b43b9866916b4208c4792 |
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