TFEB overexpression promotes glycogen clearance of Pompe disease iPSC-derived skeletal muscle

Pompe disease (PD) is a lysosomal disorder caused by acid α-glucosidase (GAA) deficiency. Progressive muscular weakness is the major symptom of PD, and enzyme replacement therapy can improve the clinical outcome. However, to achieve a better clinical outcome, alternative therapeutic strategies are b...

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Detalles Bibliográficos
Autores principales: Sato, Yohei, Kobayashi, Hiroshi, Higuchi, Takashi, Shimada, Yohta, Ida, Hiroyuki, Ohashi, Toya
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4980109/
https://www.ncbi.nlm.nih.gov/pubmed/27556060
http://dx.doi.org/10.1038/mtm.2016.54

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4980109/
https://www.ncbi.nlm.nih.gov/pubmed/27556060
http://dx.doi.org/10.1038/mtm.2016.54

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