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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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書目詳細資料
發表在:Mol Ther Methods Clin Dev
Main Authors: Sato, Yohei, Kobayashi, Hiroshi, Higuchi, Takashi, Shimada, Yohta, Ida, Hiroyuki, Ohashi, Toya
格式: Artigo
語言:Inglês
出版: Nature Publishing Group 2016
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC4980109/
https://ncbi.nlm.nih.gov/pubmed/27556060
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2016.54
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