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Article

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

1 Jan 2016

Abstract excerpt

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 being investigated, including gene therapy and pharmacological chaperones. We previously used lentiviral...

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PMID: 27556060
DOI: 10.1038/mtm.2016.54

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