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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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| Publicado no: | Mol Ther Methods Clin Dev |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group
2016
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| Assuntos: | |
| Acesso em linha: | 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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