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Murine Muscle Cell Models for Pompe Disease and Their Use in Studying Therapeutic Approaches
Lysosomes filled with glycogen are a major pathologic feature of Pompe disease, a fatal myopathy and cardiomyopathy caused by a deficiency of the glycogen-degrading lysosomal enzyme, acid α-glucosidase (GAA). To facilitate studies germane to this genetic disorder, we developed two in vitro Pompe mod...
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| Main Authors: | , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2009
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2680079/ https://ncbi.nlm.nih.gov/pubmed/19167256 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2008.12.012 |
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