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Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice
Pompe disease is an autosomal recessive genetic disorder characterized by a deficiency of the enzyme responsible for degradation of lysosomal glycogen (acid α-glucosidase (GAA)). Cardiac dysfunction and respiratory muscle weakness are primary features of this disorder. To attenuate the progressive a...
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| Veröffentlicht in: | Mol Ther Methods Clin Dev |
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| Hauptverfasser: | , , , , , , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Nature Publishing Group
2015
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4445006/ https://ncbi.nlm.nih.gov/pubmed/26029718 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2015.7 |
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