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Spinal Delivery of AAV Vector Restores Enzyme Activity and Increases Ventilation in Pompe Mice
Pompe disease is a form of muscular dystrophy due to lysosomal storage of glycogen caused by deficiency of acid α-glucosidase (GAA). Respiratory failure in Pompe disease has been attributed to respiratory muscle dysfunction. However, evaluation of spinal tissue from Pompe patients and animal models...
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| Hlavní autoři: | , , , , |
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| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
Nature Publishing Group
2012
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| Témata: | |
| On-line přístup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3255594/ https://ncbi.nlm.nih.gov/pubmed/22008916 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.214 |
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