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Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease
Pompe disease is a neuromuscular disease resulting from deficiency in acid α-glucosidase (GAA), results in cardiac, skeletal muscle, and central nervous system (CNS) pathology. Enzyme replacement therapy (ERT) has been shown to partially correct cardiac and skeletal muscle dysfunction. However, ERT...
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| 主要な著者: | , , , , , , , |
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| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Nature Publishing Group
2013
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| 主題: | |
| オンライン・アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3776643/ https://ncbi.nlm.nih.gov/pubmed/23732990 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.96 |
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