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Progress and challenges of gene therapy for Pompe disease
Pompe disease (PD) is a monogenic disorder caused by mutations in the acid alpha-glucosidase gene (Gaa). GAA is a lysosomal enzyme essential for the degradation of glycogen. Deficiency of GAA results in a severe, systemic disorder that, in its most severe form, can be fatal. About a decade ago, the...
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| 出版年: | Ann Transl Med |
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| 主要な著者: | , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
AME Publishing Company
2019
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6642941/ https://ncbi.nlm.nih.gov/pubmed/31392199 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.21037/atm.2019.04.67 |
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