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Liver depot gene therapy for Pompe disease
Gene therapy for Pompe disease has advanced to early phase clinical trials, based upon proof-of-concept data indicating that gene therapy could surpass the benefits of the current standard of care, enzyme replacement therapy (ERT). ERT requires frequent infusions of large quantities of recombinant h...
保存先:
| 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/PMC6642935/ https://ncbi.nlm.nih.gov/pubmed/31392200 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.21037/atm.2019.05.02 |
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