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Gene therapy for alpha-1 antitrypsin deficiency
Alpha-1 antitrypsin (AAT) deficiency is a common single-gene disorder among Northern Europeans and North Americans. The carrier frequency for the common missense mutation (Z-AAT) ranges from 4% in the US to nearly 25% in the Republic of Ireland. Severe AAT deficiency (plasma levels below 11 μm) is m...
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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: |
Oxford University Press
2011
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3095063/ https://ncbi.nlm.nih.gov/pubmed/21498872 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddr156 |
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