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Correction of murine ADAMTS13 deficiency by hematopoietic progenitor cell–mediated gene therapy
ADAMTS13, a metalloprotease primarily synthesized in liver and endothelial cells, cleaves von Willebrand factor (VWF) at the central A2 domain, thereby reducing the sizes of circulating VWF multimers. Genetic or acquired deficiency of plasma ADAMTS13 activity leads to a potentially fatal syndrome, t...
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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: |
American Society of Hematology
2009
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| 主題: | |
| オンライン・アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2652365/ https://ncbi.nlm.nih.gov/pubmed/19141866 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2008-08-173021 |
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