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一次性解决:治疗B型血友病的增强性第九因子基因治疗

David Lillicrap1

  • 1Department of Pathology & Molecular Medicine, Richardson Laboratory, Queen's University, Kingston, Ontario K7L 3N6, Canada.

Cell
|December 16, 2017
PubMed
概括

用AAV载体治疗B型血友病的基因治疗显示了持续的9系因子水平,几乎可以在一年多的时间内消除出血,而且没有严重的副作用. 这代表了血友病治疗的重大进步.

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