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血友 B の ため の 強化 さ れ た 因子 IX の 遺伝子 療法

David Lillicrap1

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

Cell
|December 16, 2017
PubMed
まとめ

AAVベクトルを用いたB型血友症の遺伝子治療は 持続的なファクターIXレベルを示し,深刻な副作用なしに 1年以上出血をほぼ排除します. これは血友病の治療における 重要な進歩を表しています

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