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Updated: Jun 21, 2025

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罗克塔维亚基因治疗血友病A的基因疗法

Benjamin J Samelson-Jones1,2,3, Juliana C Small1, Lindsey A George1,2,3

  • 1Clinical In Vivo Gene Therapy and Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA.

Blood advances
|July 11, 2024
PubMed
概括

瓦洛克托基因roxaparvovec (Roctavian) 是一个具有里程碑意义的血友病A基因疗法,但因子VIII表达的不明原因下降需要进一步调查以确定未来的治疗方法.

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