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Updated: Jun 24, 2025

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克服血友病A基因治疗的局限性,用增强功能因素VIII变异克服血友病A基因治疗的局限性

Anna R Sternberg, Cristina Martos-Rus, Robert Davidson

    bioRxiv : the preprint server for biology
    |June 3, 2024
    PubMed
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    血友病A的基因疗法面临的挑战是持久的第八因子 (FVIII) 表达. 一种新的FVIII变体 (FVIII-QQ) 显示出在较低,更稳定的表达水平上正常化血液静止的希望.

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