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General Review Article

血友病基因治疗：从长凳到床侧的新发展

卷 19, 期 4, 2019

页: [264 - 273] 页: 10

弟呕挨: 10.2174/1566523219666190924121836

摘要

近年来，新的基因治疗策略改变了许多遗传性疾病的预后。 遗传工具和研究模型的新发展使我们更接近彻底治愈血友病。 这篇综述将介绍血友病A和血友病B的最新基因疗法研究，包括基因疗法工具，遗传策略和动物模型。 它还总结了最近的临床试验结果。 讨论了有关血友病基因治疗当前障碍的潜在解决方案。

关键词: 基因治疗，慢病毒载体，血友病，隐性疾病，新型基因治疗，止血。

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图形摘要

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