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Review Article

我们的立场：血友病A的基因治疗

卷 20, 期 2, 2020

页: [142 - 151] 页: 10

弟呕挨: 10.2174/1566523220666200806110849

价格: $65

摘要

血友病A (HA)是一种遗传性出血性疾病，由于缺乏凝血因子VIII (FVIII)在血浆中。HA患者常发生关节和肌肉自发性反复出血，甚至出现脑出血，可导致残疾或死亡。虽然目前可以通过提供来源于血浆或重组的FVIII来控制该病，但这种方法成本高昂，而且可能在很大一部分患者体内产生中和抗体，这使得治疗方案无效和难以获得。考虑到HA的单基因性以及FVIII的轻微升高可显著缓解其表型，HA被认为是一种合适的基因治疗靶向疾病。因此，通过病毒或非病毒载体将功能F8基因拷贝导入适当的靶细胞，包括通过基因组编辑方法进行基因校正，最终可能为HA患者提供一种有效的治疗方法。在这次审查中,我们讨论基因治疗的最新进展与病毒和病毒的HA交付向量,包括piggyBac（转座因子）、慢病毒和腺相关病毒载体,以及新的提高涉及肝毒性的问题,预先存在的病毒的中和抗体的方法,为病毒的靶细胞类型的选择。

关键词: 血友病A，基因治疗，BDD-F8，慢病毒，腺相关病毒，非病毒

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