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血友病B是由于凝血因子Ⅸ缺乏所致的一种严重出血性遗传病,其临床治疗主要依靠输血或补充人Ⅸ因子浓缩制剂,但这种替代疗法不仅疗效短,费用昂贵,而且使患者面临着爱滋病毒及肝炎病毒感染的威胁。目前,利用基因转移技术进行遗传病基因治疗的研究已取得令人注目的进展。在先前的血友病B基因治疗的研究中,我们构建了几个带有不同启动子控制
Hemophilia B is a severe hemorrhagic genetic disease due to lack of coagulation factor IX. Its clinical treatment mainly depends on blood transfusions or human factor IX enrichment preparations. However, this alternative therapy is not only ineffective and expensive, but also enables patients Faced with the threat of HIV and hepatitis virus infection. Currently, the use of gene transfer technology for genetic disease gene therapy research has made remarkable progress. In a previous study of Hemophilia B gene therapy, we constructed several vectors with different promoter controls