乙型血友病又称为因子Ⅸ缺乏症或血浆凝血活酶成分缺乏症,是由于凝血因子Ⅸ编码基因突变导致该凝血因子功能缺陷而致病。目前对该病的治疗方法是定期注射凝血因子Ⅸ。最近Nathwani等采用基因疗法治疗乙型血友病取得了初步成功。小规模临床试验表明,患者只需要接受一次注射,自我凝血的能力就能大幅改善,且暂未发现副作用。 Hemophilia B, also known as factor IX deficiency or plasma thromboplastin deficiency, is caused by a defect in the function of the coagulation factor due to a mutation in the gene encoding coagulation factor IX. The current treatment of the disease is regular injection of coagulation factor Ⅸ. Nathwani and other recently adopted gene therapy for hemophilia B achieved initial success. Small-scale clinical trials have shown that patients who receive only one injection have a much improved ability to self-clot and have yet to find any side effects.