血友病B基因治疗的研究进展  

Research progress in gene therapy of hemophilia B

作　　者：王馨 闫振宇 Wang Xin;Yah Zhenyu(North China University of Science and Technology,Tangshan 063000,Hebei Province,China（Wang X;Department of Hematology,Affiliated Hospital of North China University of Science and Technology,Tangshan 063000,Hebei Province,China（Yah ZY)

机构地区：[1]华北理工大学,河北唐山063000 [2]华北理工大学附属医院血液内科

出　　处：《国际输血及血液学杂志》2018年第4期317-321,共5页International Journal of Blood Transfusion and Hematology

基　　金：2016年河北省政府临床医学优秀人才基础与培养项目（361036）

摘　　要：血友病B为临床上常见的出血性疾病,为X染色体连锁隐性遗传性疾病,以男性患者多见,女性患者极其罕见.我国血友病B患者数量众多,目前主要治疗方法为输注凝血因子FⅨ制剂以达到止血目的.但是由于反复输注凝血因子FⅨ制剂而产生凝血因子FⅨ抑制物、相关制剂价格高昂等问题,多数患者未能得到有效治疗,影响患者的生存质量.血友病B为单基因遗传性疾病,是基因治疗遗传性疾病的理想模型.因此,基因治疗为最有望治愈血友病B的方法.近年来,随着多种病毒载体的成功构建、定点切割的核酸酶的发现,将基因治疗血友病B的各方面研究进一步向前推进.笔者拟就血友病B基因治疗的研究进展进行综述.Hemophilia B is a common hemorrhagic disease in clinic and a X chromosome-linked recessive genetic disorder.It is common in male patients,and rare in female patients.The number of patients with hemophilia B in China is numerous,the main treatment is infusing coagulation factor FⅨ to achieve hemostasis.But coagulation factor FⅨ inhibitors caused by repeating infusion of coagulation factor FⅨ and costly price of coagulation factor FⅨ products,the majority of patients have not been effectively treated,which influence the life quality of the patients with hemophilia B.Hemophilia B is a monogenetic inherited disease and is an ideal model for genetic treatment.Therefore,gene therapy has become the most promising approach to cure hemophilia B.In recent years,with the successful construction of a variety of viral vectors and the discovery of site-specific nuclease,progress of gene therapy for hemophilia B has been further promoted.In this paper,the progress of gene therapy for hemophilia B is reviewed.

关 键 词：血友病B 基因治疗 凝血因子Ⅸ CRISPR-Cas系统 基因载体 

分 类 号：R554.1[医药卫生—血液循环系统疾病]