血友病A的基因治疗  

Gene therapy for hemophilia A

作　　者：刘慕君[1] 刘艳平[1] 刘雄昊[1] 

机构地区：[1]中南大学生物科学与技术学院

出　　处：《中国临床药理学与治疗学》2010年第8期932-937,共6页Chinese Journal of Clinical Pharmacology and Therapeutics

基　　金：国家自然科学基金资助项目(30700458;30900801)

摘　　要：血友病A是一种X-连锁隐性遗传病,在男性中的发病率约为(1～2)/10000。血友病A的临床症状主要是自发性关节、软组织或其他组织的出血、血肿,常可致残,甚至危及生命。其致病机制是由于编码凝血因子Ⅷ的基因先天性异常而导致的凝血因子Ⅷ缺乏或功能缺陷。血友病A是基因治疗的首选疾病之一。本文就血友病A基因治疗中所采用的载体和靶细胞作一综述。Hemophilia A is an X chromosome-linked recessive inheritable bleeding disorder with an estimated incidence of 1-2 per 10000 males.Patients with Hemophilia A experience spontaneous bleeding into joints,soft tissues and other sites.Uncontrolled haemorrhagic episodes are crippling and potentially life threatening.Hemophilia A is caused by a deficiency or abnormality in factor Ⅷ,which is one of the most attractive candidates for gene therapy.In this article,the research of vectors and target cell in gene therapy for hemophilia A was reviewed.

关 键 词：人凝血因子Ⅷ 血友病A 基因治疗 

分 类 号：R554.1[医药卫生—血液循环系统疾病]