中文摘要：

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。

英文摘要：

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.