血友病A基因治疗载体研究现状

Advancements in Researches on Vectors of the Gene Therapy for Hemophilia A

血友病A是X染色体隐性遗传出血性疾病。其发病原因是患者血液中先天缺乏凝血因子FⅧ。用于血友病A基因治疗研究的载体有病毒载体和非病毒载体，目前研究较多的是病毒载体，主要有逆转录病毒载体和慢病毒载体，腺病毒载体及腺相关病毒载体等。非病毒载体主要有质粒、脂质体、转座子等。文章拟对血友病A基因治疗各载体的特点和研究进展作一综述。

Hemophilia A is an X-linked recessive bleeding disorder caused by a congenital deficiency of clotting factor VIU in patients＇ blood. The vectors applied in researches on the gene therapy for hemophilia A include viral vectors and nonviral vectors. Recently, more researches have been done in the development of viral vectors system, such as retrovirus vectors, lentivirus vectors, adenovirus vectors, and adeno-associated vectors. Nonviral vectors mainly include plasmids, lipo fectamine, transposon, etc. This article will review the progress in researches on the vectors of gene therapy for hemophilia A.