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血友病A基因治疗载体研究现状
引用本文:王晴,颜景斌,曾溢滔.血友病A基因治疗载体研究现状[J].国外医学:分子生物学分册,2011(5):437-442.
作者姓名:王晴  颜景斌  曾溢滔
作者单位:上海市儿童医院,上海交通大学医学遗传研究所,上海市200040
摘    要: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。

关 键 词:血友病A  载体  基因治疗  人凝血因子Ⅷ

Advancements in Researches on Vectors of the Gene Therapy for Hemophilia A
Authors:WANG Qing  YAN Jingbin  ZENG Yitao
Institution:Children' s Hospital of Shanghai, Institute of Medical Genetics, Shanghai Jiaotong University, Shanghai, 200040, China
Abstract: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.
Keywords:hemophilia A  vector  gene therapy  human coagulation factor Ⅷ
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