血友病A基因治疗载体研究现状 |
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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. |
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作者姓名: | 王晴 颜景斌 曾溢滔 |
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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 |
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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。
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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 |
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Authors: | WANG Qing YAN Jingbin ZENG Yitao |
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Institution: | Children' s Hospital of Shanghai, Institute of Medical Genetics, Shanghai Jiaotong University, Shanghai, 200040, China |
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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. |
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Keywords: | hemophilia A vector gene therapy human coagulation factor Ⅷ |
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