关键词: 腺相关病毒, 基因治疗, 杆状病毒表达系统, 脂蛋白脂肪酶缺乏症, 人类视网膜疾病

Abstract: With the continuous approval of gene therapy products into the clinical research, the viral vectors which are used for gene therapy have attracted extensive attention. Adeno-associated virus is the most promising viral vector of many other viral vectors because of its low pathogenicity, low immunogenicity and the advantage of the host range and stable expression. It has gotten a wide range of applications in clinical research. Currently, two drugs related to recombinant adenovirus vectors have approved for clinical application with significant effects. But its lacking of mass production technology limits its research and wide applications. This review summarized the structure and function of adeno-associated virus, its production mode and recent clinical application in gene therapy to support reference for its widely application in the market.

Key words: adeno-associated virus, gene therapy, baculovirus expression system, LPLD, human retinal disease