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

摘要： 随着基因治疗产品不断得到批准用于临床研究，基因治疗相关的病毒载体也受到广泛关注，其中腺相关病毒载体因其低致病性、低免疫原性、宿主范围广泛且稳定表达等独特优势成为最有前景的病毒载体，广泛应用于临床研究，目前已有2种重组腺相关病毒载体的药品得到批准且效果显著，但由于大规模生产技术的缺乏限制了重组腺相关病毒载体的研究和广泛应用。就腺相关病毒的结构和功能、载体的生产方式及其现阶段在基因治疗中的应用进行了综述，以期为今后以腺相关病毒为载体的基因治疗大规模应用于市场提供参考。

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