腺相关病毒(Adeno-associated virus,AAV)是单链DNA病毒。AAV基因载体由于安全性好、宿主细胞范围广(分裂和非分裂细胞)、免疫原性低,在体内表达外源基因时间长等特点,被视为最有前途的基因转移载体之一,在世界范围内的基因治疗和疫苗研究中得到广泛应用。AAV编码的非结构蛋白Rep具有抑制肿瘤和病毒的作用,并可以对宿主细胞周期和增殖产生重要影响。Rep蛋白也可以对许多异源启动子产生抑制效果,并同时对胞内许多转录原件产生影响。文章就AAV的基本生物学特性和其Rep蛋白在肿瘤治疗方向的最新进展进行了综述。 Adeno-associated virus (AAV) is a single-stranded DNA virus. AAV gene vector is regarded as one of the most promising gene transfer vectors due to its good safety, wide range of host cells (split and non-dividing cells), low immunogenicity and long time expression of foreign genes in vivo. Worldwide gene therapy and vaccine research is widely used. The AAV-encoded non-structural protein Rep has the effect of inhibiting tumors and viruses and can have a major impact on the host cell cycle and proliferation. The Rep protein can also exert an inhibitory effect on many heterologous promoters and simultaneously affect many intracellular transcriptional elements. This review summarizes the basic biological characteristics of AAV and the recent progress of its Rep protein in the treatment of cancer.