腺相关病毒介导的肝细胞癌靶向治疗策略thestrategiesof-药学学报.pdfVIP

腺相关病毒介导的肝细胞癌靶向治疗策略 * 高三星，蔡双凤 ，许瑞安 （华侨大学生物医学学院，分子药物教育部工程研究中心，福建省分子医学重点实验室， 厦门市海洋与基因工程药物重点实验室，福建 厦门 361021 ） 摘要: 肝细胞癌是最为常见的肝脏原发性肿瘤，也是致死率居第二位的癌症。随着分子生 物学的发展，基因治疗成为有治愈癌症潜力的新途径。腺相关病毒以其较高的感 率、低致病 性和低免疫原性等优势成为最有前景的基因治疗载体。靶向基因治疗能实现目的基因的靶向表 达，避免对非靶向器官的毒性作用而弥补了传统腺相关病毒在基因治疗中的不足。本文就近年 来肝细胞癌发病机制、腺相关病毒的生物学特点及其介导的靶向治疗肝细胞癌策略做一综述， 为肝细胞癌靶向基因治疗的进一步研究提供参考。 关键词: 肝细胞癌；分子机制；腺相关病毒；靶向治疗；基因治疗 中图分类号: R735.7 文献标识码: A 文章编号: 0513-4870(2017) The strategies of targeting therapy of hepatocellular carcinoma by adeno - associated virus * GAO San-xing, CAI Shuang-feng , XU Rui-an （Engineering Research Center of Molecular Medicine of Ministry of Education, Key Labo ratory of Fujian Molecular Medicine, Key Laboratory of Xiamen Marine and Gene Drugs, School of Biomedical Sciences, Huaqiao University, Xiamen 361021, China ） Abstract ：Hepatocellular carcinoma (HCC) is the most common liver primary tumor, which is also the second leading cause of death in cancer. With the development of molecular biology and technology, gene therapy has become a new potential method to cure cancer. As to the gene therapy vector, the adeno-associated virus (AAV) is the most promising vector for its high efficiency of infection, low pathogenicity and low immunogenicity. However, AAV has so wide range of host that can produce side effects. Targeted therapy can achieve higher site-specificity about where you want the gene to transfer or express, avoiding systemic and non-targeted organs toxicity, thereby improving the safety and efficacy of gene therapy. In this review, we overview what has been 收稿日期: 2017-04-01; 修回日期: 2017-05-09. 基金项目： 国家自然科学基金资助项目 (81271692