Center for Regenerative Medicine, University of Modena and Reggio Emilia, Via Gottardi 100, Modena, Italy.
Curr Gene Ther. 2011 Oct;11(5):399-405. doi: 10.2174/156652311797415809.
Inserting genetic information at precise locations into the human genome has been the goal of gene transfer technology for almost two decades. The spectacular progress of mammalian genetics has led to the development of technology for genome editing and homologous recombination in human somatic cells that is finally approaching efficiency compatible with clinical application. Site-specific integration, or the insertion of genes at known locations by enzymes with target recognition capacity, has progressed slowly but steadily in recent years, and could very well be the basis of the next generation of gene transfer technology. This review focuses on the use of Rep, the replicase/integrase of the adeno-associated virus (AAV), to insert genes at the natural AAV integration site on human chromosome 19. This region (AAVS1) has characteristics that make it an ideal target for somatic transgenesis.
将近二十年来，将遗传信息精确地插入人类基因组一直是基因转移技术的目标。哺乳动物遗传学的惊人进展导致了人类体细胞基因组编辑和同源重组技术的发展，其最终效率接近临床应用的要求。近年来，定点整合，即通过具有目标识别能力的酶将基因插入已知位置的技术，一直在缓慢而稳定地发展，它很可能成为下一代基因转移技术的基础。本文综述了利用腺相关病毒（AAV）的复制酶/整合酶 Rep 将基因插入人类 19 号染色体上天然的 AAV 整合位点的方法。该区域（AAVS1）具有使其成为体转基因理想靶标的特征。
