Department of Human Genome Research, Kazusa DNA Research Institute, 2-6-7 Kazusa-Kamatari, Kisarazu, Chiba 292-0818, Japan.
Drug Discov Today. 2010 Mar;15(5-6):198-202. doi: 10.1016/j.drudis.2010.01.006. Epub 2010 Jan 29.
The emergence of induced pluripotent stem (iPS) cell technology has shifted gene correction therapy toward reality. Crucial issues are ensuring the safety of using iPS cell technology in patients and discovering how best to transfer genetically manipulated iPS cells back into patients. One key issue that has hindered progress of gene correction therapy, however, is the inability to achieve efficient homologous recombination in human iPS cells. This review focuses on recently developed technologies that aim to improve homologous recombination in human embryonic stem cells and on their application to iPS cells.
诱导多能干细胞（iPS）技术的出现将基因矫正治疗推向了现实。关键问题是确保在患者中使用 iPS 细胞技术的安全性，并发现将遗传修饰的 iPS 细胞最佳地回输到患者体内的方法。然而，阻碍基因矫正治疗进展的一个关键问题是无法在人 iPS 细胞中实现有效的同源重组。本综述重点介绍了最近开发的旨在提高人胚胎干细胞同源重组效率的技术及其在 iPS 细胞中的应用。
