Li Wan-Ying, Gao Qing-Ping, Liu Hui
Department of Hematology, People's Hospital of Wuhan University, Wuhan 430000, Hubei Province, China.
Department of Hematology, People's Hospital of Wuhan University, Wuhan 430000, Hubei Province, China E-mail:
Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2018 Dec;26(6):1863-1867. doi: 10.7534/j.issn.1009-2137.2018.06.048.
In recent years, with the development of gene editing technology, the site-specific genome can be modified. The curability of genetic disease may be achieved by the use of gene editing techniques. As the simplicity, high specificity and economical efficiency, much attention has been paid to the CRISPR/Cas9 system, which was been widely used in research of molecular biology and other fields of life science. In this review, the mechanism for CR1SPR/Cas9 system and the progress of gene therapy, such as for hemophilia, betathalassaemia and chronic myeloid leukemia were summarized briefly.
近年来，随着基因编辑技术的发展，特定基因组位点能够被修饰。利用基因编辑技术或许可以实现对遗传疾病的治愈。由于具有操作简便、特异性高和经济高效等特点，CRISPR/Cas9系统备受关注，已广泛应用于分子生物学研究及生命科学的其他领域。本文简要综述了CRISPR/Cas9系统的作用机制以及在血友病、β地中海贫血和慢性髓性白血病等疾病基因治疗方面的研究进展。
