CRISPR/Cas9 System and Its Advances in Gene Therapy of Hematologic Diseases--Review.
10.7534/j.issn.1009-2137.2018.06.048
- Author:
Wan-Ying LI
1
;
Qing-Ping GAO
2
;
Hui LIU
3
Author Information
1. Department of Hematology, People's Hospital of Wuhan University, Wuhan 430000, Hubei Province, China.
2. Department of Hematology, People's Hospital of Wuhan University, Wuhan 430000, Hubei Province, China E-mail: 13006126258@163.com.
3. Department of Hematology, People's Hospital of Wuhan University, Wuhan 430000, Hubei Province, China E-mail:459660786@qq.com.
- Publication Type:Journal Article
- MeSH:
CRISPR-Cas Systems;
Gene Editing;
Genetic Therapy;
Hematologic Diseases;
therapy;
Humans;
Molecular Biology
- From:
Journal of Experimental Hematology
2018;26(6):1863-1867
- CountryChina
- Language:Chinese
-
Abstract:
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.