Base editors: a powerful tool for generating animal models of human diseases
Authors:Zongyang Lu1,2 and Xingxu Huang1,3
1 School of Life Science and Technology, ShanghaiTech University, 100 Haike Rd., Pudong New Area, Shanghai 201210, China.
2 University of Chinese Academy of Sciences, 100049 Beijing, China.
3 CAS Center for Excellence in Molecular Cell Science, Shanghai Institute of Biochemistry and Cell Biology, Chinese Academy of Sciences; University of Chinese Academy of Sciences, 320 Yueyang Road, Shanghai 200031, China.
Keywords:
base editors, disease models, genome engineering, mice, non-human primates.
Corresponding Author(s):
Conflict of interest statement:
The authors declare no conflict of interest.
Please cite this article as:
Zongyang Lu and Xingxu Huang (2018). Base editors: a powerful tool for generating animal models of human diseases. Cell Stress 2(10): 242-245. doi: 10.15698/cst2018.10.156
© 2018 Lu and Huang. This is an open-access article released under the terms of the Creative Commons Attribution (CC BY) license, which allows the unrestricted use, distribution, and reproduction in any medium, provided the original author and source are acknowledged.
Abstract:
Myriads of genetic mutations, including base substitutions, deletions, and insertions as well as chromosome structural variations, have been detected in many human diseases. Although current combination of genomics and bioinformatics has contributed greatly to understanding the genetics of these disorders, it remains challenging to ensure the causal functions of each mutation, and then to further investigate the underlying mechanism and to develop therapeutic strategies. Animal models generated by genome engineering are the key to address these issues. In this review, we will first revisit the limitation of conventional gene editing tools and mouse models generated in the past. We will then introduce a novel tool, base editors (BEs), which present a new promising approach to establish pathogenically relevant animal models. Finally, we will discuss the application of BEs in non-human primates and share our perspectives on future development of base-editing techniques.