Application of CRISPR-Cas9 genome editing for constructing animal models of human diseases.
10.3760/cma.j.issn.1003-9406.2016.04.030
- VernacularTitle:CRISPR-Cas9基因编辑技术在构建动物疾病模型中的应用
- Author:
Zhanhui OU
1
;
Xiaofang SUN
Author Information
1. Key Laboratory for Major Obstetric Diseases of Guangdong Province, Key Laboratory of Reproduction and Genetics of Guangdong Higher Education Institutes, The Third Affiliated Hospital of Guangzhou Medical University, Guangzhou, Guangdong 510150, China. Email: xiaofangsun@gzhmu.edu.cn.
- Publication Type:Journal Article
- MeSH:
Animals;
Clustered Regularly Interspaced Short Palindromic Repeats;
genetics;
Disease Models, Animal;
Humans;
RNA Editing;
genetics
- From:
Chinese Journal of Medical Genetics
2016;33(4):559-563
- CountryChina
- Language:Chinese
-
Abstract:
The CRISPR-Cas9 system is a new targeted nuclease for genome editing, which can directly introduce modifications at the targeted genomic locus. The system utilizes a short single guide RNA (sgRNA) to direct the endonuclease Cas9 in the genome. Upon targeting, Cas9 can generate DNA double-strand breaks (DSBs). As such DSBs are repaired by non-homologous end joining (NHEJ) or homology directed repair (HDR), therefore facilitates introduction of random or specific mutations, repair of endogenous mutations, or insertion of DNA elements. The system has been successfully used to generate gene targeted cell lines including those of human, animals and plants. This article reviews recent advances made in this rapidly evolving technique for the generation of animal models for human diseases.