CRISPR/Cas9 and its application in drugs development
10.16438/j.0513-4870.2017-0644
- VernacularTitle:CRISPR/Cas9技术及其在药物研发中的应用
- Author:
Di LU
1
;
Li LI
1
;
Xian-ming DENG
1
Author Information
1. State-Province Joint Engineering Laboratory of Targeted Drugs from Natural Products, School of Life Sciences, Xiamen University, Xiamen 361102, China
- Publication Type:REVIEWS
- Keywords:
CRISPR/Cas9 system;
genome editing;
drug-resistant mutation;
target validation;
drug development
- From:
Acta Pharmaceutica Sinica
2018;53(1):11-20
- CountryChina
- Language:Chinese
-
Abstract:
CRISPR/Cas9 system, consisting of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins, is a prokaryotic immune system that confers resistance to foreign genetic elements such as those present within plasmids and phages. A simple version of the CRISPR/Cas system, type Ⅱ CRISPR, has been modified to edit genomes. By delivering the Cas9 nuclease together with a synthetic guide RNA (sgRNA) into cells, genome can be edited at desired loci site. CRISPR/Cas genome editing techniques have been widely implemented in various species and research areas. In this review, we summarize the several applications of CRISPR/Cas9 in the field of drug discovery and development, which include target gene screening and editing, drug target screening and validation, generation of animal models and treatment of genetic disease, etc. The defects and improvements of CRISPR/Cas9 technology is discussed as well.
