《生命科学》 2021, 33(11): 1370-1381

CRISPR/Cas9技术及其在肿瘤研究与治疗中的应用

马孟丹^1,2 , 杨育宾^1,2 , 陈延萍^1,2 , 吴　果^1,2 , 刘宇辰^1,* , 黄卫人^1,*

¹深圳合成生物创新研究院，转化医学研究院，深圳市第二人民医院，深圳 518036 ²汕头大学医学院，汕头 515041

摘　要：

CRISPR/Cas 系统可以进行基因敲入、敲除、单碱基突变和表观遗传调控，在生物医学领域有广阔的发展前景，为探索疾病发生和发展的机制、发现药物靶点提供了新思路。该文对CRISPR/Cas 系统、Cas9 的功能和在肿瘤中的应用，包括模型构建、机制研究、分子诊断、生物治疗和药物研发与筛选方面进行了概述，同时讨论了CRISPR/Cas9 在肿瘤基础研究和临床治疗中的不足，并展望其应用前景。

通讯作者：刘宇辰 , Email:liuyuchenmdcg@163.com 黄卫人 , Email:pony8980@163.com

CRISPR/Cas9 and its application in cancer research and therapy

MA Meng-Dan^1,2 , YANG Yu-Bin^1,2 , CHEN Yan-Ping^1,2 , WU Guo^1,2 , LIU Yu-Chen^1,* , HUANG Wei-Ren^1,*

¹Shenzhen Institute of Synthetic Biology, Institute of Translational Medicine, Shenzhen Second Hospital, Shenzhen 518036, China ²Shantou University Medical College, Shantou 515041, China

Abstract:

The recent developed CRISPR/Cas system can be used in gene knock-in, knock-out, single base mutation and epigenetic regulation. Technologies based on CRISPR/Cas have a wide developmental prospect in biomedicine. It puts forward new ways for exploring the mechanism of disease development, uncovering high-confidence biomarkers and screening target drugs. This paper focused on the revolutionary CRISPR/Cas system and the application of CRISPR/Cas9 in cancer, including model construction, mechanism research, molecular diagnosis, biotherapy, drug development and screening. The limitations and prospects of CRISPR/Cas9 in cancer research and clinical therapy were also discussed.

Communication Author：LIU Yu-Chen , Email:liuyuchenmdcg@163.com HUANG Wei-Ren , Email:pony8980@163.com