《生命科学》 2021, 33(7): 912-920

CRISPR/Cas9基因编辑技术在阿尔茨海默病研究中的应用

孙谕莹 , 周鹤妍 , 黄汉昌^*

北京联合大学生物活性物质与功能食品北京市重点实验室，北京 100191

摘　要：

阿尔茨海默病(Alzheimer’s disease, AD) 是一种与基因和环境等多因素相关的神经退行性疾病，其发病机理复杂，目前也鲜有针对AD 的治疗药物。CRISPR/Cas9 是基于DNA 重组修复原理的第三代基因编辑技术，该技术已经成功地应用于斑马鱼、啮齿类动物基因组的编辑，将其应用于AD 有助于推动AD 发病机理和治疗方法的研究。该文在介绍CRISPR/Cas9 基因编辑技术的基础上，综述了该技术在AD 病理模型构建、致病风险因素筛查、治疗靶标寻找和靶向治疗等方面的研究和应用进展。

通讯作者：黄汉昌 , Email:hanchang@buu.edu.cn

Application of CRISPR/Cas9 gene-editing technology on the research in Alzheimer’s disease 

SUN Yu-Ying , ZHOU He-Yan , HUANG Han-Chang^*

Beijing Key Laboratory of Bioactive Substances and Functional Food, Beijing Union University, Beijing 100191, China

Abstract:

Alzheimer’s disease (AD) is a neurodegenerative disease, which etiopathogenesis is related to multiple factors, such as genetic and environmental factors. The pathological mechanism of AD is quite complex, and there are only a few drugs developed for AD treatment. CRISPR/Cas9 is the third generation of gene-editing technology based on the principle of DNA recombination and repair. This technology has been successfully used in the genome modification from lower animals to mammals, such as zebrafish and rodents. The application of CRISPR/Cas9 technology in AD research might contribute to the discovery of pathological mechanism and the methodologic development of AD treatment. This article introduced the technology of CRISPR/Cas9 and reviewed the progress on research and application of this technology in AD, including the construction of pathological models, the screening of pathogenic risk factors, and the research for the therapeutic targets and the targeted therapy.

Communication Author：HUANG Han-Chang , Email:hanchang@buu.edu.cn