CRISPR/Cas9基因编辑技术在疾病治疗中的应用与展望

doi:10.19586/j.2095-2341.2024.0148

生物技术进展 ›› 2025, Vol. 15 ›› Issue (1): 35-42.DOI: 10.19586/j.2095-2341.2024.0148

CRISPR/Cas9基因编辑技术在疾病治疗中的应用与展望

李仪扬¹(), 周执政²(), 王淑菲², 刘博雅², 刘宇飞², 李孝彦², 隋宏书³(), 刘东巍²()

^1.山东第一医科大学实验动物学院，济南 250000
^2.山东第一医科大学临床与基础医学院，济南 250000
^3.山东第一医科大学临床与基础医学院组织学与胚胎学系，济南 250000

收稿日期:2024-09-10 接受日期:2024-11-01 出版日期:2025-01-25 发布日期:2025-03-07
通讯作者: 隋宏书,刘东巍
作者简介:李仪扬 E-mail： 2916536631@qq.com；
周执政 E-mail：15615571667@163.com第一联系人： §为本文共同第一作者。
基金资助:
国家自然科学基金项目(81670004);山东第一医科大学教育教学改革项目(JXGGYJ-22243253);山东省研究生优质课程建设项目(SDYKC21148)

Application and Prospect of CRISPR/Cas9 Gene Editing Technology in Disease Treatment

Yiyang LI¹(), Zhizheng ZHOU²(), Shufei WANG², Boya LIU², Yufei LIU², Xiaoyan LI², Hongshu SUI³(), Dongwei LIU²()

^1.College of Laboratory Animals，Shandong First Medical University，Jinan 250000，China
^2.College of Clinical and Basic Medicine，Shandong First Medical University，Jinan 250000，China
^3.Department of Histology and Embryology，College of Clinical and Basic Medicine，Shandong First Medical University，Jinan 250000，China

Received:2024-09-10 Accepted:2024-11-01 Online:2025-01-25 Published:2025-03-07
Contact: Hongshu SUI,Dongwei LIU

摘要/Abstract

摘要：

成簇规律间隔短回文重复序列相关蛋白（clustered regularly interspaced short palindromic repeats-CRISPR-associated proteins, CRISPR）系统是原核生物的一种获得性免疫系统，基于细菌免疫系统CRISPR改造发展而来的CRISPR/Cas9系统正在改变着生物学和基础医学研究，是现有基因编辑和基因修饰技术中效率最高、最简便、成本最低的技术之一。然而，目前缺乏在体内将CRISPR系统有效递送到患病细胞的策略，具有靶标识别功能的非病毒载体可能是未来研究的重点，疾病发病引起的病理和生理变化有望作为靶向递送或基因编辑靶标的识别因素。概述了现有的基因编辑工具以及CRISPR/Cas9系统的优势，总结了CRISPR/Cas9在治疗领域的应用进展，并讨论了在CRISPR/Cas9介导的治疗中所遇到的问题和挑战，以期能够促进CRISPR/Cas9治疗技术的进步，并为治疗其他复杂疾病提供新的视角。

关键词: CRISPR/Cas9, 基因编辑, 基因治疗

Abstract:

The clustered regularly interspaced short palindromic repeats associated proteins （CRISPR) system is an acquired immune system of prokaryotes. The CRISPR/Cas9 system， developed based on the bacterial immune system CRISPR， is changing biology and basic medical research， and is one of the most efficient， simplest and cost-effective gene editing and modification technologies available. However， there is currently a lack of strategies for effectively delivering CRISPR systems to diseased cells in vivo， and non-viral vectors with target recognition capabilities may be the focus of future research， with pathological and physiological changes caused by disease onset promising as identifying factors for targeted delivery or gene editing targets. This article provided an overview of existing gene editing tools and the advantages of the CRISPR/Cas9 system， summarized the application of CRISPR/Cas9 in the field of therapy， and discussed the problems and challenges encountered in CRISPR/Cas9-mediated therapy， in order to promote the advancement of CRISPR/Cas9 therapeutical technology and provide new perspectives for treating other complex diseases.

Key words: CRISPR/Cas9, gene editing, gene therapy

中图分类号:

李仪扬, 周执政, 王淑菲, 刘博雅, 刘宇飞, 李孝彦, 隋宏书, 刘东巍. CRISPR/Cas9基因编辑技术在疾病治疗中的应用与展望[J]. 生物技术进展, 2025, 15(1): 35-42.

Yiyang LI, Zhizheng ZHOU, Shufei WANG, Boya LIU, Yufei LIU, Xiaoyan LI, Hongshu SUI, Dongwei LIU. Application and Prospect of CRISPR/Cas9 Gene Editing Technology in Disease Treatment[J]. Current Biotechnology, 2025, 15(1): 35-42.

图/表 1

参考文献 47

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CRISPR/Cas9基因编辑技术在疾病治疗中的应用与展望

Application and Prospect of CRISPR/Cas9 Gene Editing Technology in Disease Treatment

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