The power and the promise of CRISPR/Cas9 genome editing for clinical application with gene therapy

Ning Guo; Ji-Bin Liu; Wen Li; Yu-Shui Ma; Da Fu

doi:10.1016/j.jare.2021.11.018

The power and the promise of CRISPR/Cas9 genome editing for clinical application with gene therapy

J Adv Res. 2022 Sep:40:135-152. doi: 10.1016/j.jare.2021.11.018. Epub 2021 Dec 4.

Authors

Ning Guo¹, Ji-Bin Liu², Wen Li³, Yu-Shui Ma⁴, Da Fu⁵

Affiliations

¹ National Engineering Laboratory for Deep Process of Rice and Byproducts, College of Food Science and Engineering, Central South University of Forestry and Technology, Changsha 410004, Hunan, China.
² Institute of Oncology, Affiliated Tumor Hospital of Nantong University, Nantong 226631, China.
³ National Engineering Laboratory for Deep Process of Rice and Byproducts, College of Food Science and Engineering, Central South University of Forestry and Technology, Changsha 410004, Hunan, China. Electronic address: liwendream@163.com.
⁴ Institute of Oncology, Affiliated Tumor Hospital of Nantong University, Nantong 226631, China; Department of Nuclear Medicine, Shanghai Tenth People's Hospital, Tongji University School of Medicine, Shanghai 200072, China. Electronic address: mayushui@tongji.edu.cn.
⁵ National Engineering Laboratory for Deep Process of Rice and Byproducts, College of Food Science and Engineering, Central South University of Forestry and Technology, Changsha 410004, Hunan, China; General Surgery, Ruijin Hospital & Institute of Pancreatic Diseases, Shanghai Jiaotong University School of Medicine, Shanghai 200025, China. Electronic address: fu800da900@126.com.

Abstract

Background: Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is derived from the bacterial innate immune system and engineered as a robust gene-editing tool. Due to the higher specificity and efficiency of CRISPR/Cas9, it has been widely applied to many genetic and non-genetic disease, including cancers, genetic hemolytic diseases, acquired immunodeficiency syndrome, cardiovascular diseases, ocular diseases, and neurodegenerative diseases, and some X-linked diseases. Furthermore, in terms of the therapeutic strategy of cancers, many researchers used the CRISPR/Cas9 technique to cure or alleviate cancers through different approaches, such as gene therapy and immune therapy.

Aim of review: Here, we conclude the recent application and clinical trials of CRISPR/Cas9 in non-cancerous diseases and cancers and pointed out some of the problems to be solved.

Key scientific concepts of review: CRISPR/Cas9, derived from the microbial innate immune system, is developed as a robust gene-editing tool and has been applied widely. Due to its high accuracy and efficiency, CRISPR/Cas9 techniques may provide a great chance to treat some gene-related diseases by disrupting, inserting, correcting, replacing, or blocking genes for clinical application with gene therapy.

Keywords: CRISPR/Cas9; Cancer; Clinical application; Gene therapy; Immune therapy.

Publication types

Review
Research Support, Non-U.S. Gov't

MeSH terms

CRISPR-Cas Systems / genetics
Cardiovascular Diseases* / genetics
Gene Editing / methods
Genetic Therapy
Humans
Neoplasms* / genetics
Neoplasms* / therapy