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

Journal of Advanced Research (Sep 2022)

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

Affiliations

Ning Guo: 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
Ji-Bin Liu: Institute of Oncology, Affiliated Tumor Hospital of Nantong University, Nantong 226631, China
Wen Li: 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; Corresponding authors at: Institute of Oncology, Affiliated Tumor Hospital of Nantong University, Nantong 226631, China (Y. Ma). 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 (D. Fu).
Yu-Shui Ma: 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; Corresponding authors at: Institute of Oncology, Affiliated Tumor Hospital of Nantong University, Nantong 226631, China (Y. Ma). 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 (D. Fu).
Da Fu: 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; Corresponding authors at: Institute of Oncology, Affiliated Tumor Hospital of Nantong University, Nantong 226631, China (Y. Ma). 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 (D. Fu).

Journal volume & issue: Vol. 40
pp. 135 – 152

Abstract

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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.

Published in Journal of Advanced Research

ISSN: 2090-1232 (Print); 2090-1224 (Online)
Publisher: Elsevier
Country of publisher: Netherlands
LCC subjects: Medicine: Medicine (General); Science: Science (General)
Website: http://www.journals.elsevier.com/journal-of-advanced-research/

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