Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy

Man-Ling Zhang; Hong-Bin Li; Yong Jin

doi:10.3389/fgene.2024.1364742

Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy

Front Genet. 2024 Apr 11:15:1364742. doi: 10.3389/fgene.2024.1364742. eCollection 2024.

Authors

Man-Ling Zhang^{1

2}, Hong-Bin Li^{1

2}, Yong Jin^{1

2}

Affiliations

¹ Department of Rheumatology and Immunology, The Affiliated Hospital of Inner Mongolia Medical University, Hohhot, China.
² Inner Mongolia Key Laboratory for Pathogenesis and Diagnosis of Rheumatic and Autoimmune Diseases, The Affiliated Hospital of Inner Mongolia Medical University, Hohhot, China.

Abstract

The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) mediated Cas9 nuclease system has been extensively used for genome editing and gene modification in eukaryotic cells. CRISPR/Cas9 technology holds great potential for various applications, including the correction of genetic defects or mutations within the human genome. The application of CRISPR/Cas9 genome editing system in human disease research is anticipated to solve a multitude of intricate molecular biology challenges encountered in life science research. Here, we review the fundamental principles underlying CRISPR/Cas9 technology and its recent application in neurodegenerative diseases, cardiovascular diseases, autoimmune related diseases, and cancer, focusing on the disease modeling and gene therapy potential of CRISPR/Cas9 in these diseases. Finally, we provide an overview of the limitations and future prospects associated with employing CRISPR/Cas9 technology for diseases study and treatment.

Keywords: CRISPR/Cas9; autoimmune-related diseases; cancer; cardiovascular diseases; diseases modeling; gene therapy; neurodegenerative diseases.

Publication types

Review

Grants and funding

The author(s) declare that financial support was received for the research, authorship, and/or publication of this article. This work was supported by the National Natural Science Foundation of China (82260138, 82260327); Natural Science Foundation of Inner Mongolia (2022QN08022); The Scientific Research Project of Colleges and Universities, Inner Mongolia Department of Education (NJZZ22678, NJZZ23014); The Inner Mongolia Health Commission’s Medical and Health Science and Technology Program (Class A) (202201267).