Clustered Regularly Interspaced Short Palindromic Repeats and Clustered Regularly Interspaced Short Palindromic Repeats-Associated Protein 9 System: Factors Affecting Precision Gene Editing Efficiency and Optimization Strategies

Hum Gene Ther. 2023 Dec;34(23-24):1190-1203. doi: 10.1089/hum.2023.115. Epub 2023 Oct 17.

Abstract

The clustered regularly interspaced short palindromic repeat (CRISPR)-CRISPR-associated (Cas) system is a powerful genomic DNA editing tool. The increased applications of gene editing tools, including the CRISPR-Cas system, have contributed to recent advances in biological fields, such as genetic disease therapy, disease-associated gene screening and detection, and cancer therapy. However, the major limiting factor for the wide application of gene editing tools is gene editing efficiency. This review summarizes the recent advances in factors affecting the gene editing efficiency of the CRISPR-Cas9 system and the CRISPR-Cas9 system optimization strategies. The homology-directed repair efficiency-related signal pathways and the form and delivery method of the CRISPR-Cas9 system are the major factors that influence the repair efficiency of gene editing tools. Based on these influencing factors, several strategies have been developed to improve the repair efficiency of gene editing tools. This review provides novel insights for improving the repair efficiency of the CRISPR-Cas9 gene editing system, which may enable the development and improvement of gene editing tools.

Keywords: CRISPR-Cas9; gene delivery; gene editing efficiency; homology-directed repair.

Publication types

  • Review

MeSH terms

  • CRISPR-Associated Protein 9 / genetics
  • CRISPR-Cas Systems*
  • Gene Editing* / methods
  • Genetic Therapy
  • Recombinational DNA Repair

Substances

  • CRISPR-Associated Protein 9