Advances in Off-Target Detection for CRISPR-Based Genome Editing

Hum Gene Ther. 2023 Feb;34(3-4):112-128. doi: 10.1089/hum.2022.198.

Abstract

The CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)-based genome editing system exhibits marked potential for both gene editing and gene therapy, and its continuous improvement contributes to its great clinical potential. However, the largest hindrance to its application in clinical practice is the presence of off-target effects (OTEs). Thus, in addition to continuous optimization of the CRISPR system to reduce and eventually eliminate OTEs, further development of unbiased genome-wide detection of OTEs is key for its successful clinical application. This article summarizes detection strategies for OTEs of different CRISPR systems, to provide detailed guidance for the detection of OTEs in CRISPR-based genome editing.

Keywords: CRISPR/Cas nucleases; base editors; genome editing; off-target detection; prime editors.

Publication types

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

MeSH terms

  • CRISPR-Cas Systems*
  • Gene Editing*
  • Genetic Therapy
  • Genome