Off-target analysis of CRISPR/Cas9-edited rat model

A rat model of Cockayne syndrome is generated by using CRISPR/Cas9, a nonsense mutation is introduced into CSB gene. Despite widespread use of CRISPR, frequency and impact of Cas9-mediated off-targets in modified human cells and rodents have always been a concern. We used whole genome sequencing to comprehensively access on- and off-target mutations in the rat model, and we found that CRISPR/Cas9-based gene editing was active on the expected genomic sites without producing off-target modifications in other functional regions of the rat genome.