CRISPR/Cas9 is poised to become the gene editing tool of choice in clinical contexts, but the full spectrum of on-target damage remains unknown. Thus far, exploration of Cas9 induced genetic alteratio
RNA-guided genome editing with the CRISPR-Cas9 system has great potential for basic and clinical research, but the determinants of targeting specificity and the extent of off-target cleavage remain in
The CRISPR system is widely used in genome editing for biomedical research. Here, using either dual paired Cas9D10A nickases or paired Cas9 nuclease we characterise unintended larger deletions at on-t
The aim of this study is to assess the safety expression profile of different CRISPR/Cas9 gene-disruption strategies for the upregulation of gamma-globin, and subsequent fetal hemoglobin (HbF). KLF1,