PRJNA834963
Off-target cleavage analysis of AAVS1-specific Cas9 nucleases
In this study, we show that fully viral gene-deleted adenoviral vector delivery of donors prone to HMEJ and HR, together with matched CRISPR-Cas9 complexes, achieves targeted chromosomal integration of large genetic payloads in human cells. This was exploit for the genetic complementation of defective DMD genes in human muscle progenitor cells.