Off-target effects and the feasibility for AAV-mediated delivery are the major barriers impeding the clinical in vivo application of base editors. Here, the authors report the small size AAV-deliverable Cas9-ABE variant that has improved on-target editing efficiency and reduced RNA-off target footprint.
- Minh Thuan Nguyen Tran
- Mohd Khairul Nizam Mohd Khalid
- Alex W. Hewitt