Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

Levy et al. (2020). Nat Biomed Eng DOI: 10.1038/s41551-019-0501-5 Citations: 510

Parts used

BE3.9max N-term BE3.9max C-term SaCBE C-term SaCBE-KKH C-term

Key findings

Developed split-intein dual-AAV system for in vivo base editing, achieving therapeutically relevant efficiencies in mouse brain (59%), liver (38%), retina (38%), heart (20%), and skeletal muscle (9%). Demonstrated disease correction in Niemann-Pick type C mice.