In vivo genome editing using Staphylococcus aureus Cas9

Ran et al. (2015). Nature DOI: 10.1038/nature14299 Citations: 3465

Key findings

First demonstration of SaCas9 for single-AAV in vivo genome editing. Achieved >40% indel frequency in mouse liver targeting Pcsk9. Established SaCas9 as viable alternative to SpCas9 for gene therapy.

Parts used

SaCas9 Nuclease