A compact, high-accuracy Cas9 with a dinucleotide PAM for in vivo genome editing
Key findings
Identified Nme2Cas9 (1082aa) from Neisseria meningitidis recognizing simple N4CC dinucleotide PAM with high target site density. All-in-one AAV delivery targeting Pcsk9 in adult mouse liver achieved efficient genome editing and reduced serum cholesterol with exceptionally high specificity, expanding options for compact Cas9 orthologs.