bioRxiv
Compact type II-C Cas9 nucleases with expanded PAM access and high fidelity for therapeutic genome editing
Preprint: experiments in cellsInterventions
Abstract
Compact type II-C Cas9 nucleases are attractive for therapeutic genome editing because their small size enables packaging into adeno-associated viral (AAV) vectors, and their extended protospacer-adjacent motifs (PAMs) reduce off-target cleavage while expanding targeting scope.
The paper
King Abdullah University of Science and Technology
bioRxiv, 20 Aug 2026, CC BY, Preprint, not peer-reviewed