Iterative engineering of a compact Cas9 ortholog for in vivo gene editing via single AAV delivery
Alessandro Barenghi, Jamie A. Hackett, Neil E. Humphreys, James A. Sawitzke
The paper engineers a compact Cas9 ortholog, enCme2, for efficient in vivo gene editing via single AAV delivery, showing high specificity and minimal off-target activity.
bioRxiv · Jan 12, 2026 · PreprintMolecular Biology