In vivo base editing via single myotrophic adeno-associated viruses in dystrophic mouse muscle and satellite cells
Kuan‐Hung Lin, Amy Lam, Samual Ooijen, Madeline Maier, Gracia Kassis, Regan Ellis, Kathleen A. Messemer, Jennifer Martin, Ramzi Khairallah, Amy J. Wagers
This study demonstrates that single-vector adenine base editing via MyoAAV can recode a DMD-causing mutation in mice, with optimal therapeutic outcomes achieved in juvenile animals.
bioRxiv · May 10, 2026 · PreprintCell Biology