Cell BiologybioRxivPreprint
In vivo base editing via single myotrophic adeno-associated viruses in dystrophic mouse muscle and satellite cells
Kuan‐Hung Lin, Amy Lam, Samual Ooijen…·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.
DOI 10.64898/2026.05.09.721064