Compact adenine base editors to enable therapeutic rescue of Duchenne muscular dystrophy
Kartik Lakshmi Rallapalli, Joanne M. L. Ho, Isabel Nocedal…·Cristina N. Butterfield
This paper develops compact adenine base editors for in vivo gene editing, demonstrating their application in Duchenne muscular dystrophy by enabling exon skipping through targeted splice-site disruption.