#gene editing
Sep 2026
Development of an AAV-Encoded Adenine Base Editor for Duchenne Muscular Dystrophy.
It is suggested that even modest levels of base editing-mediated exon skipping may ameliorate the DMD cardiac phenotype toward a BMD-like state, supporting the translational potential of this dual rAAV base editing approach for DMD cardiomyopathy.
Ina Luksch, Christine M. Poch, Aylin Mayer et al.
· Human Gene Therapy · 0 citations