Molecular BiologybioRxivPreprint
RNA trans-splicing treatment for Duchenne muscular dystrophy
Ryan H. Hsu, Claire E. Williams, Geraldine Maier…·Samuel L. Pfaff
The paper develops a multi-vector RNA End Joining system for Duchenne muscular dystrophy that enables efficient expression of large therapeutic proteins with minimal off-target effects.
DOI 10.64898/2026.09.13.751281