RNA trans-splicing treatment for Duchenne muscular dystrophy
Ryan H. Hsu, Claire E. Williams, Geraldine Maier, Miriam Gullo, Karen Lettieri, Cindy J. Alvarez, Kip J. Hermann, Stella Kramer, Satchidananda Panda, Lukas C. Bachmann, 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.
bioRxiv · Sep 16, 2026 · PreprintMolecular Biology