Kavli Affiliate: Samuel Pfaff | Authors: 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 and Samuel L. Pfaff | Summary: Duchenne muscular dystrophy (DMD) is a fatal disorder caused by dystrophin mutations, leading to progressive muscle degeneration and cardiac failure. […]
Continue.. RNA trans-splicing treatment for Duchenne muscular dystrophy