Open access
Sep 2026
Engineering suppressor tRNAs for effective treatment of Duchenne muscular dystrophy
It is shown that systemic delivery of these sup-tRNAs restores full-length dystrophin and recovers muscle strength and motor function in mice at lower doses than AAV microdystrophin therapies, establishing sup-tRNAs as a promising therapeutic platform for DMD and other disorders.
Julien Oury, Ying-Hsin Chen, Sean Mcfarland et al.
· Science Advances · 0 citations