Posted innews Otorhinolaryngology Pediatrics
DB-OTO Restores and Sustains Hearing in Otoferlin-Deficient Mice: A Hair-Cell–Targeted Dual-AAV Strategy Advances to Clinical Testing
DB-OTO, a hair-cell-specific dual-AAV gene therapy encoding full-length human otoferlin, achieved dose-dependent and sustained auditory restoration in OTOF-deficient mice, supporting an ongoing Phase I/II pediatric trial.