Multicentre gene therapy for OTOF -related deafness followed up to 2.5 years - Nature

A multicentre trial of AAV1-hOTOF gene therapy in 42 participants (ages 0.8–32.3 years) with autosomal recessive DFNB9 deafness found the treatment to be safe and tolerated, and reported improvements in hearing thresholds and speech perception. Younger participants and those with residual cochlear function (measured by distortion product otoacoustic emissions) tended to have better outcomes. The findings support continued development of AAV-based gene replacement approaches for inherited hearing loss and illustrate broader clinical progress in gene therapies for genetic neurological disorders.