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

Nature, Published online: 22 April 2026; doi:10.1038/s41586-026-10393-y

In a multicentre trial of AAV1-hOTOF gene therapy involving 42 participants aged 0.8–32.3 years with autosomal recessive deafness 9, treatment demonstrated safety, tolerability, and improved hearing and speech perception, with age and distortion product otoacoustic emissions associated with treatment outcomes.