Re-administration of AAV-mediated gene therapy for OTOF-related deafness: a single-arm trial

Nature Medicine, Published online: 26 June 2026; doi:10.1038/s41591-026-04505-4

As part of a clinical trial, re-administration of AAV gene therapy in four patients with congenital deafness, who had previously received a first gene therapy dose and developed neutralizing antibodies, was safe and led to further hearing improvements.