The gene therapy involved using a synthetic adeno-associated virus (AAV) to deliver a functional version of the OTOF gene to the inner ear via a single injection through a membrane at the base of the cochlea called the round window.
The effect of the gene therapy was rapid and the majority of the patients recovered some hearing after just one month. A six-month follow-up showed considerable hearing improvement in all participants, the average volume of perceptible sound improving from 106 decibels to 52.
The younger patients, especially those between the ages of five and eight, responded best to the treatment. One of the participants, a seven-year-old girl, quickly recovered almost all her hearing and was able to hold daily conversations with her mother four months afterwards. However, the therapy also proved effective in adults.
Deafness reversed: Single injection brings hearing back within weeks
— Professor Erwin Loh (@erwinloh) July 5, 2025
A cutting-edge gene therapy has significantly restored hearing in children and adults with congenital deafness, showing dramatic results just one month after a single injection.
Researchers used a virus to… pic.twitter.com/O8hrV1nflx