Scientists for the first time have achieved a historic breakthrough, restoring hearing in people born deaf by correcting one defective gene.
The groundbreaking treatment, delivered through viral gene therapy, is being hailed as a historic leap in medicine and a life-changing breakthrough for millions with hereditary hearing loss.
The therapy works by delivering a corrected version of the OTOF gene directly into the inner ear.
This gene is responsible for producing otoferlin, a crucial protein that allows sound signals to travel from the ear to the brain.
Without it, patients are unable to process sound.
After treatment, participants who had never heard a sound before began to recognize voices, music, and everyday noises.
In clinical trials, patients ranging from one to 24 years old experienced remarkable improvements.
Within just a month of receiving the therapy, most participants regained partial hearing.
Six months later, all ten participants showed significant progress.
The average sound level they could detect improved from 106 decibels a level of profound deafness to 52 decibels, considered within the range of functional hearing.
“This is a huge step forward in the genetic treatment of deafness, one that can be life-changing for children and adults,” said Maoli Duan, consultant and docent at Karolinska Institutet in Sweden and one of the study’s corresponding authors.
Researchers used an adeno-associated virus (AAV), a harmless viral carrier, to deliver the corrected OTOF gene into the cochlea.
The procedure involved a single injection through the round window, a delicate membrane at the base of the inner ear.
Moreover, scientists observed that younger patients responded more quickly, highlighting the brain’s ability to adapt during early development.
However, adults in the trial also reported noticeable improvements, proving the therapy’s broad potential.
Beyond restoring hearing, the study highlights the vast possibilities of gene therapy for other inherited conditions.
Instead of managing genetic disorders, this approach aims to cure them at the source.
The trial was a collaboration between Karolinska Institutet in Sweden, Zhongda Hospital in China, and several universities.
It was partly funded by Otovia Therapeutics Inc., the company behind the therapy.
The breakthrough signals a future where hereditary deafness could become preventable.
