In a major scientific breakthrough, researchers have successfully restored hearing in both children and adults with congenital deafness through a single gene therapy injection.
The international study, published in Nature Medicine, was led by scientists from the Karolinska Institutet in Sweden in collaboration with Chinese partners. The team used a harmless virus to deliver a functional copy of the OTOF gene into the inner ear. This gene produces otoferlin, a protein critical for transmitting sound from the ear to the brain.
The trial involved ten participants aged between 1 and 24, all of whom had OTOF-related deafness. Just one month after treatment, all participants experienced significant hearing improvements. In one striking case, a 7-year-old girl regained near-normal hearing. Overall, the average hearing threshold among participants improved from 106 decibels to 52 decibels.
The therapy proved most effective in younger children but also showed measurable benefits in adults. No serious side effects were reported during the trial.
This marks the first time gene therapy has been successfully used to treat OTOF-related hearing loss in both children and adults. Researchers say the findings open the door to developing similar treatments for more widespread genetic forms of deafness, such as those caused by mutations in the GJB2 and TMC1 genes.
Experts believe the study could signal the beginning of a new era in treating deafness, offering hope to millions of people affected by hearing loss worldwide.
