Groundbreaking Gene Therapy Successfully Restores Hearing in Congenitally Deaf Patients
A groundbreaking gene therapy trial has successfully restored hearing in all 10 patients with congenital deafness caused by mutations in the OTOF gene. This marks a historic breakthrough in treating genetic hearing loss. The study was spearheaded by researchers at Karolinska Institutet, in collaboration with Chinese hospitals, and represents the first successful gene therapy treatment for adult patients born completely deaf.
The innovative therapy employs a synthetic virus to deliver healthy copies of the OTOF gene directly to the inner ear. Patients, ranging in age from 1 to 24, showed dramatic improvements, with hearing levels improving from an average of 106 decibels to 52 decibels. Most remarkably, younger patients responded particularly well, with one seven-year-old regaining near-normal hearing and conversational ability within just one month.
“This is a huge step forward in the genetic treatment of deafness, one that can be life-changing for children and adults,” said Dr. Maoli Duan from Karolinska Institutet.
The treatment was well-tolerated with no serious side effects reported during 6-12 months of follow-up. Researchers are now expanding their work to target other common deafness genes, including GJB2 and TMC1. Animal studies are already showing promising results. The study was published in Nature Medicine on July 2, 2025.