A gene therapy has shown remarkable success in reversing congenital hearing loss with just a single injection, according to new research published in Nature Medicine.
Conducted by scientists at Sweden’s Karolinska Institutet, the clinical trial demonstrated significant hearing restoration in both children and adults with a genetic form of deafness.
The therapy targets mutations in the OTOF gene, which result in the absence of otoferlin, a protein essential for transmitting sound signals from the ear to the brain. Using a harmless synthetic virus, researchers delivered a healthy copy of the OTOF gene directly into the inner ear of 10 participants, ranging from young children to adults.
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In just one month, most participants showed measurable improvements, with hearing thresholds improving dramatically—from an average of 106 decibels (severe hearing loss) to 52 decibels (mild impairment) after six months. Children between ages 5 and 8 responded best to the treatment. In one standout case, a 7-year-old girl regained nearly complete hearing and was able to engage in daily conversations within four months.
“This is the first time the method has been tested in teenagers and adults,” said Dr. Maoli Duan, co-author of the study. “Hearing was greatly improved in many of the participants, which can have a profound effect on their quality of life.”
Importantly, the treatment was found to be safe and well-tolerated. No serious side effects were reported during a 6 to 12-month follow-up period, with the most common reaction being a temporary reduction in neutrophils, a type of white blood cell.
“We are already working on therapies targeting other common deafness genes like GJB2 and TMC1,” said Dr. Duan. “While these may be more complex to treat, early animal studies are promising.”
