07/07/2025
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Researchers say this type of treatment for deafness is ‘just the beginning.’
A single injection of a breakthrough gene therapy may soon be able to reverse hearing loss within weeks, according to new research from Sweden’s Karolinska Institutet.
The therapy was tested in a small clinical trial involving children and adults with congenital deafness or severe hearing impairment caused by mutations in the OTOF gene. This gene is responsible for producing otoferlin, a protein essential for transmitting sound signals from the ear to the brain. Without it, individuals are left unable to process sound despite having otherwise healthy hearing structures.
In the trial, a harmless, synthetic version of the adeno-associated virus was used to deliver a healthy copy of the OTOF gene directly into the inner ear via a single injection. The therapy led to rapid and significant improvements. Within just one month, most participants began to recover their hearing, and by six months, all ten showed measurable gains.
On average, the volume of perceptible sound improved dramatically - from 106 decibels (near total deafness) to 52 decibels (mild hearing loss). Children between the ages of five and eight responded the best, with one seven-year-old girl regaining nearly full hearing and holding regular conversations just four months post-treatment.
Importantly, the study, published in Nature Medicine, found that the treatment was both safe and well-tolerated. No serious adverse effects were reported during the six- to twelve-month follow-up period, with the most common reaction being a temporary reduction in neutrophils, a type of white blood cell. This marks the first time this gene therapy approach has been tested in teenagers and adults, expanding the potential reach of the treatment.
Dr. Maoli Duan, one of the lead researchers, emphasized the life-changing potential of the therapy and noted that OTOF is just the beginning. The team is now exploring treatments for other common genetic causes of deafness, including GJB2 and TMC1. While these present more complex challenges, early animal studies have been promising, and researchers are hopeful that a wide range of genetic hearing disorders will eventually become treatable.