0
0
A single injection of gene therapy has restored hearing in children and adults born with severe genetic deafness, according to a groundbreaking international study published in Nature Medicine. The clinical trial, led by researchers at Sweden’s Karolinska Institutet in collaboration with several Chinese hospitals and universities, marks a significant advance in the treatment of hereditary hearing loss.
The therapy targets patients with mutations in the OTOF gene, which is responsible for producing otoferlin—a protein crucial for transmitting sound signals from the ear to the brain. Researchers used a harmless adeno-associated virus (AAV) to deliver a healthy copy of the OTOF gene directly into the inner ear through a single injection at the base of the cochlea.
Dramatic Results in Both Children and Adults
All ten participants, aged 1 to 24, experienced noticeable improvement in hearing. Most began to recover some hearing within a month, and after six months, the average sound level they could detect improved from 106 decibels to 52 decibels—a major step toward functional hearing. The therapy proved especially effective in children aged five to eight, with one seven-year-old girl regaining almost full hearing and able to hold daily conversations just four months after treatment. Adults in the study also benefited significantly.
Dr. Maoli Duan of Karolinska Institutet, a corresponding author of the study, called the results “a huge step forward in the genetic treatment of deafness, one that can be life-changing for children and adults”.
The treatment was well-tolerated, with no serious adverse reactions reported during the six- to twelve-month follow-up. The most common side effect was a temporary reduction in neutrophils, a type of white blood cell. Researchers are now expanding their work to target other genes responsible for more common forms of genetic deafness, such as GJB2 and TMC1, with early animal studies showing promise.
“OTOF is just the beginning. We are confident that patients with different kinds of genetic deafness will one day be able to receive treatment,” said Dr. Duan.
Disclaimer: This article is based on early-stage clinical research. While results are promising, gene therapy for hearing loss is not yet widely available and further studies are needed to confirm long-term safety and effectiveness. Readers should consult qualified healthcare professionals for medical advice or treatment options.
