A New Gene Therapy May Restore Hearing In Weeks, Give People Born Deaf A Chance To Hear Again

The treatment targets a specific type of inherited deafness caused by mutations in a gene called OTOF.

04 Apr 2026, 11:58 · www.ndtv.com

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Gene therapy restored hearing in 10 deaf patients by delivering a healthy OTOF gene
Patients' hearing improved from profound deafness to normal conversation levels
Most participants noticed hearing gains within one month, stable by six months

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A new gene therapy is giving people born deaf the chance to hear for the first time. In a small trial, scientists used a single injection into the inner ear to deliver a working copy of a critical hearing gene. Ten patients between the ages one and 24 showed clear improvement in hearing, with some starting to respond to sounds within a month. If confirmed in larger studies, this could mark a turning point for tens of thousands of people with a genetic form of deafness, offering them hope. The study was conducted by researchers at Karolinska Institutet, who worked with hospitals and universities in China. The findings were published in the journal Nature Medicine.

Maoli Duan, consultant and docent at the Department of Clinical Science, Intervention and Technology, Karolinska Institutet, Sweden, and one of the study's corresponding authors, said, "This is a huge step forward in the genetic treatment of deafness, one that can be life-changing for children and adults."

What The New Therapy Does

The treatment targets a specific type of inherited deafness caused by mutations in a gene called OTOF. Normally, this gene instructs the body to make a protein called otoferlin, which is essential for passing sound signals from the inner ear to the brain. When the OTOF gene is faulty, this 'messenger' protein is missing or broken, so even though the inner ear may pick up vibrations, the brain never receives them. As a result, a person is born profoundly deaf or loses almost all hearing early in life.

To fix this, researchers used a synthetic virus known as an adeno-associated virus (AAV) as a tiny biological delivery truck. They loaded the virus with a healthy version of the OTOF gene and injected it once into the inner ear through a small membrane at the base of the cochlea. The virus then shuttles the functional gene into key cells, allowing them to produce normal otoferlin and restart the pathway that sends sound to the brain.

Fast Improvements In Hearing

In this study, all ten patients, treated at five hospitals in China, experienced measurable hearing improvement after the single injection. On average, the level of sound they could detect changed from about 106 decibels (similar to a very loud chainsaw or rock concert) to around 52 decibels (about the level of normal conversation). That drop means they went from hearing nothing at all, even very loud sounds, to being able to pick up everyday speech and environmental noises.

Most participants began to notice some response to sound within one month and by six months, the results were stable and clear. One of the most striking cases was a seven-year-old girl whose hearing improved so much that she was able to have normal conversations with her mother just four months after treatment. Even though teenagers and adults also showed meaningful gains, most of the improvements were seen in younger children, especially those between five and eight years old.

Dr. Duan said, "Smaller studies in China have previously shown positive results in children, but this is the first time that the method has been tested in teenagers and adults, too. Hearing was greatly improved in many of the participants, which can have a profound effect on their life quality. We will now be following these patients to see how lasting the effect is."

Safety And Possible Side Effects

The therapy was reported to be safe and well-tolerated during the follow-up period of six to twelve months. The most common side effect was a temporary decrease in neutrophils, a type of white blood cell that helps fight infections. However, this did not lead to serious illness or require stopping the treatment. No major adverse effects were directly linked to the gene therapy itself. However, this is an early-stage study, and the long-term effects are not yet fully known. Doctors will need to keep monitoring patients for years to see whether the restored hearing remains stable.

Why "OTOF Is Just The Beginning"

The researchers emphasise that this success with OTOF is only the first step in a broader effort. Genetic deafness can be caused by mutations in many different genes, such as GJB2 and TMC1, which are more common than OTOF-related deafness. These genes are more complex to treat, but animal studies so far have shown promising results, and teams are already working on similar gene therapies tailored to them.

Dr. Duan said, "OTOF is just the beginning. We and other researchers are expanding our work to other, more common genes that cause deafness, such as GJB2 and TMC1. These are more complicated to treat, but animal studies have so far returned promising results. We are confident that patients with different kinds of genetic deafness will one day be able to receive treatment."

Disclaimer: This content including advice provides generic information only. It is in no way a substitute for a qualified medical opinion. Always consult a specialist or your own doctor for more information. NDTV does not claim responsibility for this information.

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