New gene therapy shows potential for restoring hearing loss

IANS July 5, 2025 498 views

A groundbreaking gene therapy has successfully restored hearing in patients with genetic deafness. The treatment, tested in China and Sweden, used a synthetic virus to deliver a functional OTOF gene. Younger patients showed the most dramatic improvements, with some regaining near-normal hearing within months. Researchers confirm the therapy was safe and effective across all age groups.

"This is a huge step forward in the genetic treatment of deafness, one that can be life-changing for children and adults." – Maoli Duan, Karolinska Institutet
New gene therapy shows potential for restoring hearing loss
New Delhi, July 5: In a significant scientific breakthrough, an international team of researchers has developed a novel gene therapy that can improve hearing in children and adults with congenital deafness or severe hearing impairment.

Key Points

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Gene therapy restored hearing in patients with OTOF mutations

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Treatment involved AAV-delivered OTOF gene via cochlear injection

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Young patients aged 5-8 responded best to therapy

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Results showed safe and lasting hearing improvement in 6 months

In the study, Swedish and Chinese used gene therapy and improved hearing in 10 patients, and the treatment was well-tolerated.

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

The study, published in the journal Nature Medicine, comprised 10 patients between the ages of 1 and 24 at five hospitals in China, all of whom had a genetic form of deafness or severe hearing impairment caused by mutations in a gene called OTOF.

These mutations cause a deficiency of the protein otoferlin, which plays a critical part in transmitting auditory signals from the ear to the brain.

The gene therapy involved using a synthetic adeno-associated virus (AAV) to deliver a functional version of the OTOF gene to the inner ear via a single injection through a membrane at the base of the cochlea called the round window.

The gene therapy worked rapidly and helped the majority of the patients to recover some hearing in just a month.

A six-month follow-up showed considerable hearing improvement in all participants, with the average volume of perceptible sound improving from 106 decibels to 52.

The younger patients, especially those between the ages of five and eight, responded best to the treatment.

One of the participants, a seven-year-old girl, quickly recovered almost all her hearing and was able to hold daily conversations with her mother four months afterward. However, the therapy also proved effective in adults.

“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,” Duan said.

“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,” the expert said.

Importantly, the results also show that the treatment was safe and well-tolerated.

Reader Comments

R
Rohit P
Great breakthrough but I wonder about the cost. Most Indian families won't be able to afford such advanced treatments. Hope our scientists at AIIMS and other institutes can develop more affordable versions.
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Arjun K
As someone working in biotech, I must say this is remarkable! But we need more Indian participation in such global research. Our CSIR and DBT should collaborate with these international teams.
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Sarah B
The part about younger patients responding better is interesting. This shows how important early intervention is. Indian parents should get their children screened for hearing issues as early as possible.
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Vikram M
While this is promising, I'm concerned about long-term effects. Gene therapy is still relatively new. We need more studies before making it widely available. Safety first!
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Kavya N
Imagine being able to hear your child say "Mummy" for the first time! 😭 This technology could bring so much joy to families. Hope Indian hospitals start clinical trials soon.

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Tags:
gene therapy hearing loss deafness treatment OTOF mutation Maoli Duan Karolinska Institutet