Breakthrough Gene Therapy Enhances Hearing in Deaf Children and Adults

Significant Advancement in Hearing Restoration

New Delhi, July 5: An international collaboration of researchers has achieved a remarkable milestone in medical science by creating a new gene therapy aimed at enhancing hearing capabilities in individuals suffering from congenital deafness or severe hearing loss.

The research, conducted by teams from Sweden and China, successfully utilized gene therapy to improve auditory function in 10 patients, with the treatment being well-received.

Maoli Duan, a consultant and docent at the Department of Clinical Science, Intervention and Technology at Karolinska Institutet in Sweden, remarked, “This represents a significant advancement in the genetic treatment of hearing loss, potentially transforming lives for both children and adults.”

Published in the journal Nature Medicine, the study involved 10 participants aged between 1 and 24, all of whom were treated at five hospitals in China. Each had a genetic form of hearing loss linked to mutations in the OTOF gene.

These genetic mutations lead to a lack of otoferlin protein, essential for transmitting sound signals from the ear to the brain.

The innovative gene therapy employed a synthetic adeno-associated virus (AAV) to introduce a functional OTOF gene into the inner ear through a single injection at the round window membrane located at the cochlea's base.

Results were promising, with most patients experiencing some hearing recovery within just a month.

A follow-up after six months indicated significant improvements in hearing across all participants, with the average sound perception level rising from 106 decibels to 52.

Younger patients, particularly those aged five to eight, showed the most favorable responses to the therapy.

One notable case involved a seven-year-old girl who regained nearly all her hearing and was able to engage in daily conversations with her mother just four months post-treatment. The therapy also yielded positive results in adult patients.

Duan noted, “While smaller studies in China have previously indicated positive outcomes in children, this is the first instance where the method has been applied to teenagers and adults.”

“The substantial improvement in hearing among many participants can significantly enhance their quality of life. We will continue to monitor these patients to assess the long-term effects,” the expert added.

Crucially, the findings confirm that the treatment is both safe and well-tolerated.