Gene therapy in both ears proves a success for deaf children

Five children with congenital deafness have had their hearing significantly improved after receiving gene therapy, the Eye, Ear, Nose and Throat Hospital of Fudan University said on Wednesday.

The children, between the ages of 1 and 11 all suffered hereditary deafness due to a genetic mutation.

Around 30,000 children are born deaf in China each year with 60 percent of cases related to genetic defects, seriously impacting their language, cognition and intelligence development. There is no effective medication so far.

Gene therapy is a promising treatment for hereditary deafness, taking effect by injecting properly functioning genes directly into the inner ear.

Dr Shu Yilai led his team at the hospital to develop a genetic medicine targeting deafness due to the mutation with a precise and minimally invasive drug delivery route and equipment.

Since December 2022, six children have received an injection to one ear. The first, who has been followed for a year and five months, has been able to develop proper daily communication.

To achieve a bigger benefit to patients and help them regain their hearing and build 3D and accurate auditory localization, doctors started gene therapy on patients' both ears in July 2023.

The world's first gene therapy on deafness genes in both ears has testified to the safety and effects of gene therapy in congenitally deaf people and greatly boosts its development, showing a strong potential of gene therapy in congenital deafness treatment, experts said.

The research was published by world-leading journal Nature Medicine.