Gene Therapy

Gene Therapy Restores Hearing in Recent Trial

Gene therapy is showing promise in restoring hearing for children born deaf due to genetic conditions. Recent research published in Nature Medicine reports on a trial involving five children with autosomal recessive deafness 9 (DFNB9). This condition is caused by mutations in the OTOF gene, which leads to a lack of the protein necessary for hearing.

In a trial at the Eye & ENT Hospital of Fudan University in Shanghai, China six children were treated with a new gene therapy. The researchers,  co-led by researchers from Mass Eye and Ear, demonstrated that a gene therapy restores hearing in children suffering from hereditary deafness. The children treated had a specific form of autosomal recessive deafness caused by a specific gene. This gene mutation causes a failure to produce a functioning otoferlin protein, which is needed to transmit sound signals from the ear to the brain.

The first patient was treated in December 2022. Their results were published January 24 in The Lancet.