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.
