Gene Therapy Restores Hearing in Children With Hereditary Deafness

A study that was co-led by investigators from Mass Eye and Ear, a member of mass General Brigham, demonstrated the effectiveness of gene therapy towards restoring hearing function for children that suffer from hereditary deafness. In a trial of six children that took place at the Eye & ENT Hospital of Fudan University located in Shanghai, China, researchers found that novel gene therapy was considered to be effective treatment for patients with a specific form of autosomal recessive deafness, which is caused by mutation of the otoferlin (OTOF) gene, also known as DFNB9. With their first patient that was treated in December 2022, this research represented the first human clinical trial to administer gene therapy for treating this condition with the most patients treated and longest follow-up to date. Their results were later published in “The Lancet” on January 24.   For more information, click here.