Gene Therapy Successfully Restores Hearing in Children with Hereditary Deafness

In a groundbreaking clinical trial, five out of six children showed significant hearing recovery and improved speech perception.

Gene therapy has successfully restored hearing in children suffering from hereditary deafness caused by mutations of the OTOF gene, according to a study co-led by investigators from Mass Eye and Ear and the Eye & ENT Hospital of Fudan University.
The trial, which is the first human clinical trial to administer gene therapy for treating this condition, observed six children over a 26-week period, with five demonstrating hearing recovery and significant improvements in speech perception.
The therapy involves the use of an adeno-associated virus (AAV) carrying a version of the human OTOF gene, which is introduced into the inner ears of the patients through a special surgical procedure.
No dose-limiting toxicity was observed in the trial, and the majority of the 48 adverse events noted were low grade and transitory with no long-term impact.
The researchers plan to expand the trial to a larger sample size and track outcomes over a longer timeline, marking a significant milestone in the treatment of genetic hearing loss.