An experimental gene therapy restored at least some level hearing in most patients with a rare form of inherited deafness, a study found.
The study, published April 22 in Nature, evaluated 42 children and adults with autosomal recessive deafness 9, a condition caused by mutations in the OTOF gene that prevent sound signals from reaching the brain. The condition affects about 50 babies each year in the U.S., though researchers said the findings could support development of gene therapies for other forms of genetic hearing loss.
Researchers used a virus to deliver a functional version of the gene into the inner ear. About 90% of patients experienced hearing improvement, with some achieving near-normal hearing levels and benefits lasting more than two years.
Patients began to detect sound within weeks of treatment, with hearing continuing to improve over about six months. Some were able to develop speech after gaining hearing.
The findings build on earlier findings. In 2024, an 11-year-old boy in the U.S. became the first patient to receive a successful gene therapy for congenital deafness through a clinical trial at Children’s Hospital of Philadelphia.
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