Deafness Reversed: Hearing Restored in Weeks
Scientists have achieved a major breakthrough in treating hearing loss. A single gene therapy injection can restore hearing in children and adults. Researchers at Karolinska Institutet, together with Chinese hospitals and universities, tested ten patients. Every participant experienced hearing improvement. The treatment was also well-tolerated, showing no serious side effects. The results appeared in Nature Medicine.
Targeting the OTOF Gene
The trial focused on patients with mutations in the OTOF gene. These mutations prevent the body from making enough otoferlin, a protein essential for sending sound signals from the inner ear to the brain. Ten patients aged 1 to 24 were treated at five hospitals in China. Doctors used a synthetic adeno-associated virus (AAV) to deliver a functional OTOF gene directly into the inner ear. The injection went through the cochlea’s round window membrane.
Rapid Results and Strong Gains
Patients regained some hearing within a month. After six months, all showed clear improvement. On average, their hearing improved from 106 decibels to 52. Children aged five to eight saw the most dramatic results. One seven-year-old girl regained nearly full hearing and could converse normally with her mother after just four months. Adults also experienced meaningful improvements.
Safe and Expanding Therapy
The therapy was safe, with the main side effect being a temporary decrease in white blood cells. Researchers plan to expand gene therapy to other deafness genes, such as GJB2 and TMC1. Animal studies are promising. If successful, more patients with different genetic forms of deafness could benefit in the future. Funding came from Chinese research programs and Otovia Therapeutics Inc., the company that developed the therapy. Researchers will continue monitoring patients to see how long the improvements last.
