A single gene therapy injection has been shown to restore hearing in both children and adults with inherited deafness. In a groundbreaking study involving ten individuals with mutations in the OTOF gene, a gene crucial for sound signal transmission from the ear to the brain, researchers used a virus to deliver a healthy gene copy directly into the round window of the cochlea. Most regained hearing within a month, with the best outcomes seen in children aged 5 – 8 years. By six months, all patients had significant improvement— detecting sounds as soft as 52 decibels compared to 106 decibels at baseline. The treatment was safe and well-tolerated, marking a major milestone toward reversing genetic deafness and paving the way for future therapies targeting other hearing-related genes. ⁹

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