Researchers at Karolinska Institutet, collaborating with Chinese institutions, have unveiled a promising gene therapy that restores hearing in patients with congenital deafness caused by OTOF gene mutations. Using a synthetic virus to deliver a healthy gene into the inner ear, the treatment improved hearing thresholds dramatically—from severe impairment to much clearer sound—in just six months. Children aged 5 to 8 showed the most remarkable progress, including a 7-year-old girl who nearly regained full hearing. Adults also experienced benefits, marking a milestone in genetic deafness treatment. The therapy was safe and well-tolerated, paving the way for future advances targeting other common deafness genes.
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