Stockholm’s Karolinska Institutet, in collaboration with Chinese hospitals and universities, has reported a medical breakthrough in Nature Medicine: gene therapy has successfully restored hearing in patients with congenital deafness caused by mutations in the OTOF gene. The trial involved ten patients aged 1–24. Using an adeno-associated viral vector, researchers delivered a healthy copy of the OTOF gene into the inner ear. Most patients showed significant hearing improvement within a month, with younger children responding best. One seven-year-old girl regained nearly all her hearing within four months and could hold conversations with her mother. No severe side effects were reported during the 6–12 month follow-up. “This is a huge step forward in the genetic treatment of deafness,” said Maoli Duan of Karolinska Institutet. “We are optimistic this approach could transform the lives of children and adults.” Researchers now aim to expand gene therapy to other genes linked to hereditary deafness, such as GJB2 and TMC1.
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