Gene therapies restore hearing in several kids with inherited deafness


Several children born with a rare, inherited form of deafness can now hear thanks to two new gene therapies, clinical trial results show.

Both therapies target the gene for otoferlin, a protein in the inner ear that lets nerve cells translate vibrations from sound into electrical signals that can be interpreted by the brain. Mutations in the otoferlin gene cause about 1% to 8% of cases of congenital deafness, in which a child is born deaf. Still, mutations in the gene are fairly rare, affecting an estimated 200,000 people worldwide.


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