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.
The new gene therapies use harmless, modified viruses to deliver working otoferlin genes directly into the inner ear. Early data suggest that the treatments work for most patients — although much more research is needed to get the therapies fully approved.
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