Gene Therapy Restores Hearing in Breakthrough Trial

Sweden has demonstrated that a single gene therapy injection can restore hearing in people born with a genetic form of deafness, offering a potential long-term treatment.

“This is a huge step forward in the genetic treatment of deafness, one that can be life-changing for children and adults,” said Maoli Duan, consultant at Karolinska Institutet.

The treatment targets mutations in the OTOF gene, which produces otoferlin, a protein essential for transmitting sound signals from the inner ear to the brain. In a trial of 10 patients aged one to 24, all showed improved hearing after receiving a working copy of the gene delivered into the cochlea via a viral vector. Hearing thresholds improved from 106 to 52 decibels on average within six months, with many patients responding within one month. Children showed the strongest gains, including a 7-year-old who regained near-normal hearing. Therapy was well-tolerated, with no serious adverse effects.