Pioneering gene therapy restores UK girl’s hearing

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A Groundbreaking Gene Therapy Restores Hearing for a UK Girl Born Deaf

In a remarkable medical breakthrough, a young girl from the UK born deaf can now hear unaided, thanks to a revolutionary gene therapy treatment. Opal Sandy, who was treated shortly before her first birthday, can now hear sounds as soft as a whisper and is starting to speak her first words, including “Mama,” “Dada,” and “uh-oh.”

The therapy, which is delivered as an infusion into the ear, replaces the faulty DNA responsible for Opal’s inherited deafness. Opal is part of a clinical trial recruiting patients in the UK, US, and Spain, and similar treatments are being explored in other countries, including China.

Opal’s parents, Jo and James, from Oxfordshire, describe the results as “mind-blowing,” though they admit that allowing their daughter to be the first to test this treatment, developed by Regeneron, was an extremely tough decision.

“It was really scary, but I think we’d been given this unique opportunity,” Jo said.

Opal’s older sister, Nora, who is also deaf, manages well with a cochlear implant, which provides the “sensation” of hearing by directly stimulating the auditory nerve, bypassing the damaged sound-sensing hair cells in the inner ear. In contrast, the new gene therapy uses a modified, harmless virus to deliver a working copy of the Otof gene into these cells.

After receiving the gene therapy in her right ear, along with a cochlear implant in her left ear, Opal was able to hear loud sounds, such as clapping, within just a few weeks. And after six months, her doctors at Addenbrooke’s Hospital in Cambridge confirmed that her right ear had almost normal hearing for soft sounds, including very quiet whispers.

“It’s wonderful seeing her respond to sound,” said chief investigator and ear surgeon Prof. Manohar Bance. “It’s a very joyful time.”

Experts hope this therapy could also benefit those with other types of profound hearing loss, as more than half of hearing loss cases in children have a genetic cause. Prof. Bance is optimistic that the trial could lead to gene therapy being used to restore hearing in young children, potentially eliminating the need for cochlear implants and other technologies that require replacement.

“The younger we can restore hearing, the better for all children because the brain starts to shut down its plasticity [adaptability] after the age of about three or so,” he said.

Opal’s story and the scientific data from the trial will be presented at the American Society of Gene and Cell Therapy conference in Baltimore, US. Martin McLean, from the National Deaf Children’s Society, welcomed the new treatment options, stating that “with the right support from the start, deafness should never be a barrier to happiness or fulfilment.

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