Pioneering gene therapy restores UK girl’s hearing | bbc

A UK girl born deaf can now hear unaided, after a groundbreaking gene-therapy treatment.

Opal Sandy was treated shortly before her first birthday – and six months on, can hear sounds as soft as a whisper and is starting to talk, saying words such as “Mama”, “Dada” and “uh-oh”.

Given as an infusion into the ear, the therapy replaces faulty DNA causing her type of inherited deafness.

Opal is part of a trial recruiting patients in the UK, US and Spain.

Doctors in other countries, including China, are also exploring very similar treatments for the Otof gene mutation Opal has.

Her parents, Jo and James, from Oxfordshire, say the results have been mind-blowing – but allowing Opal to be the first to test this treatment, made by Regeneron, was extremely tough.

“It was really scary, but I think we’d been given this unique opportunity,” Jo told me in an exclusive TV interview for BBC Breakfast News.

Her sister, Nora, five, has the same type of deafness and manages well wearing an electrical cochlear implant.

Rather than making sound louder, like a hearing aid, it gives the “sensation” of hearing, by directly stimulating the auditory nerve that communicates with the brain, bypassing the damaged sound-sensing hair cells in a part of the inner ear known as the cochlea.

In contrast, the therapy uses a modified, harmless virus to deliver a working copy of the Otof gene into these cells.

Opal had the therapy in her right ear, under general anaesthetic, and a cochlear implant put into her left.

Just a few weeks later, she could hear loud sounds, such as clapping, in her right ear.

And after six months, her doctors, at Addenbrooke’s Hospital, in Cambridge, confirmed that ear had almost normal hearing for soft sounds – even very quiet whispers.

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