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UK toddler regains hearing in gene therapy breakthrough

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STORY: Banging on blocks and playing the flute, Opal Sandy looks like an average energetic toddler.

:: May 1, 2024

It would be hard to tell she was born deaf – and unable to hear just months earlier.

:: Oxfordshire, England

She’s the first patient in England to regain her hearing through gene therapy.

:: Jo Sandy, Mother

“That was 24 weeks post-surgery, and we heard the phrase ‘near-normal hearing’, and she was turning to really soft sounds.”

:: James Sandy, Father

“Yeah, they played us the sounds that she was turning to and we were quite mind-blown by how soft it was, how quiet it was. They were sounds that in day-to-day life you might not even notice yourself, sort of thing.”

Opal received treatment as part of a groundbreaking global gene therapy trial, which doctors say can combat various genetic conditions that cause hearing loss in children.

:: Manohar Bance, Surgeon

“We can start to use gene therapy in young children, restore hearing for a variety of different kinds of genetic hearing loss, and then have a more ‘one and done’ type approach where we actually restore the hearing, we don’t have to have cochlear implants and other technologies that have to be replaced.”

Opal’s parents have described the results as mind-blowing.

(Jo Sandy)

“I think we’ve been given a really unique opportunity with no real evidence that any harm or adverse effects were likely to come to her and I think a lot of parents, regardless of the difficulties their children face, to be given an opportunity to potentially make obstacles easier for her to overcome was a risk definitely worth taking.”

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