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The first patient in a pioneering gene therapy trial had her hearing restored.

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Opal Sandy, who was born completely deaf because of a rare genetic condition, and can now hear unaided for the first time after receiving ground-breaking gene therapy at 11-months-old, at her home in Eynsham, Oxfordshire

All Ears

The Story

The first patient in a pioneering gene therapy trial had her hearing restored.

Tell me more.

Opal Sandy, an 18-month-old from the UK, was born deaf due to auditory neuropathy, a condition caused by a faulty gene that doesn’t allow the ear to communicate sound to the brain properly. As part of the trial, researchers injected a harmless virus with a working copy of the gene into Opal’s ear. After nearly six months, clinicians said she had almost normal hearing, even the ability to hear whispers. She can now respond to her parents’ voices and has started saying “Dada,” and “bye-bye.” Experts say they’re hopeful this gene therapy could work for many types of hearing loss.

What do you mean?

About one in 500 babies are born with hearing loss or develop it in early childhood, and roughly half of those cases are caused by genetics, according to the CDC. The trial, which began last year, will continue to enroll patients under the age of 18 from the UK, the US, and Spain. They will be followed for up to five years to see how much their hearing has improved. Meanwhile, doctors in other countries, including China, are working on similar treatments for deafness caused by the gene variation that Opal has.

theSkimm

The first results from an international trial aimed at treating hearing loss through gene therapy showed groundbreaking and better than expected results. Now, experts say this type of treatment could be a game changer for genetic deafness.

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