Deaf child from Oxfordshire receives groundbreaking gene therapy treatment, hears for the first time
Opal Sandy, a deaf child from Oxfordshire, UK, has recently made history by becoming the youngest child in the world to receive groundbreaking gene therapy treatment that allowed her to hear for the first time. Opal, who was born deaf due to a rare genetic condition known as auditory neuropathy caused by a variation on the OTOF gene, is also the first British patient to undergo this treatment.
The condition, although rare, affects approximately 1 to 3 children per 10,000 births. Opal’s older sister also has the same genetic condition, which allowed doctors to identify it in Opal at just 3 weeks old. The gene therapy treatment involved an infusion of a harmless virus and a working copy of the OTOF gene into Opal’s right ear, where she was also fitted with a cochlear implant during the same surgery.
Opal’s case has shown “spectacular” results, with her now able to hear soft sounds and whispers in her treated ear. At just 18-months-old, Opal is responding to her parents’ voices and communicating words, indicating restored hearing to normal levels. The trial, named CHORD, which started in May 2023, could have a global impact on children with specific genetic causes of deafness.
The National Deaf Children’s Society hopes that the trial results will provide insights into the effectiveness of gene therapy for genetic causes of deafness. Opal’s case will be presented at the American Society of Gene and Cell Therapy meeting in Baltimore, showcasing the potential breakthrough in treating genetic deafness in children. This treatment could revolutionize the way certain genetic causes of deafness are addressed, offering hope to families worldwide.
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