English toddler has hearing restored in world’s first gene therapy trialEnglish toddler has hearing restored in world’s first gene therapy trial

“A British toddler has had her hearing restored after becoming the first person in the world to take part in a pioneering gene therapy trial, in a development that doctors say marks a new era in treating deafness. Opal Sandy was born unable to hear anything due to auditory neuropathy, a condition that disrupts nerve impulses traveling from the inner ear to the brain and can be caused by a faulty gene. But after receiving an infusion containing a working copy of the gene during groundbreaking surgery that took just 16 minutes, the 18-month-old can hear almost perfectly and enjoys playing with toy drums.”

The Guardian reports that the “girl, from Oxfordshire, was treated at Addenbrooke’s hospital, part of Cambridge university hospitals NHS foundation trust, which is running the Chord trial. More deaf children from the UK, Spain and the US are being recruited to the trial and will all be followed up for five years….A second child has also recently received the gene therapy treatment at Cambridge university hospitals, with positive results. The overall Chord trial consists of three parts, with three deaf children including Opal receiving a low dose of gene therapy in one ear only. A different set of three children will get a high dose on one side. Then, if that is shown to be safe, more children will receive a dose in both ears at the same time. In total, 18 children worldwide will be recruited to the trial.”

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LU Staff

LU Staff

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