Opal Sandy, aged 18 months, was born completely deaf due to the condition auditory neuropathy, which is caused by the disruption of nerve impulses travelling from the inner ear to the brain.
Professor Manohar Bance, an ear surgeon at the trust and chief investigator for the trial, told the PA news agency the results were “better than I hoped or expected” and may point to a cure for patients with this type of deafness.
“Certainly since February, we’ve noticed her sister [Nora] waking her up in the morning because she’s running around on the landing, or someone rings on the door so her nap’s cut short.
A second child has also received the gene therapy treatment at Cambridge University Hospitals, with positive results seen recently, six weeks after surgery.
The overall phase 1/2 Chord trial consists of three parts, with three deaf children, including Opal, receiving a low dose of gene therapy in one ear only.
“It also supports the development of other gene therapies that may prove to make a difference in other genetic-related hearing conditions, many of which are more common than auditory neuropathy.”
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