1 week Ago By Jane Kirby
A British girl has had her hearing restored after becoming the first person in the world to take part in a groundbreaking new gene therapy trial. 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. Now, thanks to a “one and done” gene therapy being trialled in the UK and worldwide, Opal’s hearing is almost normal – and could even improve further.
The little girl, from Oxfordshire , who has a genetic form of auditory neuropathy, was treated at Addenbrooke’s Hospital, which is part of Cambridge University Hospitals NHS Foundation Trust. 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 cure patients with this type of deafness. He said: “We have results from (Opal) which are very spectacular – so close to normal hearing restoration.
So we do hope it could be a potential cure.” Auditory neuropathy can be caused by a fault in the OTOF gene, which is responsible for making a protein called otoferlin. This enables cells in the ear to communicate with the hearing nerve.
To overcome the fault, the “new era” gene therapy – from biotech firm Regeneron – delivers a working copy of the gene to the ear. In Opal’s case, she received an infusion containing the working gene to her right ear during surgery.
