Opal Sandy was born deaf due to a rare genetic condition but can now hear unaided after receiving ground-breaking gene therapy. Photograph: Andrew Matthews/PA WireA 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.
Her parents were left “gobsmacked” when they realised she could hear for the first time after the treatment. “I couldn’t really believe it,” Opal’s mother, Jo Sandy, said. “It was ... bonkers.”Israeli officials weigh sharing power with Arab states in postwar Gaza Prof Manohar Bance, an ear surgeon at the trust and chief investigator for the trial, said the initial results were “better than I hoped or expected” and could cure patients with this type of deafness.
Auditory neuropathy can be caused by a fault in the OTOF gene, which makes a protein called otoferlin. This enables cells in the ear to communicate with the hearing nerve. To overcome the fault, the new therapy from biotech firm Regeneron sends a working copy of the gene to the ear. 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.
Source: Law Daily Report (lawdailyreport.net)
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