Similar treatments targeting a rare genetic mutation causing hereditary deafness have shown promising results in trials conducted by medical teams in China and the United States.
London: An 18-month-old British girl, born completely deaf, is now believed to be the youngest individual to regain her hearing through pioneering gene therapy. This therapy, developed by US biotech firm Regeneron, marks a significant breakthrough in the treatment of hereditary deafness, focusing on a rare genetic mutation.
Dr. Manohar Bance, an ear surgeon from the UK, stated that Opal, the toddler, is the first person globally to undergo this therapy, marking a milestone in medical history. Opal received treatment at Addenbrooke’s Hospital in Cambridge, part of Cambridge University Hospitals NHS Foundation Trust.
Opal suffers from a genetic form of auditory neuropathy, resulting from a disruption in nerve impulses from the inner ear to the brain. This condition is often caused by a fault in the OTOF gene, responsible for producing a crucial protein called otoferlin, essential for communication between ear cells and the hearing nerve.
The gene therapy developed by Regeneron aims to rectify this fault by delivering a functional copy of the gene directly to the ear. Opal’s surgery, conducted last September, has shown remarkable results, bringing her hearing close to normal, with further improvements expected.
Notably, Opal is the first participant in a gene therapy trial led by Dr. Bance in Cambridge. The trial, which involves three parts, aims to assess the safety and efficacy of the therapy. Up to 18 deaf children from the UK, Spain, and the United States will be recruited for the trial, with a follow-up period of five years.
Dr. Bance emphasized that the current treatment for auditory neuropathy involves implants. Reflecting on the breakthrough, he expressed awe at witnessing the tangible success of gene therapy, a concept long deemed to be on the horizon.
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