Groundbreaking gene therapy restores hearing in 1-year-old UK girl
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London: An 18-month-old British girl, born deaf, has become the youngest person to have her hearing restored through pioneering gene therapy.
This groundbreaking treatment, developed by US biotech firm Regeneron, marks a significant milestone in the field of medical science.
Opal, the toddler from Oxfordshire, England, underwent revolutionary therapy at Addenbrooke's Hospital in Cambridge, making her the first person worldwide to receive this specific treatment at such a young age, reported AFP.
According to UK ear surgeon Manohar Bance, Opal's surgery resulted in "spectacular" outcomes, bringing her hearing close to normal. This achievement signifies a major advancement in the treatment of hereditary deafness, offering hope for a potential cure.
Opal suffers from a genetic form of auditory neuropathy, caused by a disruption in nerve impulses between the inner ear and the brain. This condition is linked to a fault in the OTOF gene, responsible for producing a crucial protein called otoferlin, essential for auditory function.
The gene therapy developed by Regeneron aims to overcome this genetic fault by delivering a functional copy of the gene directly to the ear, restoring normal hearing function.
Following her surgery in September, Opal's hearing has shown remarkable improvement, with further enhancements expected in the future. Another child who underwent the same gene therapy in Cambridge also experienced positive results within six weeks post-surgery.
While similar gene therapy trials are underway in China and the United States, Opal's case stands out due to her age and the success of the treatment. The trial in Cambridge involves three stages, with different doses administered to deaf children to assess safety and efficacy.
Up to 18 children from the UK, Spain, and the US are participating in the trial, with long-term follow-up planned for five years. This pioneering approach offers new hope for individuals with auditory neuropathy, potentially revolutionizing the treatment landscape for deafness.
Reflecting on this groundbreaking achievement, Bance expressed his awe and excitement, noting the significant impact of gene therapy in transforming the lives of individuals with auditory impairments.