A groundbreaking gene therapy trial has given Opal Sandy, an 18-month-old from England, a hearing. Opal, born deaf, suffers from auditory neuropathy, a faulty gene that prevents sound transmission from her ear to the brain.
During only a 16-minute procedure under general anesthesia, doctors injected a harmless virus into her right ear, and she soon after started hearing.
This virus carried a functional copy of the faulty gene, aiming to replace the defective one and restore proper hearing function.
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Opal was treated at Addenbrooke’s Hospital, a part of Cambridge University Hospitals NHS Foundation Trust.
According to a press release from Britain’s National Health Service within four weeks, Opal began responding to sounds, and after 24 weeks, her hearing was “close to normal.” She can hear whispering in her treated ear, respond to her parents’ voices, and even begin saying words such as “daddy” and “bye-bye.”
50% of all children’s hearing loss and 66% of newborn hearing loss result from genetic causes. Current hearing screening programs can only detect hearing loss beyond 35 dB. Thus, genetic screening can help identify children with mild SNHL that are missed with conventional screening
This successful treatment, the first of its kind, not only offers hope for millions of children born with genetic hearing loss but also paves the way for a potential new solution for auditory neuropathy: gene therapy.
