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ABC News reports that a 3-year-old girl in England, Opal Sandy, was born with profound hearing loss caused by a mutation in the OTOF gene, a condition where the inner ear cannot transmit sound signals to the brain even though the rest of the ear structures are formed. She entered a clinical trial at 11 months old and received a one-time gene therapy injection in her right ear.
The treatment delivered a working copy of the OTOF gene to her inner ear cells. Her left ear was fitted with a cochlear implant. Over the next months her parents noticed steady improvements and, by age three, she could respond to sound from the treated ear even when her implant was off.
The trial, run by Regeneron Pharmaceuticals across several countries, has reported that most participating children show measurable hearing gains within weeks. Researchers say they need longer follow up to understand how durable the hearing improvements are and whether children will need additional interventions over time.
Read the full article here.
