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4 July 2025 – A groundbreaking clinical trial has demonstrated that a single injection of gene therapy can significantly restore hearing in individuals with congenital deafness, with improvements observed in just weeks. The study, published in Nature Medicine, involved ten participants aged 1 to 24, all with mutations in the OTOF gene, which impairs the production of otoferlin—a protein essential for transmitting sound signals from the ear to the brain.
🔬 How It Works
Researchers used a synthetic adeno-associated virus (AAV) to deliver a healthy copy of the OTOF gene directly into the inner ear via a minimally invasive procedure. The therapy rapidly improved auditory function, with average hearing thresholds improving from 106 decibels to 52 decibels within six months.
👧 Remarkable Results in Children
Children aged 5 to 8 responded most dramatically. One seven-year-old girl regained near-normal hearing and was able to hold daily conversations within four months of treatment. Adults also showed meaningful gains, expanding the potential age range for future therapies.
✅ Safety and Tolerability
The treatment was well-tolerated, with no serious adverse effects reported. The most common reaction was a temporary reduction in neutrophil counts, a type of white blood cell.
🔭 Next Steps in Genetic Hearing Loss Research
Lead researcher Dr. Maoli Duan of Karolinska Institutet emphasized that this is “just the beginning.” Teams are now exploring therapies targeting other common deafness-related genes such as GJB2 and TMC1, which pose greater complexity but show promise in animal models.
You can read the full study summary on ScienceDaily or The Independent.
