According to a new study published in the journal Nature, an experimental gene therapy has demonstrated high efficacy and safety in restoring hearing for patients born with genetic deafness. The large-scale study, led by researcher Zheng-Yi Chen of Mass Eye and Ear in Boston, involved 42 patients. As a result, 90% of the participants showed significant hearing improvement, with some even regaining the ability to hear whispers.
The research focused on autosomal recessive deafness (DFNB9), caused by mutations in the OTOF gene. This gene is responsible for producing the protein otoferlin, which is essential for transmitting sound signals from the inner ear cells to the brain. Using an adeno-associated virus (AAV), scientists delivered a healthy version of the gene into the ear, restoring the cells’ ability to produce the protein.
The effects of the treatment became apparent within a few weeks of injection, with improvements continuing over approximately six months. Significantly, patients not only heard sounds but also began learning to speak, fundamentally transforming their social integration. Experts describe this as a “game-changer” in otolaryngology, as it paves the way for using gene therapy to treat more common forms of hearing loss.
Scientists emphasize the importance of early screening, noting that the earlier the intervention occurs, the better the results. Currently, a similar therapy developed by Regeneron Pharmaceuticals may become the first to receive official FDA authorization. Researchers hope that this one-time procedure will provide patients with lifelong benefits.
