Gene Therapy Successfully Treats Toddler’s Hearing Loss

An 18-month-old in London, England, is one of the youngest children to receive gene therapy to treat hearing loss caused by a mutation in the OTOF gene. This gene produces a protein called Otoferlin that allows for communication between cells of the cochlea and the brain.

The recipient received an infusion of a functional copy of the OTOF in her ear. The procedure was completed in just 16 minutes. Reportedly, it only took a few weeks for the child to begin to hear and respond to sounds without the help of her cochlear implant.

References

Afzali K. (2024) Toddler born deaf can hear after gene therapy trial breakthrough her parents call “mind-blowing.”CBS News(accessed May 13, 2024).