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Toddler Born Deaf Can Hear After Gene Therapy Trial Breakthrough Her Parents Call "Mind-Blowing"

Posted on: 20 May 2024, source: CBS News
One of the youngest children in the world to receive a new type of gene therapy to treat genetic deafness can now hear for the first time in her life. The family of the toddler taking part in a medical trial has called the change in their daughter "mind-blowing." Opal Sandy, now 18 months old, was born with total deafness due to a fault in the OTOF gene, which makes a protein called Otoferlin. Otoferlin enables communication between cells of the inner ear, or cochlea, and the brain. As part of a trial run by Cambridge University, Opal received an infusion of a working copy of the OTOF gene in her right ear. The surgical procedure took only 16 minutes and was carried out just before she reached her first birthday.