FIRST-EVER GENE THERAPY TRIAL TO CURE FORM OF DEAFNESS BEGINS

Summary from the TLDR newsletter

Up to 18 children from the UK, Spain, and the US are being recruited for the world's first trial of a gene therapy to cure auditory neuropathy, a condition caused by the disruption of nerve impulses traveling from the inner ear to the brain. The condition can be caused by a variation in a single gene that produces a protein that allows inner hair cells in the ear to communicate with the hearing nerve. The study aims to deliver a working copy of the gene using a modified non-pathogenic virus. Participants will be monitored for five years to gauge whether the treatment works.