GENE THERAPY RESTORES HEARING IN TODDLERS AND TEENAGERS BORN WITH CONGENITAL DEAFNESS – NEW RESEARCH

Summary from the TLDR newsletter

Researchers have developed a form of gene therapy that can successfully restore hearing in toddlers and young adults born with OTOF-related deafness, a condition caused by mutations in the OTOF gene. People with this condition have healthy hearing structures in their inner ear, but one crucial gene isn't working properly - fixing the faulty gene restores hearing. The researchers used a virus as a delivery system to carry a working copy of the OTOF gene directly to the inner ear's hearing cells, restoring hearing sometimes to near-normal levels.