GENETIC THERAPY AIMS TO BRING HEARING TO THOSE BORN DEAF

Summary from the TLDR newsletter

OTOF-related hearing loss results from a genetic change in a single critical protein. The auditory nerve fails to receive the proper signals from the hair cells in the ear due to the lack of the proper protein, resulting in profound deafness. DB-OTO is a virus-based gene therapy that can deliver a working version of the OTOF gene. It uses a non-pathogenic virus to carry the proper genetic code that produces the protein. In an early trial of the DB-OTO treatment, eleven out of twelve patients developed improved hearing within weeks, and nine patients no longer required cochlear implants.