Guei-Sheung Liu

RNA editing therapy for Usher syndrome

Usher syndrome causes irreversible vision loss alongside early-onset hearing impairment. This project is developing an RNA editing therapy that directly corrects the genetic cause of the disease and restores essential retinal proteins. By targeting the root cause rather than managing symptoms, the approach has the potential to halt retinal degeneration and transform outcomes for up to 300,000 people worldwide.