Small study tests goggle-assisted gene therapy in people blind from retinitis pigmentosa
In a small open-label NEJM safety study, meaning everyone knew the treatment, 10 people blind from advanced retinitis pigmentosa, an inherited disease that destroys retinal cells, received therapy in the worse-seeing eye. A virus gave retinal nerve cells instructions for a light-sensitive protein; goggles activated it, with the untreated eye patched. Goggle-assisted light sensitivity rose 2.0- to 62.3-fold in 7; 6 achieved the study's meaningful threshold of about fourfold or more. Nine had 34 eye adverse events: 23 mild, 10 moderate and one severe central retinal artery blockage immediately after injection that medication resolved within minutes. GenSight funded it; authors called for more safety and efficacy research.
Safety was the study's primary outcome, while goggle-assisted light sensitivity was a secondary outcome.
Written by the Genomes desk from the primary source cited and linked above and checked against it. Research use only; not medical advice. Corrections: editor@genome.news.