Miguel Seabra’s recently published work in Nature Medicine gives a new hope to patients with Choroideremia, as it reveals that gene therapy can sustain and improve the vision of these patients, pointing to the potential of this procedure to cure common causes of blindness.
This new study shows the final results of a clinical trial in individuals with Choroideremia, a retinal degeneration disease that leads to progressive blindness due to deficiency of a protein – the REP1 protein. Through subretinal injection, these individuals were administered a vehicle that expressed the protein REP1. After 2 years, these individuals that were administered the vehicle presented improved vision.
“This study reports excellent results of gene therapy in the hereditary blinding disease, Choroideremia. The patients showed improvements in vision and quality of life, especially the most advanced cases and the treatment is sustained given that some patients were treated over 5 years ago” says Miguel Seabra, Principal Investigator of the Molecular Mechanisms of Disease Lab.
For more information, please check the article “Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia”.