Health and Medicine Technology

Gene Therapy Is Successfully Restoring Vision To Genetic Blindness Patients

blindness gene therapy

New research from the University of Oxford has found a way to use gene therapy in order to restore partial vision to those suffering from rare genetic blindness. As of yet, vision was successfully improved for up to four years. This offers promise into the future of curing vision loss on a much wider scale.

During the study, a virus was injected into the eyes of patients with vision problems. The concoction contained healthy genes that work to replace genes that are missing. Individuals with choroideremia have benefitted from up to four years of visual improvement. Professor Robert MacLaren from the Nuffield Department of Clinical Neurosciences says long term efficiency of this gene therapy have been questioned but he says there is now clear-cut evidence that single injections prove to be quite viable.

This latest research is the best evidence thus far linking gene therapy to a single treatment cure for a variety of forms of blindness, such as retinitis pigmentosa which affects people of all ages. In a trial that was funded by the Department of Health and the Wellcome Trust, University of Oxford doctors tracked six patients and their vision changes for four years following their gene therapy. The treatments were created to slow down or completely stop vision degeneration. What was most surprising were the results of two patients who actually showed improvements in their vision without additional treatment. Three of those treated had vision that remained the same, while the final patient experienced very slow vision decline.

The study‘s lead investigator Professor Robert MacLaren says this new technique for vision treatment shows a lot of potential. As genetics are more and more understood, scientists have discovered that the best form of treatment may lie in correcting damaged or altered genes before diseases begin. Gene therapy works by taking harmful properties within a virus and placing their DNA in a cell, replacing the ‘bad’ cell with DNA that has been reprogrammed in order to heal or ‘correct’ faulty genes. Dr. Stephen Caddick, Director of Innovation at the Wellcome Trust hopes to one day be able to use this new form of treatment to restore sight to those who have inherited blindness. He says we are one step closer to incorporating gene therapy into the routine care provided to sufferers.

The first patient in the world to be treated by gene therapy still has vision problems but has doubled the vision in one of his eyes thanks to the groundbreaking treatment. Jonathan Wyatt is now 68 years old and has had vision problems since he was just 20 years old. Wyatt says he feels honored to work with the John Radcliffe research group and is thankful to be able to be a part of a project that is going to help others. Because of his treatment, he has become more independent and is able to easily leave the house which isn’t something he would have been able to do without assistance.

The most recent patient to partake in gene therapy treatment is a 24 year old teacher from Croydon whose name is Joe Pepper. He says he can see much better than before the operation. He says reading is easier than it has ever been and he feels much less stress on his eyes and is now able to see defined outlines rather than a blurry sense of shapes in the distance. Pepper says thanks to the work of MacLaren he has hope for the future of his vision, releasing a stress of blindness he has struggled with for over a decade.

The Efficacy and Mechanism Evaluation Programme is currently funding a follow up trial which includes 30 patients.