BBC Medical Science — Surgeons in Oxford have used a gene therapy technique to improve the vision of six patients who would otherwise have gone blind. The operation involved inserting a gene into the eye, a treatment that revived light-detecting cells. The doctors involved believe that the treatment could in time be used to treat common forms of blindness.
Prof Robert MacLaren, the surgeon who led the research, said he was “absolutely delighted” at the outcome. “We really couldn’t have asked for a better result,” he said. …
If the improvements seen in the patients continue, the aim will be to offer the treatment to younger choroideremia patients to prevent them from losing their sight. The condition is relatively rare: it is thought to affect a thousand people in the UK. But Professor MacLaren believes that success with choroideremia demonstrates the principle that gene therapy could be used to cure other forms of genetic blindness including age-related macular degeneration. This condition causes blindness in 300,000 people in Britain and causes a deterioration in the vision of one in four people over the age of 75.
“The mechanisms of choroideremia and what we are trying to do with the treatment would broadly be applicable to more common causes of blindness,” the professor explained. “Choroideremia shows some similarities with macular degeneration in that we are targeting the same cells. We don’t yet know which genes to target for macular degeneration but we do know now how to do it and how to put the genes back in.” (01/20/2014)