Dennis Thompson HealthDay Reporter Posted: Thursday, January 16, 2014, 2:00 PM THURSDAY, Jan. 16, 2014 (HealthDay News) -- A new gene therapy that successfully treated a rare eye disease in clinical trials could prove the key to preventing more common inherited causes of blindness, researchers say. In six male patients, doctors used a virus to repair a defective gene that causes choroideremia, a degenerative eye disease that can lead to complete blindness by middle age, according to a clinical trial report published online Jan. 16 in The Lancet. Vision improved for all the patients following the gene therapy, and particularly for two patients with advanced choroideremia, said lead author Robert MacLaren of the Nuffield Laboratory of Ophthalmology at the University of Oxford, and a consultant surgeon at the Oxford Eye Hospital, in England. "In truth, we did not expect to see such dramatic improvements in visual acuity and so we contacted both patients' home opticians to get current and historical data on their vision in former years, long before the gene therapy trial started," MacLaren said in a university news release. "These readings confirmed exactly what we had seen in our study and provided an independent verification." While choroideremia is … Continue reading →

Scientists hope early intervention with the surgical treatment will halt progression of the devastating disorder, choroideremia, before patients are robbed of their sight. It is the first time gene therapy has successfully been applied to the light-sensitive photoreceptors of the retina, the digital camera at the back of the eye. Preliminary results from the first six patients taking part in a Phase One trial surprised and delighted the Oxford University team. Although the trial was only designed to test safety and dosages, two men with relatively advanced disease experienced dramatic improvements to their eyesight. The researchers are now planning a larger Phase II trial that will focus on the therapy's effectiveness. Professor Robert MacLaren, who led the gene therapy operations at Oxford Eye Hospital, said: "We're absolutely delighted with the results so far. "It is still too early to know if the gene therapy treatment will last indefinitely, but we can say that the vision improvements have been maintained for as long as we have been following up the patients, which is two years in one case. "In truth, we did not expect to see such dramatic improvements in visual acuity and so we contacted both patients' home opticians to … Continue reading →

Scientists believe they may have found a cure for some forms of age-related blindness. Photo: Michele Mossop London: Thousands of people suffering from common forms of blindness could have their sight restored by a pioneering treatment. Researchers at Oxford University have discovered that by replacing a missing protein in the retina they can prevent cells from degenerating. The therapy even improves the sight of those who have already begun to go blind in results that have "surpassed expectations". Two men who were already at an advanced stage of vision loss have experienced "dramatic improvements" in their sight which, so far, have lasted for two years. Professor Robert MacLaren, of the Nuffield Laboratory of Ophthalmology at the University of Oxford, said: "We're absolutely delighted with the results so far. In truth, we did not expect to see such dramatic improvements This has huge implications for anyone with a genetic retinal disease such as age-related macular degeneration or retinitis pigmentosa because it has, for the first time, shown, that gene therapy can be applied safely before the onset of vision loss." Advertisement The trial was carried out on patients suffering from choroideremia - a rare inherited cause of blindness which affects about … Continue reading →

By delivering gene therapies to patients before they go blind, doctors may be able to prevent the loss of many important light-detecting cells. Light preserver: Robert MacLaren performs retinal surgery on a patient participating in a gene therapy experiment at the Oxford University Eye Hospital. A new kind of gene therapy has reversed some vision loss in people born with a degenerative eye disease for which there is no existing treatment. In a first for the field, the treatment can be given to some participants who still had 20/20 vision, albeit in a limited field of vision. By delivering gene therapy at an earlier stage, researchers hope to save more light-sensing cells in the retina. We need to push gene therapy forward, to apply it before vision is gone, says Robert MacLaren, an ophthalmologist at the University of Oxford who led the study. When retinal damage gets to a certain point, its beyond repair. MacLaren says earlier treatment could also be particularly important for conditions such as retinitis pigmentosa and age-related macular degeneration. The surgical procedure employed put the precious remaining vision of patients in the trial at risk because it involved detaching delicate retina tissue in one of each … Continue reading →