British scientists using a new gene therapy failed to restore a degree of vision in six patients aged 35-63 years suffering from a rare congenital disease, the chorioeiderimia, causing blindness. It was the first clinical trial of treatment and was a relative success.
Doctors are optimistic that in the future a similar treatment could be used in the most common forms of blindness such as macular degeneration due to aging, which affects about one in four people over 75 years and will researchers, led by Professor Robert MacLaren surgery, they made after publication in the medical journal “Lancet”, according to the BBC and the “New Scientist”, replaced-with the help of a genetically modified virus harmless ‘vehicle’ – a defective gene in cells (photoreceptors) eye of patients, which made the latter sensitive to light again.
Patients with chorioeiderimia (which affects about one person in 50,000) photo-sensitive cells retinal lens gradually destroyed and the patient slowly loses his sight. The disease, which primarily affects males, is caused by a defective gene, CHM, on chromosome X. The new treatment-that “works” only if the cells of the eye are not completely destroyed by the disease are not fully re-vision, but the significantly improved. Six patients monitored n c if the improvement of vision proved permanent, then the gene therapy will be implemented promptly and in younger patients, in order not to lose almost all their light. Although future clinical trials are successful (the next, with 30 patients, will start in 2015), then the new treatment is expected to be formally approved after about five years. As McLaren said, “the chorioeiderimia shows some similarities with the macular degeneration, as well as the therapeutic goal is the same cells.
We do not know yet in which genes to focus our attention on macular degeneration, but now we know how to do. “