Gene therapy restores vision partially blind patients

Transplanting genes led to a partial recovery of vision in nearly blind, four patients with congenital eye disease. These results were obtained in two independent teams of scientists from the U.S. and the UK, according to New England Journal of Medicine. Altogether the study, six adult patients with liver amaurosis. This is a disease that occurs due to genetic defects, manifested progressive degeneration of the retina. For 20 or 30 years, patients lose almost all of his eyes. In tests by ophthalmologists Murfilds hospitals and researchers at the University of Pennsylvania conducted patients were involved with a defect in the RPE65 gene. This gene encodes a protein that is responsible for the normal function of the retina. During surgery, patients received a copy of the damaged gene runs in the back of the eye. "Delivery" gene in the DNA of the pigment cells was carried out with a harmless adenovirus. After four to six months after surgery, U.S. researchers reported that gene therapy was successful in all three of the study participants - women aged 19 and 26 years old and 26-year-old male. Previously, only patients to distinguish hand movements, and are now able to see the lines on the eye test table, the head of research Albert Maguire (Albert M. Maguire). A British team of scientists led by Robin Ali was able to achieve success in one patient out of three. A 17-year-old boy has improved night vision: through gene therapy, he could safely walk the half-dark labyrinth. According to the researchers, the gene therapy did not result in serious side effects. None of the patients in particular have not an inflammation of the retina. For the near future, the researchers, the new method on children suffer from an earlier stage of the disease to test and have a better chance of recovery.