Gene Therapy Creates a New Fovea

Gene therapy for an inherited form of blindness shows promise, a U.S. study shows.

According to a study published in the Aug. online edition of Human Gene Therapy and in a letter to the editor in the Aug. 13 issue of the New England Journal of Medicine, experimental gene therapy appeared to improve vision in three patients with Leber congenital amaurosis. In this condition an abnormal protein in sufferers' photoreceptors severely impairs their sensitivity to light. "It's like wearing several pairs of sunglasses in a dark room," says Artur Cideciyan, a researcher at the University of Pennsylvania in Philadelphia, who oversaw the trial

For the study, researchers from the University of Pennsylvania injected a gene encoding a functional copy of a light-sensitivity protein into a small part of one eye of three patients, all in their twenties and blind since birth. Three months after treatment, all three patients showed substantial improvements in their ability to detect light. Notably, one year after treatment, one patient discovered that she could read an illuminated clock in the family car for the first time in her life.

The authors suggested that the brain can adapt to new sensory capacity, even in people who have been blind since birth

Posted in: Blindness, Gene, Gene therapy, Leber Congenital Amaurosis, New England Journal of Medicine, Photophobia, University of Pennsylvania