Gene Therapy Benefit

By delivering gene therapies to patients before they go blind, doctors may be able to prevent the loss of many important light-detecting cells.
Robert MacLaren, an ophthalmologist at the University of Oxford led the study. “When retinal damage gets to a certain point, it’s beyond repair.”

Gene Trial Makes Eye Disease Breakthrough

The experimental treatment adds a working copy of the culpable gene to the retinal cells of the patients born with a defective copy. The trial also involved an experimental way of delivering gene therapy to the eye.  Each patient's retina was first lifted, and the gene therapy was injected into the space created under the retina.  MacLaren and colleagues report on the condition of six patients in a study published on 16th January in the Lancet.