Doctors said they suspect that similar gene therapy treatments could prove curative for many of the 150,000 Americans suffering from that broad family of diseases, all of which involve a deterioration of the light-detecting retina in the back of the eye. But that said, it's hard not to get very excited about this because it's a very dramatic result. I mean, basically these dogs were blind and now they are not blind anymore. Children with LCA have little or no vision at birth, and what little may be there is lost over time. It occurs when there is a defect in any of several genes that help convert light into electrical signals in the eye.
Parents carrying the defect on one copy of the gene have normal vision.
Jean Bennett, Maguire's wife and co-worker at the Schieie Institute's F.M. Kirby research center, led the new study, in which thousands of viruses were injected directly into the eyes of three blind Briards. But the right eyes got injections directly behind the retina, very close to the so-called retinal pigment epithelial cells where the rpe gene does its job. A video shows the dogs navigating around a cluttered room, only bumping into objects when they are on the animals' left side. Electrical measurements of the retina, resembling an EKG for the heart, appear normal. It's just beautiful work.