Leber congenital amaurosis is a rare genetic disease that causes near total blinness in young children. Mutations in at least 3 genes can cause LCA and studies in mice that that have mutations in one of these genes showed increasing blindness over time.
Now scientists working with dogs that have a naturally occurring mutation in one of these genes, and go blind in much the same way as young children, have managed to restore the sight in the affected eyes.
The scientists used gene therapy to introduce normal gene into the retinas of the dogs and now hope that this technique may work for human sufferers.
Nature Genetics, May 2001
