A real eye-opener

Gene therapy restores sight to dogs with rare inherited condition

source: Philip Cohen
New Scientist May 5 2001

Researchers at the University of Pennsylvania, Philadelphia, have restored the sight of dogs with blindness caused by Leber congenital amaurosis, an inherited condition which affects the retina, and which is also found in humans. The dogs were found to have a defective gene for RPE65, a protein. A properly functioning copy of this gene was engineered into a virus, and then injected into the dogs' retinas. Pupil function and electrical activity improved, and this was not the case with control animals, which just had the gene injected into the fluid of their eyes, or no injections. The dogs that had the treatment were also able to navigate obstacles better than the controls could. This research could help with developing treatments for blindness in people.