In gene therapy first, scientists restore vision to dogs born blind

GAINESVILLE, Fla. --- During World War I, the French army dispatched ammunition-carrying Briard dogs to the front lines. The Red Cross turned to the shaggy-haired sheepdogs to haul first-aid supplies.

And now Briards once again have been called to service: For the first time ever, animals that were born blind gained the ability to see after undergoing gene therapy, according to research from the University of Florida, Cornell University and the University of Pennsylvania. The findings, published in the May issue of Nature Genetics, open the door to developing treatment in coming years for people with a rare, inherited eye disorder.

The scientists reported their success in three dogs with a condition closely resembling Leber congenital amaurosis, or LCA, a blinding disease caused by mutations in a gene important for eye function. About 2,000 people in the United States have LCA, a currently untreatable condition that typically results in blindness from birth or shortly thereafter.

"In gene therapy with other eye diseases, we've been able to prevent worsening of the condition in animals, but this is the first time we've been able to recover a lost function," said William W. Hauswirth a contributing author who is the Rybaczki-Bullard professor of molecular genetics and microbiology at UF's College of Medicine.

"This is also really the first study that shows you can use gene therapy to get a response in a human-sized eye, that you can effectively treat enough of the retina to have an effect on the animal's visual function."

Dr. Jean Bennett, a senior author of the Nature Genetics paper and a scientist at the F.M. Kirby Center for Molecular Ophthalmology at Penn's Scheie Eye Institute, said the study results expand the possible treatment strategies for degenerative retinal diseases.

"We have worked hard for many, many years trying to develop a treatment for retinal degeneration, and this is the biggest leap forward yet," she said.

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Contact: Victoria White
University of Florida

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