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UF scientists restore sight to chickens with blinding disease

ome photoreceptors."

Infants with LCA1 would receive an injection of the gene transfer agents directly into the eyeball during the first couple of years of life, bypassing embryonic treatment. That's important, researchers say, because a diagnosis of LCA1 is often not made until months after a child is born.

"There are only a few clues that an infant may have this disease," Semple-Rowland said. "Often parents will notice that their child doesn't seem to be smiling at or looking at faces. Children may also poke or rub their eyes, behaviors clinically known as oculo-digital signs that may produce sensations of sight."

Work remains to refine the viral delivery system that transfers the healthy genes to the photoreceptor cells. In addition, solutions have to be found to make the treatment long-lasting -- scientists have restored sight and slowed degeneration, but the retinal cells still degenerate.

But Semple-Rowland thinks the time necessary to turn these research results into a treatment for patients will be a fraction of the 20 years that have gone into discovering the genetic defect and developing a therapy for it.

"We can do amazing things in animal models," Semple-Rowland said, "but this work can't be done quickly. That's the hardest thing -- knowing there are people who need these treatments now. But we work as fast as we can. You'll see the first treatments for some of these genetic eye diseases soon, especially after the groundwork for an approved therapy is laid and the therapy works."


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Source:University of Florida


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