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Gene therapy works in mice to prevent blindness that strikes boys

elp the brain process light and color - were spared from the disease and the connections between the layers of the retinas were intact.

In addition, the protein appears capable of moving within the retina to its target sites and the beneficial changes appear to be long lasting, researchers said. Especially encouraging were signs the treatment may be able to repair retinal damage.

The treatment has promising implications for other genetic eye diseases that involve the eye's ability to process light, including retinitis pigmentosa, which affects about 200,000 people in the United States and is one of the most common inherited causes of blindness in people between the ages of 20 and 60.

"We've been very successful in curing a disease in mice that has a direct copy in humans," said Hauswirth, who, in conjunction with UF, has interest in a biotechnology company that may seek to market some of the research technology. "It may take two to five years before we try this in human patients because of the need for safety studies, but we feel based on success so far, we will be able to provide formal evidence for safety that will allow us to get treatment into the clinic."

UF researchers worked with Bernhard Weber, Ph.D., at the Institute of Human Genetics in Regensburg, Germany, and Robert Molday, Ph.D., director of the Center for Macular Research at the University of British Columbia in Vancouver.

The Foundation Fighting Blindness, the National Institutes of Health and the Macula Vision Research Foundation supported the research.

"We now have proof of principle that gene therapy can basically prevent retinoschisis," said Stephen Rose, Ph.D., chief research officer for the Maryland-based Foundation Fighting Blindness. "Furthermore, this therapy apparently demonstrates that even if disease has begun, there is a healing that takes place. That raises hope for suffering patients that we may be able to offer something that can improve
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Source:University of Florida


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