Gene therapy may be a tool to prevent blindness

Reduces blinding blood vessel growth by up to 90 percent in laboratory mice

Gene therapy may one day be used to halt or even prevent the overgrowth of blood vessels in the eye that blinds patients with macular degeneration and diabetic retinopathy, according to two recent studies led by researchers at Johns Hopkins' Wilmer Eye Institute.

In the studies, performed with laboratory mice, two different genes that were separately injected into the tail veins or eyes of the animals reduced new blood vessel growth by up to 90 percent. One gene is for endostatin, which inhibits blood vessel growth; the other is a substance that increases cell survival. The mice had conditions similar to macular degeneration and diabetic retinopathy, the leading causes of blindness in older and younger people, respectively, in developed countries.

"These studies show that when therapeutic genes are introduced before the stimulus for abnormal blood vessel growth, they partially prevent the abnormal vessels from growing," says Peter A. Campochiaro, M.D., senior author of the studies and a professor of ophthalmology and neuroscience at Hopkins. "While the substances we tested certainly need further study, results are very exciting and demonstrate that gene therapy might provide a promising approach for the treatment of these conditions."

Current therapies for macular degeneration and diabetic retinopathy include laser treatment or surgery to eliminate the abnormal blood vessels. But, in addition to risks associated with surgery, the treatments do nothing to attack the underlying stimuli for blood vessel growth, Campochiaro says.

"As a result, the blood vessels tend to come back," he says. "Even with initially successful treatments, many patients still end up with severe loss of vision."

For the first study, published in the July issue of the American Journal of Pathology, Campochiaro and colleagues packaged the gene

Contact: Karen Blum
Johns Hopkins Medical Institutions

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