Researchers find way to prevent blindness in research model for retinitis pigmentosa

June 11, 2010

Researchers at the University of Oklahoma Health Sciences Center have found a way to use a radical new type of gene therapy to prevent blindness caused by retinitis pigmentosa, giving hope to the estimated 100,000 Americans who suffer from this debilitating disease.

The study appears in the Journal of the Federation of American Societies for Experimental Biology (FASEB).

The research, led by Muna Naash, Ph.D., at the University of Oklahoma Health Sciences Center, with collaborators in Cleveland and Buffalo, discovered a way to deliver known gene therapies directly to the light-sensitive cells affected by this disease.

The discovery already is being used to develop new treatments for another disease - , the leading cause of blindness in the United States.

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Muna Naash, Ph.D., principal investigator, Robert Leonard, M.D., Dean McGee Eye Institute, and patient Tracie Harris, Oklahoma City, talk about Dr. Naash's breakthrough work to prevent blindness. Credit: University Hospitals Authority and Trust

"I am thrilled about it. That's why we have been working so hard to get this as quickly as possible through the necessary experiments, so we can publish our findings and take it out to the patients," Naash said. "We hope the results of our study will be instrumental in generating a cure for the debilitating blindness associated with and other inherited and acquired retinal diseases. We want to give Oklahomans and others suffering from these diseases renewed independence and quality of life."

Utilizing nanoparticle technology, scientists created a microscopic capsule capable of carrying genetic therapies to their destination inside cells of the retina. The tiny delivery vehicle is being tested with a variety of gene therapies in animal models with the potential of treating several diseases from to diabetes. The capsules have proven very effective, carrying therapies to the designated location in the eye within 15 minutes of delivery and spreading the genetic repair message quickly to nearby cells.

"This is an incredible breakthrough in terms of being able to treat with gene therapy," said Robert E. Leonard, M.D., an ophthalmologist at the Dean McGee Eye Institute. "Outside of , we are at a loss to be able to treat these patients, so this is incredibly important research. It's breathtaking, very exciting."

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2 comments

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fixer
5 / 5 (2) Jun 12, 2010
Let's hope this one gets off the ground running.
A lot of people are waiting for this.
OzkarNiftybootz
3 / 5 (2) Jun 12, 2010
We need to have a cure for Retinitis Pigmentosa and other blinding diseases by 2020. I hope this would entail a reversal of previous genetic retinal damage as well as stopping the degeneration process itself.

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