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Saturday, May 26, 2012
OUHSC researchers seek to restore sight
by   |  June 14, 2010  |  

For 40-year-old Tracie Harris, happiness is as simple as being able to see lightning bugs and stars again.

A debilitating eye disease known as retinitis pigmentosa has taken away not only her vision, but also her ability to do and enjoy many of the things she always has in her life.

But now, thanks to OU Health Sciences Center researcher Muna Naash and her team, a non-viral gene therapy utilizing nanoparticle technology is giving Harris and others hope to stop and reverse the damage of the hereditary disease.

Harris, like others, first discovered her ailment during childhood. Her mother also had the disease.

With no treatment options, the Oklahoma resident said she could see her vision deteriorating as she got older.

“When I go to a movie theater, I can see the lights but I can’t see the stairs. I have to have someone help me,” she said. “One thing I miss is seeing lightning bugs and stars. But you learn to take the bumps and the bruises.”

The disease blinds an estimated 100,000 Americans, according to a press release.

“This strategy of non-viral gene therapy that we are using is very efficient,” Naash said at a press conference June 9. “There are so many ways we could use this technology.”

Scientists created a microscopic capsule capable of carrying genetic therapies to their destination inside cells of the retina within 15 minutes, according to the release.

This technique could eventually be used to treat diabetes and certain types of cancers. It also is been used effectively to treat cystic fibrosis and some brain diseases in the laboratory, but hasn’t yet moved to patients, Naash said.

“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 want to give Oklahomans and others suffering from these diseases renewed independence and quality of life.”

There are many different genes involved in retinitis pigmentosa, and it can be a mild or very aggressive form, said Robert E. Leonard, an ophthalmologist at the Dean McGee Eye Institute.

“The disease typically manifests itself in early adulthood,” Leonard said. “Symptoms such as night blindness and loss of peripheral vision is noticed.”

Naash said she is hopes this treatment process will be taken to patients in the near future. She said the research has been an outcome of many years of hard work.

Leonard said the development is a breakthrough.

“This disease currently is managed with no specific treatment, other than vitamin treatment, which is controversial,” Leonard said.

Harris said she looks forward to the days when she can recover some of the vision she has been missing.

“To know that something like this is happening, it’s a great thing,” Harris said.

For more information on retinitis pigmentosa, visit www.nlm.nih.gov.

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