Looking for a cure

OUHSC researcher receives grant for blindness research

Tuesday, August 28, 2012

by Sarah Terry-Cobo

The Journal Record

OKLAHOMA CITY -- Muna Naash has been researching causes of and possible treatments for blindness at the University of Oklahoma Health Sciences Center since 2000. She recently received a $300,000 grant that she said she hopes will take her research from the laboratory to animal trials, the step necessary before human clinical trials.

The three-year grant from the Foundation Fighting Blindness will help provide funding for research on a type of hereditary blindness that is incurable.

"Because of the (genetic) mutations, the damaged proteins (are) unavailable or don't form," Naash said. "If we deliver that missing gene or protein, we can correct the situation."

The grant helps identify the process to deliver that missing gene for the particular genetic mutation called Usher-2A, which is a type of vision loss that is accompanied with hearing loss. According to the National Institutes of Health, severe type II Usher syndrome can lead to retinitis pigmentosa, vision loss that includes decreased night and peripheral vision and, in advanced cases, complete vision loss.

Naash's research is important because although retinitis pigmentosa is a rare condition affecting about 1 in 4,000 people in the U.S., there is no effective treatment for the disease. Naash said she hopes her lab's research can discover a permanent treatment through the use of gene therapy. Because that portion of the retina is already damaged, her lab is working on a way to deliver gene therapy in a single injection, rather than continued treatment.

"Our attempt is to go to the soul (of the problem), rather than patch it," she said. "Even when you deliver regular drugs, you have to deliver them regularly, all the time. Our therapy is (delivered) once, all the same time; that is the beauty of gene therapy."

Naash and her team are working on a number of research projects for several types of diseases that cause blindness, including diabetes. The most recent grant she received is only a portion of the funds necessary to complete the research project on Usher syndrome, Naash said. Before Naash's team can advance to clinical trials, they must test on baboons, the closest non-primate mammals with similar eye structures to humans.

Though the OU Health Sciences Center has one of the largest baboon test facilities in the nation, testing on these animals is expensive: $8,000 per baboon. She said she hopes that the work the lab will do in the next three years will produce results that can enable her to receive funding from the National Institutes of Health.