Retinitis Pigmentosa Blindness Prevented in Mice

A group of investigators has recently managed a breakthrough that is bound to bring hope to the millions of people suffering from a degenerative eye condition known as retinitis pigmentosa. The disease is characterized by dysfunctions in the retina's photoreceptor cells, which are shaped either as cones or rods. Night blindness and tunnel vision are common symptoms, and they are often followed by central vision loss later in life. In the new set of experiments, scientists managed to successfully test a new gene therapy that prevents complete blindness from settling in.

The work was conducted on unsuspecting mice models, all of which had conditions similar to retinitis pigmentosa. The results could directly affect more than 100,000 Americans who suffer from this set of genetic disorders, say researchers from the University of Oklahoma Health Sciences Center, who are behind the study. Details of their investigation appear in the latest issue of the esteemed scientific Journal of the Federation of American Societies for Experimental Biology (FASEB), PhysOrg reports.

“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. We hope the results of our study will be instrumental in generating a cure for the debilitating blindness associated with retinitis pigmentosa and other inherited and acquired retinal diseases. We want to give Oklahomans and others suffering from these diseases renewed independence and quality of life,” explains Muna Naash PhD, the UO expert that led the new investigation. Science teams from Cleveland and Buffalo also contributed to the work.

The group explains that their new approach is already undergoing various adaptations, which would make it fit for use in patients suffering from another widespread disease, macular degeneration. “This is an incredible breakthrough in terms of being able to treat with gene therapy. Outside of gene therapy, we are at a loss to be able to treat these patients, so this is incredibly important research. It's breathtaking, very exciting,” concludes Dean McGee Eye Institute ophthalmologist Robert E. Leonard, MD. The active ingredients in the new therapy are delivered to the retina encase in nanoscale capsules, which are able to make the “trip” within less than 15 minutes, the researchers say.