Gene Therapy Used in Retinitis Pigmentosa

OKLAHOMA CITY, June 15 (UPI) — U.S. medical investigators say they have developed a gene therapy technique that can prevent and possibly cure blindness caused by retinitis pigmentosa.

University of Oklahoma Health Sciences Center researchers said they found a way to use a radical new type of gene therapy that gives hope to the estimated 100,000 U.S. citizens who suffer from retinitis pigmentosa — an eye disease in which genetic damage to the retina gradually becomes worse and can eventually lead to blindness.


Researchers, led by Professor Muna Naash, discovered a way to deliver gene therapies directly to the light-sensitive cells affected by the disease. The scientists said their discovery already is being used to develop new treatments for another disease — macular degeneration — which is the leading cause of blindness in the United States.

Utilizing nanoparticle technology, Naash and her team created a microscopic capsule capable of carrying genetic therapies to 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 other diseases from bladder cancer to diabetes.

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

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