GENE THERAPY FOR RETINOPATHY OF PREMATURITY

Retinopathy of prematurity (ROP) is a leading cause of childhood blindness worldwide. It is becoming more common as emerging countries develop technology to save preterm infants, but lack resources to provide optimal care. In the United States, 14 percent of childhood blindness is attributed to ROP, while in some developing nations estimates surpass 20 percent. Treatments of severe ROP include 1) laser ablation of peripheral avascular retina, which destroys developing retina; or 2) intravitreal anti- VEGF agents, which can lead to persistent avascular retina and even blindness.

The University of Utah is developing a gene therapy based novel approach by targeting STAT3, that unlike anti-VEGF treatment, will not interfere with physiologic retinal vascular development. This approach would stop the growth of abnormal blood vessels in the eye, prevent retinal detachment, and preserve vision.