A groundbreaking new study by John A. Moran Eye Center researcher Mary Elizabeth Hartnett, MD, moves physicians one step closer to better treatments for premature babies at risk for retinopathy of prematurity (ROP)—the leading cause of childhood vision loss and blindness.
Current therapies use anti-vascular endothelial growth factor (VEGF) treatments to curb ROP by inhibiting abnormal blood vessel growth in the eye. The problem: VEGF also plays an important role in the development of the retina—and the infant.
In a new study published this week in Angiogenesis, Gene Therapy Knockdown of VEGFR2 in Retinal Endothelial Cells to Treat Retinopathy, Hartnett tested a more targeted approach. Hartnett used gene therapy to knockdown VEGF receptor 2 in endothelial cells in a model of oxygen-induced retinopathy. The approach inhibited abnormal blood vessel growth, but allowed the retina to continue to develop with no alterations in its function.
"These studies provide insights into mechanisms and safety in the management of ROP," said Hartnett. "More work is needed to find safe drug delivery approaches and to clearly discern the mechanisms of action through the receptor—this method will now enable us to test mechanisms in a model highly representative of human ROP. Furthermore, the outcomes may translate to other retinopathies that are related to overactivation of VEGF signaling, including diabetic retinopathy."
In addition to Hartnett, the study was conducted by Moran researchers Aaron B. Simmons, PhD; Colin A. Bretz, PhD; Haibo Wang, PhD; Eric Kunz; Kassem Hajj; Carson Kennedy; and Zhihong Yang. Other collaborators included Thipparat Suwanmanee, PhD, and Tal Kafri, MD, PhD from the University of North Carolina at Chapel Hill.
The study was funded by a grant from the National Eye Institute and an unrestricted grant from Research to Prevent Blindness.