New Therapeutic Approach May Help Treat Age-Related Macular Degeneration (AMD) Effectively–Inhibiting Gene (RUNX1) Involved in Abnormal Growth of Blood Vessels in Certain Ocular Disorders May Reduce Retinal Neovascularization

Runt-related transcription factor 1 (RUNX1) has been linked to retinal neovascularization and the development of abnormal blood vessels, which result in vision loss in diabetic retinopathy. Now, scientists have found that RUNX1 inhibition presents a new therapeutic approach in the treatment of age-related macular degeneration (AMD), which is the leading cause of blindness in the elderly worldwide. The results were first reported online on December 23, 2020 are published in the March 1, 2021 issue of The American Journal of Pathology, published by Elsevier ( The open-access article is titled “Treatment of Experimental Choroidal Neovascularization via RUNX1 Inhibition.” Abnormal growth of blood vessels, or aberrant angiogenesis, arises from the choroid, a part of the eye located behind the retina. This condition, known as choroidal neovascularization (CNV), is present in several ocular diseases that lead to blindness such as AMD. This study is the first to implicate RUNX1 in CNV and to test RUNX1 inhibition therapy for treating CNV. Researchers found that application of a RUNX1 inhibitor, alone or in combination with a standard treatment for AMD, may represent an important therapeutic advance. “Incomplete response to anti–vascular endothelial growth factor (VEGF) drugs is a critical problem that hinders visual outcomes in CNV. RUNX1 represents a promising therapeutic target that may help address current limitations of anti-VEGF therapy,” explains first author Lucia Gonzalez-Buendia, MD, a retina specialist at Puerta de Hierro-Majadahonda University Hospital (Spain), and former postdoctoral fellow at the Schepens Eye Research Institute of Mass Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, Massachusetts, USA.
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