Mina Pantcheva, MD

Gene Therapy for Glaucoma

This project is dedicated to addressing the critical requirement for novel therapeutic solutions aimed at preventing the degeneration of retinal ganglion cells (RGCs) and safeguarding vision in individuals with glaucoma, a leading cause of blindness globally. Current IOP-lowering therapies have limitations, and about 10% of patients do not respond to them. The researchers propose an innovative approach utilizing adeno-associated virus serotype 2 (AAV-2) for targeted delivery of small heat shock proteins (sHSPs) specifically to RGCs, as sHSPs have anti-apoptotic, antioxidant, and anti-inflammatory properties. 

The core objective is to explore the potential of AAV-2-mediated expression of sHSPs in shielding RGCs within animal models of glaucoma. This investigation is undertaken with the ultimate objective of developing a novel therapeutic strategy to combat this vision-threatening disease. Collaborators: Ram Nagaraj, PhD and Mi-Hyun Nam, PhD

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