Neurotrophic Biofactories for the Treatment of Glaucoma

Anna Demetriades, MD, PhD Joan and Sanford I. Weill Medical College of Cornell University


Glaucoma is the result of retinal ganglion cell (RGC) death in the eye which ultimately leads to vision loss in patients. RGCs are the nerve cells that extend down the optic nerve and connect the eye to the brain. The aim of this study is to develop a novel treatment for patients by identifying a new way to slow down and prevent RGC death. Dr. Demetriades’ team will use gene therapy to deliver neuroprotective proteins to specific cells within the retina and determine whether it is best to deliver these proteins directly to RGCs or to adjacent cells in order to optimally improve their survival.

Project Details

The goal of this project is to develop a new gene therapy treatment for glaucoma patients aimed at slowing down or stopping disease progression.

Dr. Demetriades’ team will use a gene therapy approach to deliver neuroprotective proteins to specific cells within the retina. Retinal ganglion cell (RGC) death is the hallmark of glaucoma. The gene therapy method will deliver therapeutic proteins to RGCs and adjacent cells within the retina to improve their survival.

The innovative aspect of this proposal is the use of a cell-specific gene therapy approach. Dr. Demetriades will develop gene therapy carrier systems that enable delivery of a therapeutic protein to a specific cell type within the retina. This research could lead to the creation of a novel delivery system for the eye.

Current treatments control eye pressure but vision loss may still occur. Dr. Demetriades’ study will provide new insights into the potential of cell-specific gene therapy for the treatment of glaucoma. Their goal through this work is to develop an adjuvant therapy that slows or stops disease progression in order to prevent vision loss.