TGF-β and Glaucoma Progression in a Spontaneous Model

Project Summary

A growing body of evidence implicates a chemical growth factor, TGF-β, in damage to the optic nerve at the back of the eye and subsequent loss of vision in glaucoma. The goal of our research is to repurpose an existing drug to target TGF-β to slow or prevent permanent vision loss.

In our first aim, we will assess the ability of an oral medication that affects TGF-β signaling, to prevent progressive damage to the optic nerve. To accomplish this aim, we will study effects of treatment on optic nerve structure and function in a highly relevant large animal model of inherited glaucoma that has many similarities to human disease. 

In our second aim, we will determine the effects of this same medication on expression of different genes and proteins in the eye tissues that are implicated in this scarring process. Our results will aid in confirmation and discovery of genes and pathways that contribute to loss of vision in glaucoma, and will provide insight into how these genes and pathways are modified by therapy.

Our studies will provide proof of concept that an existing drug can be repurposed to target TGF-β, as a complement to conventional treatments, and will accelerate transfer of this innovative strategy to clinical trials in human patients in order to slow or prevent the inexorable vision loss from glaucoma that many people currently face.