Two translational studies at the Vanderbilt Eye Institute are targeting photoreceptors and retinal ganglion cells (RGCs) to restore vision through regeneration of the retina. The research is funded by separate, five-year grants from the Audacious Goals Initiative (AGI), an ongoing program of the National Eye Institute.
Vision loss due to RGC death is irreversible, leading to blindness in a high percentage of diagnosed patients. Vision restoration through cell replacement therapy, a major focus of the AGI, could be a potential solution.
“The ultimate goal is to inject these stem cell-derived ganglion cells into the eye, form connections and really integrate into the retina,” said David Calkins, Ph.D., Denis M. O’Day Professor, vice chair and director for research at the Vanderbilt Eye Institute. He is leading an AGI U24 grant to develop translation-enabling models for evaluating survival and integration of regenerated photoreceptors and RGCs.
Tonia Rex, Ph.D., associate vice chair for research at the Vanderbilt Eye Institute, is lead principal investigator on another U24 grant. The objective of her study is to optimize allogenic transplantation of induced pluripotent stem cell derived RGCs in two different models of optic neuropathy.
“Understanding the pathophysiologies of these two optic neuropathies is going to be important to the success of the cell replacement therapy,” Rex said. “There are a lot of challenges to overcome, but the exciting thing is how much we have learned as a field and how many tools we now have available to make it actually feasible.”