Embryonic Stem Cells for Corneal Endothelial Degeneration

Here we proposed to do foundational research towards developing a stem cell-based therapy corneal diseases. We successfully completed all proposed experiments. Through Aim 1 we discovered that corneal endothelium-like cells could be generated by differentiation of human stem cells. Novel compounds were tested that were discovered to improve the cells' morphology and function, with or without affecting cells’ proliferation and migration. Taking this data forward into Aim 2 for in vivo experiments in a relevant animal model, we explored successfully human stem cell-to-corneal endothelial cell differentiation and transplantation, and discovered a risk of immune rejection not previously anticipated. These data suggest that for proof-of-concept studies in the future, immunosuppression may be preferred to move such cell therapy through future proof-of-concept (POC) and FDA-enabling studies.