Treatment of Corneal Blindness Using Human Stem Cell Derived Tissue

Funding Type: 
New Faculty Physician Scientist
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

The goal of this proposal is to develop stem cell based therapies for treatment of blindness due to corneal endothelial dysfunction. Cornea is the anterior-most, transparent structure of the eye, and the corneal endothelium, the inner layer of the cornea, functions as a pump that is necessary for maintaining corneal transparency and clear vision. Corneal endothelial dysfunction, a frequent cause of corneal blindness, is a common indication for corneal transplantation in developed nations. The latest eye bank statistics show that over 60,000 donor corneas were made available for transplantation in 2010 in the US, and the demand is expected to rise due to increasing age of the baby boomers. Stem cell based therapies offers a means of addressing the impending rise in demand fro donor corneas, but it is hindered by poorly understood corneal endothelial cell (CEC) development. This proposal will investigate the feasibility of using CEC precursors derived from human embryonic stem cells for transplantation. The CEC precursors will be identified from spontaneously differentiated hESC, grown in the laboratory, and tested to determine their utility for transplantation. The results of this proposal can potentially provide an unlimited supply of CEC for treatment of blindness secondary to corneal endothelial dysfunction.

Statement of Benefit to California: 

California, in addition to being the most populous state, is also the state with the highest number of Cornea & External Disease subspecialists in the union. This indicates that there is a substantial population of patients with cornea problems in California, many of whom need cornea transplants. Successful translation of the results of this proposal will result in increased availability of corneas for transplantation, directly benefiting the people of California needing cornea transplants. This proposal will also help the state economy by relying on California suppliers whenever possible.