Dr. Srinivasa Sripathi from Johns Hopkins University School of Medicine awarded $70,000 Knights Templar Eye Foundation Grant for Leber Congenital Amaurosis Research

Dr. Srinivasa Sripathi from the Wilmer Ophthalmological Institute, Johns Hopkins University School of Medicine was awarded a $70,000 grant entitled: Inhibiting Epithelial Mesenchymal Transition (EMT) as an Approach to Improving RPE Transplantation for Treatment of the Juvenile Macular Dystrophies.

Leber congenital amaurosis (LCA) and stargardt disease are the most common forms of inherited retinal dystrophies in children characterized by severe vision loss or blindness at birth. Primarily, vision impairment occurs due to progressive degeneration and death of photoreceptor (PR) and retinal pigment epithelium (RPE). For afflicted children, this disease can be devastating and there are currently no treatments to prevent, let alone cure this disease. However, in last decade there has been much excitement in the development of human stem cell derived retinal cell transplantation clinical trial approaches to treat these diseases.

In this application, Dr. Sripathi will focus his efforts to help improve the preparation of human stem cell derived RPE cell for transplantation for the treatment of stargardt disease and LCA. RPE cells can also be transplanted on a substrate, but indeed, even a mild perturbation of the monolayer or cell environment can induce hRPE de-differentiation and epithelial to mesenchymal transition (EMT), which includes loss of the stereotypical RPE cobblestone morphology.

In order to aid in the treatment of childhood blindness, he will propose to use the tools we have developed to study hRPE EMT to further develop and apply small molecule EMT inhibitors to help improve the safety and efficacy of hRPE cells for the treatment of diseases such as stargart disease and LCA. His goal is to inhibit enzymatic dissociation induced RPE EMT using bioactive small molecules and better use them for sub-retinal transplantation.