Induced to cure: Engineering iPS cell derived RPE scaffolds to treat degenerative eye diseases

The recent success with embryonic stem (ES) cell derived retinal pigment epithelium (RPE) has provided hope for a cure for degenerative eye diseases. Induced pluripotent stem (iPS) cells are an autologous source of stem cells potentially with fewer immune-challenges as compared to ES cells. Using a developmentally guided differentiation protocol we have developed fully polarized RPE tissue from iPS cells. The RPE monolayer along with its secreted ECM and a degradable scaffold form a tissue that mimics the native tissue in structural and functional properties. This RPE tissue performs several key RPE functions like phagocytosis of photoreceptor outer segments, ability to transport water from apical to basal side, and the ability to secrete cytokines in a polarized fashion. Currently, we are testing the safety and the efficacy of this tissue in animal models. We have begun Phase I Investigational New Drug (IND) enabling studies with the goal to transplant autologous iPS cell derived RPE in patients in advanced Geographic Atrophy stage of age-related macular degeneration (AMD), one of the leading blinding diseases in the US. Our work will provide a potential personalized cell therapy for AMD patients.