Pluripotent stem cell-based models to investigate retinal pigmented epithelium function and disease

Animal models do not always recapitulate or translate to human diseases. Pluripotent stem cells provide a self-renewing resource to generate mature cell types, including those of the retina. Furthermore, induced pluripotent stem cells derived from patients with genetic diseases can be differentiated into specialized cells to create in vitro disease models with precise pathogenic genotypes. Recent progress in deriving functional RPE cells from these disease-carrying stem cells in vitro can provide insights into disease pathobiology, thus providing a platform for discovery of new pharmacological interventions.