Use of human pluripotent stem cells to study and treat retinopathies

Figure 1 Schematic diagram summarizing the issues to consider when modeling retinal diseases with human pluripotent stem cells. Choice of disease (monogenic vs polygenic); Type of pluripotent stem cells (hESCs and/or iPSCs); for iPSCs: selection of cell type to reprogram (fibroblasts, keratinocytes, blood cells), selection of reprogramming method (retrovirus mediated, non-integrative); choice of quality control for iPSCs characterization (genetic analysis, pluripotency, teratoma assay); differentiation to relevant cell types (photoreceptors, RPE); disease phenotype characterization (molecular characterization, expression studies, genetic rescue); applications (drug screening, toxicological testing, new molecular mechanisms). hESCs: Human embryonic stem cells; iPSCs: Induced pluripotent stem cells; RPE: Retinal pigment epithelium.