PURPOSE To provide a concise review of induced pluripotent stem cells (iPSCs) and retinal organoids (ROs) as models for human retinal diseases and their role in gene discovery and treatment… Click to show full abstract
PURPOSE To provide a concise review of induced pluripotent stem cells (iPSCs) and retinal organoids (ROs) as models for human retinal diseases and their role in gene discovery and treatment of inherited retinal diseases (IRDs). METHODS A Pubmed literature review was performed for models of human retinal disease, including animal models and human pluripotent stem cell-derived models. RESULTS There is a growing body of research on retinal disease utilizing human pluripotent stem cells. This is a significant change from just a decade ago when the vast majority of research was performed on animal models. The advent of iPSCs has permitted not only the generation of 2-dimensional human cell cultures such as RPE, but also more recently the generation of 3-dimensional retinal organoids that better reflect the multicellular laminar architecture of the human retina. CONCLUSION Modern stem cell techniques are improving our ability to model human retinal disease in vitro especially with the use of patient-derived iPSCs. In the future, a personalized approach may be employed in which the individual's unique genotype can be modeled in 2-dimensional culture or 3-dimensional organoids and then rescued with an optimized therapy prior to treating the patient.
               
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