Human pluripotent stem cells: A toolbox to understand and treat retinal degeneration

Age-related Macular Degeneration (AMD) and Retinitis Pigmentosa (RP) are retinal degenerative disorders that dramatically damage the retina. As there is no therapeutic option for the majority of patients, vision is progressively and irremediably lost. Owing to their unlimited renewal and potency to give rise to any cell type of the human adult body, human pluripotent stem cells (hPSCs) have been extensively studied in recent years to develop more physiologically relevant in vitro cellular models. Such models open new perspectives to investigate the pathological molecular mechanisms of AMD and RP but also in drug screening. Moreover, proof-of-concept of hPSC-derived retinal cell therapy in animal models have led to first clinical trials. This review outlines the recent advances in the use of hPSCs in pathological modeling of retinal degeneration and their use in regenerative medicine. We also address the associated limitations and challenges that need to be overcome when using hPSCs. •Pathological mechanisms of AMD and RP are not fully understood.•Patient hPSC derived retinal cells recapitulate the hallmarks of AMD and RP.•AMD/RP hPSC retinal cells could be used for drug screening.•hPSC-RPE cell therapy is currently evaluated in clinical trials.•hPSC derived PR therapy needs improvements before clinical evaluation.