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.

年龄相关性黄斑变性 (AMD) 和色素性视网膜炎 (RP) 是严重损害视网膜的视网膜退行性疾病。由于大多数患者没有治疗选择，视力会逐渐且不可修复地丧失。由于人类多能干细胞 (hPSC) 具有无限的更新能力和产生成人身体任何细胞类型的能力，近年来已对其进行了广泛的研究，以在体外开发更具生理相关性的细胞细胞模型。这些模型为研究 AMD 和 RP 的病理分子机制以及药物筛选开辟了新的视角。此外，动物模型中 hPSC 衍生的视网膜细胞疗法的概念验证已导致首次临床试验。本综述概述了 hPSC 在视网膜变性病理模型中的使用及其在再生医学中的应用的最新进展。我们还解决了使用 hPSC 时需要克服的相关限制和挑战。