人类胚胎干细胞（hESCs）和诱导多能干细胞（hiPSCs）衍生的视网膜器官样结构（ROs）是三维层状结构，能重现人类视网膜的发育轨迹。ROs为基础科学研究、眼病模型构建、治疗开发和组织/细胞替代生物库提供了一个迷人的工具。在本文中，我们回顾了为RO技术铺平道路的先前研究，介绍了最广泛认可的两个标准化生成ROs的方案，并讨论了ROs在医学发现中的应用。本综述从基础科学研究、再生医学中的移植、疾病建模以及药物筛选和基因疗法的治疗开发等多个角度进行。ROs为新技术开辟了道路，如组装体、与其他器官样结构共培养、血管系统或免疫细胞、微流控装置（器官-芯片）、细胞外囊泡用于药物传递、生物材料工程、先进成像技术和人工智能（AI）。然而，目前ROs的一些缺点限制了其在医学应用中的转化，并对未来的研究提出了挑战。尽管存在这些局限性，ROs仍然是研究视网膜疾病的功能性研究和治疗策略的强大工具。© 2023. 作者，独家授权给Springer Nature Switzerland AG。

Human embryonic stem cells (hESCs)- and induced pluripotent stem cells (hiPSCs)-derived retinal organoids (ROs) are three-dimensional laminar structures that recapitulate the developmental trajectory of the human retina. The ROs provide a fascinating tool for basic science research, eye disease modeling, treatment development, and biobanking for tissue/cell replacement. Here we review the previous studies that paved the way for RO technology, the two most widely accepted, standardized protocols to generate ROs, and the utilization of ROs in medical discovery. This review is conducted from the perspective of basic science research, transplantation for regenerative medicine, disease modeling, and therapeutic development for drug screening and gene therapy. ROs have opened avenues for new technologies such as assembloids, coculture with other organoids, vasculature or immune cells, microfluidic devices (organ-on-chip), extracellular vesicles for drug delivery, biomaterial engineering, advanced imaging techniques, and artificial intelligence (AI). Nevertheless, some shortcomings of ROs currently limit their translation for medical applications and pose a challenge for future research. Despite these limitations, ROs are a powerful tool for functional studies and therapeutic strategies for retinal diseases.© 2023. The Author(s), under exclusive license to Springer Nature Switzerland AG.