人类胚胎干细胞(hESC)和诱导多能干细胞(hiPSC)衍生的视网膜类器官(RO)是三维层状结构,可概括人类视网膜的发育轨迹。RO为基础科学研究提供了一个迷人的工具,眼部疾病建模,治疗发展,和用于组织/细胞置换的生物样本。在这里,我们回顾了以前为RO技术铺平了道路的研究,最广泛接受的两个,生成RO的标准化协议,以及RO在医学发现中的应用。本文从基础科学研究的角度进行综述,再生医学移植,疾病建模,以及药物筛选和基因治疗的治疗开发。RO为组装等新技术开辟了道路,与其他类器官共培养,脉管系统或免疫细胞,微流体装置(芯片上的器官),用于药物递送的细胞外囊泡,生物材料工程,先进的成像技术,人工智能(AI)。然而,目前RO的一些缺点限制了它们在医学应用中的翻译,并对未来的研究提出了挑战。尽管有这些限制,RO是视网膜疾病功能研究和治疗策略的强大工具。
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.