Department of Ophthalmology, Karalis Johnson Retina Center, University of Washington, Seattle, Washington.
Department of Ophthalmology, Seattle Children's Hospital, Seattle, Washington.
Retina. 2022 Oct 1;42(10):1829-1835. doi: 10.1097/IAE.0000000000003571.
To provide a concise review of induced pluripotent stem cells (iPSCs) and retinal organoids as models for human retinal diseases and their role in gene discovery and treatment of inherited retinal diseases (IRDs).
A PubMed literature review was performed for models of human retinal disease, including animal models and human pluripotent stem cell-derived models.
There is a growing body of research on retinal disease using human pluripotent stem cells. This is a significant change from just a decade ago when most research was performed on animal models. The advent of induced pluripotent stem cells has permitted not only the generation of two-dimensional human cell cultures such as RPE but also more recently the generation of three-dimensional retinal organoids that better reflect the multicellular laminar architecture of the human retina.
Modern stem cell techniques are improving our ability to model human retinal disease in vitro, especially with the use of patient-derived induced pluripotent stem cells. In the future, a personalized approach may be used in which the individual's unique genotype can be modeled in two-dimensional culture or three-dimensional organoids and then rescued with an optimized therapy before treating the patient.
简要回顾诱导多能干细胞（iPSCs）和视网膜类器官作为人类视网膜疾病模型及其在基因发现和遗传性视网膜疾病（IRDs）治疗中的作用。
对包括动物模型和人多能干细胞衍生模型在内的人类视网膜疾病模型进行了 PubMed 文献回顾。
利用人多能干细胞进行视网膜疾病研究的文献越来越多。这与仅仅十年前大多数研究都在动物模型上进行的情况相比有了显著的变化。诱导多能干细胞的出现不仅允许生成二维的人细胞培养物，如 RPE，而且最近还允许生成更能反映人视网膜的多层层状结构的三维视网膜类器官。
现代干细胞技术正在提高我们在体外模拟人类视网膜疾病的能力，特别是使用患者来源的诱导多能干细胞。未来，可能会采用个性化方法，在二维培养物或三维类器官中对个体独特的基因型进行建模，然后用优化的治疗方法进行挽救，再对患者进行治疗。
