多能干细胞衍生视网膜疾病模型:阐明发病机制、评估新型疗法和估计毒性

IF 18.6 1区 医学 Q1 OPHTHALMOLOGY Progress in Retinal and Eye Research Pub Date : 2024-02-16 DOI:10.1016/j.preteyeres.2024.101248
Marzena Kurzawa-Akanbi , Nikolaos Tzoumas , Julio C. Corral-Serrano , Rosellina Guarascio , David H. Steel , Michael E. Cheetham , Lyle Armstrong , Majlinda Lako
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引用次数: 0

摘要

失明是一个日益严峻的全球性挑战,约 26% 的失明病例归因于退行性视网膜疾病。虽然基因疗法、光遗传学工具、光敏开关和视网膜假体为视力恢复带来了希望,但这些高成本疗法只能惠及少数患者。因此,了解视网膜疾病是推进有效治疗的关键,这就需要复制病理的体外模型,并对药物发现进行定量评估。多能干细胞(PSCs)提供了一种独特的解决方案,因为它们具有无限的供应能力和分化成包括所有细胞类型的光反应视网膜组织的能力。本综述重点介绍利用多能干细胞生成感光细胞和视网膜色素上皮细胞(RPE)的历史和现状。我们探讨了这项技术在疾病建模、实验治疗测试、生物标记物鉴定和毒性研究中的应用。我们考虑了可扩展性、标准化和可重复性方面的挑战,并强调了将血管和免疫细胞纳入视网膜有机体的重要性。我们提倡在数据采集和分析中实现高通量自动化,并强调先进微观生理系统的价值,该系统能充分捕捉神经视网膜、RPE 和绒毛膜之间的相互作用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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Pluripotent stem cell-derived models of retinal disease: Elucidating pathogenesis, evaluating novel treatments, and estimating toxicity

Blindness poses a growing global challenge, with approximately 26% of cases attributed to degenerative retinal diseases. While gene therapy, optogenetic tools, photosensitive switches, and retinal prostheses offer hope for vision restoration, these high-cost therapies will benefit few patients. Understanding retinal diseases is therefore key to advance effective treatments, requiring in vitro models replicating pathology and allowing quantitative assessments for drug discovery. Pluripotent stem cells (PSCs) provide a unique solution given their limitless supply and ability to differentiate into light-responsive retinal tissues encompassing all cell types. This review focuses on the history and current state of photoreceptor and retinal pigment epithelium (RPE) cell generation from PSCs. We explore the applications of this technology in disease modelling, experimental therapy testing, biomarker identification, and toxicity studies. We consider challenges in scalability, standardisation, and reproducibility, and stress the importance of incorporating vasculature and immune cells into retinal organoids. We advocate for high-throughput automation in data acquisition and analyses and underscore the value of advanced micro-physiological systems that fully capture the interactions between the neural retina, RPE, and choriocapillaris.

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来源期刊
CiteScore
34.10
自引率
5.10%
发文量
78
期刊介绍: Progress in Retinal and Eye Research is a Reviews-only journal. By invitation, leading experts write on basic and clinical aspects of the eye in a style appealing to molecular biologists, neuroscientists and physiologists, as well as to vision researchers and ophthalmologists. The journal covers all aspects of eye research, including topics pertaining to the retina and pigment epithelial layer, cornea, tears, lacrimal glands, aqueous humour, iris, ciliary body, trabeculum, lens, vitreous humour and diseases such as dry-eye, inflammation, keratoconus, corneal dystrophy, glaucoma and cataract.
期刊最新文献
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