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使用人类诱导的多能干细胞衍生光受体细胞和视网膜色素上皮细胞模型遗传视网膜疾病

Ivan Seah^1,2,3, Debbie Goh⁴, Animesh Banerjee¹

¹Translational Retinal Research Laboratory, Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (A*STAR), Singapore, Singapore.

Frontiers in medicine

|July 16, 2024

概括

诱导多能干细胞 (iPSC) 技术使遗传视网膜疾病 (IRD) 的建模成为可能. 本综述提供了一个工具包,并讨论了一致和可靠的iPSC衍生IRD模型的质量控制.

科学领域:

干细胞生物学干细胞生物学
眼科医生眼科眼科
遗传学是一个遗传学.

背景情况:

诱导多能干细胞 (iPSC) 技术已经彻底改变了疾病建模.
遗传性视网膜疾病 (IRD) 的细胞模型对于理解病原和药物发现至关重要.
一致性和质量控制对于iPSC衍生疾病模型的实用性至关重要,但经常被忽视.

研究的目的:

为生成iPSC衍生的光受体,视网膜色素上皮细胞和IRDs的器官模型提供一个全面的工具包.
讨论开发可靠和可重复的基于iPSC的IRD模型的关键考虑因素.
审查当前的方法,确定科学障碍,并概述基于iPSC的IRD建模的未来方向.

主要方法:

对iPSC衍生和差异化的现有文献和方法的审查.
对基于iPSC的疾病建模质量控制指标和策略的分析.
剖析各种iPSC衍生的IRD模型及其应用.

主要成果:

介绍了一套用于生成iPSC衍生的光受体,RPE和有机体模型的工具包.
讨论了包括iPSC起源,重编程,质量控制,控制和差异化在内的重要考虑因素.
分析了当前基于iPSC的IRD模型,突出了它们的优点和局限性.

结论:

标准化质量控制指标对于提高iPSC衍生的IRD模型的可靠性至关重要.
解决基于iPSC的疾病建模中的科学障碍将加速IRD的目标发现和验证.
本综述为利用iPSC技术创造有效的视网膜遗传疾病细胞模型提供了一份路线图.

关键词:

诱导的多能干细胞干细胞.遗传性视网膜疾病遗传性视网膜疾病有机生物有机物摄影受体细胞是一种光受体细胞.视网膜色素表皮质是视网膜色素表皮质.

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