研究动态
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使用人类诱导多能干细胞衍生的感光细胞和视网膜色素上皮细胞模拟遗传性视网膜疾病。

Modeling inherited retinal diseases using human induced pluripotent stem cell derived photoreceptor cells and retinal pigment epithelial cells.

发表日期:2024
作者: Ivan Seah, Debbie Goh, Animesh Banerjee, Xinyi Su
来源: Stem Cell Research & Therapy

摘要:

自从发现诱导多能干细胞 (iPSC) 技术以来,人们进行了许多尝试来创建遗传性视网膜疾病 (IRD) 的细胞模型,用于研究致病过程,以促进靶点发现和验证活动。一致性仍然是确定这些发现的效用的关键。尽管一致性很重要,但质量控制指标仍未得到广泛使用。在这篇综述中,提供了一个利用 iPSC 技术生成光感受器、视网膜色素上皮细胞和类器官疾病模型的工具包。讨论了开发 iPSC 衍生 IRD 模型时的注意事项,例如 iPSC 起源、重编程方法、质量控制指标、控制策略和分化协议。对各种 iPSC IRD 模型进行了剖析,并讨论了基于 iPSC 的疾病建模的科学障碍,以概述该领域的当前方法和未来方向。版权所有 © 2024 Seah、Goh、Banerjee 和 Su。
Since the discovery of induced pluripotent stem cell (iPSC) technology, there have been many attempts to create cellular models of inherited retinal diseases (IRDs) for investigation of pathogenic processes to facilitate target discovery and validation activities. Consistency remains key in determining the utility of these findings. Despite the importance of consistency, quality control metrics are still not widely used. In this review, a toolkit for harnessing iPSC technology to generate photoreceptor, retinal pigment epithelial cell, and organoid disease models is provided. Considerations while developing iPSC-derived IRD models such as iPSC origin, reprogramming methods, quality control metrics, control strategies, and differentiation protocols are discussed. Various iPSC IRD models are dissected and the scientific hurdles of iPSC-based disease modeling are discussed to provide an overview of current methods and future directions in this field.Copyright © 2024 Seah, Goh, Banerjee and Su.