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诱导多能干细胞模型与治疗外视网膜遗传性退行性疾病:3D 类器官的局限性与生物工程解决方案。

Inducible Pluripotent Stem Cells to Model and Treat Inherited Degenerative Diseases of the Outer Retina: 3D-Organoids Limitations and Bioengineering Solutions.

机构信息

Department of Biology, University of Pisa, 56127 Pisa, Italy.

Department of Pharmacy, University of Pisa, 56126 Pisa, Italy.

出版信息

Cells. 2021 Sep 20;10(9):2489. doi: 10.3390/cells10092489.

Abstract

Inherited retinal degenerations (IRD) affecting either photoreceptors or pigment epithelial cells cause progressive visual loss and severe disability, up to complete blindness. Retinal organoids (ROs) technologies opened up the development of human inducible pluripotent stem cells (hiPSC) for disease modeling and replacement therapies. However, hiPSC-derived ROs applications to IRD presently display limited maturation and functionality, with most photoreceptors lacking well-developed outer segments (OS) and light responsiveness comparable to their adult retinal counterparts. In this review, we address for the first time the microenvironment where OS mature, i.e., the subretinal space (SRS), and discuss SRS role in photoreceptors metabolic reprogramming required for OS generation. We also address bioengineering issues to improve culture systems proficiency to promote OS maturation in hiPSC-derived ROs. This issue is crucial, as satisfying the demanding metabolic needs of photoreceptors may unleash hiPSC-derived ROs full potential for disease modeling, drug development, and replacement therapies.

摘要

遗传性视网膜退行性疾病(IRD)影响光感受器或色素上皮细胞,导致进行性视力丧失和严重残疾,甚至完全失明。视网膜类器官(RO)技术为疾病建模和替代疗法的人类诱导多能干细胞(hiPSC)的发展开辟了道路。然而,hiPSC 衍生的 RO 在 IRD 中的应用目前显示出有限的成熟度和功能,大多数光感受器缺乏发育良好的外节(OS)和与成年视网膜相当的光响应性。在这篇综述中,我们首次讨论了 OS 成熟的微环境,即视网膜下腔(SRS),并讨论了 SRS 在光感受器代谢重编程中的作用,这是 OS 生成所必需的。我们还讨论了生物工程问题,以提高培养系统的效率,促进 hiPSC 衍生的 RO 中 OS 的成熟。这个问题至关重要,因为满足光感受器的高代谢需求可能会释放 hiPSC 衍生的 RO 在疾病建模、药物开发和替代疗法方面的全部潜力。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1e00/8471616/eb59a2bf3e5e/cells-10-02489-g001.jpg

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