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生成 ESC 衍生的 RGC 用于细胞替代治疗。

Generating ESC-Derived RGCs for Cell Replacement Therapy.

机构信息

Department of Ophthalmology, Louis J. Fox Center for Vision Restoration, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.

Department of Neurobiology, Center of Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.

出版信息

Methods Mol Biol. 2025;2848:187-196. doi: 10.1007/978-1-0716-4087-6_12.

DOI:10.1007/978-1-0716-4087-6_12
PMID:39240524
Abstract

In several ocular diseases, degeneration of retinal neurons can lead to permanent blindness. Transplantation of stem cell (SC)-derived RGCs has been proposed as a potential therapy for RGC loss. Although there are reports of successful cases of SC-derived RGC transplantation, achieving long-distance regeneration and functional connectivity remains a challenge. To address these hurdles, retinal organoids are being used to study the regulatory mechanism of stem cell transplantation. Here we present a modified protocol for differentiating human embryonic stem cells (ESCs) into retinal organoids and transplanting organoid-derived RGCs into the murine eyes.

摘要

在一些眼部疾病中,视网膜神经元的退化可能导致永久性失明。干细胞(SC)衍生的 RGC 移植已被提议作为治疗 RGC 损失的一种潜在疗法。尽管有报道称 SC 衍生的 RGC 移植成功的案例,但实现远距离再生和功能连接仍然是一个挑战。为了解决这些障碍,视网膜类器官正被用于研究干细胞移植的调节机制。在这里,我们提出了一种改良的方案,用于将人胚胎干细胞(ESCs)分化为视网膜类器官,并将类器官衍生的 RGC 移植到小鼠眼中。

相似文献

1
Generating ESC-Derived RGCs for Cell Replacement Therapy.生成 ESC 衍生的 RGC 用于细胞替代治疗。
Methods Mol Biol. 2025;2848:187-196. doi: 10.1007/978-1-0716-4087-6_12.
2
Retinal Ganglion Cell Transplantation: Differentiation and Quantification Strategies.视网膜神经节细胞移植:分化与定量策略。
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引用本文的文献

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Damage and repair in retinal degenerative diseases: Molecular basis through clinical translation.视网膜退行性疾病中的损伤与修复:从分子基础到临床转化
Neural Regen Res. 2026 Apr 1;21(4):1383-1395. doi: 10.4103/NRR.NRR-D-24-01016. Epub 2025 Feb 24.
2
Trends and challenges in organoid modeling and expansion with pluripotent stem cells and somatic tissue.多能干细胞和体细胞组织在类器官建模与扩增方面的趋势及挑战
PeerJ. 2024 Nov 27;12:e18422. doi: 10.7717/peerj.18422. eCollection 2024.
3
Retinal Organoids from Induced Pluripotent Stem Cells of Patients with Inherited Retinal Diseases: A Systematic Review.

本文引用的文献

1
Retinal organoid light responsivity: current status and future opportunities.视网膜类器官的光反应性:现状与未来机遇。
Transl Res. 2022 Dec;250:98-111. doi: 10.1016/j.trsl.2022.06.001. Epub 2022 Jun 9.
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Ocular Neurodegenerative Diseases: Interconnection between Retina and Cortical Areas.眼部神经退行性疾病:视网膜与皮质区的连接。
Cells. 2021 Sep 12;10(9):2394. doi: 10.3390/cells10092394.
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Stem Cell-Based Regeneration and Restoration for Retinal Ganglion Cell: Recent Advancements and Current Challenges.基于干细胞的视网膜神经节细胞再生和修复:最新进展和当前挑战。
来自遗传性视网膜疾病患者诱导多能干细胞的视网膜类器官:一项系统综述。
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Cell transplantation of retinal ganglion cells derived from hESCs.人胚胎干细胞来源的视网膜神经节细胞的细胞移植。
Restor Neurol Neurosci. 2020;38(2):131-140. doi: 10.3233/RNN-190941.
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Optic nerve regeneration: A long view.视神经再生:长远视角。
Restor Neurol Neurosci. 2019;37(6):525-544. doi: 10.3233/RNN-190960.
7
Retinal Ganglion Cell Replacement: Current Status and Challenges Ahead.视网膜神经节细胞替代:现状与挑战
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8
Enhanced Stem Cell Differentiation and Immunopurification of Genome Engineered Human Retinal Ganglion Cells.增强基因编辑的人视网膜神经节细胞的干细胞分化和免疫纯化。
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