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

Kurzawa-Akanbi, Marzena; Tzoumas, Nik; Corral-Serrano, Julio C; Guarascio, Rosellina; Steel, David; Cheetham, Michael E; Armstrong, Lyle; (2024) Pluripotent stem cell-derived models of retinal disease: Elucidating pathogenesis, evaluating novel treatments, and estimating toxicity. Progress in Retinal and Eye Research , 100 , Article 101248. 10.1016/j.preteyeres.2024.101248. Green open access

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Abstract

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 and organoids 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 the choriocapillaris.

Type: Article
Title: Pluripotent stem cell-derived models of retinal disease: Elucidating pathogenesis, evaluating novel treatments, and estimating toxicity
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.preteyeres.2024.101248
Publisher version: http://dx.doi.org/10.1016/j.preteyeres.2024.101248
Language: English
Additional information: Copyright © 2024 The Authors. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/)
Keywords: AMD, Antisense oligonucleotides, Biomarker, CEP290, CRISPR-Cas9, Cilia, ESCs, Gene therapy, MELAS, Mitochondria, PRPFs, PSCs, RP, RPE, Retina, Retinoblastoma, Toxicity, iPSCs
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > Institute of Ophthalmology
URI: https://discovery.ucl.ac.uk/id/eprint/10187523
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