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Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases

Inherited retinal diseases (IRDs) can induce severe sight-threatening retinal degeneration and impose a considerable economic burden on patients and society, making efforts to cure blindness imperative. Transgenic animals mimicking human genetic diseases have long been used as a primary research too...

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Autores principales: Liang, Yuqin, Sun, Xihao, Duan, Chunwen, Tang, Shibo, Chen, Jiansu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10683306/
https://www.ncbi.nlm.nih.gov/pubmed/38012786
http://dx.doi.org/10.1186/s13287-023-03564-5
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author Liang, Yuqin
Sun, Xihao
Duan, Chunwen
Tang, Shibo
Chen, Jiansu
author_facet Liang, Yuqin
Sun, Xihao
Duan, Chunwen
Tang, Shibo
Chen, Jiansu
author_sort Liang, Yuqin
collection PubMed
description Inherited retinal diseases (IRDs) can induce severe sight-threatening retinal degeneration and impose a considerable economic burden on patients and society, making efforts to cure blindness imperative. Transgenic animals mimicking human genetic diseases have long been used as a primary research tool to decipher the underlying pathogenesis, but there are still some obvious limitations. As an alternative strategy, patient-derived induced pluripotent stem cells (iPSCs), particularly three-dimensional (3D) organoid technology, are considered a promising platform for modeling different forms of IRDs, including retinitis pigmentosa, Leber congenital amaurosis, X-linked recessive retinoschisis, Batten disease, achromatopsia, and best vitelliform macular dystrophy. Here, this paper focuses on the status of patient-derived iPSCs and organoids in IRDs in recent years concerning disease modeling and therapeutic exploration, along with potential challenges for translating laboratory research to clinical application. Finally, the importance of human iPSCs and organoids in combination with emerging technologies such as multi-omics integration analysis, 3D bioprinting, or microfluidic chip platform are highlighted. Patient-derived retinal organoids may be a preferred choice for more accurately uncovering the mechanisms of human retinal diseases and will contribute to clinical practice.
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spelling pubmed-106833062023-11-30 Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases Liang, Yuqin Sun, Xihao Duan, Chunwen Tang, Shibo Chen, Jiansu Stem Cell Res Ther Review Inherited retinal diseases (IRDs) can induce severe sight-threatening retinal degeneration and impose a considerable economic burden on patients and society, making efforts to cure blindness imperative. Transgenic animals mimicking human genetic diseases have long been used as a primary research tool to decipher the underlying pathogenesis, but there are still some obvious limitations. As an alternative strategy, patient-derived induced pluripotent stem cells (iPSCs), particularly three-dimensional (3D) organoid technology, are considered a promising platform for modeling different forms of IRDs, including retinitis pigmentosa, Leber congenital amaurosis, X-linked recessive retinoschisis, Batten disease, achromatopsia, and best vitelliform macular dystrophy. Here, this paper focuses on the status of patient-derived iPSCs and organoids in IRDs in recent years concerning disease modeling and therapeutic exploration, along with potential challenges for translating laboratory research to clinical application. Finally, the importance of human iPSCs and organoids in combination with emerging technologies such as multi-omics integration analysis, 3D bioprinting, or microfluidic chip platform are highlighted. Patient-derived retinal organoids may be a preferred choice for more accurately uncovering the mechanisms of human retinal diseases and will contribute to clinical practice. BioMed Central 2023-11-27 /pmc/articles/PMC10683306/ /pubmed/38012786 http://dx.doi.org/10.1186/s13287-023-03564-5 Text en © The Author(s) 2023 https://creativecommons.org/licenses/by/4.0/Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Review
Liang, Yuqin
Sun, Xihao
Duan, Chunwen
Tang, Shibo
Chen, Jiansu
Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases
title Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases
title_full Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases
title_fullStr Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases
title_full_unstemmed Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases
title_short Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases
title_sort application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10683306/
https://www.ncbi.nlm.nih.gov/pubmed/38012786
http://dx.doi.org/10.1186/s13287-023-03564-5
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