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Pluripotent stem cell‐derived retinal organoids for disease modeling and development of therapies
Retinal diseases constitute a genetically and phenotypically diverse group of clinical conditions leading to vision impairment or blindness with limited treatment options. Advances in reprogramming of somatic cells to induced pluripotent stem cells and generation of three‐dimensional organoids resem...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley & Sons, Inc.
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7586922/ https://www.ncbi.nlm.nih.gov/pubmed/32506758 http://dx.doi.org/10.1002/stem.3239 |
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author | Kruczek, Kamil Swaroop, Anand |
author_facet | Kruczek, Kamil Swaroop, Anand |
author_sort | Kruczek, Kamil |
collection | PubMed |
description | Retinal diseases constitute a genetically and phenotypically diverse group of clinical conditions leading to vision impairment or blindness with limited treatment options. Advances in reprogramming of somatic cells to induced pluripotent stem cells and generation of three‐dimensional organoids resembling the native retina offer promising tools to interrogate disease mechanisms and evaluate potential therapies for currently incurable retinal neurodegeneration. Next‐generation sequencing, single‐cell analysis, advanced electrophysiology, and high‐throughput screening approaches are expected to greatly expand the utility of stem cell‐derived retinal cells and organoids for developing personalized treatments. In this review, we discuss the current status and future potential of combining retinal organoids as human models with recent technologies to advance the development of gene, cell, and drug therapies for retinopathies. |
format | Online Article Text |
id | pubmed-7586922 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | John Wiley & Sons, Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-75869222020-10-30 Pluripotent stem cell‐derived retinal organoids for disease modeling and development of therapies Kruczek, Kamil Swaroop, Anand Stem Cells Regenerative Medicine Retinal diseases constitute a genetically and phenotypically diverse group of clinical conditions leading to vision impairment or blindness with limited treatment options. Advances in reprogramming of somatic cells to induced pluripotent stem cells and generation of three‐dimensional organoids resembling the native retina offer promising tools to interrogate disease mechanisms and evaluate potential therapies for currently incurable retinal neurodegeneration. Next‐generation sequencing, single‐cell analysis, advanced electrophysiology, and high‐throughput screening approaches are expected to greatly expand the utility of stem cell‐derived retinal cells and organoids for developing personalized treatments. In this review, we discuss the current status and future potential of combining retinal organoids as human models with recent technologies to advance the development of gene, cell, and drug therapies for retinopathies. John Wiley & Sons, Inc. 2020-06-07 2020-10-01 /pmc/articles/PMC7586922/ /pubmed/32506758 http://dx.doi.org/10.1002/stem.3239 Text en ©2020 The Authors. stem cells published by Wiley Periodicals LLC on behalf of AlphaMed Press 2020 This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made. |
spellingShingle | Regenerative Medicine Kruczek, Kamil Swaroop, Anand Pluripotent stem cell‐derived retinal organoids for disease modeling and development of therapies |
title | Pluripotent stem cell‐derived retinal organoids for disease modeling and development of therapies |
title_full | Pluripotent stem cell‐derived retinal organoids for disease modeling and development of therapies |
title_fullStr | Pluripotent stem cell‐derived retinal organoids for disease modeling and development of therapies |
title_full_unstemmed | Pluripotent stem cell‐derived retinal organoids for disease modeling and development of therapies |
title_short | Pluripotent stem cell‐derived retinal organoids for disease modeling and development of therapies |
title_sort | pluripotent stem cell‐derived retinal organoids for disease modeling and development of therapies |
topic | Regenerative Medicine |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7586922/ https://www.ncbi.nlm.nih.gov/pubmed/32506758 http://dx.doi.org/10.1002/stem.3239 |
work_keys_str_mv | AT kruczekkamil pluripotentstemcellderivedretinalorganoidsfordiseasemodelinganddevelopmentoftherapies AT swaroopanand pluripotentstemcellderivedretinalorganoidsfordiseasemodelinganddevelopmentoftherapies |