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Towards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics

Our understanding of neurological diseases has been tremendously enhanced over the past decade by the application of new technologies. Genome-wide association studies have highlighted glial cells as important players in diseases. Single-cell profiling technologies are providing descriptions of disea...

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Autores principales: Leng, Kun, Kampmann, Martin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9673433/
https://www.ncbi.nlm.nih.gov/pubmed/36401300
http://dx.doi.org/10.1186/s13073-022-01134-7
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author Leng, Kun
Kampmann, Martin
author_facet Leng, Kun
Kampmann, Martin
author_sort Leng, Kun
collection PubMed
description Our understanding of neurological diseases has been tremendously enhanced over the past decade by the application of new technologies. Genome-wide association studies have highlighted glial cells as important players in diseases. Single-cell profiling technologies are providing descriptions of disease states of neurons and glia at unprecedented molecular resolution. However, significant gaps remain in our understanding of the mechanisms driving disease-associated cell states, and how these states contribute to disease. These gaps in our understanding can be bridged by CRISPR-based functional genomics, a powerful approach to systematically interrogate gene function. In this review, we will briefly review the current literature on neurological disease-associated cell states and introduce CRISPR-based functional genomics. We discuss how advances in CRISPR-based screens, especially when implemented in the relevant brain cell types or cellular environments, have paved the way towards uncovering mechanisms underlying neurological disease-associated cell states. Finally, we will delineate current challenges and future directions for CRISPR-based functional genomics to further our understanding of neurological diseases and potential therapeutic strategies.
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spelling pubmed-96734332022-11-19 Towards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics Leng, Kun Kampmann, Martin Genome Med Review Our understanding of neurological diseases has been tremendously enhanced over the past decade by the application of new technologies. Genome-wide association studies have highlighted glial cells as important players in diseases. Single-cell profiling technologies are providing descriptions of disease states of neurons and glia at unprecedented molecular resolution. However, significant gaps remain in our understanding of the mechanisms driving disease-associated cell states, and how these states contribute to disease. These gaps in our understanding can be bridged by CRISPR-based functional genomics, a powerful approach to systematically interrogate gene function. In this review, we will briefly review the current literature on neurological disease-associated cell states and introduce CRISPR-based functional genomics. We discuss how advances in CRISPR-based screens, especially when implemented in the relevant brain cell types or cellular environments, have paved the way towards uncovering mechanisms underlying neurological disease-associated cell states. Finally, we will delineate current challenges and future directions for CRISPR-based functional genomics to further our understanding of neurological diseases and potential therapeutic strategies. BioMed Central 2022-11-18 /pmc/articles/PMC9673433/ /pubmed/36401300 http://dx.doi.org/10.1186/s13073-022-01134-7 Text en © The Author(s) 2022 https://creativecommons.org/licenses/by/4.0/Open AccessThis 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
Leng, Kun
Kampmann, Martin
Towards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics
title Towards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics
title_full Towards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics
title_fullStr Towards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics
title_full_unstemmed Towards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics
title_short Towards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics
title_sort towards elucidating disease-relevant states of neurons and glia by crispr-based functional genomics
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9673433/
https://www.ncbi.nlm.nih.gov/pubmed/36401300
http://dx.doi.org/10.1186/s13073-022-01134-7
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