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Targeting circular RNAs as a therapeutic approach: current strategies and challenges
Significant progress has been made in circular RNA (circRNA) research in recent years. Increasing evidence suggests that circRNAs play important roles in many cellular processes, and their dysregulation is implicated in the pathogenesis of various diseases. CircRNAs are highly stable and usually exp...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8137869/ https://www.ncbi.nlm.nih.gov/pubmed/34016945 http://dx.doi.org/10.1038/s41392-021-00569-5 |
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author | He, Alina T. Liu, Jinglei Li, Feiya Yang, Burton B. |
author_facet | He, Alina T. Liu, Jinglei Li, Feiya Yang, Burton B. |
author_sort | He, Alina T. |
collection | PubMed |
description | Significant progress has been made in circular RNA (circRNA) research in recent years. Increasing evidence suggests that circRNAs play important roles in many cellular processes, and their dysregulation is implicated in the pathogenesis of various diseases. CircRNAs are highly stable and usually expressed in a tissue- or cell type-specific manner. Therefore, they are currently being explored as potential therapeutic targets. Gain-of-function and loss-of-function approaches are typically performed using circRNA expression plasmids and RNA interference-based strategies, respectively. These strategies have limitations that can be mitigated using nanoparticle and exosome delivery systems. Furthermore, recent developments show that the cre-lox system can be used to knockdown circRNAs in a cell-specific manner. While still in the early stages of development, the CRISPR/Cas13 system has shown promise in knocking down circRNAs with high specificity and efficiency. In this review, we describe circRNA properties and functions and highlight their significance in disease. We summarize strategies that can be used to overexpress or knockdown circRNAs as a therapeutic approach. Lastly, we discuss major challenges and propose future directions for the development of circRNA-based therapeutics. |
format | Online Article Text |
id | pubmed-8137869 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Nature Publishing Group UK |
record_format | MEDLINE/PubMed |
spelling | pubmed-81378692021-06-03 Targeting circular RNAs as a therapeutic approach: current strategies and challenges He, Alina T. Liu, Jinglei Li, Feiya Yang, Burton B. Signal Transduct Target Ther Review Article Significant progress has been made in circular RNA (circRNA) research in recent years. Increasing evidence suggests that circRNAs play important roles in many cellular processes, and their dysregulation is implicated in the pathogenesis of various diseases. CircRNAs are highly stable and usually expressed in a tissue- or cell type-specific manner. Therefore, they are currently being explored as potential therapeutic targets. Gain-of-function and loss-of-function approaches are typically performed using circRNA expression plasmids and RNA interference-based strategies, respectively. These strategies have limitations that can be mitigated using nanoparticle and exosome delivery systems. Furthermore, recent developments show that the cre-lox system can be used to knockdown circRNAs in a cell-specific manner. While still in the early stages of development, the CRISPR/Cas13 system has shown promise in knocking down circRNAs with high specificity and efficiency. In this review, we describe circRNA properties and functions and highlight their significance in disease. We summarize strategies that can be used to overexpress or knockdown circRNAs as a therapeutic approach. Lastly, we discuss major challenges and propose future directions for the development of circRNA-based therapeutics. Nature Publishing Group UK 2021-05-21 /pmc/articles/PMC8137869/ /pubmed/34016945 http://dx.doi.org/10.1038/s41392-021-00569-5 Text en © The Author(s) 2021 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 license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license 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 license, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . |
spellingShingle | Review Article He, Alina T. Liu, Jinglei Li, Feiya Yang, Burton B. Targeting circular RNAs as a therapeutic approach: current strategies and challenges |
title | Targeting circular RNAs as a therapeutic approach: current strategies and challenges |
title_full | Targeting circular RNAs as a therapeutic approach: current strategies and challenges |
title_fullStr | Targeting circular RNAs as a therapeutic approach: current strategies and challenges |
title_full_unstemmed | Targeting circular RNAs as a therapeutic approach: current strategies and challenges |
title_short | Targeting circular RNAs as a therapeutic approach: current strategies and challenges |
title_sort | targeting circular rnas as a therapeutic approach: current strategies and challenges |
topic | Review Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8137869/ https://www.ncbi.nlm.nih.gov/pubmed/34016945 http://dx.doi.org/10.1038/s41392-021-00569-5 |
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