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Updated: Nov 5, 2025
Identification of Circular RNAs using RNA Sequencing
Published on: November 14, 2019
Targeting circular RNAs as a therapeutic approach: current strategies and challenges
Alina T He1, Jinglei Liu2, Feiya Li1,3
1Sunnybrook Research Institute, Toronto, ON, Canada.
Abstract:
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.
Insights
Circular RNAs (circRNAs) are key in cellular processes and disease. New methods using nanoparticles, exosomes, and CRISPR/Cas13 offer therapeutic potential for manipulating circRNA levels.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- Circular RNAs (circRNAs) are increasingly recognized for their roles in cellular functions.
- Dysregulation of circRNAs is linked to various disease pathologies.
- Their stability and specificity make circRNAs promising therapeutic targets.
Purpose of the Study:
- To review the properties and functions of circRNAs.
- To highlight the significance of circRNAs in disease pathogenesis.
- To summarize current and emerging strategies for circRNA manipulation in therapeutics.
Main Methods:
- Discussion of gain-of-function (expression plasmids) and loss-of-function (RNA interference) strategies.
- Exploration of nanoparticle and exosome delivery systems to enhance therapeutic delivery.
- Overview of advanced knockdown techniques including cre-lox and CRISPR/Cas13 systems.
Main Results:
- CircRNAs exhibit unique stability and tissue-specific expression patterns.
- Various methods exist for modulating circRNA expression, each with advantages and limitations.
- Novel delivery systems and gene-editing tools show promise for precise circRNA targeting.
Conclusions:
- CircRNAs hold significant therapeutic potential due to their disease relevance and unique characteristics.
- Advancements in delivery systems and gene-editing technologies are crucial for developing effective circRNA-based therapies.
- Further research is needed to overcome challenges and realize the full therapeutic promise of circRNAs.
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