Targeted RNA base editing for therapeutic: mechanisms and advances

Abstract

RNA base editing, which enables RNA base modification through effector proteins guided by targeting systems, is a powerful technology to correct disease-associated point mutations. Although overshadowed by CRISPR-based genome editing, RNA editing has seen rapid development in recent years, with significant improvements in efficiency and precision. In this review, we summarize the core components of RNA base editing systems (RNAtargeting systems and effector proteins) and describe major RNA editing types, including A-to-I, C-to-U, A-tom⁶ A/m⁶ A-to-A, and U-to-$\mathrm{\Psi }$ base editors, along with their research progress. In addition, we systematically summarize the delivery methods of the developed RNA editors and their initial exploration in treating diseases caused by nonsense mutations. Finally, combining the current development status of the RNA editing related field, we reflect on the problems encountered in the current development of the RNA editing field and offer our own insights on the future development direction.

Keywords

RNA editing / Point mutation / CRISPR-Cas / RNA modification

CRediT authorship contribution statement

Weikai Yan: Writing – original draft, Resources. Xiaocheng Weng: Writing – review & editing, Supervision.

Ethics approval

Not applicable.

Declaration of generative AI in scientific writing

No generative AI tools have been used throughout the entire writing process of this manuscript.

Funding information

This work was supported financially by the Noncommunicable Chronic Diseases-National Science and Technology Major Project (2023ZD0507700,2023ZD0507701), the National Natural Science Foundation of China [92253202 and 22177087 to X. W.].

Data availability

Not applicable.

Declaration of competing interest

The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Acknowledgments

This work utilized BioGDP.com for the generation of scientific figures, and the authors acknowledge its contribution to the visual presentation of this review.

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