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. 2022 Apr 19;54(4):455–465. doi: 10.1038/s12276-022-00757-5

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© The Author(s) 2022

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/.

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Fig. 2 — RNA-based drugs can target various steps involved in the expression of both protein-coding and noncoding genes. Splicing can be modulated by antisense oligonucleotides (ASOs), and mature messenger RNAs (mRNAs) can be targeted by ASOs or small interfering RNAs (siRNAs). In addition, noncoding RNAs (ncRNAs), including small ncRNAs and long ncRNAs (lncRNAs), can be suppressed by ASOs or siRNAs. Protein function can be modulated by aptamer binding. Finally, exogenous mRNAs can be used to introduce specific proteins into cells to replenish a deficient enzyme or act as antigens to elicit a targeted immune response.