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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. 3 — a Primary mechanism of action for antisense oligonucleotide (ASO)-based RNA drugs in human cells. ASOs can modulate splicing or induce RNase H-mediated degradation of the target mRNA. b Mipomersen, a drug used in the treatment of homozygous familial hypercholesterolemia, induces the degradation of apolipoprotein B-100 (ApoB-100) mRNA. c Nusinersen binds to the intronic splicing silencer element in the intron of survival motor neuron 2 (SMN2) pre-mRNA, which blocks the exon 7 skipping associated with SMN type diseases. Consequently, SMN2 mRNA is spliced like SMN1 mRNA, resulting in the production of stable SMN proteins. d In some patients with Duchenne muscular dystrophy, a mutation at exon 51 of dystrophin (DMD) mRNA induces premature termination of translation, preventing the production of functional protein. Eteplirsen binds to the exonic splicing enhancer in this exon and induces exon 51 skipping. This results in the production of smaller amounts of fully functional proteins.