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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. 4 — a Mechanism of action of siRNA drugs in mammalian cells. siRNAs interact with the Argonaute (AGO) protein to produce a binding complex that recognizes its target mRNA and then induces its sequence-specific cleavage in a process called RNA interference (RNAi). b Patisiran binds to the 3ʹ untranslated region (UTR) of transthyretin (TTR) mRNA. Because mutation occurs within the coding region of this gene, patisiran suppresses the expression of both wild-type and mutant TTR mRNAs. c Givosiran suppresses the expression of aminolevulinic acid (ALA) synthase 1 (ALAS1) mRNA. This results in a reduction in ALAS1 protein, which is required for the production of ALA and porphobilinogen (PBG). Notably, givosiran contains the N-acetylgalactosamine (GalNAc) conjugate within the 3ʹ end of its passenger strand. d Inclisiran induces the cleavage of the mRNA encoding proprotein convertase subtilisin/kexin type 9 (PCSK9), a promising target for the therapeutic management of patients with high cholesterol. Reductions in PCSK9 protein result in reduced endocytosis of low-density lipoprotein (LDL) receptors.