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. 2021 Feb 26;10(1):375–389. doi: 10.1007/s40120-021-00235-6

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© The Author(s) 2021, corrected publication 2021

Open Access This article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial 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 licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence 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 licence, visit http://creativecommons.org/licenses/by-nc/4.0/.

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Fig. 1 — ASGPR mediates the uptake of the GalNAc₃-conjugated ASO AKCEA-TTR-L_Rx by hepatocytes, where it binds to the TTR mRNA through Watson–Crick hybridization and prevents production of TTR protein via RNase H1-mediated degradation of the target TTR mRNA. ASGPR asialoglycoprotein receptor, ASO antisense oligonucleotide, DNA deoxyribonucleic acid, mRNA messenger ribonucleic acid, RNase ribonuclease, TTR transthyretin