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. 2021 Mar 18;9:628137. doi: 10.3389/fbioe.2021.628137

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Copyright © 2021 Damase, Sukhovershin, Boada, Taraballi, Pettigrew and Cooke.

This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

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Schematic illustrating the mechanism of action of small interfering RNA (siRNA) drug, patisiran. The drugs are encapsulated in lipid nanoparticles and administered intravenously. After administration, the drugs finally reach hepatocyte and released into the cytoplasm, where it is loaded onto the RISC. The antisense strand hybridizes with target mRNA to suppress the production of target protein (TTR) (Kristen et al., 2018). TTR, transthyretin; wt, wild type; RISC, RNA-induced silencing complex; mRNA, messenger RNA; RNA, ribonucleic acid.