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The therapeutic prospects of N-acetylgalactosamine-siRNA conjugates

RNA interference has become increasingly used for genetic therapy following the rapid development of oligonucleotide drugs. Significant progress has been made in its delivery system and implementation in the treatment of target organs. After a brief introduction of RNA interference technology and si...

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Autores principales: Zhang, Lei, Liang, Yayu, Liang, Guohui, Tian, Zhili, Zhang, Yue, Liu, Zhihui, Ji, Xinying
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9794871/
https://www.ncbi.nlm.nih.gov/pubmed/36588695
http://dx.doi.org/10.3389/fphar.2022.1090237
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author Zhang, Lei
Liang, Yayu
Liang, Guohui
Tian, Zhili
Zhang, Yue
Liu, Zhihui
Ji, Xinying
author_facet Zhang, Lei
Liang, Yayu
Liang, Guohui
Tian, Zhili
Zhang, Yue
Liu, Zhihui
Ji, Xinying
author_sort Zhang, Lei
collection PubMed
description RNA interference has become increasingly used for genetic therapy following the rapid development of oligonucleotide drugs. Significant progress has been made in its delivery system and implementation in the treatment of target organs. After a brief introduction of RNA interference technology and siRNA, the efficiency and stability of GalNAc-siRNA conjugates are highlighted since several oligonucleotide drugs of GalNAc have been approved for clinical use in recent years. The structure and features of GalNAc-siRNA conjugates are studied and the clinical efficiency and limitations of oligonucleotide-based drugs are summarized and investigated. Furthermore, another delivery system, lipid nanoparticles, that confer many advantages, is concluded, includ-ing stability and mass production, compared with GalNAc-siRNA conjugates. Importantly, developing new approaches for the use of oligonucleotide drugs brings hope to genetic therapy.
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spelling pubmed-97948712022-12-29 The therapeutic prospects of N-acetylgalactosamine-siRNA conjugates Zhang, Lei Liang, Yayu Liang, Guohui Tian, Zhili Zhang, Yue Liu, Zhihui Ji, Xinying Front Pharmacol Pharmacology RNA interference has become increasingly used for genetic therapy following the rapid development of oligonucleotide drugs. Significant progress has been made in its delivery system and implementation in the treatment of target organs. After a brief introduction of RNA interference technology and siRNA, the efficiency and stability of GalNAc-siRNA conjugates are highlighted since several oligonucleotide drugs of GalNAc have been approved for clinical use in recent years. The structure and features of GalNAc-siRNA conjugates are studied and the clinical efficiency and limitations of oligonucleotide-based drugs are summarized and investigated. Furthermore, another delivery system, lipid nanoparticles, that confer many advantages, is concluded, includ-ing stability and mass production, compared with GalNAc-siRNA conjugates. Importantly, developing new approaches for the use of oligonucleotide drugs brings hope to genetic therapy. Frontiers Media S.A. 2022-12-14 /pmc/articles/PMC9794871/ /pubmed/36588695 http://dx.doi.org/10.3389/fphar.2022.1090237 Text en Copyright © 2022 Zhang, Liang, Liang, Tian, Zhang, Liu and Ji. https://creativecommons.org/licenses/by/4.0/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.
spellingShingle Pharmacology
Zhang, Lei
Liang, Yayu
Liang, Guohui
Tian, Zhili
Zhang, Yue
Liu, Zhihui
Ji, Xinying
The therapeutic prospects of N-acetylgalactosamine-siRNA conjugates
title The therapeutic prospects of N-acetylgalactosamine-siRNA conjugates
title_full The therapeutic prospects of N-acetylgalactosamine-siRNA conjugates
title_fullStr The therapeutic prospects of N-acetylgalactosamine-siRNA conjugates
title_full_unstemmed The therapeutic prospects of N-acetylgalactosamine-siRNA conjugates
title_short The therapeutic prospects of N-acetylgalactosamine-siRNA conjugates
title_sort therapeutic prospects of n-acetylgalactosamine-sirna conjugates
topic Pharmacology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9794871/
https://www.ncbi.nlm.nih.gov/pubmed/36588695
http://dx.doi.org/10.3389/fphar.2022.1090237
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