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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...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2022
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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. |
format | Online Article Text |
id | pubmed-9794871 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
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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