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Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development
Antisense oligonucleotides (ASOs) bind sequence specifically to the target RNA and modulate protein expression through several different mechanisms. The ASO field is an emerging area of drug development that targets the disease source at the RNA level and offers a promising alternative to therapies...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7355792/ https://www.ncbi.nlm.nih.gov/pubmed/32604776 http://dx.doi.org/10.3390/jcm9062004 |
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author | Dhuri, Karishma Bechtold, Clara Quijano, Elias Pham, Ha Gupta, Anisha Vikram, Ajit Bahal, Raman |
author_facet | Dhuri, Karishma Bechtold, Clara Quijano, Elias Pham, Ha Gupta, Anisha Vikram, Ajit Bahal, Raman |
author_sort | Dhuri, Karishma |
collection | PubMed |
description | Antisense oligonucleotides (ASOs) bind sequence specifically to the target RNA and modulate protein expression through several different mechanisms. The ASO field is an emerging area of drug development that targets the disease source at the RNA level and offers a promising alternative to therapies targeting downstream processes. To translate ASO-based therapies into a clinical success, it is crucial to overcome the challenges associated with off-target side effects and insufficient biological activity. In this regard, several chemical modifications and diverse delivery strategies have been explored. In this review, we systematically discuss the chemical modifications, mechanism of action, and optimized delivery strategies of several different classes of ASOs. Further, we highlight the recent advances made in development of ASO-based drugs with a focus on drugs that are approved by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for clinical applications. We also discuss various promising ASO-based drug candidates in the clinical trials, and the outstanding opportunity of emerging microRNA as a viable therapeutic target for future ASO-based therapies. |
format | Online Article Text |
id | pubmed-7355792 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-73557922020-07-23 Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development Dhuri, Karishma Bechtold, Clara Quijano, Elias Pham, Ha Gupta, Anisha Vikram, Ajit Bahal, Raman J Clin Med Review Antisense oligonucleotides (ASOs) bind sequence specifically to the target RNA and modulate protein expression through several different mechanisms. The ASO field is an emerging area of drug development that targets the disease source at the RNA level and offers a promising alternative to therapies targeting downstream processes. To translate ASO-based therapies into a clinical success, it is crucial to overcome the challenges associated with off-target side effects and insufficient biological activity. In this regard, several chemical modifications and diverse delivery strategies have been explored. In this review, we systematically discuss the chemical modifications, mechanism of action, and optimized delivery strategies of several different classes of ASOs. Further, we highlight the recent advances made in development of ASO-based drugs with a focus on drugs that are approved by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for clinical applications. We also discuss various promising ASO-based drug candidates in the clinical trials, and the outstanding opportunity of emerging microRNA as a viable therapeutic target for future ASO-based therapies. MDPI 2020-06-26 /pmc/articles/PMC7355792/ /pubmed/32604776 http://dx.doi.org/10.3390/jcm9062004 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Dhuri, Karishma Bechtold, Clara Quijano, Elias Pham, Ha Gupta, Anisha Vikram, Ajit Bahal, Raman Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development |
title | Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development |
title_full | Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development |
title_fullStr | Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development |
title_full_unstemmed | Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development |
title_short | Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development |
title_sort | antisense oligonucleotides: an emerging area in drug discovery and development |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7355792/ https://www.ncbi.nlm.nih.gov/pubmed/32604776 http://dx.doi.org/10.3390/jcm9062004 |
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