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The Challenges and Opportunities in the Development of MicroRNA Therapeutics: A Multidisciplinary Viewpoint
microRNAs (miRs) are emerging as attractive therapeutic targets because of their small size, specific targetability, and critical role in disease pathogenesis. However, <20 miR targeting molecules have entered clinical trials, and none progressed to phase III. The difficulties in miR target ident...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8619171/ https://www.ncbi.nlm.nih.gov/pubmed/34831320 http://dx.doi.org/10.3390/cells10113097 |
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author | Momin, Mohammad Yahya Gaddam, Ravinder Reddy Kravitz, Madeline Gupta, Anisha Vikram, Ajit |
author_facet | Momin, Mohammad Yahya Gaddam, Ravinder Reddy Kravitz, Madeline Gupta, Anisha Vikram, Ajit |
author_sort | Momin, Mohammad Yahya |
collection | PubMed |
description | microRNAs (miRs) are emerging as attractive therapeutic targets because of their small size, specific targetability, and critical role in disease pathogenesis. However, <20 miR targeting molecules have entered clinical trials, and none progressed to phase III. The difficulties in miR target identification, the moderate efficacy of miR inhibitors, cell type-specific delivery, and adverse outcomes have impeded the development of miR therapeutics. These hurdles are rooted in the functional complexity of miR’s role in disease and sequence complementarity-dependent/-independent effects in nontarget tissues. The advances in understanding miR’s role in disease, the development of efficient miR inhibitors, and innovative delivery approaches have helped resolve some of these hurdles. In this review, we provide a multidisciplinary viewpoint on the challenges and opportunities in the development of miR therapeutics. |
format | Online Article Text |
id | pubmed-8619171 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-86191712021-11-27 The Challenges and Opportunities in the Development of MicroRNA Therapeutics: A Multidisciplinary Viewpoint Momin, Mohammad Yahya Gaddam, Ravinder Reddy Kravitz, Madeline Gupta, Anisha Vikram, Ajit Cells Review microRNAs (miRs) are emerging as attractive therapeutic targets because of their small size, specific targetability, and critical role in disease pathogenesis. However, <20 miR targeting molecules have entered clinical trials, and none progressed to phase III. The difficulties in miR target identification, the moderate efficacy of miR inhibitors, cell type-specific delivery, and adverse outcomes have impeded the development of miR therapeutics. These hurdles are rooted in the functional complexity of miR’s role in disease and sequence complementarity-dependent/-independent effects in nontarget tissues. The advances in understanding miR’s role in disease, the development of efficient miR inhibitors, and innovative delivery approaches have helped resolve some of these hurdles. In this review, we provide a multidisciplinary viewpoint on the challenges and opportunities in the development of miR therapeutics. MDPI 2021-11-09 /pmc/articles/PMC8619171/ /pubmed/34831320 http://dx.doi.org/10.3390/cells10113097 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/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 (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Momin, Mohammad Yahya Gaddam, Ravinder Reddy Kravitz, Madeline Gupta, Anisha Vikram, Ajit The Challenges and Opportunities in the Development of MicroRNA Therapeutics: A Multidisciplinary Viewpoint |
title | The Challenges and Opportunities in the Development of MicroRNA Therapeutics: A Multidisciplinary Viewpoint |
title_full | The Challenges and Opportunities in the Development of MicroRNA Therapeutics: A Multidisciplinary Viewpoint |
title_fullStr | The Challenges and Opportunities in the Development of MicroRNA Therapeutics: A Multidisciplinary Viewpoint |
title_full_unstemmed | The Challenges and Opportunities in the Development of MicroRNA Therapeutics: A Multidisciplinary Viewpoint |
title_short | The Challenges and Opportunities in the Development of MicroRNA Therapeutics: A Multidisciplinary Viewpoint |
title_sort | challenges and opportunities in the development of microrna therapeutics: a multidisciplinary viewpoint |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8619171/ https://www.ncbi.nlm.nih.gov/pubmed/34831320 http://dx.doi.org/10.3390/cells10113097 |
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