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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...

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Autores principales: Momin, Mohammad Yahya, Gaddam, Ravinder Reddy, Kravitz, Madeline, Gupta, Anisha, Vikram, Ajit
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
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.
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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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