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: Mohammad Yahya Momin, Ravinder Reddy Gaddam, Madeline Kravitz, Anisha Gupta, Ajit Vikram
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Publicado: MDPI AG 2021
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spelling oai:doaj.org-article:8d2384e466034a058851f9185a4627982021-11-25T17:11:28ZThe Challenges and Opportunities in the Development of MicroRNA Therapeutics: A Multidisciplinary Viewpoint10.3390/cells101130972073-4409https://doaj.org/article/8d2384e466034a058851f9185a4627982021-11-01T00:00:00Zhttps://www.mdpi.com/2073-4409/10/11/3097https://doaj.org/toc/2073-4409microRNAs (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.Mohammad Yahya MominRavinder Reddy GaddamMadeline KravitzAnisha GuptaAjit VikramMDPI AGarticlepeptide nucleic acidsmiR deliverymiRNAplant-derived miRsBiology (General)QH301-705.5ENCells, Vol 10, Iss 3097, p 3097 (2021)
institution DOAJ
collection DOAJ
language EN
topic peptide nucleic acids
miR delivery
miRNA
plant-derived miRs
Biology (General)
QH301-705.5
spellingShingle peptide nucleic acids
miR delivery
miRNA
plant-derived miRs
Biology (General)
QH301-705.5
Mohammad Yahya Momin
Ravinder Reddy Gaddam
Madeline Kravitz
Anisha Gupta
Ajit Vikram
The Challenges and Opportunities in the Development of MicroRNA Therapeutics: A Multidisciplinary Viewpoint
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 article
author Mohammad Yahya Momin
Ravinder Reddy Gaddam
Madeline Kravitz
Anisha Gupta
Ajit Vikram
author_facet Mohammad Yahya Momin
Ravinder Reddy Gaddam
Madeline Kravitz
Anisha Gupta
Ajit Vikram
author_sort Mohammad Yahya Momin
title 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_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_sort challenges and opportunities in the development of microrna therapeutics: a multidisciplinary viewpoint
publisher MDPI AG
publishDate 2021
url https://doaj.org/article/8d2384e466034a058851f9185a462798
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