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The infinite possibilities of RNA therapeutics

Although the study of ribonucleic acid (RNA) therapeutics started decades ago, for many years, this field of research was overshadowed by the growing interest in DNA-based therapies. Nowadays, the role of several types of RNA in cell regulation processes and the development of various diseases have...

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Autores principales: Mollocana-Lara, Evelyn C, Ni, Ming, Agathos, Spiros N, Gonzales-Zubiate, Fernando A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8788720/
https://www.ncbi.nlm.nih.gov/pubmed/34463324
http://dx.doi.org/10.1093/jimb/kuab063
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author Mollocana-Lara, Evelyn C
Ni, Ming
Agathos, Spiros N
Gonzales-Zubiate, Fernando A
author_facet Mollocana-Lara, Evelyn C
Ni, Ming
Agathos, Spiros N
Gonzales-Zubiate, Fernando A
author_sort Mollocana-Lara, Evelyn C
collection PubMed
description Although the study of ribonucleic acid (RNA) therapeutics started decades ago, for many years, this field of research was overshadowed by the growing interest in DNA-based therapies. Nowadays, the role of several types of RNA in cell regulation processes and the development of various diseases have been elucidated, and research in RNA therapeutics is back with force. This short literature review aims to present general aspects of many of the molecules currently used in RNA therapeutics, including in vitro transcribed mRNA (IVT mRNA), antisense oligonucleotides (ASOs), aptamers, small interfering RNAs (siRNAs), and microRNAs (miRNAs). In addition, we describe the state of the art of technologies applied for synthetic RNA manufacture and delivery. Likewise, we detail the RNA-based therapies approved by the FDA so far, as well as the ongoing clinical investigations. As a final point, we highlight the current and potential advantages of working on RNA-based therapeutics and how these could lead to a new era of accessible and personalized healthcare.
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spelling pubmed-87887202022-06-08 The infinite possibilities of RNA therapeutics Mollocana-Lara, Evelyn C Ni, Ming Agathos, Spiros N Gonzales-Zubiate, Fernando A J Ind Microbiol Biotechnol Biotechnology Methods Although the study of ribonucleic acid (RNA) therapeutics started decades ago, for many years, this field of research was overshadowed by the growing interest in DNA-based therapies. Nowadays, the role of several types of RNA in cell regulation processes and the development of various diseases have been elucidated, and research in RNA therapeutics is back with force. This short literature review aims to present general aspects of many of the molecules currently used in RNA therapeutics, including in vitro transcribed mRNA (IVT mRNA), antisense oligonucleotides (ASOs), aptamers, small interfering RNAs (siRNAs), and microRNAs (miRNAs). In addition, we describe the state of the art of technologies applied for synthetic RNA manufacture and delivery. Likewise, we detail the RNA-based therapies approved by the FDA so far, as well as the ongoing clinical investigations. As a final point, we highlight the current and potential advantages of working on RNA-based therapeutics and how these could lead to a new era of accessible and personalized healthcare. Oxford University Press 2021-08-31 /pmc/articles/PMC8788720/ /pubmed/34463324 http://dx.doi.org/10.1093/jimb/kuab063 Text en © The Author(s) 2021. Published by Oxford University Press on behalf of Society of Industrial Microbiology and Biotechnology. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs licence (https://creativecommons.org/licenses/by-nc-nd/4.0/), which permits non-commercial reproduction and distribution of the work, in any medium, provided the original work is not altered or transformed in any way, and that the work is properly cited. For commercial re-use, please contact journals.permissions@oup.com
spellingShingle Biotechnology Methods
Mollocana-Lara, Evelyn C
Ni, Ming
Agathos, Spiros N
Gonzales-Zubiate, Fernando A
The infinite possibilities of RNA therapeutics
title The infinite possibilities of RNA therapeutics
title_full The infinite possibilities of RNA therapeutics
title_fullStr The infinite possibilities of RNA therapeutics
title_full_unstemmed The infinite possibilities of RNA therapeutics
title_short The infinite possibilities of RNA therapeutics
title_sort infinite possibilities of rna therapeutics
topic Biotechnology Methods
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8788720/
https://www.ncbi.nlm.nih.gov/pubmed/34463324
http://dx.doi.org/10.1093/jimb/kuab063
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