An overview of structural approaches to study therapeutic RNAs
RNAs provide considerable opportunities as therapeutic agent to expand the plethora of classical therapeutic targets, from extracellular and surface proteins to intracellular nucleic acids and its regulators, in a wide range of diseases. RNA versatility can be exploited to recognize cell types, perf...
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Format: | Article |
Language: | English |
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Frontiers Media S.A.
2022-10-01
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Series: | Frontiers in Molecular Biosciences |
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Online Access: | https://www.frontiersin.org/articles/10.3389/fmolb.2022.1044126/full |
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author | Luca Mollica Francesca Anna Cupaioli Grazisa Rossetti Federica Chiappori |
author_facet | Luca Mollica Francesca Anna Cupaioli Grazisa Rossetti Federica Chiappori |
author_sort | Luca Mollica |
collection | DOAJ |
description | RNAs provide considerable opportunities as therapeutic agent to expand the plethora of classical therapeutic targets, from extracellular and surface proteins to intracellular nucleic acids and its regulators, in a wide range of diseases. RNA versatility can be exploited to recognize cell types, perform cell therapy, and develop new vaccine classes. Therapeutic RNAs (aptamers, antisense nucleotides, siRNA, miRNA, mRNA and CRISPR-Cas9) can modulate or induce protein expression, inhibit molecular interactions, achieve genome editing as well as exon-skipping. A common RNA thread, which makes it very promising for therapeutic applications, is its structure, flexibility, and binding specificity. Moreover, RNA displays peculiar structural plasticity compared to proteins as well as to DNA. Here we summarize the recent advances and applications of therapeutic RNAs, and the experimental and computational methods to analyze their structure, by biophysical techniques (liquid-state NMR, scattering, reactivity, and computational simulations), with a focus on dynamic and flexibility aspects and to binding analysis. This will provide insights on the currently available RNA therapeutic applications and on the best techniques to evaluate its dynamics and reactivity. |
first_indexed | 2024-04-12T01:13:55Z |
format | Article |
id | doaj.art-09435f8de6ca4304b97b1e7dfa55c604 |
institution | Directory Open Access Journal |
issn | 2296-889X |
language | English |
last_indexed | 2024-04-12T01:13:55Z |
publishDate | 2022-10-01 |
publisher | Frontiers Media S.A. |
record_format | Article |
series | Frontiers in Molecular Biosciences |
spelling | doaj.art-09435f8de6ca4304b97b1e7dfa55c6042022-12-22T03:54:01ZengFrontiers Media S.A.Frontiers in Molecular Biosciences2296-889X2022-10-01910.3389/fmolb.2022.10441261044126An overview of structural approaches to study therapeutic RNAsLuca Mollica0Francesca Anna Cupaioli1Grazisa Rossetti2Federica Chiappori3Department of Medical Biotechnologies and Translational Medicine, L.I.T.A/University of Milan, Milan, ItalyNational Research Council—Institute for Biomedical Technologies, Milan, ItalyIFOM ETS—The AIRC Institute of Molecular Oncology, Milan, ItalyNational Research Council—Institute for Biomedical Technologies, Milan, ItalyRNAs provide considerable opportunities as therapeutic agent to expand the plethora of classical therapeutic targets, from extracellular and surface proteins to intracellular nucleic acids and its regulators, in a wide range of diseases. RNA versatility can be exploited to recognize cell types, perform cell therapy, and develop new vaccine classes. Therapeutic RNAs (aptamers, antisense nucleotides, siRNA, miRNA, mRNA and CRISPR-Cas9) can modulate or induce protein expression, inhibit molecular interactions, achieve genome editing as well as exon-skipping. A common RNA thread, which makes it very promising for therapeutic applications, is its structure, flexibility, and binding specificity. Moreover, RNA displays peculiar structural plasticity compared to proteins as well as to DNA. Here we summarize the recent advances and applications of therapeutic RNAs, and the experimental and computational methods to analyze their structure, by biophysical techniques (liquid-state NMR, scattering, reactivity, and computational simulations), with a focus on dynamic and flexibility aspects and to binding analysis. This will provide insights on the currently available RNA therapeutic applications and on the best techniques to evaluate its dynamics and reactivity.https://www.frontiersin.org/articles/10.3389/fmolb.2022.1044126/fulltherapeutic RNAsRNA structureRNA bindingRNA dynamics and flexibilityRNA selectivity and specificity |
spellingShingle | Luca Mollica Francesca Anna Cupaioli Grazisa Rossetti Federica Chiappori An overview of structural approaches to study therapeutic RNAs Frontiers in Molecular Biosciences therapeutic RNAs RNA structure RNA binding RNA dynamics and flexibility RNA selectivity and specificity |
title | An overview of structural approaches to study therapeutic RNAs |
title_full | An overview of structural approaches to study therapeutic RNAs |
title_fullStr | An overview of structural approaches to study therapeutic RNAs |
title_full_unstemmed | An overview of structural approaches to study therapeutic RNAs |
title_short | An overview of structural approaches to study therapeutic RNAs |
title_sort | overview of structural approaches to study therapeutic rnas |
topic | therapeutic RNAs RNA structure RNA binding RNA dynamics and flexibility RNA selectivity and specificity |
url | https://www.frontiersin.org/articles/10.3389/fmolb.2022.1044126/full |
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