Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders

Disruptions in RNA processing play critical roles in the pathogenesis of neurological diseases. In this Perspective, we discuss recent progress in the development of RNA-targeting therapeutic modalities. We focus on progress, limitations, and opportunities in a new generation of therapies engineered...

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Detalles Bibliográficos
Autores principales: Morelli, Kathryn H., Smargon, Aaron A., Yeo, Gene W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory Press 2023
Materias:
Perspectives
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10019361/
https://www.ncbi.nlm.nih.gov/pubmed/36693761
http://dx.doi.org/10.1261/rna.079519.122
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author Morelli, Kathryn H.
Smargon, Aaron A.
Yeo, Gene W.
author_facet Morelli, Kathryn H.
Smargon, Aaron A.
Yeo, Gene W.
author_sort Morelli, Kathryn H.
collection PubMed
description Disruptions in RNA processing play critical roles in the pathogenesis of neurological diseases. In this Perspective, we discuss recent progress in the development of RNA-targeting therapeutic modalities. We focus on progress, limitations, and opportunities in a new generation of therapies engineered from RNA binding proteins and other endogenous RNA regulatory macromolecules to treat human neurological disorders.
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spelling pubmed-100193612023-04-01 Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders Morelli, Kathryn H. Smargon, Aaron A. Yeo, Gene W. RNA Perspectives Disruptions in RNA processing play critical roles in the pathogenesis of neurological diseases. In this Perspective, we discuss recent progress in the development of RNA-targeting therapeutic modalities. We focus on progress, limitations, and opportunities in a new generation of therapies engineered from RNA binding proteins and other endogenous RNA regulatory macromolecules to treat human neurological disorders. Cold Spring Harbor Laboratory Press 2023-04 /pmc/articles/PMC10019361/ /pubmed/36693761 http://dx.doi.org/10.1261/rna.079519.122 Text en © 2023 Morelli et al.; Published by Cold Spring Harbor Laboratory Press for the RNA Society https://creativecommons.org/licenses/by-nc/4.0/This article, published in RNA, is available under a Creative Commons License (Attribution-NonCommercial 4.0 International), as described at http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) .
spellingShingle Perspectives
Morelli, Kathryn H.
Smargon, Aaron A.
Yeo, Gene W.
Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders
title Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders
title_full Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders
title_fullStr Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders
title_full_unstemmed Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders
title_short Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders
title_sort programmable macromolecule-based rna-targeting therapies to treat human neurological disorders
topic Perspectives
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10019361/
https://www.ncbi.nlm.nih.gov/pubmed/36693761
http://dx.doi.org/10.1261/rna.079519.122
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