Nucleic acid-based therapeutics for the treatment of central nervous system disorders

Nucleic acid-based therapeutics (NBTs) are an emerging class of drugs with potential for the treatment of a wide range of central nervous system conditions. To date, pertaining to CNS indications, there are two commercially available NBTs and a large number of ongoing clinical trials. However, these...

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Autores principales: McCartan, Robyn, Khorkova, Olga, Volmar, Claude-Henry, Wahlestedt, Claes
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Genetics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10468602/
https://www.ncbi.nlm.nih.gov/pubmed/37662844
http://dx.doi.org/10.3389/fgene.2023.1250276
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author McCartan, Robyn
Khorkova, Olga
Volmar, Claude-Henry
Wahlestedt, Claes
author_facet McCartan, Robyn
Khorkova, Olga
Volmar, Claude-Henry
Wahlestedt, Claes
author_sort McCartan, Robyn
collection PubMed
description Nucleic acid-based therapeutics (NBTs) are an emerging class of drugs with potential for the treatment of a wide range of central nervous system conditions. To date, pertaining to CNS indications, there are two commercially available NBTs and a large number of ongoing clinical trials. However, these NBTs are applied directly to the brain due to very low blood brain barrier permeability. In this review, we outline recent advances in chemical modifications of NBTs and NBT delivery techniques intended to promote brain exposure, efficacy, and possible future systemic application.
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spelling pubmed-104686022023-09-01 Nucleic acid-based therapeutics for the treatment of central nervous system disorders McCartan, Robyn Khorkova, Olga Volmar, Claude-Henry Wahlestedt, Claes Front Genet Genetics Nucleic acid-based therapeutics (NBTs) are an emerging class of drugs with potential for the treatment of a wide range of central nervous system conditions. To date, pertaining to CNS indications, there are two commercially available NBTs and a large number of ongoing clinical trials. However, these NBTs are applied directly to the brain due to very low blood brain barrier permeability. In this review, we outline recent advances in chemical modifications of NBTs and NBT delivery techniques intended to promote brain exposure, efficacy, and possible future systemic application. Frontiers Media S.A. 2023-08-16 /pmc/articles/PMC10468602/ /pubmed/37662844 http://dx.doi.org/10.3389/fgene.2023.1250276 Text en Copyright © 2023 McCartan, Khorkova, Volmar and Wahlestedt. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Genetics
McCartan, Robyn
Khorkova, Olga
Volmar, Claude-Henry
Wahlestedt, Claes
Nucleic acid-based therapeutics for the treatment of central nervous system disorders
title Nucleic acid-based therapeutics for the treatment of central nervous system disorders
title_full Nucleic acid-based therapeutics for the treatment of central nervous system disorders
title_fullStr Nucleic acid-based therapeutics for the treatment of central nervous system disorders
title_full_unstemmed Nucleic acid-based therapeutics for the treatment of central nervous system disorders
title_short Nucleic acid-based therapeutics for the treatment of central nervous system disorders
title_sort nucleic acid-based therapeutics for the treatment of central nervous system disorders
topic Genetics
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10468602/
https://www.ncbi.nlm.nih.gov/pubmed/37662844
http://dx.doi.org/10.3389/fgene.2023.1250276
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