Genetic therapies for neurological disorders

In recent years, it has become increasingly apparent that many neurological disorders are underpinned by a genetic aetiology. This has resulted in considerable efforts to develop therapeutic strategies which can treat the disease-causing mutation, either by supplying a functional copy of the mutated...

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Detalles Bibliográficos
Autores principales: FitzPatrick, Laura, Bird, Adrian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8607967/
https://www.ncbi.nlm.nih.gov/pubmed/34807307
http://dx.doi.org/10.1007/s00439-021-02399-5
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author FitzPatrick, Laura
Bird, Adrian
author_facet FitzPatrick, Laura
Bird, Adrian
author_sort FitzPatrick, Laura
collection PubMed
description In recent years, it has become increasingly apparent that many neurological disorders are underpinned by a genetic aetiology. This has resulted in considerable efforts to develop therapeutic strategies which can treat the disease-causing mutation, either by supplying a functional copy of the mutated gene or editing the genomic sequence. In this review, we will discuss the main genetic strategies which are currently being explored for the treatment of monogenic neurological disorders, as well as some of the challenges they face. In addition, we will address some of the ethical difficulties which may arise.
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spelling pubmed-86079672021-11-23 Genetic therapies for neurological disorders FitzPatrick, Laura Bird, Adrian Hum Genet Review In recent years, it has become increasingly apparent that many neurological disorders are underpinned by a genetic aetiology. This has resulted in considerable efforts to develop therapeutic strategies which can treat the disease-causing mutation, either by supplying a functional copy of the mutated gene or editing the genomic sequence. In this review, we will discuss the main genetic strategies which are currently being explored for the treatment of monogenic neurological disorders, as well as some of the challenges they face. In addition, we will address some of the ethical difficulties which may arise. Springer Berlin Heidelberg 2021-11-22 2022 /pmc/articles/PMC8607967/ /pubmed/34807307 http://dx.doi.org/10.1007/s00439-021-02399-5 Text en © The Author(s) 2021 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) .
spellingShingle Review
FitzPatrick, Laura
Bird, Adrian
Genetic therapies for neurological disorders
title Genetic therapies for neurological disorders
title_full Genetic therapies for neurological disorders
title_fullStr Genetic therapies for neurological disorders
title_full_unstemmed Genetic therapies for neurological disorders
title_short Genetic therapies for neurological disorders
title_sort genetic therapies for neurological disorders
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8607967/
https://www.ncbi.nlm.nih.gov/pubmed/34807307
http://dx.doi.org/10.1007/s00439-021-02399-5
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