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Gene Editing and Alzheimer's Disease: Is There Light at the End of the Tunnel?

Alzheimer's disease continues to be a fatal, incurable neurodegenerative disease, despite many years of efforts to find approaches to its treatment. Here we review recent studies on Alzheimer's disease as a target for gene therapy and specifically, gene editing technology. We also review t...

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Autor principal: Stepanichev, Mikhail
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8525398/
https://www.ncbi.nlm.nih.gov/pubmed/34713213
http://dx.doi.org/10.3389/fgeed.2020.00004
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author Stepanichev, Mikhail
author_facet Stepanichev, Mikhail
author_sort Stepanichev, Mikhail
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description Alzheimer's disease continues to be a fatal, incurable neurodegenerative disease, despite many years of efforts to find approaches to its treatment. Here we review recent studies on Alzheimer's disease as a target for gene therapy and specifically, gene editing technology. We also review the opportunities and limitations of modern methods of gene therapy based on the CRISPR editing system. The opportunities of using this approach for modeling, including cellular and animal models, studying on pathogenesis and disease correction mechanisms, as well as limitations for its therapeutic use are discussed.
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spelling pubmed-85253982021-10-27 Gene Editing and Alzheimer's Disease: Is There Light at the End of the Tunnel? Stepanichev, Mikhail Front Genome Ed Genome Editing Alzheimer's disease continues to be a fatal, incurable neurodegenerative disease, despite many years of efforts to find approaches to its treatment. Here we review recent studies on Alzheimer's disease as a target for gene therapy and specifically, gene editing technology. We also review the opportunities and limitations of modern methods of gene therapy based on the CRISPR editing system. The opportunities of using this approach for modeling, including cellular and animal models, studying on pathogenesis and disease correction mechanisms, as well as limitations for its therapeutic use are discussed. Frontiers Media S.A. 2020-06-03 /pmc/articles/PMC8525398/ /pubmed/34713213 http://dx.doi.org/10.3389/fgeed.2020.00004 Text en Copyright © 2020 Stepanichev. 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 Genome Editing
Stepanichev, Mikhail
Gene Editing and Alzheimer's Disease: Is There Light at the End of the Tunnel?
title Gene Editing and Alzheimer's Disease: Is There Light at the End of the Tunnel?
title_full Gene Editing and Alzheimer's Disease: Is There Light at the End of the Tunnel?
title_fullStr Gene Editing and Alzheimer's Disease: Is There Light at the End of the Tunnel?
title_full_unstemmed Gene Editing and Alzheimer's Disease: Is There Light at the End of the Tunnel?
title_short Gene Editing and Alzheimer's Disease: Is There Light at the End of the Tunnel?
title_sort gene editing and alzheimer's disease: is there light at the end of the tunnel?
topic Genome Editing
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8525398/
https://www.ncbi.nlm.nih.gov/pubmed/34713213
http://dx.doi.org/10.3389/fgeed.2020.00004
work_keys_str_mv AT stepanichevmikhail geneeditingandalzheimersdiseaseistherelightattheendofthetunnel