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CRISPR-Cas9: A Promising Genome Editing Therapeutic Tool for Alzheimer’s Disease—A Narrative Review

Alzheimer’s disease (AD) is a chronic and irreversible neurodegenerative disorder characterized by cognitive deficiency and development of amyloid-β (Aβ) plaques and neurofibrillary tangles, comprising hyperphosphorylated tau. The number of patients with AD is alarmingly increasing worldwide; curren...

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Autores principales: Barman, Nirmal Chandra, Khan, Niuz Morshed, Islam, Maidul, Nain, Zulkar, Roy, Rajib Kanti, Haque, Anwarul, Barman, Shital Kumar
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Healthcare 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7606404/
https://www.ncbi.nlm.nih.gov/pubmed/33089409
http://dx.doi.org/10.1007/s40120-020-00218-z
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author Barman, Nirmal Chandra
Khan, Niuz Morshed
Islam, Maidul
Nain, Zulkar
Roy, Rajib Kanti
Haque, Anwarul
Barman, Shital Kumar
author_facet Barman, Nirmal Chandra
Khan, Niuz Morshed
Islam, Maidul
Nain, Zulkar
Roy, Rajib Kanti
Haque, Anwarul
Barman, Shital Kumar
author_sort Barman, Nirmal Chandra
collection PubMed
description Alzheimer’s disease (AD) is a chronic and irreversible neurodegenerative disorder characterized by cognitive deficiency and development of amyloid-β (Aβ) plaques and neurofibrillary tangles, comprising hyperphosphorylated tau. The number of patients with AD is alarmingly increasing worldwide; currently, at least 50 million people are thought to be living with AD. The mutations or alterations in amyloid-β precursor protein (APP), presenilin-1 (PSEN1), or presenilin-2 (PSEN2) genes are known to be associated with the pathophysiology of AD. Effective medication for AD is still elusive and many gene-targeted clinical trials have failed to meet the expected efficiency standards. The genome editing tool clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 has been emerging as a powerful technology to correct anomalous genetic functions and is now widely applied to the study of AD. This simple yet powerful tool for editing genes showed the huge potential to correct the unwanted mutations in AD-associated genes such as APP, PSEN1, and PSEN2. So, it has opened a new door for the development of empirical AD models, diagnostic approaches, and therapeutic lines in studying the complexity of the nervous system ranging from different cell types (in vitro) to animals (in vivo). This review was undertaken to study the related mechanisms and likely applications of CRISPR-Cas9 as an effective therapeutic tool in treating AD.
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spelling pubmed-76064042020-11-10 CRISPR-Cas9: A Promising Genome Editing Therapeutic Tool for Alzheimer’s Disease—A Narrative Review Barman, Nirmal Chandra Khan, Niuz Morshed Islam, Maidul Nain, Zulkar Roy, Rajib Kanti Haque, Anwarul Barman, Shital Kumar Neurol Ther Review Alzheimer’s disease (AD) is a chronic and irreversible neurodegenerative disorder characterized by cognitive deficiency and development of amyloid-β (Aβ) plaques and neurofibrillary tangles, comprising hyperphosphorylated tau. The number of patients with AD is alarmingly increasing worldwide; currently, at least 50 million people are thought to be living with AD. The mutations or alterations in amyloid-β precursor protein (APP), presenilin-1 (PSEN1), or presenilin-2 (PSEN2) genes are known to be associated with the pathophysiology of AD. Effective medication for AD is still elusive and many gene-targeted clinical trials have failed to meet the expected efficiency standards. The genome editing tool clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 has been emerging as a powerful technology to correct anomalous genetic functions and is now widely applied to the study of AD. This simple yet powerful tool for editing genes showed the huge potential to correct the unwanted mutations in AD-associated genes such as APP, PSEN1, and PSEN2. So, it has opened a new door for the development of empirical AD models, diagnostic approaches, and therapeutic lines in studying the complexity of the nervous system ranging from different cell types (in vitro) to animals (in vivo). This review was undertaken to study the related mechanisms and likely applications of CRISPR-Cas9 as an effective therapeutic tool in treating AD. Springer Healthcare 2020-10-21 /pmc/articles/PMC7606404/ /pubmed/33089409 http://dx.doi.org/10.1007/s40120-020-00218-z Text en © The Author(s) 2020 Open Access This article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial 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-nc/4.0/.
spellingShingle Review
Barman, Nirmal Chandra
Khan, Niuz Morshed
Islam, Maidul
Nain, Zulkar
Roy, Rajib Kanti
Haque, Anwarul
Barman, Shital Kumar
CRISPR-Cas9: A Promising Genome Editing Therapeutic Tool for Alzheimer’s Disease—A Narrative Review
title CRISPR-Cas9: A Promising Genome Editing Therapeutic Tool for Alzheimer’s Disease—A Narrative Review
title_full CRISPR-Cas9: A Promising Genome Editing Therapeutic Tool for Alzheimer’s Disease—A Narrative Review
title_fullStr CRISPR-Cas9: A Promising Genome Editing Therapeutic Tool for Alzheimer’s Disease—A Narrative Review
title_full_unstemmed CRISPR-Cas9: A Promising Genome Editing Therapeutic Tool for Alzheimer’s Disease—A Narrative Review
title_short CRISPR-Cas9: A Promising Genome Editing Therapeutic Tool for Alzheimer’s Disease—A Narrative Review
title_sort crispr-cas9: a promising genome editing therapeutic tool for alzheimer’s disease—a narrative review
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7606404/
https://www.ncbi.nlm.nih.gov/pubmed/33089409
http://dx.doi.org/10.1007/s40120-020-00218-z
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