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Understanding the Potential of Genome Editing in Parkinson’s Disease
CRISPR is a simple and cost-efficient gene-editing technique that has become increasingly popular over the last decades. Various CRISPR/Cas-based applications have been developed to introduce changes in the genome and alter gene expression in diverse systems and tissues. These novel gene-editing tec...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8430539/ https://www.ncbi.nlm.nih.gov/pubmed/34502143 http://dx.doi.org/10.3390/ijms22179241 |
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author | Arango, David Bittar, Amaury Esmeral, Natalia P. Ocasión, Camila Muñoz-Camargo, Carolina Cruz, Juan C. Reyes, Luis H. Bloch, Natasha I. |
author_facet | Arango, David Bittar, Amaury Esmeral, Natalia P. Ocasión, Camila Muñoz-Camargo, Carolina Cruz, Juan C. Reyes, Luis H. Bloch, Natasha I. |
author_sort | Arango, David |
collection | PubMed |
description | CRISPR is a simple and cost-efficient gene-editing technique that has become increasingly popular over the last decades. Various CRISPR/Cas-based applications have been developed to introduce changes in the genome and alter gene expression in diverse systems and tissues. These novel gene-editing techniques are particularly promising for investigating and treating neurodegenerative diseases, including Parkinson’s disease, for which we currently lack efficient disease-modifying treatment options. Gene therapy could thus provide treatment alternatives, revolutionizing our ability to treat this disease. Here, we review our current knowledge on the genetic basis of Parkinson’s disease to highlight the main biological pathways that become disrupted in Parkinson’s disease and their potential as gene therapy targets. Next, we perform a comprehensive review of novel delivery vehicles available for gene-editing applications, critical for their successful application in both innovative research and potential therapies. Finally, we review the latest developments in CRISPR-based applications and gene therapies to understand and treat Parkinson’s disease. We carefully examine their advantages and shortcomings for diverse gene-editing applications in the brain, highlighting promising avenues for future research. |
format | Online Article Text |
id | pubmed-8430539 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-84305392021-09-11 Understanding the Potential of Genome Editing in Parkinson’s Disease Arango, David Bittar, Amaury Esmeral, Natalia P. Ocasión, Camila Muñoz-Camargo, Carolina Cruz, Juan C. Reyes, Luis H. Bloch, Natasha I. Int J Mol Sci Review CRISPR is a simple and cost-efficient gene-editing technique that has become increasingly popular over the last decades. Various CRISPR/Cas-based applications have been developed to introduce changes in the genome and alter gene expression in diverse systems and tissues. These novel gene-editing techniques are particularly promising for investigating and treating neurodegenerative diseases, including Parkinson’s disease, for which we currently lack efficient disease-modifying treatment options. Gene therapy could thus provide treatment alternatives, revolutionizing our ability to treat this disease. Here, we review our current knowledge on the genetic basis of Parkinson’s disease to highlight the main biological pathways that become disrupted in Parkinson’s disease and their potential as gene therapy targets. Next, we perform a comprehensive review of novel delivery vehicles available for gene-editing applications, critical for their successful application in both innovative research and potential therapies. Finally, we review the latest developments in CRISPR-based applications and gene therapies to understand and treat Parkinson’s disease. We carefully examine their advantages and shortcomings for diverse gene-editing applications in the brain, highlighting promising avenues for future research. MDPI 2021-08-26 /pmc/articles/PMC8430539/ /pubmed/34502143 http://dx.doi.org/10.3390/ijms22179241 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Arango, David Bittar, Amaury Esmeral, Natalia P. Ocasión, Camila Muñoz-Camargo, Carolina Cruz, Juan C. Reyes, Luis H. Bloch, Natasha I. Understanding the Potential of Genome Editing in Parkinson’s Disease |
title | Understanding the Potential of Genome Editing in Parkinson’s Disease |
title_full | Understanding the Potential of Genome Editing in Parkinson’s Disease |
title_fullStr | Understanding the Potential of Genome Editing in Parkinson’s Disease |
title_full_unstemmed | Understanding the Potential of Genome Editing in Parkinson’s Disease |
title_short | Understanding the Potential of Genome Editing in Parkinson’s Disease |
title_sort | understanding the potential of genome editing in parkinson’s disease |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8430539/ https://www.ncbi.nlm.nih.gov/pubmed/34502143 http://dx.doi.org/10.3390/ijms22179241 |
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