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CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease
Parkinson’s disease (PD) and other chronic and debilitating neurodegenerative diseases (NDs) impose a substantial medical, emotional, and financial burden on individuals and society. The origin of PD is unknown due to a complex combination of hereditary and environmental risk factors. However, over...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9229276/ https://www.ncbi.nlm.nih.gov/pubmed/35745824 http://dx.doi.org/10.3390/pharmaceutics14061252 |
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author | Rahman, Mujeeb ur Bilal, Muhammad Shah, Junaid Ali Kaushik, Ajeet Teissedre, Pierre-Louis Kujawska, Małgorzata |
author_facet | Rahman, Mujeeb ur Bilal, Muhammad Shah, Junaid Ali Kaushik, Ajeet Teissedre, Pierre-Louis Kujawska, Małgorzata |
author_sort | Rahman, Mujeeb ur |
collection | PubMed |
description | Parkinson’s disease (PD) and other chronic and debilitating neurodegenerative diseases (NDs) impose a substantial medical, emotional, and financial burden on individuals and society. The origin of PD is unknown due to a complex combination of hereditary and environmental risk factors. However, over the last several decades, a significant amount of available data from clinical and experimental studies has implicated neuroinflammation, oxidative stress, dysregulated protein degradation, and mitochondrial dysfunction as the primary causes of PD neurodegeneration. The new gene-editing techniques hold great promise for research and therapy of NDs, such as PD, for which there are currently no effective disease-modifying treatments. As a result, gene therapy may offer new treatment options, transforming our ability to treat this disease. We present a detailed overview of novel gene-editing delivery vehicles, which is essential for their successful implementation in both cutting-edge research and prospective therapeutics. Moreover, we review the most recent advancements in CRISPR-based applications and gene therapies for a better understanding of treating PD. We explore the benefits and drawbacks of using them for a range of gene-editing applications in the brain, emphasizing some fascinating possibilities. |
format | Online Article Text |
id | pubmed-9229276 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-92292762022-06-25 CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease Rahman, Mujeeb ur Bilal, Muhammad Shah, Junaid Ali Kaushik, Ajeet Teissedre, Pierre-Louis Kujawska, Małgorzata Pharmaceutics Review Parkinson’s disease (PD) and other chronic and debilitating neurodegenerative diseases (NDs) impose a substantial medical, emotional, and financial burden on individuals and society. The origin of PD is unknown due to a complex combination of hereditary and environmental risk factors. However, over the last several decades, a significant amount of available data from clinical and experimental studies has implicated neuroinflammation, oxidative stress, dysregulated protein degradation, and mitochondrial dysfunction as the primary causes of PD neurodegeneration. The new gene-editing techniques hold great promise for research and therapy of NDs, such as PD, for which there are currently no effective disease-modifying treatments. As a result, gene therapy may offer new treatment options, transforming our ability to treat this disease. We present a detailed overview of novel gene-editing delivery vehicles, which is essential for their successful implementation in both cutting-edge research and prospective therapeutics. Moreover, we review the most recent advancements in CRISPR-based applications and gene therapies for a better understanding of treating PD. We explore the benefits and drawbacks of using them for a range of gene-editing applications in the brain, emphasizing some fascinating possibilities. MDPI 2022-06-13 /pmc/articles/PMC9229276/ /pubmed/35745824 http://dx.doi.org/10.3390/pharmaceutics14061252 Text en © 2022 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 Rahman, Mujeeb ur Bilal, Muhammad Shah, Junaid Ali Kaushik, Ajeet Teissedre, Pierre-Louis Kujawska, Małgorzata CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease |
title | CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease |
title_full | CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease |
title_fullStr | CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease |
title_full_unstemmed | CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease |
title_short | CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease |
title_sort | crispr-cas9-based technology and its relevance to gene editing in parkinson’s disease |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9229276/ https://www.ncbi.nlm.nih.gov/pubmed/35745824 http://dx.doi.org/10.3390/pharmaceutics14061252 |
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