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...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2022-06-01
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| Series: | Pharmaceutics |
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| Online Access: | https://www.mdpi.com/1999-4923/14/6/1252 |
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| author | Mujeeb ur Rahman Muhammad Bilal Junaid Ali Shah Ajeet Kaushik Pierre-Louis Teissedre Małgorzata Kujawska |
| author_facet | Mujeeb ur Rahman Muhammad Bilal Junaid Ali Shah Ajeet Kaushik Pierre-Louis Teissedre Małgorzata Kujawska |
| author_sort | Mujeeb ur Rahman |
| collection | DOAJ |
| 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. |
| first_indexed | 2024-03-09T22:46:00Z |
| format | Article |
| id | doaj.art-f11c05cf65ea49489ba07155c2d6762f |
| institution | Directory Open Access Journal |
| issn | 1999-4923 |
| language | English |
| last_indexed | 2024-03-09T22:46:00Z |
| publishDate | 2022-06-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceutics |
| spelling | doaj.art-f11c05cf65ea49489ba07155c2d6762f2023-11-23T18:30:44ZengMDPI AGPharmaceutics1999-49232022-06-01146125210.3390/pharmaceutics14061252CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s DiseaseMujeeb ur Rahman0Muhammad Bilal1Junaid Ali Shah2Ajeet Kaushik3Pierre-Louis Teissedre4Małgorzata Kujawska5Department of Toxicology, Faculty of Pharmacy, Poznan University of Medical Sciences, Dojazd 30, 60-631 Poznan, PolandCollege of Biotechnology, Tianjin University of Science and Technology, Tianjin 300457, ChinaCollege of Life Sciences, Jilin University, Changchun 130012, ChinaNanoBioTech Laboratory, Health System Engineering, Department of Environmental Engineering, Florida Polytechnic University, Lakeland, FL 33805, USAInstitut des Sciences de la Vigne et du Vin, Université de Bordeaux, EA 4577, Œnologie, 210 Chemin de Leysotte, F-33140 Villenave d’Ornon, FranceDepartment of Toxicology, Faculty of Pharmacy, Poznan University of Medical Sciences, Dojazd 30, 60-631 Poznan, PolandParkinson’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.https://www.mdpi.com/1999-4923/14/6/1252Parkinson’sCRISPR-Cas9gene therapydeliveryapplications |
| spellingShingle | Mujeeb ur Rahman Muhammad Bilal Junaid Ali Shah Ajeet Kaushik Pierre-Louis Teissedre Małgorzata Kujawska CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease Pharmaceutics Parkinson’s CRISPR-Cas9 gene therapy delivery applications |
| 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 | Parkinson’s CRISPR-Cas9 gene therapy delivery applications |
| url | https://www.mdpi.com/1999-4923/14/6/1252 |
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