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...
| Main Authors: | , , , , , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2021-08-01
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| Series: | International Journal of Molecular Sciences |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1422-0067/22/17/9241 |
| _version_ | 1797521398046916608 |
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| author | David Arango Amaury Bittar Natalia P. Esmeral Camila Ocasión Carolina Muñoz-Camargo Juan C. Cruz Luis H. Reyes Natasha I. Bloch |
| author_facet | David Arango Amaury Bittar Natalia P. Esmeral Camila Ocasión Carolina Muñoz-Camargo Juan C. Cruz Luis H. Reyes Natasha I. Bloch |
| author_sort | David Arango |
| collection | DOAJ |
| 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. |
| first_indexed | 2024-03-10T08:12:02Z |
| format | Article |
| id | doaj.art-c4272a785de74e30a45784e5eb092319 |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-10T08:12:02Z |
| publishDate | 2021-08-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-c4272a785de74e30a45784e5eb0923192023-11-22T10:40:12ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672021-08-012217924110.3390/ijms22179241Understanding the Potential of Genome Editing in Parkinson’s DiseaseDavid Arango0Amaury Bittar1Natalia P. Esmeral2Camila Ocasión3Carolina Muñoz-Camargo4Juan C. Cruz5Luis H. Reyes6Natasha I. Bloch7Department of Biomedical Engineering, Universidad de los Andes, Bogotá 111711, ColombiaDepartment of Biomedical Engineering, Universidad de los Andes, Bogotá 111711, ColombiaDepartment of Biomedical Engineering, Universidad de los Andes, Bogotá 111711, ColombiaGrupo de Diseño de Productos y Procesos, Department of Chemical and Food Engineering, Universidad de los Andes, Bogotá 111711, ColombiaDepartment of Biomedical Engineering, Universidad de los Andes, Bogotá 111711, ColombiaDepartment of Biomedical Engineering, Universidad de los Andes, Bogotá 111711, ColombiaGrupo de Diseño de Productos y Procesos, Department of Chemical and Food Engineering, Universidad de los Andes, Bogotá 111711, ColombiaDepartment of Biomedical Engineering, Universidad de los Andes, Bogotá 111711, ColombiaCRISPR 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.https://www.mdpi.com/1422-0067/22/17/9241gene editingCRISPR/CasParkinson’s diseasedelivery vehiclesnanostructured materialsclinical trials |
| spellingShingle | David Arango Amaury Bittar Natalia P. Esmeral Camila Ocasión Carolina Muñoz-Camargo Juan C. Cruz Luis H. Reyes Natasha I. Bloch Understanding the Potential of Genome Editing in Parkinson’s Disease International Journal of Molecular Sciences gene editing CRISPR/Cas Parkinson’s disease delivery vehicles nanostructured materials clinical trials |
| 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 | gene editing CRISPR/Cas Parkinson’s disease delivery vehicles nanostructured materials clinical trials |
| url | https://www.mdpi.com/1422-0067/22/17/9241 |
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