Journal article
CRISPR System: A High-throughput Toolbox for Research and Treatment of Parkinson’s Disease
Cellular and molecular neurobiology, Vol.40(4), pp.477-493
05/01/2020
DOI: 10.1007/s10571-019-00761-w
PMCID: PMC11448816
PMID: 31773362
Abstract
In recent years, the innovation of gene-editing tools such as the CRISPR/Cas9 system improves the translational gap of treatments mediated by gene therapy. The privileges of CRISPR/Cas9 such as working in living cells and organs candidate this technology for using in research and treatment of the central nervous system (CNS) disorders. Parkinson’s disease (PD) is a common, debilitating, neurodegenerative disorder which occurs due to loss of dopaminergic neurons and is associated with progressive motor dysfunction. Knowledge about the pathophysiological basis of PD has altered the classification system of PD, which manifests in familial and sporadic forms. The first genetic linkage studies in PD demonstrated the involvement of Synuclein alpha (SNCA) mutations and SNCA genomic duplications in the pathogenesis of PD familial forms. Subsequent studies have also insinuated mutations in leucine repeat kinase-2 (LRRK2), Parkin, PTEN-induced putative kinase 1 (PINK1), as well as DJ-1 causing familial forms of PD. This review will attempt to discuss the structure, function, and development in genome editing mediated by CRISP/Cas9 system. Further, it describes the genes involved in the pathogenesis of PD and the pertinent alterations to them. We will pursue this line by delineating the PD linkage studies in which CRISPR system was employed. Finally, we will discuss the pros and cons of CRISPR employment vis-à-vis the process of genome editing in PD patients’ iPSCs.
Details
- Title: Subtitle
- CRISPR System: A High-throughput Toolbox for Research and Treatment of Parkinson’s Disease
- Creators
- Fatemeh Safari - Shiraz University of Medical SciencesGholamreza Hatam - Shiraz University of Medical SciencesAbbas Behzad Behbahani - Shiraz University of Medical SciencesVahid Rezaei - Shiraz University of Medical SciencesMazyar Barekati‑Mowahed - Case Western Reserve UniversityPeyman Petramfar - Shiraz University of Medical SciencesFarzaneh Khademi - Shiraz University of Medical Sciences
- Resource Type
- Journal article
- Publication Details
- Cellular and molecular neurobiology, Vol.40(4), pp.477-493
- DOI
- 10.1007/s10571-019-00761-w
- PMID
- 31773362
- PMCID
- PMC11448816
- NLM abbreviation
- Cell Mol Neurobiol
- ISSN
- 0272-4340
- eISSN
- 1573-6830
- Publisher
- Springer US
- Number of pages
- 17
- Language
- English
- Date published
- 05/01/2020
- Academic Unit
- Neurology
- Record Identifier
- 9985014706902771
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