CRISPR System: A High-throughput Toolbox for Research and Treatment of Parkinson’s Disease

  • Fatemeh Safari
  • Gholamreza Hatam
  • Abbas Behzad Behbahani
  • Vahid Rezaei
  • Mazyar Barekati‑Mowahed
  • Peyman Petramfar
  • Farzaneh KhademiEmail author
Review Paper


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.


Parkinson disease Gene editing CRISPR-associated protein 9 Induced pluripotent stem cells Neuroinflammation 



It is certified that no funding has been received for the preparation of this manuscript

Compliance with Ethical Standards

Conflict of interest

The authors declare that they have no conflict of interest.

Ethical Approval

This article does not contain any studies with human participants or animals performed by any of the authors.

Informed Consent

As no human participants are involved in this study no informed consent had been obtained.


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Authors and Affiliations

  1. 1.Department of Medical Biotechnology, School of Advanced Medical Sciences and TechnologiesShiraz University of Medical SciencesShirazIran
  2. 2.Basic Sciences in Infectious Diseases Research Center, School of Advanced Medical Sciences and TechnologiesShiraz University of Medical SciencesShirazIran
  3. 3.Diagnostic Laboratory Sciences and Technology Research Center, School of Paramedical SciencesShiraz University of Medical SciencesShirazIran
  4. 4.Department of Medical Nanotechnology, School of Advanced Medical Sciences and TechnologiesShiraz University of Medical SciencesShirazIran
  5. 5.Department of Physiology & Biophysics, School of MedicineCase Western Reserve UniversityOhioUSA
  6. 6.Department of Neurology, Clinical Neurology Research CenterShiraz University of Medical SciencesShirazIran
  7. 7.Department of Tissue Engineering and Applied Cell Sciences, School of Advanced Medical Sciences and TechnologiesShiraz University of Medical SciencesShirazIran

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