Mutations, oxidative stress and PD
In another study, researchers used CRISPR / Cas9 to delete PARKIN( PRKN), DJ-1( PARK7) and ATP13A2( PARK9) in isogenic lines of nigral dopaminergic neurons— populations with identical genes. Transcriptomic and proteomic analyses showed dysregulation of oxidative-stress pathways across all the edited lines. Oxidative stress— an imbalance between reactive oxygen species( ROS) accumulating in cells and the body’ s ability to detoxify them— can damage cells, and the degeneration of dopaminergic neurons in the substantia nigra is the immediate cause of PD. Finding the genetic mutations that drive this degeneration, and potentially fixing them, could therefore be hugely valuable. In future, CRISPR – Cas9 might be used to change or eliminate these genes.
CRISPR has thus proved invaluable for understanding the molecular basis of PD, revealing the correlation between mutations, oxidative stress and disease. But therapeutic application remains early-stage and risky: PD is caused by mutations in several genes, not one; delivering edits precisely to affected regions such as the substantia nigra is difficult; and unwanted side effects— including unintended genome alterations that could lead to cancer— cannot yet be fully avoided. CRISPR also raises ethical concerns, and is currently restricted to a few therapeutic uses such as treating sickle-cell anaemia.
Conclusion
CRISPR uses the Cas9 enzyme to cut DNA and to add, change or remove genetic material. Applied to Parkinson’ s— a disease that damages dopaminergic neurons in the substantia nigra— it offers the chance to edit the harmful, protein-coding genes so their mutations are never expressed in a patient. So far CRISPR has been a research tool, identifying which genes drive symptoms such as oxidative stress; in future it could edit those genes directly to treat the disease. Significant obstacles remain— the risk of off-target mutations, imprecise delivery, and lingering ethical questions— but CRISPR is a promising candidate to one day treat one of the world’ s most widespread neurodegenerative diseases.
References
01 Akbar, A. et al.( 2025). CRISPR in Neurodegenerative Diseases Treatment. aGenes, 16( 8), 850.
02 Arango, D. et al.( 2021). Understanding the Potential of Genome Editing in Parkinson’ s Disease. Int. J. Mol. Sci., 22( 17), 9241.
03 Baugh, L. S.( 2024). CRISPR | Description, Technology, Uses & Ethical Concerns. Britannica.
04 Rahman, M. et al.( 2022). CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’ s Disease. Pharmaceutics, 14( 6), 1252.
05 Pinjala, P. et al.( 2023). CRISPR / Cas9 assisted stem cell therapy in Parkinson’ s disease. Biomaterials Research, 27( 1), 46.
06 Samuelson, K.( 2025). New key genes in Parkinson’ s disease identified using CRISPR technology. Northwestern.
07 Dickson, D. W.( 2018). Neuropathology of Parkinson disease. Parkinsonism & Related Disorders, 46( 1), S30 – S33.
08 NHS( 2022). Parkinson’ s Disease— Overview and Treatment.
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