The Application and Potential of CRISPR-Cas9 in the Treatment of Parkinson's Disease

Parkinson's disease (PD) is a progressive neurodegenerative disorder that contains the significant characters of gradual degeneration of dopaminergic neurons in the substantia nigra of the human brain. This disease significantly affects patients lives significantly affecting the degeneration of movement and consciousness. Despite a large amount of research results, there is still no way that can fully cure PD. The genetic factor has played a crucial role in the pathogenesis of PD, with mutations in genes such as SNCA, Parkin, PINK1, DJ-1 and LRRK2 being closely related to the disease. It highlights the potential of gene targeting therapy. CRISPR-Cas 9 is a revolutionary gene editing tool, first found in bacteria. Its flexibility and feasibility give hope to the treatment of PD. The review will discuss how CRISPR-Cas9 can be used in the PD treatment and its application with these gene editing targets. In addition, CRISPR-Cas 9, this kind of gene therapy, is also facing some big challenges like off-target activity, human body inflammation, and ethical problems. In conclusion, to make CRISPR-Cas 9 and PD combined treatment can have a good development for the clinic, it still needs to overcome a lot of technological barriers and moral controversy.

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Publication Details

Journal
Theoretical and Natural Science
Published
2025-07-24
DOI
https://doi.org/10.54254/2753-8818/2025.au25412
Primary Topic
CRISPR and Genetic Engineering
Type
article
Field-Weighted Citation Impact
0.00
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article

The Application and Potential of CRISPR-Cas9 in the Treatment of Parkinson's Disease

Peng Dai
Theoretical and Natural Science
CRISPR and Genetic Engineering
article

The Application and Potential of CRISPR-Cas9 in the Treatment of Parkinson's Disease

Peng Dai
article en

Abstract

Parkinson's disease (PD) is a progressive neurodegenerative disorder that contains the significant characters of gradual degeneration of dopaminergic neurons in the substantia nigra of the human brain. This disease significantly affects patients lives significantly affecting the degeneration of movement and consciousness. Despite a large amount of research results, there is still no way that can fully cure PD. The genetic factor has played a crucial role in the pathogenesis of PD, with mutations in genes such as SNCA, Parkin, PINK1, DJ-1 and LRRK2 being closely related to the disease. It highlights the potential of gene targeting therapy. CRISPR-Cas 9 is a revolutionary gene editing tool, first found in bacteria. Its flexibility and feasibility give hope to the treatment of PD. The review will discuss how CRISPR-Cas9 can be used in the PD treatment and its application with these gene editing targets. In addition, CRISPR-Cas 9, this kind of gene therapy, is also facing some big challenges like off-target activity, human body inflammation, and ethical problems. In conclusion, to make CRISPR-Cas 9 and PD combined treatment can have a good development for the clinic, it still needs to overcome a lot of technological barriers and moral controversy.

Theoretical and Natural ScienceVol. 126(1)
Chaminade College Preparatory School (US)
Openalex Percentile: Top 84%
CRISPR and Genetic Engineering
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The Application and Potential of CRISPR-Cas9 in the Treatment of Parkinson's Disease — Peng Dai · Theoretical and Natural Science (2025) | TGRS Research Map | TGRS