CRISPR-Cas9 and Parkinson’s Disease: A Review of Gene Editing Strategies and Therapeutic Potential

Parkinson’s Disease (PD) is a progressive neurodegenerative disorder characterized by the loss of dopaminergic neurons, leading to motor impairments such as tremors, rigidity, and bradykinesia, as well as cognitive decline. Current treatments, including dopaminergic medications and deep brain stimulation, manage symptoms but do not stop disease progression. Genetic mutations in SNCA, LRRK2, and PARKIN, along with environmental factors, contribute to PD’s development. CRISPR Cas9 presents a potential therapeutic avenue by enabling precise gene modifications to correct PD related mutations, reduce toxic protein accumulation, and improve mitochondrial function. It also enhances stem cell therapies, offering new possibilities for disease modification. However, challenges such as targeted gene delivery, off-target effects, and ethical concerns must be addressed before clinical implementation. This paper explores PD’s underlying mechanisms, current treatment limitations, and CRISPR’s role in developing novel therapies. It also examines the scientific and ethical challenges that must be navigated to bring CRISPR-based interventions into practical use for PD patients.

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Publication Details

Journal
American journal of student research.
Published
2025-01-01
DOI
https://doi.org/10.70251/hyjr2348.35525535
Primary Topic
CRISPR and Genetic Engineering
Type
article
Field-Weighted Citation Impact
0.00

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article

CRISPR-Cas9 and Parkinson’s Disease: A Review of Gene Editing Strategies and Therapeutic Potential

Kiara L. Rodríguez-Acevedo, Muhammad Zeshan
American journal of student research.
CRISPR and Genetic Engineering
article

CRISPR-Cas9 and Parkinson’s Disease: A Review of Gene Editing Strategies and Therapeutic Potential

Kiara L. Rodríguez-Acevedo, Muhammad Zeshan
article en

Abstract

Parkinson’s Disease (PD) is a progressive neurodegenerative disorder characterized by the loss of dopaminergic neurons, leading to motor impairments such as tremors, rigidity, and bradykinesia, as well as cognitive decline. Current treatments, including dopaminergic medications and deep brain stimulation, manage symptoms but do not stop disease progression. Genetic mutations in SNCA, LRRK2, and PARKIN, along with environmental factors, contribute to PD’s development. CRISPR Cas9 presents a potential therapeutic avenue by enabling precise gene modifications to correct PD related mutations, reduce toxic protein accumulation, and improve mitochondrial function. It also enhances stem cell therapies, offering new possibilities for disease modification. However, challenges such as targeted gene delivery, off-target effects, and ethical concerns must be addressed before clinical implementation. This paper explores PD’s underlying mechanisms, current treatment limitations, and CRISPR’s role in developing novel therapies. It also examines the scientific and ethical challenges that must be navigated to bring CRISPR-based interventions into practical use for PD patients.

American journal of student research.
National Science Foundation, Yale University
Openalex Percentile: Top 76%
CRISPR and Genetic Engineering
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