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image of Advancement in Gene Therapy for the Treatment of Parkinson’s Disease: A Comprehensive Review

Abstract

Parkinson’s Disease (PD) is a neurological disease marked by the buildup of α-synuclein. The main symptom of the disease is the degeneration of dopaminergic neurons in the substantia nigra pars compacta (SNc). Gene therapy may be a treatment option for PD and has been used in clinical trials to treat a variety of illnesses in the human brain. Currently, the majority of gene therapy clinical studies are being conducted to treat PD. The primary objective is to enhance medications that address motor issues. Patients with PD have been the subjects of several gene therapy treatment techniques that have been developed and tested. Genes are typically transported to neurons in brain regions relevant to PD, such as the striatum, using viral vectors. It may only be necessary to administer these gene delivery methods once, and they may induce expression to persist for an extended time. Several neurotrophic factors, including neurturin, GDNF, BDNF, CDNF, and VEGF-A, have demonstrated promising outcomes in preclinical models as potential disease-modifying targets that may slow disease development. Currently available treatment regimens for PD mostly comprise the administration of levodopa (L-DOPA), dopamine agonists or MAO-B inhibitors, or surgery in the form of deep brain stimulation or neuroablative surgery, among other options. Many different targeting moieties for PD treatment, as well as current treatment techniques and gene therapy methodologies, are covered in this review article. The research reviewed the relevant literature on the potential role of gene therapy for the treatment of PD. The research articles are obtained through various databases, including ScienceDirect, Scopus, PubMed, and Google Scholar. This review includes various targeting moieties for the treatment of PD, current PD treatment strategies, PD treatment using gene therapy, comparison of risk-benefit ratios of gene therapy vs. DBS/drugs, and gene vector technology in the treatment of PD. This review compiles data on Parkinson's disease, its current treatment strategies, and the potential role of gene therapy in its treatment.

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2025-11-04
2026-02-22

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Advancement in Gene Therapy for the Treatment of Parkinson’s Disease: A Comprehensive Review
Bentham Science Publishers ; https://doi.org/10.2174/0113895575405524251008112752
/content/journals/mrmc/10.2174/0113895575405524251008112752
/content/journals/mrmc/10.2174/0113895575405524251008112752
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  • Article Type:     Review Article
Keywords: dopamine ; Parkinson’s disease ; BDNF ; neurotrophic factors ; GDNF ; gene therapy

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