Abstract
Parkinson's Disease (PD), a progressive neurodegenerative disorder characterized by dopaminergic neuron degeneration, leads to motor and non-motor symptoms that significantly impair quality of life. Conventional treatments, such as levodopa, provide symptom relief but are associated with long-term complications and do not halt disease progression. This review systematically evaluates the efficacy and safety of gene therapy targeting the aromatic L-amino acid decarboxylase (AADC) enzyme in treating PD and AADC deficiency, focusing on sustained dopamine production and motor function improvement. A systematic literature review was conducted using databases like PubMed, Scopus, and Embase, covering studies published over the past 32 years. Out of 52 articles identified, 25 met inclusion criteria, providing data on motor outcomes, dopamine production, and safety profiles. Findings suggest that AADC gene therapy may offer a durable therapeutic approach, reducing reliance on conventional dopamine replacement therapies and mitigating related complications. This review highlights gene therapy's potential in clinical practice, emphasizing a shift towards targeted treatment strategies for dopamine-deficient neurodegenerative disorders. Further research should address long-term efficacy and safety across diverse patient groups. Limitations include potential selection bias and language restrictions.
Published Version
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