Alternative Delivery Systems in Gene Therapy for Parkinson's Disease

Parkinson’s Disease (PD) is a progressive neurodegenerative disorder characterized by motor and non-motor impairments resulting from dopaminergic neuron loss in the substantia nigra. Gene therapy presents a promising approach to restoring dopamine synthesis and alleviating PD symptoms. This study reviews and evaluates current gene therapy strategies, including the use of adeno-associated virus (AAV) and lentiviral vectors, as well as direct infusion of neurotrophic growth factors. By analyzing 50 peer-reviewed articles from 1993 to 2024, the project explores the therapeutic potential and limitations of these delivery systems. AAV and lentiviral vectors show promise in increasing dopaminergic function and sustained protein expression in the striatum, but immune responses and limited vector dispersion reduce their clinical efficacy. Direct infusion of growth factors enhances neurotrophic factor expression yet poses challenges in precise targeting and invasive delivery methods. This review underscores the need for improved delivery technologies to overcome immune and anatomical barriers. These findings contribute to the development of safer, more effective gene therapies for neurodegenerative disorders.