Gene therapy for Parkinson’s Disease: advances, challenges and future directions
DOI:
https://doi.org/10.34119/bjhrv8n5-288Keywords:
Parkinson Disease, genetic therapy, gene expressionAbstract
Introduction: Parkinson’s Disease (PD) is considered the second most frequent neurodegenerative disease in the world, affecting approximately 8.5 million people. Numerous efforts remain in the development of therapies that overcome the limitations of the treatments used for PD, Thus, gene therapy appears to be a promising technique for the treatment of this disease. Objective: To understand the most recent advances in the development of gene therapies for the treatment of PD. Method: The search was carried out in PubMed, Scopus and Cochrane databases using the terms “Genetic Therapy” AND “Parkinson Disease”. The inclusion criteria were complete, free and original articles (clinical trials, cohort, postmortem and in vitro studies) published between 2019 and 2024. Reviews, case series, case-reports, abstracts, letters to the editors, commentaries and animal studies were excluded. Results: neurturin infusion resulted in positive tyrosine hydroxylase staining, dopaminergic activity in substantia nigra and putamen and dopaminergic neuron survival. However, it shows limitations to the sites where it was injected and does not address underlying Lewy body accumulation and α-synuclein particles. Glia cell line-derived neurotrophic factor infusion increased tyrosine hydroxylase concentration and restored neural pathways. VY-AADC01 administration improved responses to levodopa therapy and lower dose requirements to control symptoms. Therapy on exon skipping the mutated PRKN2 gene can be used to maintain neuron function and avoid degradation, a potentially therapy for juvenile-onset PD. Conclusion: Gene therapy offers disease modifying and non-modifying approaches to PD treatment, showing significant advancements that may provide long-lasting treatment alternatives in the future.
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