Development of advanced therapies based on viral vector-mediated overexpression of therapeutic molecules and knockdown of disease-related genes for Parkinson’s disease

Abstract

The last decade witnessed the translation of several gene-based therapeutic approaches from experimental studies to early clinical trials. Studies targeting the treatment of Parkinson’s disease (PD) were among the forefront of trials in the CNS. In this article, we overview three major strategies for the treatment of PD: the enzyme-replacement strategies are based on well-defined principles of functional restoration and are well suited for treatment of patients with advanced disease who would typically experience complications due to side effects of pharmacotherapy. Neurotrophic factor delivery, on the other hand, aims to delay the disability and eventually modifiy disease progression. Finally, we present an outlook to a completely new way of interfering with the disease process, which is taking advantage of recently discovered RNAi mechanisms in cells. Gene therapy is now becoming a reality in the clinics and developments in the next decade will help uncover the true potential of this approach for not only the treatment of PD patients, but also many other neurological disorders.