Gene Therapy for Parkinson’s Disease

Gene therapy is a promising future tool for treatment of Parkinson’s disease (PD) and several different strategies are currently being evaluated. Although many of these strategies have shown promising results in animal models of PD and parkinsonian patients, some have been less effective and caused adverse side effects. A vector system with high specificity and appropriate expression level of the transgene is needed to make gene therapy for PD as safe and beneficial as possible. The vector should also be relevant for the disease. Here, we present a method to design promoters relevant for PD, using microarray data from patients, and validation of these in vivo. The method also includes fine-tuning of promoter candidates by adding miRNA target sites to increase cell specificity. (Less)