Gene Therapy for Parkinson's Disease

Abstract

Gene therapy is a clinical tool that may eventually provide therapeutic benefit to patients suffering from movement disorders through a few potential mechanisms: direct correction of the pathogenic mechanism, neuroprotection, neurorestoration or symptom control. The therapeutic mechanism is therefore dependent on knowledge of disease pathogenesis and the required temporal and spatial specificities of gene expression. An additional critical challenge is achieving the most complete transduction of the target structure while avoiding leakage into neighboring regions or perivascular spaces. Although critical clinical work is ongoing to optimize the direct intracerebral delivery of transgenes to the brain, the field has recently entered a new technological era, where interventional-MRI-guided convection-enhanced delivery is the gold standard for verifying accurate vector delivery in real-time.