Restorative gene therapy approaches to Parkinson's disease

The application of in vivo and ex vivo gene transfer technologies to Parkinson's disease has advanced significantly over the past decade. As a result, it is likely that clinical trials of one or more of these systems will be initiated before the end of this century. These approaches are focused on using genetic information to guide the synthesis of desired proteins in the human brain that can either increase dopaminergic tone or provide a neuroregenerative/neuroprotective milieu. These systems provide a revolutionary concept based on altering the genetic framework of a cell, bypassing the extracellular receptor-drug interactions on which current pharmacological approaches rely.