Gene therapy for Parkinson's disease: where are we now and where are we going?

The number of patients worldwide who have received some kind of gene therapy is now in the thousands. A subset of that number have received intracranial injections of adeno-associated viruses encoding various therapeutic genes directed at ameliorating Parkinson's disease (PD). In this article we briefly examine the current status of Phase I and Phase II trials of gene therapy for PD and preview some of the improvements in delivery technology that promise to make adeno-associated-virus-based gene therapy for PD safer and more accessible to interventional neurologists around the world.