Gene therapy for Parkinson's disease: where are we now and where are we going?. (December 2010)

• Record Type: Journal Article
• Authors: Forsayeth, John; Bankiewicz, Krystof S; Aminoff, Michael J
• Is Part Of: Expert review of neurotherapeutics. Volume 10:Number 12(2010)
• Page Start: 1839
• Page End: 1845
• Publication Date: 2010-12
• Subjects: AAV2–DDNF -- AAV2–hAADC -- AAV–GAD -- adeno-associated virus -- CERE-120 -- gene therapy -- MRI -- Parkinson's disease; Neuropharmacology -- Periodicals; 615.7805
• DOI: 10.1586/ern.10.161
• Languages: English
• ISSNs: 1473-7175

Abstract

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.