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

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.

Keywords::

• AAV2–DDNF
• AAV2–hAADC
• AAV–GAD
• adeno-associated virus
• CERE-120
• gene therapy
• MRI
• Parkinson’s disease

Financial & competing interests disclosure

Michael Aminoff was the primary investigator on a Phase I study of the AADC gene, funded by Genzyme. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.