An Update on Gene Therapy Approaches for Parkinson’s Disease: Restoration of Dopaminergic Function
Amber D. Van Laar, Victor S. Van Laar, Waldy San Sebastián, Aristide Merola, J. Bradley Elder, Russell R. Lonser, Krystof S. Bankiewicz
Abstract
At present there is a significant unmet need for clinically available treatments for Parkinson's disease (PD) patients to stably restore balance to dopamine network function, leaving patients with inadequate management of symptoms as the disease progresses. Gene therapy is an attractive approach to impart a durable effect on neuronal function through introduction of genetic material to reestablish dopamine levels and/or functionally recover dopaminergic signaling by improving neuronal health. Ongoing clinical gene therapy trials in PD are focused on enzymatic enhancement of dopamine production and/or the restoration of the nigrostriatal pathway to improve dopaminergic network function. In this review, we discuss data from current gene therapy trials for PD and recent advances in study design and surgical approaches.