Next Article in Journal
a-Anilinoketones, Esters and Amides: A Chemical Study
Next Article in Special Issue
Physical Factors Affecting Plasmid DNA Compaction in Stearylamine-Containing Nanoemulsions Intended for Gene Delivery
Previous Article in Journal
Cannabidiol in Humans—The Quest for Therapeutic Targets
Previous Article in Special Issue
The Signalling Role of the avβ5-Integrin Can Impact the Efficacy of AAV in Retinal Gene Therapy
Article Menu

Export Article

Open AccessReview
Pharmaceuticals 2012, 5(6), 553-590; doi:10.3390/ph5060553

Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future Indications

1
Translational NeuroTherapy Center, Department of Neurological Surgery, University of California San Francisco, 1855 Folsom Street, Mission Center Building, San Francisco, CA 94103, USA
2
Departments of Neurology and Neuroscience, Georgetown University Medical Center, 4000 Reservoir Road, Washington, DC 20007, USA
*
Author to whom correspondence should be addressed.
Received: 17 April 2012 / Revised: 18 May 2012 / Accepted: 23 May 2012 / Published: 4 June 2012
(This article belongs to the Special Issue Gene Therapy)
View Full-Text   |   Download PDF [442 KB, uploaded 4 June 2012]   |  

Abstract

The pharmaceutical industry’s development of therapeutic medications for the treatment of Parkinson’s disease (PD) endures, as a result of the continuing need for better agents, and the increased clinical demand due to the aging population. Each new drug offers advantages and disadvantages to patients when compared to other medical offerings or surgical options. Deep brain stimulation (DBS) has become a standard surgical remedy for the effective treatment of select patients with PD, for whom most drug regimens have failed or become refractory. Similar to DBS as a surgical option, gene therapy for the treatment of PD is evolving as a future option. In the four different PD gene therapy approaches that have reached clinical trials investigators have documented an excellent safety profile associated with the stereotactic delivery, viral vectors and doses utilized, and transgenes expressed. In this article, we review the clinically relevant gene therapy strategies for the treatment of PD, concentrating on the published preclinical and clinical results, and the likely mechanisms involved. Based on these presentations, we advance an analysis of how the nature of the gene therapy used may eventually expand the scope and utility for the management of PD. View Full-Text
Keywords: convection-enhanced delivery; enzyme-replacement therapy; gene therapy; neurotrophic factor; Parkinson’s disease; viral vector convection-enhanced delivery; enzyme-replacement therapy; gene therapy; neurotrophic factor; Parkinson’s disease; viral vector
Figures

This is an open access article distributed under the Creative Commons Attribution License (CC BY 3.0).

Scifeed alert for new publications

Never miss any articles matching your research from any publisher
  • Get alerts for new papers matching your research
  • Find out the new papers from selected authors
  • Updated daily for 49'000+ journals and 6000+ publishers
  • Define your Scifeed now

SciFeed Share & Cite This Article

MDPI and ACS Style

Fiandaca, M.S.; Bankiewicz, K.S.; Federoff, H.J. Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future Indications. Pharmaceuticals 2012, 5, 553-590.

Show more citation formats Show less citations formats

Related Articles

Article Metrics

Article Access Statistics

1

Comments

[Return to top]
Pharmaceuticals EISSN 1424-8247 Published by MDPI AG, Basel, Switzerland RSS E-Mail Table of Contents Alert
Back to Top