1. ¹Department of Neuroscience, McKnight Brain Institute, University of Florida College of Medicine, Gainesville, FL, USA
2. ²The Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA
3. ³Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL, USA

Correspondence: Dr RJ Mandel, Department of Neuroscience, McKnight Brain Institute, University of Florida College of Medicine, PO Box 100244, Gainesville, FL 32610-0024, USA. E-mail: rmandel@mbi.ufl.edu

Received 21 June 2005; Revised 16 September 2005; Accepted 19 September 2005; Published online 3 November 2005.

Abstract

Parkinson's disease is a prevalent progressive degenerative disorder of the elderly. There is a current need for novel therapeutic strategies because the standard levodopa pharmacotherapy is only temporarily efficacious. Recently, there have been some high-profile successful preclinical results obtained in animal models of neurological disorders using small interfering RNAs delivered by viral vectors. RNA interference can theoretically be applied to Parkinson's disease since over-expression of various proteins is known to kill the dopamine neurons of the substantia nigra in animal models and in familial forms of Parkinson's disease. Potential RNA interfering strategies and caveats are discussed in this review.