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Gene Ther. 2006 Mar;13(6):517-24.

RNA knockdown as a potential therapeutic strategy in Parkinson's disease.

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Department of Neuroscience, McKnight Brain Institute, University of Florida College of Medicine, Gainesville, FL 32610-0024, USA.


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.

[Indexed for MEDLINE]

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