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

Gene Therapy volume 13, pages 517–524 (2006)Cite this article

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.

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Acknowledgements

This work was supported by a Fast-track grant from the Michael J Fox Foundation to RJM and ASL.

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Authors and Affiliations

  1. Department of Neuroscience, McKnight Brain Institute, University of Florida College of Medicine, Gainesville, FL, USA

    F P Manfredsson & R J Mandel

  2. The Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA

    F P Manfredsson, A S Lewin & R J Mandel

  3. Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL, USA

    A S Lewin

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Correspondence to R J Mandel.

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Manfredsson, F., Lewin, A. & Mandel, R. RNA knockdown as a potential therapeutic strategy in Parkinson's disease. Gene Ther 13, 517–524 (2006). https://doi.org/10.1038/sj.gt.3302669

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