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J Neurol. 2011 Feb;258(2):179-88. doi: 10.1007/s00415-010-5796-9. Epub 2010 Oct 21.

Gene therapy: a viable therapeutic strategy for Parkinson's disease?

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  • 1Department of Molecular Neuroscience, UCL Institute of Neurology, Queen Square, London, WC1N 3BG, UK.


Gene therapy represents a potentially useful additional technique to ameliorate the motor symptoms of Parkinson's disease (PD), and the motor complications of its treatment. The neurodegenerative process itself, as well as the non-motor symptoms of PD, both remain less amenable to most of the current gene therapy approaches. This review presents an overview of the four gene therapies in phase I/II clinical trials, outlines some of the challenges they face, and proposes additional alternative strategies that might improve the clinical prospects of gene therapy for PD. In so doing, we hope to highlight the issue of the current absence of effective treatment for non-motor symptoms of PD and the potential of further candidate targets for gene therapy intervention that might improve upon this, for both specific individuals with genetic forms of PD as well as "sporadic" PD patients.

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