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Gene therapy for Parkinson's disease.

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Author

Summary, in English

The once fantastic theoretical concept that patients with Parkinson's disease (PD) would receive gene therapy in an attempt to alleviate their symptoms and potentially modify the course of their disease has become a reality. On the basis of positive preclinical data, four different gene therapy approaches are currently in Phase I or Phase II clinical trials. Some approaches are intended to increase levels of endogenous dopamine or enhance the function of the prodrug levodopa. Others are intended to normalize basal ganglia circuitry by reducing the PD-related overactivity of specific brain structures such as the subthalamic nucleus. Each is intended for symptomatic benefit. Finally, gene delivery of trophic factors that not only augment dopaminergic function but are potentially disease modifying has a strong preclinical database and are also in clinical trials. Each of these approaches is discussed in the present review.

Publishing year

2010

Language

English

Pages

161-173

Publication/Series

Movement Disorders

Volume

25 Suppl 1

Document type

Journal article

Publisher

John Wiley & Sons Inc.

Topic

  • Neurology

Status

Published

Research group

  • Molecular Neuromodulation
  • Brain Repair and Imaging in Neural Systems (BRAINS)

ISBN/ISSN/Other

  • ISSN: 0885-3185