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Gene therapy progress and prospects: Parkinson's disease

Gene Therapy volume 10, pages 1721–1727 (2003)Cite this article

  • Inhibition of apoptosis by gene delivery prevents development of the disease phenotype in animal models

  • Transgene-mediated expression of glial cell line-derived neurotrophic factor may prevent progression after an initial insult, and may even be restorative in animal models

  • Combination of antiapoptotic and glial cell line-derived neurotrophic factor (GDNF) gene therapy protects dopaminergic neurons against a toxic insult, more effectively than either intervention alone

  • Transgene-mediated production of the inhibitory neurotransmitter γ-amino butyric acid (GABA) in neurons of the subthalamic nucleus ameliorates the behavioral phenotype and may be neuroprotective, in an animal model

  • Delivery of transgenes encoding enzymes involved in dopamine biosynthesis enhances dopamine production in the striatum

  • Stem cells may be driven to differentiate into functioning dopaminergic cells by genetic modification

  • Isolation of genes implicated in rare genetic forms of Parkinson's disease (PD) has allowed generation of new animal models and identification of new candidate targets for intervention

  • One human gene therapy trial is about to commence in PD

  • The optimal vector remains uncertain

Inhibition of apoptosis by gene delivery prevents development of the disease phenotype in animal models

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Author information

Authors and Affiliations

  1. Department of Clinical Neurology, University of Oxford, Radcliffe Infirmary, Oxford, UK

    E A Burton

  2. Department of Molecular Genetics and Biochemistry, University of Pittsburgh, USA

    J C Glorioso

  3. Department of Neurology, University of Pittsburgh, Pittsburgh, PA, USA

    D J Fink

  4. Geriatric Research, Education and Clinical Center (GRECC), Pittsburgh VA Healthcare System, Pittsburgh, PA, USA

    D J Fink

Authors
  1. E A Burton
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  2. J C Glorioso
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  3. D J Fink
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Burton, E., Glorioso, J. & Fink, D. Gene therapy progress and prospects: Parkinson's disease. Gene Ther 10, 1721–1727 (2003). https://doi.org/10.1038/sj.gt.3302116

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