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Novel disease-specific promoters for use in gene therapy for Parkinson's disease.

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Author

Summary, in English

Gene therapy is a promising therapeutic tool for Parkinson's disease (PD), but there is a lack of evaluated cell specific promoters that are relevant for the disease. We have chosen PD relevant promoter candidates for gene therapy vectors based on either previous studies; Drd1a, Drd2 and pDyn, or from a microarray study on parkinsonian patients; ACE, DNAJC3, GALNS, MAP1a and RNF25. These candidates have been evaluated in rat striatum to determine their suitability for use in cell specific vectors. The promoters had a neuronal specificity of 91-100%. The efficiency of the promoters was variable, but RNF25, DNAJC3 and MAP1a were comparable to widely used ubiquitous promoters. MAP1a was also affected by dopamine depletion.

Publishing year

2012

Language

English

Pages

29-34

Publication/Series

Neuroscience Letters

Volume

530

Issue

1

Document type

Journal article

Publisher

Elsevier

Topic

  • Neurosciences

Keywords

  • Promoter
  • Parkinson's disease
  • Gene therapy
  • Neuron-specific

Status

Published

Research group

  • CNS Gene Therapy

ISBN/ISSN/Other

  • ISSN: 0304-3940