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Gene Therapy for Parkinson’s Disease

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Viral Vector Approaches in Neurobiology and Brain Diseases

Part of the book series: Neuromethods ((NM,volume 82))

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Abstract

Gene therapy is a promising future tool for treatment of Parkinson’s disease (PD) and several different strategies are currently being evaluated. Although many of these strategies have shown promising results in animal models of PD and parkinsonian patients, some have been less effective and caused adverse side effects. A vector system with high specificity and appropriate expression level of the transgene is needed to make gene therapy for PD as safe and beneficial as possible. The vector should also be relevant for the disease. Here, we present a method to design promoters relevant for PD, using microarray data from patients, and validation of these in vivo. The method also includes fine-tuning of promoter candidates by adding miRNA target sites to increase cell specificity.

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Acknowledgments

The authors are indebted to Christina Isaksson, Ulla Jarl, Anneli Josephsson, and Michael Sparrenius for their technical expertise. The work was supported by the Swedish Research Council (Grants # 2010-4496 and 2007-8626).

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Authors and Affiliations

  1. Department of Experimental Medical Science, CNS Gene Therapy Unit, Wallenberg Neuroscience Center, Lund University, Lund, Sweden

    Erika Elgstrand Wettergren, Luis Quintino, Giuseppe Manfré & Cecilia Lundberg

Authors
  1. Erika Elgstrand Wettergren
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  2. Luis Quintino
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  3. Giuseppe Manfré
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  4. Cecilia Lundberg
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Editor information

Editors and Affiliations

  1. Institute of Experimental Neurology, San Raffaele Scientific Institute Division of Neuroscience, Milan, Italy

    Riccardo Brambilla

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Wettergren, E.E., Quintino, L., Manfré, G., Lundberg, C. (2014). Gene Therapy for Parkinson’s Disease. In: Brambilla, R. (eds) Viral Vector Approaches in Neurobiology and Brain Diseases. Neuromethods, vol 82. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-62703-610-8_10

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  • DOI: https://doi.org/10.1007/978-1-62703-610-8_10

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  • Publisher Name: Humana Press, Totowa, NJ

  • Print ISBN: 978-1-62703-609-2

  • Online ISBN: 978-1-62703-610-8

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