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The therapeutic potential of siRNA in gene therapy of neurodegenerative disorders

  • Conference paper
Neuropsychiatric Disorders An Integrative Approach

Part of the book series: Journal of Neural Transmission. Supplementa ((NEURALTRANS,volume 72))

Abstract

RNA interference using small inhibitory RNA (siRNA) has become a powerful tool to downregulate mRNA levels by cellular nucleases that become activated when a sequence homology between the siRNA and a respective mRNA molecule is detected. Therefore siRNA can be used to silence genes involved in the pathogenesis of various diseases associated with a known genetic background. As for many neurodegenerative disorders a causative therapy is unavailable, siRNA holds a promising option for the development of novel therapeutic strategies. Here we discuss different siRNA target strategies aiming for an allele-specific degradation of disease-inducing mRNA and we review the literature in the field of siRNA and its application in animal models of neurodegenerative diseases, including Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS), Huntingtons disease (HD) and spinocerebellar ataxia (SCA1).

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Authors and Affiliations

  1. Institute of Virology and Immunobiology, University of Würzburg, Versbacher Strasse 7, 97078, Würzburg, Germany

    E. Koutsilieri, A. Rethwilm & PD Dr. C. Scheller

Authors
  1. E. Koutsilieri
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  2. A. Rethwilm
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  3. PD Dr. C. Scheller
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Editor information

Editors and Affiliations

  1. Klinik und Poliklinik für Kinder- und Jugendpsychiatrie und Psychotherapie, Würzburg, Germany

    M. Gerlach

  2. Klinik und Poliklinik für Psychiatrie und Psychotherapie, Würzburg, Germany

    Jürgen Deckert

  3. Prince of Wales Medical Research Institute, Sydney, Australia

    K. Double

  4. Institut für Virologie und Immunobiologie, Würzburg, Germany

    E. Koutsilieri

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© 2007 Springer-Verlag

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Koutsilieri, E., Rethwilm, A., Scheller, C. (2007). The therapeutic potential of siRNA in gene therapy of neurodegenerative disorders. In: Gerlach, M., Deckert, J., Double, K., Koutsilieri, E. (eds) Neuropsychiatric Disorders An Integrative Approach. Journal of Neural Transmission. Supplementa, vol 72. Springer, Vienna. https://doi.org/10.1007/978-3-211-73574-9_7

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  • DOI: https://doi.org/10.1007/978-3-211-73574-9_7

  • Publisher Name: Springer, Vienna

  • Print ISBN: 978-3-211-73573-2

  • Online ISBN: 978-3-211-73574-9

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