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Adeno-Associated Virus Vectors pp 209–233Cite as

Subpial AAV Delivery for Spinal Parenchymal Gene Regulation in Adult Mammals

Part of the Methods in Molecular Biology book series (MIMB,volume 1950)

Abstract

The use of adeno-associated virus (AAV) vectors has become an attractive method for treatment of a variety of neurodegenerative disorders by permitting targeted gene upregulation or silencing in the CNS. Systemic and intrathecal infusion, while preferable routes of vector delivery, have shown encouraging but variable efficacy due to the poor permeability of AAV into spinal cord and brain parenchyma in adult mammals. Recently we have developed a novel and relatively noninvasive technique of spinal subpial vector delivery. This technique confers widespread transgene expression throughout the spinal parenchyma, including both white and gray matter. We have demonstrated that this technique can be performed safely, with a high level of accuracy, and is effective in both small (mouse or rat) and large preclinical (adult pig or nonhuman primate) animal models. In this chapter we provide a comprehensive description of the subpial vector delivery technique in adult rodents (mouse and rat) and large preclinical animals (adult pig and nonhuman primates).

Key words

  • Subpial delivery
  • Gene therapy
  • Mice
  • Rats
  • Pigs
  • Nonhuman primates
  • Adeno-associated virus
  • Spinal cord

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  • DOI: 10.1007/978-1-4939-9139-6_12
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Authors and Affiliations

  1. Neuroregeneration Laboratory, Department of Anesthesiology, University of California, San Diego, La Jolla, CA, USA

    Mariana Bravo-Hernández, Takahiro Tadokoro & Martin Marsala

Authors
  1. Mariana Bravo-Hernández
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  2. Takahiro Tadokoro
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  3. Martin Marsala
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Corresponding author

Correspondence to Martin Marsala .

Editor information

Editors and Affiliations

  1. Department of Neurosciences, University of California, San Diego, La Jolla, CA, USA

    Ph.D. Michael J. Castle

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Bravo-Hernández, M., Tadokoro, T., Marsala, M. (2019). Subpial AAV Delivery for Spinal Parenchymal Gene Regulation in Adult Mammals. In: Castle, M. (eds) Adeno-Associated Virus Vectors. Methods in Molecular Biology, vol 1950. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-9139-6_12

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  • DOI: https://doi.org/10.1007/978-1-4939-9139-6_12

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  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-9138-9

  • Online ISBN: 978-1-4939-9139-6

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