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Adeno-Associated Viral Vectors for Gene Therapy of Inherited Retinal Degenerations

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Retinal Degeneration

Part of the book series: Methods in Molecular Biology ((MIMB,volume 935))

Abstract

Adeno-associated virus (AAV) vectors are in wide use for in vivo gene transfer for the treatment of inherited retinal disease. AAV vectors have been tested in many animal models and have demonstrated efficacy with low toxicity. In this chapter we describe some of the recent methods for small-scale production of these vectors for use in a laboratory setting in volumes and purity appropriate for testing in small and large animals.

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Author information

Authors and Affiliations

  1. Neuroscience Division, Department of Molecular and Cell Biology, Helen Wills Neuroscience Institute, University of California, Berkeley, CA, USA

    John G. Flannery & Meike Visel

Authors
  1. John G. Flannery
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  2. Meike Visel
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Corresponding author

Correspondence to John G. Flannery .

Editor information

Editors and Affiliations

  1. , Institute of Human Genetics, University of Regensburg, Franz-Josef-Strauss-Allee 11, Regensburg, 93053, Germany

    Bernhard H.F. Weber

  2. INSTITUTE OF HUMAN GENETICS, INSTITUTE OF HUMAN GENETICS, FRANZ-JOSEF-STRAUSS-ALLEE 11, 93053 REGENSBURG, Germany

    THOMAS LANGMANN

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Flannery, J.G., Visel, M. (2012). Adeno-Associated Viral Vectors for Gene Therapy of Inherited Retinal Degenerations. In: Weber, B., LANGMANN, T. (eds) Retinal Degeneration. Methods in Molecular Biology, vol 935. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-62703-080-9_25

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  • DOI: https://doi.org/10.1007/978-1-62703-080-9_25

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  • Publisher Name: Humana Press, Totowa, NJ

  • Print ISBN: 978-1-62703-079-3

  • Online ISBN: 978-1-62703-080-9

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