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Prenatal Gene Therapy pp 55–84Cite as

Vector Systems for Prenatal Gene Therapy: Principles of Adenovirus Design and Production

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Part of the book series: Methods in Molecular Biology ((MIMB,volume 891))

Abstract

Adenoviruses have many attributes, which have made them one of the most widely investigated vectors for gene therapy applications. These include ease of genetic manipulation to produce replication-deficient vectors, ability to readily generate high titer stocks, efficiency of gene delivery into many cell types, and ability to encode large genetic inserts. Recent advances in adenoviral vector engineering have included the ability to genetically manipulate the tropism of the vector by engineering of the major capsid proteins, particularly fiber and hexon. Furthermore, simple replication-deficient adenoviral vectors deleted for expression of a single gene have been complemented by the development of systems in which the majority of adenoviral genes are deleted, generating sophisticated Ad vectors which can mediate sustained transgene expression following a single delivery. This chapter outlines methods for developing simple transgene over expressing Ad vectors and detailed strategies to engineer mutations into the major capsid proteins.

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Author information

Authors and Affiliations

  1. Institute of Cardiovascular and Medical Sciences, University of Glasgow, Glasgow, UK

    Raul Alba, Andrew H. Baker PhD & Stuart A. Nicklin BSc (Hons) PhD

Authors
  1. Raul Alba
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  2. Andrew H. Baker PhD
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  3. Stuart A. Nicklin BSc (Hons) PhD
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Corresponding author

Correspondence to Stuart A. Nicklin BSc (Hons) PhD .

Editor information

Editors and Affiliations

  1. National Heart & Lung Inst., Molecular & Cellular Medicine Sec., Imperial College London, Imperial College Road, London, SW7 2AZ, United Kingdom

    Charles Coutelle

  2. Reader in Gene Transfer Technology, Gene Transfer Technology Group, University College London, Chenies Mews 86-96, London, WC1E 6HX, United Kingdom

    Simon N. Waddington

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© 2012 Springer Science+Business Media, LLC.

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Alba, R., Baker, A.H., Nicklin, S.A. (2012). Vector Systems for Prenatal Gene Therapy: Principles of Adenovirus Design and Production. In: Coutelle, C., Waddington, S. (eds) Prenatal Gene Therapy. Methods in Molecular Biology, vol 891. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-61779-873-3_4

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  • DOI: https://doi.org/10.1007/978-1-61779-873-3_4

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  • Publisher Name: Humana Press, Totowa, NJ

  • Print ISBN: 978-1-61779-872-6

  • Online ISBN: 978-1-61779-873-3

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