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Prenatal Gene Therapy pp 273–290Cite as

Choice of Surrogate and Physiological Markers for Prenatal Gene Therapy

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

Abstract

Surrogate genetically encoded markers have been utilized in order to analyze gene transfer efficacy, location, and persistence. These marker genes have greatly accelerated the development of gene transfer vectors for the ultimate application of gene therapy using therapeutic genes. They have also been used in many other applications, such as gene marking in order to study developmental cell lineages, to track cell migration, and to study tumor growth and metastasis. This chapter aims to describe the analysis of several commonly used marker genes: green fluorescent protein (GFP), β-galactosidase, firefly luciferase, human factor IX, and alkaline phosphatase. The merits and disadvantages of each are briefly discussed. In addition a few short examples are provided for continual and endpoint analysis in different disease models including hemophilia, cystic fibrosis, ornithine transcarbamylase deficiency and Gaucher disease.

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Author information

Authors and Affiliations

  1. Gene Transfer Technology Group, Institute for Women’s Health, University College London, London, UK

    Juliette M. K. M. Delhove, Ahad A. Rahim PhD & Suzanne M. K. Buckley PhD

  2. Department of Endocrinology, William Harvey Research Institute, Charterhouse Square, London, EC1M 6BQ, UK

    Tristan R. McKay

  3. Institute for Women’s Health, Gene Transfer Technology Group, University College London, London, UK

    Simon N. Waddington PhD

Authors
  1. Juliette M. K. M. Delhove
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  2. Ahad A. Rahim PhD
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  3. Tristan R. McKay
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  4. Simon N. Waddington PhD
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  5. Suzanne M. K. Buckley PhD
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Corresponding author

Correspondence to Simon N. Waddington 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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Delhove, J.M.K.M., Rahim, A.A., McKay, T.R., Waddington, S.N., Buckley, S.M.K. (2012). Choice of Surrogate and Physiological Markers for Prenatal Gene Therapy. 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_13

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  • DOI: https://doi.org/10.1007/978-1-61779-873-3_13

  • Published:

  • Publisher Name: Humana Press, Totowa, NJ

  • Print ISBN: 978-1-61779-872-6

  • Online ISBN: 978-1-61779-873-3

  • eBook Packages: Springer Protocols

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