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Prenatal Gene Therapy pp 183–200Cite as

Animal Models for Prenatal Gene Therapy: Choosing the Right Model

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

Abstract

Testing in animal models is an essential requirement during development of prenatal gene therapy for ­clinical application. Some information can be derived from cell lines or cultured fetal cells, such as the efficiency of gene transfer and the vector dose that might be required. Fetal tissues can also be maintained in culture for short periods of time and transduced ex vivo. Ultimately, however, the use of animals is unavoidable since in vivo experiments allow the length and level of transgene expression to be measured, and provide an assessment of the effect of the delivery procedure and the gene therapy on fetal and neonatal development. The choice of animal model is determined by the nature of the disease and characteristics of the animal, such as its size, lifespan, and immunology, the number of fetuses and their development, parturition, and the length of gestation and the placentation. The availability of a disease model is also critical. In this chapter, we discuss the various animal models that can be used and consider how their characteristics can affect the results obtained. The projection to human application and the regulatory hurdles are also presented.

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Authors and Affiliations

  1. Prenatal Cell and Gene Therapy Group, EGA Institute for Women’s Health, University College London, London, UK

    Vedanta Mehta, Donald Peebles MD & Anna L. David PhD, MRCOG

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  1. Vedanta Mehta
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  2. Donald Peebles MD
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  3. Anna L. David PhD, MRCOG
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Corresponding author

Correspondence to Anna L. David PhD, MRCOG .

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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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Mehta, V., Peebles, D., David, A.L. (2012). Animal Models for Prenatal Gene Therapy: Choosing the Right Model. 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_9

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  • DOI: https://doi.org/10.1007/978-1-61779-873-3_9

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