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Interdisciplinary Approaches to Gene Therapy pp 17–25Cite as

Gene Therapy with Non-Viral Vector Systems

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Abstract

The concept to treat disease by the introduction of genes into affected human cells has become known as human somatic gene therapy. Several viral and non-viral vector systems are currently used in this context to transfer genes into human cells in vitro and in vivo. The ideal gene therapy vector, in particular for the treatment of inherited genetic disease, should enable the safe transfer of a gene construct to the affected cells and mediate efficient, physiologically regulated and permanent expression of the therapeutic gene. Unfortunately, none of the vector systems currently in use for gene therapy fulfils all these expectations although all of them carry some of the desirable features, but also certain disadvantages. Vims based vectors are designed to use the natural viral mechanisms to transfer genes and express them in target cells and at the same time to eliminate as far as possible negative viral features such as replication, immunogenicity and toxicity. In contrast non-viral vector systems try to use combinations of synthetic and natural molecules to mimic certain advantageous characteristics of the viral system while avoiding their negative features. The main factors influencing permanent and efficient expression of the introduced transgene sequences include the vector preparation, the routes of vector application, cell entry and the intracellular fate of the constructs, their expression and the activity and stability of the expressed protein in the target cells as well as the reactions of the host organism to the vector and transgene product.

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Author information

Authors and Affiliations

  1. Imperial College School of Medicine at St. Mary’s, London, W2 1PG, UK

    C. Coutelle

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  1. C. Coutelle
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Editors and Affiliations

  1. Altmannstraße 23, D-28209, Bremen, Germany

    Stefan Müller

  2. Linzerstraße 3, D-30519, Hannover, Germany

    Jürgen W. Simon

  3. Fundstraße 1C, D-30161, Hannover, Germany

    Jan W. Vesting

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© 1997 Springer-Verlag Berlin • Heidelberg 1997

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Coutelle, C. (1997). Gene Therapy with Non-Viral Vector Systems. In: Müller, S., Simon, J.W., Vesting, J.W. (eds) Interdisciplinary Approaches to Gene Therapy. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-60829-2_2

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  • DOI: https://doi.org/10.1007/978-3-642-60829-2_2

  • Publisher Name: Springer, Berlin, Heidelberg

  • Print ISBN: 978-3-540-63056-2

  • Online ISBN: 978-3-642-60829-2

  • eBook Packages: Springer Book Archive

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