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Principles of gene transfer and foreign protein expression for human gene therapy

  • Chapter
Recombinant Protein Drugs

Part of the book series: Milestones in Drug Therapy ((MDT))

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Abstract

The conceptual and technical bases for human gene therapy are maturing rapidly. At its inception in the early 1970s, gene therapy presumed the eventual development of efficient and targeted vectors to deliver therapeutic foreign genetic elements into defective cells for the purpose of complementing underlying genetic defects responsible for human genetic disease. The initial models for this approach were the single-gene Mendelian defects associated with classical “inborn errors of metabolism”. It was a relatively simple task to demonstrate early that normal alleles could be introduced into defective cells, that newly expressed gene products could complement genetic defects and that aspects of complex disease phenotypes could be reversed inin vitroand animal model systems.

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Author information

Authors and Affiliations

  1. Center for Molecular Genetics, UCSD School of Medicine and Program in Human Gene Therapy, La Jolla, CA, 92093, USA

    Theodore Friedmann

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  1. Theodore Friedmann
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  1. Xantos Biomedicine, Fraunhoferstr. 22, Martinsried, D-82152, Germany

    Peter Buckel

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© 2001 Springer Basel AG

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Friedmann, T. (2001). Principles of gene transfer and foreign protein expression for human gene therapy. In: Buckel, P. (eds) Recombinant Protein Drugs. Milestones in Drug Therapy. Birkhäuser, Basel. https://doi.org/10.1007/978-3-0348-8346-7_8

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  • DOI: https://doi.org/10.1007/978-3-0348-8346-7_8

  • Publisher Name: Birkhäuser, Basel

  • Print ISBN: 978-3-0348-9527-9

  • Online ISBN: 978-3-0348-8346-7

  • eBook Packages: Springer Book Archive

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