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Human Gene Therapy with Retrovirus and Lentivirus Vectors

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Lentiviral Vectors and Gene Therapy

Part of the book series: SpringerBriefs in Biochemistry and Molecular Biology ((BRIEFSBIOCHEM))

Abstract

The first human gene therapy clinical trial unsuccessfully took place in the 1970s. Despite extensive research and development in this subject, it was only approximately 10 years ago in 2000, that the results from a completely successful human gene therapy trial were published. Severe combined immunodeficiency X1 was corrected by ex vivo transduction of autologous hematopoietic stem cells with a γ-retrovirus vector encoding the therapeutic gene, followed by retransplantation. Since then, several other clinical trials using retro and lentivectors have followed. In this chapter we will briefly describe and discuss these successful trials for the correction of genetic diseases.

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Acknowledgments

David Escors is funded by an Arthritis Research UK Career Development Fellowship (18433). Holly Stephenson is funded by the Biomedical Research Centre, Institute of Child Health, UCL. Karine Breckpot is funded by the Fund for Scientific Research-Flandes. The Oxford Structural Genomics Consortium is a registered UK charity (number 1097737) that receives funds from the Canadian Institutes of Health Research, The Canadian Foundation for Innovation, Genome Canada through the Ontario Genomics Institute, GlaxoSmithKline, Karolinska Institutet, the Knut and Alice Wallenberg Foundations, the Ontario Innovation Trust, the Ontario Ministry for Research and Innovation, Merck & Co., Inc., the Novartis Research Foundation, the Swedish Foundation for Strategic Research and the Wellcome Trust.

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Authors and Affiliations

  1. Oxford Structural Genomics Consortium, University of Oxford, Old Road Campus Research Building, Roosevelt Drive, Headington, Oxford, OX3 7DQ, UK

    Grazyna Kochan

  2. Institute of Child Health, University College London, Great Ormond Street, London, WC1N 3JH, UK

    Holly Stephenson

  3. Vrije Universiteit Brussels, Brussels, Belgium

    Karine Breckpot

  4. University College London, Rayne Building, 5 University Street, London, WC1E 6JF, UK

    David Escors

Authors
  1. Grazyna Kochan
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  2. Holly Stephenson
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  3. Karine Breckpot
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  4. David Escors
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Correspondence to David Escors .

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Kochan, G., Stephenson, H., Breckpot, K., Escors, D. (2012). Human Gene Therapy with Retrovirus and Lentivirus Vectors. In: Lentiviral Vectors and Gene Therapy. SpringerBriefs in Biochemistry and Molecular Biology. Springer, Basel. https://doi.org/10.1007/978-3-0348-0402-8_6

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