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Correction of Genetic Blood Defects by Gene Transfer

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Principles of Molecular Medicine

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Abstract

Gene therapy has been proposed as an appealing tool for introducing a normal gene into affected hematopoietic stem cells to correct their inherited defect. Theoretically, in the absence of a related human leukocyte antigen identical donor, gene therapy could be an alternative given the accessibility and the information available on the hematopoietic stem cell biology. This chapter describes the progress and limits of the gene therapy approach applied to some genetic blood defects that appear to be good targets for this strategy.

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Author information

Authors and Affiliations

  1. Department of Biotherapy AP-HP, Hôpital Necker, Paris, France

    Marina Cavazzana-Calvo & Salima Hacein-Bey-Abina

  2. Molecular Immunology Unit, Institute of Child Health, London, UK

    Adrian J. Thrasher

  3. Harvard Medical School and Genetics Division, Brigham and Women’s Hospital, Boston, MA

    Philippe Leboulch

  4. Unité d’Immunologie et d’Hématologie Pédiatriques, Hôpital Necker, Paris, France

    Alain Fischer

Authors
  1. Marina Cavazzana-Calvo
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  2. Salima Hacein-Bey-Abina
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  3. Adrian J. Thrasher
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  4. Philippe Leboulch
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  5. Alain Fischer
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Editor information

Editors and Affiliations

  1. Department of Medicine, Division of Cardiology Carolina Cardiovascular Biology Center, University of North Carolina School of Medicine, Chapel Hill, NC

    Marschall S. Runge MD, PhD  & Cam Patterson MD  & 

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© 2006 Humana Press Inc.

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Cavazzana-Calvo, M., Hacein-Bey-Abina, S., Thrasher, A.J., Leboulch, P., Fischer, A. (2006). Correction of Genetic Blood Defects by Gene Transfer. In: Runge, M.S., Patterson, C. (eds) Principles of Molecular Medicine. Humana Press. https://doi.org/10.1007/978-1-59259-963-9_88

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  • DOI: https://doi.org/10.1007/978-1-59259-963-9_88

  • Publisher Name: Humana Press

  • Print ISBN: 978-1-58829-202-5

  • Online ISBN: 978-1-59259-963-9

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