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X-SCID transgene leukaemogenicity

Author

Listed:
  • Adrian J. Thrasher

    (Molecular Immunology Unit, Institute of Child Health, University College London
    Great Ormond Street Hospital for Children NHS Trust)

  • H. Bobby Gaspar

    (Molecular Immunology Unit, Institute of Child Health, University College London
    Great Ormond Street Hospital for Children NHS Trust)

  • Christopher Baum

    (Hannover Medical School
    Cincinnati Children's Hospital Medical Center)

  • Ute Modlich

    (Hannover Medical School)

  • Axel Schambach

    (Hannover Medical School)

  • Fabio Candotti

    (Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health)

  • Makoto Otsu

    (Center for Experimental Medicine, University of Tokyo)

  • Brian Sorrentino

    (St Jude Children's Research Hospital)

  • Linda Scobie

    (Institute of Comparative Medicine, Faculty of Veterinary Medicine, University of Glasgow)

  • Ewan Cameron

    (Institute of Comparative Medicine, Faculty of Veterinary Medicine, University of Glasgow)

  • Karen Blyth

    (Institute of Comparative Medicine, Faculty of Veterinary Medicine, University of Glasgow)

  • Jim Neil

    (Institute of Comparative Medicine, Faculty of Veterinary Medicine, University of Glasgow)

  • Salima Hacein-Bey Abina

    (INSERM Unit 768, Hôpital Necker, Assistance Publique–Hôpitaux de Paris
    Assistance Publique–Hôpitaux de Paris)

  • Marina Cavazzana-Calvo

    (INSERM Unit 768, Hôpital Necker, Assistance Publique–Hôpitaux de Paris
    Assistance Publique–Hôpitaux de Paris
    Faculté de Médecine, Université René Descartes)

  • Alain Fischer

    (INSERM Unit 768, Hôpital Necker, Assistance Publique–Hôpitaux de Paris
    Faculté de Médecine, Université René Descartes
    Unité d'Immuno-Hématologie Pédiatrique, Assistance Publique–Hôpitaux de Paris)

Abstract

Arising from: Woods, N.-B., Bottero, V., Schmidt, M., von Kalle, C. & Verma, I. M. Nature 440, 1123 (2006); see also communication from Pike-Overzet et al. ; Woods et al. reply Gene therapy has been remarkably effective for the immunological reconstitution of patients with severe combined immune deficiency1,2,3, but the occurrence of leukaemia in a few patients has stimulated debate about the safety of the procedure and the mechanisms of leukaemogenesis4. Woods et al.5 forced high expression of the corrective therapeutic gene IL2RG, which encodes the γ-chain of the interleukin-2 receptor, in a mouse model of the disease and found that tumours appeared in a proportion of cases. Here we show that transgenic IL2RG does not necessarily have potent intrinsic oncogenic properties, and argue that the interpretation of this observation with respect to human trials is overstated.

Suggested Citation

  • Adrian J. Thrasher & H. Bobby Gaspar & Christopher Baum & Ute Modlich & Axel Schambach & Fabio Candotti & Makoto Otsu & Brian Sorrentino & Linda Scobie & Ewan Cameron & Karen Blyth & Jim Neil & Salima, 2006. "X-SCID transgene leukaemogenicity," Nature, Nature, vol. 443(7109), pages 5-6, September.
    • Handle: RePEc:nat:nature:v:443:y:2006:i:7109:d:10.1038_nature05219
    DOI: 10.1038/nature05219
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