Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Commentary
  • Published:

20 years of gene therapy for SCID

Nature Immunology volume 11, pages 457–460 (2010)Cite this article

Subjects

Severe combined immunodeficiency conditions are devastating disorders of adaptive immunity. Although these diseases were initially treated by transplantation of allogeneic hematopoietic stem cells, the past 20 years has shown that these conditions are correctable by gene therapy.

This is a preview of subscription content, access via your institution

Relevant articles

Open Access articles citing this article.

Access options

Figure 1: Block in lymphocyte differentiation resulting from a deficiency in γc (SCID-X1) and ADA.
Figure 2: Gene therapy for SCID-X1 and ADA deficiency.

References

  1. Buckley, R.H. Annu. Rev. Immunol. 22, 625–655 (2004).

    Article  CAS  Google Scholar 

  2. Gatti, R.A., Meuwissen, H.J., Allen, H.D., Hong, R. & Good, R.A. Lancet 2, 1366–1369 (1968).

    Article  CAS  Google Scholar 

  3. Friedmann, T. & Roblin, R. Science 175, 949–955 (1972).

    Article  CAS  Google Scholar 

  4. Verma, I.M. & Weitzman, M.D. Annu. Rev. Biochem. 74, 711–738 (2005).

    Article  CAS  Google Scholar 

  5. Gaspar, H.B. et al. Blood 114, 3524–3532 (2009).

    Article  CAS  Google Scholar 

  6. Blaese, R.M. et al. Science 270, 475–480 (1995).

    Article  CAS  Google Scholar 

  7. Bordignon, C. et al. Science 270, 470–475 (1995).

    Article  CAS  Google Scholar 

  8. Kohn, D.B. et al. Nat. Med. 4, 775–780 (1998).

    Article  CAS  Google Scholar 

  9. Bousso, P. et al. Proc. Natl. Acad. Sci. USA 97, 274–278 (2000).

    Article  CAS  Google Scholar 

  10. Cavazzana-Calvo, M. et al. Science 288, 669–672 (2000).

    Article  CAS  Google Scholar 

  11. Aiuti, A. et al. Science 296, 2410–2413 (2002).

    Article  CAS  Google Scholar 

  12. Hacein-Bey-Abina, S. et al. N. Engl. J. Med. 346, 1185–1193 (2002).

    Article  CAS  Google Scholar 

  13. Gaspar, H.B. et al. Lancet 364, 2181–2187 (2004).

    Article  CAS  Google Scholar 

  14. Aiuti, A. et al. N. Engl. J. Med. 360, 447–458 (2009).

    Article  CAS  Google Scholar 

  15. Check, E. Nature 419, 545–546 (2002).

    Article  CAS  Google Scholar 

  16. Check, E. Nature 420, 116–118 (2002).

    Article  CAS  Google Scholar 

  17. Check, E. Nature 420, 595 (2002).

    Article  CAS  Google Scholar 

  18. Check, E. Nature 420, 735 (2002).

    Article  CAS  Google Scholar 

  19. Check, E. Nature 421, 305 (2003).

    Article  CAS  Google Scholar 

  20. Check, E. Nature 421, 678 (2003).

    Article  CAS  Google Scholar 

  21. Check, E. Nature 422, 7 (2003).

    Article  CAS  Google Scholar 

  22. Check, E. Nature 423, 573–574 (2003).

    Article  CAS  Google Scholar 

  23. Check, E. Nature 429, 587 (2004).

    Article  CAS  Google Scholar 

  24. Check, E. Nature 433, 561 (2005).

    PubMed  Google Scholar 

  25. Hacein-Bey-Abina, S. et al. Science 302, 415–419 (2003).

    Article  CAS  Google Scholar 

  26. Howe, S.J. et al. J. Clin. Invest. 118, 3143–3150 (2008).

    Article  CAS  Google Scholar 

  27. Hacein-Bey-Abina, S. et al. J. Clin. Invest. 118, 3132–3142 (2008).

    Article  CAS  Google Scholar 

  28. Lombardo, A. et al. Nat. Biotechnol. 25, 1298–1306 (2007).

    Article  CAS  Google Scholar 

  29. Wang, G.P. et al. Blood published online (12 March 2010).

  30. Cartier, N. et al. Science 326, 818–823 (2009).

    Article  CAS  Google Scholar 

Download references

Acknowledgements

We thank many colleagues who have taken part in this research, notably G. de Saint Basile, J. Di Santo, A. Durandy, C von Kalle and R. Bushman; A. Aiuti, F. Candotti, D.B. Kohn and A. Thrasher for providing unpublished data on their ongoing research; and the many colleagues who have contributed to the field whose important publications could not be cited here because of space constraints.

Author information

Authors and Affiliations

  1. Alain Fischer is with the Institut National de la Santé et de la Recherche Médicale, Paris, France,

    Alain Fischer

  2. Paris-Descartes University, Paris, France

    Alain Fischer, Salima Hacein-Bey-Abina & Marina Cavazzana-Calvo

  3. Unité d'Immunologie et Hématologie Pédiatrique, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants Malades, Paris, France

    Alain Fischer

  4. Salima Hacein-Bey-Abina and Marina Cavazzana-Calvo are with the Institut National de la Santé et de la Recherche Médicale, Paris, France,

    Salima Hacein-Bey-Abina & Marina Cavazzana-Calvo

  5. Department of Biotherapy, Centre d'Investigation Clinique intégré en Biothérapies, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France

    Salima Hacein-Bey-Abina & Marina Cavazzana-Calvo

Authors
  1. Alain Fischer
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Salima Hacein-Bey-Abina
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Marina Cavazzana-Calvo
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Alain Fischer.

Ethics declarations

Competing interests

The authors declare no competing financial interests.

Rights and permissions

Reprints and permissions

About this article

Cite this article

Fischer, A., Hacein-Bey-Abina, S. & Cavazzana-Calvo, M. 20 years of gene therapy for SCID. Nat Immunol 11, 457–460 (2010). https://doi.org/10.1038/ni0610-457

Download citation

  • Issue Date:

  • DOI: https://doi.org/10.1038/ni0610-457

This article is cited by

Search

Advanced search

Quick links

Nature Briefing: Translational Research

Sign up for the Nature Briefing: Translational Research newsletter — top stories in biotechnology, drug discovery and pharma.

Get what matters in translational research, free to your inbox weekly. Sign up for Nature Briefing: Translational Research