Skip to main content

Thank you for visiting 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.

Inserting optimism into gene therapy

A small clinical trial for an immunodeficiency disease provides a new way to think about gene therapy (pages 401–409).

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

Access options

Buy article

Get time limited or full article access on ReadCube.


All prices are NET prices.

Figure 1: Gene-corrected neutrophils in the blood of an individual with CGD after HSPC gene therapy.

Katie Ris


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

    Article  CAS  Google Scholar 

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

    Article  CAS  Google Scholar 

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

    Article  CAS  Google Scholar 

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

    Article  CAS  Google Scholar 

  5. Ott, M. et al. Nat. Med. 12, 401–409 (2006)

    Article  CAS  Google Scholar 

  6. Malech, H.L. et al. Proc. Natl. Acad. Sci. USA 94, 12133–12138 (1997).

    Article  CAS  Google Scholar 

  7. Baum, C., Hegewisch-Becker, S., Eckert, H.G., Stocking, C. & Ostertag, W. J. Virol. 69, 7541–7547 (1995).

    CAS  PubMed  PubMed Central  Google Scholar 

  8. Buonamici, S., Chakraborty, S., Senyuk, V. & Nucifora, G. Blood Cells Mol. Dis. 31, 206–212 (2003).

    Article  CAS  Google Scholar 

  9. Du, Y., Jenkins, N.A. & Copeland, N.G. Blood 106, 3932–3939 (2005).

    Article  CAS  Google Scholar 

  10. Calmels, B. et al. Blood 106, 2530–2533 (2005).

    Article  CAS  Google Scholar 

  11. Kustikova, O. et al. Science 308, 1171–1174 (2005).

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations


Rights and permissions

Reprints and Permissions

About this article

Cite this article

Naldini, L. Inserting optimism into gene therapy. Nat Med 12, 386–388 (2006).

Download citation

  • Issue Date:

  • DOI:

This article is cited by


Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing