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.


Towards safe therapy for immunodeficiency

A humanized mouse model of X-linked severe combined immunodeficiency suggests that the safety of correcting the human IL2RG gene in haematopoietic stem cells depends on both gene-correction frequency and the conditioning regimen used before cell transplantation.

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

Relevant articles

Open Access articles citing this article.

Access options

Rent or buy this article

Get just this article for as long as you need it


Prices may be subject to local taxes which are calculated during checkout

Fig. 1


  1. Urnov, F. D., Rebar, M. C., Holmes, M. C., Zhang, S. & Gregory, P. D. Nat. Rev. Genet.11, 636–646 (2010).

    Article  CAS  Google Scholar 

  2. Joung, J. K. & Sander, J. D. Nat. Rev. Mol. Cell Biol.14, 49–55 (2012).

    Article  Google Scholar 

  3. Sander, J. D. & Joung, J. K. Nat. Biotechnol.32, 347–355 (2014).

    Article  CAS  Google Scholar 

  4. Schiroli, J. et al. Sci. Transl. Med.9, eaan0820 (2017).

    Article  Google Scholar 

  5. Tsai, S. Q. et al. Nat. Biotechnol.33, 187–197 (2015).

    Article  CAS  Google Scholar 

  6. Martins, V. C. et al. Nature509, 465–470 (2014).

    Article  CAS  Google Scholar 

  7. Cavazzana, M., Six, E., Lagresle-Peyrou, C., André-Schmutz, S. & Hacein-Bey-Abina, S. Hum. Gene Ther.27, 108–116 (2016).

    Article  CAS  Google Scholar 

  8. Hacein-Bey-Abina, S. et al. Science302, 415–419 (2003).

    Article  CAS  Google Scholar 

  9. Aiuti, A. N. Engl. J. Med.360, 447–458 (2009).

    Article  CAS  Google Scholar 

  10. Aiuti, A. Science296, 2410–2413 (2002).

    Article  CAS  Google Scholar 

  11. Shaw, K. L. & Kohn, D. B. Sci. Transl. Med.3, 97ps36 (2011).

    Article  Google Scholar 

  12. De Ravin, S. S. et al. Sci. Transl. Med.8, 335ra57 (2016).

    Article  Google Scholar 

Download references

Author information

Authors and Affiliations


Corresponding author

Correspondence to Shengdar Q. Tsai.

Rights and permissions

Reprints and Permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Tsai, S.Q. Towards safe therapy for immunodeficiency. Nat Biomed Eng 1, 937–938 (2017).

Download citation

  • Published:

  • Issue Date:

  • DOI:

This article is cited by


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