Skip to main content

Advertisement

SpringerLink
Log in

Gene therapy

Repair and replace

  • News & Views
  • Published:

From Nature

View current issue Submit your manuscript

One approach to treating inherited diseases is repairing the defective genes, but this has proved challenging in stem cells. An optimized protocol has now been developed that allows gene repair in blood-cell precursors. See Article p.235

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Figure 1: Strategies for treating inherited diseases with gene therapy.

References

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

    Article  ADS  CAS  PubMed  Google Scholar 

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

    Article  ADS  CAS  PubMed  Google Scholar 

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

    Article  ADS  CAS  PubMed  Google Scholar 

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

    Article  ADS  CAS  PubMed  Google Scholar 

  5. Genovese, P. et al. Nature 510, 235–240 (2014).

    Article  ADS  CAS  PubMed  PubMed Central  Google Scholar 

  6. Hyongbum, K. & Jin-Soo, K. Nature Rev. Genet. 15, 321–334 (2014).

    Article  Google Scholar 

  7. Aiuti, A. et al. Science 341, 1233151 (2013).

    Article  PubMed  PubMed Central  Google Scholar 

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

    Article  ADS  CAS  PubMed  Google Scholar 

  9. Biffi, A. et al. Science 341, 1233158 (2013).

    PubMed  Google Scholar 

  10. Cavazzana-Calvo, M. et al. Nature 467, 318–322 (2010).

    Article  ADS  CAS  PubMed  PubMed Central  Google Scholar 

  11. Riddell, J. et al. Cell 157, 549–564 (2014).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Alain Fischer is at the Imagine Institute, Hôpital Necker-Enfants Malades, Paris 75015, France, and at the Collège de France, Paris.,

    Alain Fischer

Authors
  1. Alain Fischer
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Alain Fischer.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Fischer, A. Repair and replace. Nature 510, 226–227 (2014). https://doi.org/10.1038/nature13344

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/nature13344

  • Springer Nature Limited

This article is cited by

Search

Navigation