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.

  • News & Views
  • Published:

GENE THERAPY

A plethora of gene therapies for hemoglobinopathies

Nature Medicine volume 27, pages 202–204 (2021)Cite this article

Subjects

CRISPR-Cas gene editing enters the field of promising clinical approaches for mitigating hemoglobin defects in patients with hemoglobinopathies.

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

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

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

Fig. 1: Current gene therapies for hemoglobinopathies.

References

  1. Frangoul, H. et al. N. Engl. J. Med. https://doi.org/10.1056/NEJMoa2031054 (2020).

  2. Dunbar, C. E. et al. Science 359, eaan4672 (2018).

    Article  Google Scholar 

  3. Fischer, A. & Hacein-Bey-Abina, S. J. Exp. Med. 217, e20190607 (2020).

    Article  Google Scholar 

  4. Fitzhugh, C. D. et al. Blood 130, 1946–1948 (2017).

    Article  CAS  Google Scholar 

  5. Naldini, L., Trono, D. & Verma, I. M. Science 353, 1101–1102 (2016).

    Article  CAS  Google Scholar 

  6. Thompson, A. A. et al. N. Engl. J. Med. 378, 1479–1493 (2018).

    Article  CAS  Google Scholar 

  7. Marktel, S. et al. Nat. Med. 25, 234–241 (2019).

    Article  CAS  Google Scholar 

  8. Ribeil, J. A. et al. N. Engl. J. Med. 376, 848–855 (2017).

    Article  CAS  Google Scholar 

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

    Article  CAS  Google Scholar 

  10. Bauer, D. E. & Orkin, S. H. Curr. Opin. Genet. Dev. 33, 62–70 (2015).

    Article  CAS  Google Scholar 

  11. Esrick, E.B. et al. N. Engl. J. Med. https://doi.org/10.1056/NEJMoa2029392 (2020).

  12. Ferrari, S. et al. Nat. Biotechnol. 38, 1298–1308 (2020).

    Article  CAS  Google Scholar 

  13. Zeng, J. et al. Nat. Med. 26, 535–541 (2020).

    Article  CAS  Google Scholar 

Download references

Acknowledgements

C.E.D. is funded by the intramural program of the National Heart, Lung, and Blood Institute.

Author information

Authors and Affiliations

  1. Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA

    Cynthia E. Dunbar

Authors
  1. Cynthia E. Dunbar
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Cynthia E. Dunbar.

Ethics declarations

Competing interests

The author declares no competing interests.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Dunbar, C.E. A plethora of gene therapies for hemoglobinopathies. Nat Med 27, 202–204 (2021). https://doi.org/10.1038/s41591-021-01235-7

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41591-021-01235-7

Search

Advanced search

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