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A plethora of gene therapies for hemoglobinopathies

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

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Fig. 1: Current gene therapies for hemoglobinopathies.


  1. 1.

    Frangoul, H. et al. N. Engl. J. Med. (2020).

  2. 2.

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

    Article  Google Scholar 

  3. 3.

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

    Article  Google Scholar 

  4. 4.

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

    CAS  Article  Google Scholar 

  5. 5.

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

    CAS  Article  Google Scholar 

  6. 6.

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

    CAS  Article  Google Scholar 

  7. 7.

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

    CAS  Article  Google Scholar 

  8. 8.

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

    CAS  Article  Google Scholar 

  9. 9.

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

    CAS  Article  Google Scholar 

  10. 10.

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

    CAS  Article  Google Scholar 

  11. 11.

    Esrick, E.B. et al. N. Engl. J. Med. (2020).

  12. 12.

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

    CAS  Article  Google Scholar 

  13. 13.

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

    CAS  Article  Google Scholar 

Download references


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

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Correspondence to Cynthia E. Dunbar.

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The author declares no competing interests.

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Dunbar, C.E. A plethora of gene therapies for hemoglobinopathies. Nat Med 27, 202–204 (2021).

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