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Hemoglobin
international journal for hemoglobin research
Volume 43, 2019 - Issue 6: Global Globin 2020 Conference, UNESCO Paris 2019
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Keynote

Gene Therapy of the β Hemoglobinopathies: Success and Challenges

Emmanuel PayenINSERM and CEA, François Jacob Biology Institute, Paris Saclay University, Paris, France; ;CEA, François Jacob Biology Institute, University of Paris, Paris, France; Correspondence[email protected]
&
Philippe LeboulchINSERM and CEA, François Jacob Biology Institute, Paris Saclay University, Paris, France; ;CEA, François Jacob Biology Institute, University of Paris, Paris, France; ;Harvard Medical School and Brigham & Women’s Hospital, Boston, MA, USA
Page 301 | Published online: 28 Feb 2020

References

  • Thompson AA, Walters MC, Kwiatkowski J, et al. Gene therapy in patients with transfusion-dependent β-thalassemia. N Engl J Med. 2018;378(16):1479–1493.
  • Ribeil J-A, Hacein-Bey-Abina S, Payen E, et al. Gene therapy in a patient with sickle cell disease. N Engl J Med. 2017;376:848–855.

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