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Home All issues Volume 38 / No 10 (Octobre 2022) Med Sci (Paris), 38 10 (2022) 768-771 References
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Issue
Med Sci (Paris)
Volume 38, Number 10, Octobre 2022
Page(s) 768 - 771
Section Nouvelles
DOI https://doi.org/10.1051/medsci/2022120
Published online 11 October 2022
  1. Candotti F. Clinical manifestations and pathophysiological mechanisms of the Wiskott-Aldrich syndrome. J Clin Immunol 2018 ; 38 : 13–27. [CrossRef] [PubMed] [Google Scholar]
  2. Burroughs LM, Petrovic A, Brazauskas R, et al. Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. Blood 2020; 135 : 2094–105. [CrossRef] [PubMed] [Google Scholar]
  3. Albert MH, Slatter MA, Gennery AR, et al. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT inborn errors working party analysis. Blood 2022; blood.2021014687. [Google Scholar]
  4. Horak P, Uhrig S, Witzel M, et al. Comprehensive genomic characterization of gene therapy-induced T-cell acute lymphoblastic leukemia. Leukemia 2020; 34 : 2785–9. [CrossRef] [PubMed] [Google Scholar]
  5. Ferrua F, Marangoni F, Aiuti A, et al. Gene therapy for Wiskott-Aldrich syndrome: History, new vectors, future directions. J Allergy Clin Immunol 2020; 146 : 262–5. [CrossRef] [PubMed] [Google Scholar]
  6. Magnani A, Semeraro M, Adam F, et al. Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome. Nat Med 2022; 28 : 71–80. [CrossRef] [PubMed] [Google Scholar]
  7. Mahlaoui N, Pellier I, Mignot C, et al. Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome. Blood 2013 ; 121 : 1510–1516. [CrossRef] [PubMed] [Google Scholar]
  8. Cavazzana-Calvo M, Payen E, Negre O, et al. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature 2010 ; 467 : 318–322. [CrossRef] [PubMed] [Google Scholar]
  9. Negre O, Eggimann A-V, Beuzard Y, et al. Gene therapy of the β-hemoglobinopathies by lentiviral transfer of the β(A(T87Q))-globin gene. Hum Gene Ther 2016 ; 27 : 148–165. [CrossRef] [PubMed] [Google Scholar]
  10. Thompson AA, Walters MC, Kwiatkowski J, et al. Gene therapy in patients with transfusion-dependent β-thalassemia. N Engl J Med 2018 ; 378 : 1479–1493. [CrossRef] [PubMed] [Google Scholar]
  11. Magrin E, Semeraro M, Hebert N, et al. Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial. Nat Med 2022; 28 : 81–8. [CrossRef] [PubMed] [Google Scholar]
  12. Panepinto JA, Kato GJ, Smith WR. Health-related quality of life in sickle cell disease. Nat Rev Dis Primers 2019 ; 5 : 27. [CrossRef] [PubMed] [Google Scholar]
  13. 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. [CrossRef] [PubMed] [Google Scholar]
  14. Abboud M, Laver J, Blau CA. Granulocytosis causing sickle-cell crisis. Lancet 1998 ; 351 : 959. [CrossRef] [PubMed] [Google Scholar]
  15. Adler BK, Salzman DE, Carabasi MH, et al. Fatal sickle cell crisis after granulocyte colony-stimulating factor administration. Blood 2001 ; 97 : 3313–3314. [CrossRef] [PubMed] [Google Scholar]
  16. Lagresle-Peyrou C, Lefrère F, Magrin E, et al. Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion. Haematologica 2018 ; 103 : 778–786. [CrossRef] [PubMed] [Google Scholar]
  17. Matatall KA, Jeong M, Chen S, et al. Chronic infection depletes hematopoietic stem cells through stress-induced terminal differentiation. Cell Rep 2016 ; 17 : 2584–2595. [CrossRef] [PubMed] [Google Scholar]
  18. Essers MAG, Offner S, Blanco-Bose WE, et al. IFNα activates dormant haematopoietic stem cells in vivo. Nature 2009 ; 458 : 904–908. [CrossRef] [PubMed] [Google Scholar]
  19. Steinberg MH. Fetal hemoglobin in sickle cell anemia. Blood 2020; 136 : 2392–400. [CrossRef] [PubMed] [Google Scholar]

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