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Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease.
Spencer Chapman M, Cull AH, Ciuculescu MF, Esrick EB, Mitchell E, Jung H, O'Neill L, Roberts K, Fabre MA, Williams N, Nangalia J, Quinton J, Fox JM, Pellin D, Makani J, Armant M, Williams DA, Campbell PJ, Kent DG. Spencer Chapman M, et al. Nat Med. 2023 Dec;29(12):3175-3183. doi: 10.1038/s41591-023-02636-6. Epub 2023 Nov 16. Nat Med. 2023. PMID: 37973947 Free PMC article.
Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling.
De Souza DC, Hebert N, Esrick EB, Ciuculescu MF, Archer NM, Armant M, Audureau É, Brendel C, Di Caprio G, Galactéros F, Liu D, McCabe A, Morris E, Schonbrun E, Williams D, Wood DK, Williams DA, Bartolucci P, Higgins JM. De Souza DC, et al. Nat Commun. 2023 Sep 20;14(1):5850. doi: 10.1038/s41467-023-40923-5. Nat Commun. 2023. PMID: 37730674 Free PMC article.