Article
Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease.
Nature medicine - 1 Dec 2023
Spencer Chapman Michael, Cull Alyssa H, Ciuculescu Marioara F, Esrick Erica B, Mitchell Emily, Jung Hyunchul, O'Neill Laura, Roberts Kirsty, Fabre Margarete A, Williams Nicholas, Nangalia Jyoti, Quinton Joanne, Fox James M, Pellin Danilo, Makani Julie, Armant Myriam, Williams David A, Campbell Peter J, Kent David G
Abstract excerpt
Gene therapy (GT) provides a potentially curative treatment option for patients with sickle cell disease (SCD); however, the occurrence of myeloid malignancies in GT clinical trials has prompted concern, with several postulated mechanisms. Here, we used whole-genome sequencing to track hematopoietic stem cells (HSCs) from six patients with SCD at pre- and post-GT time points to map the somatic mutation and clonal...
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