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Investigators from multiple institutions conducted a phase 1–2 gene therapy trial in children with X-linked severe combined immunodeficiency (SCID-X1). Children with SCID-X1 were eligible if they were receiving care at 1 of 2 study institutions and lacked a matched sibling hematopoietic stem cell donor. Demographics and clinical status were assessed at enrollment.
Marrow was harvested from enrolled children and enriched for CD34+ cells. The cells were pre-stimulated and transduced with a lentiviral vector containing a normal copy of IL2RG, the defective gene in SCID-X1. Prior to infusion of transduced CD34+ cells, child participants received 1–2 daily doses of busulfan intravenously. Peripheral blood and bone marrow samples were obtained at various time points after infusion of transduced CD34+ cells to assess hematopoietic recovery.
The …
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