Analysis of the repopulating properties of mobilized peripheral blood CD34⁺cells from FA-A patients and healthy donors after transduction for 24 hours with a GMP-produced therapeutic lentiviral vector. The figure shows representative analyses of donor hematopoietic reconstitution in the BM of recipient NSG mice 4 weeks after infusion of FA-A (A) and HD (B) mPB CD34⁺ cells transduced for 24 hours with the therapeutic PGK-FANCA.Wpre* lentiviral vector. (C) Analysis of engraftment measured as percentage of hCD45⁺ cells in individual NSG mice after intravenous (dark red circles) or intrabone (light red circles) injection of FA or the intravenous injection (blue circles) of HD mPB CD34⁺ cells transduced under the established conditions. (D) Distribution of donor myeloid and lymphoid cell engraftment in the BM of recipient mice transplanted with transduced FA-A and HD CD34⁺ cells (symbols as in panel C). CD34⁺ cells from 4 different FA-A patients and from 1 HD, all of them treated with G-CSF and plerixafor.