Long-term repopulation ability of FA-D1 HSCs genetically corrected with BRCA2-LVs. (A) Schematic representation of the experimental protocol used to assess the long-term repopulating capacity of FA-D1 BM grafts transduced with BRCA2-LVs. Six months after the infusion of EGFP- or BRCA2-transduced FA-D1 BM cells, recipients were killed and BM cells from 2 recipients corresponding to the therapeutic group (recipients 1 and 3) and from one recipient of the control group (recipient 9) were transplanted (10⁷ BM cells per mouse) into 4 secondary FA-D1 recipients (recipients 1.1, 3.1, 3.2, and 9.1) previously irradiated with 3 Gy. (B) Analysis of the proportion of donor repopulation (cells positive for SRY sequences) in the PB of secondary recipients 9 months after BMT. (C) Proportion of donor-derived CFCs (SRY⁺; ▩) and the proportion of transduced CFCs (hBRCA2⁺ or EGFP⁺; ■) as deduced from qPCR analyses conducted with individually picked colonies. (D) Percentage of CFCs obtained from the BM of secondary recipients (9 months after BMT) that survived to 30 nM MMC added to the clonogenic cultures.