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. 2016 Dec 18;4:1–16. doi: 10.1016/j.omtm.2016.11.001

Figure 4.

Figure 4

Maintenance of VCN and WASp+ Expression in HSC and Their Progeny in 650.MND.hWASp LV-Treated Animals following Secondary Transplantation

Proportion of WASp+ cells in the BM (A) and spleen (B) and in PB platelets (C) of secondary recipients (16 weeks post-transplantation with BM from primary recipients). Data represent three individual experiments: n = 12 (KO mock), 16 (MND.hWASp), 51 (650.MND.hWASp) (n = 50 in C), and 15 (WT mock). In (C), the percentage (left) and MFI (right) of WAS+ platelets is shown. (D) Comparison of viral copy number (VCN) determined by qPCR of gDNA extracted from total BM and splenic cells of primary (1°) and secondary (2°) recipient mice; each dot represents a single animal. Data are from three independent experiments for both primary (n = 10 for MND.hWASp and n = 28 for 650.MND.hWASp) and secondary recipients (n = 16 for MND.hWASp, 51 for 650.MND.hWASp). (E) Percentage of WASp+ cells in HSCs in BM of primary (1°) and secondary (2°) gene therapy recipients. Data represents two unique experiments for both primary (n = 3 for MND.hWASp and n = 6 for 650.MND.hWASp) and secondary recipients (n = 6 for MND.hWASp and 12 for 650.MND.hWASp). In all panels, the mean ± SD value is shown. p values were calculated using the unpaired two-tailed t test. *p < 0.04, **p < 0.005.