Extended Data Fig. 2: Gene transfer efficiency into HSPC in 2-week-old C57Bl/6 mice after in vivo LV administration.
From: In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking
a,b, Mean with SEM of GFP-positive cells (a) or VCN (b) measured in the PB of C57Bl/6 mice administered LV (LV, n = 15; 3e10 TU/kg) at 2 weeks-of-age or untreated (Unt, n = 1). c, Single values and mean with SEM of the VCN measured in the indicated organs in Unt and LV mice, 16 weeks after LV administration. d, Single values and mean with SEM of the absolute count of HSCs in the spleen of 2-week-old mobilized (n = 6) or untreated control (n = 7) mice, as indicated. Mann-Whitney test. e,f, Single values and mean with SEM of GFP-positive cells in the thymus (e) or spleen (f) of mobilized (n = 6) or control (n = 7) mice, 20 wks after LV administration. Mann-Whitney test. g,h, Mean with SEM of the engraftment of CD45.2 cells in the PB of CD45.1 recipient mice over time. Controls: n = 5 recipient; Mobilized: n = 6 recipient. Mice were transplanted with 15x106 total BM cells harvested from donor mice, 20 wks after LV administration. i,j, Single values and mean with SEM of the VCN (i) or engraftment of CD45.2 cells (j) measured in CD45.1 recipient mice, 16 wks after transplant in the indicated organs. Mann-Whitney test in (i). k,l, Single values and mean with SEM of GFP-positive cells in the thymus (k), or spleen (l) of CD45.1 recipient mice, 16 wks after transplant. Mann-Whitney test.
