Figure 1. Human mobilized gene-modified CD34⁺ cells produced using point-of-care manufacturing engraft in immunodeficient mice.

(a) Human CD34⁺ cells were obtained from mobilized PB after HPC-A. Adult NSG mice ranging between 8 and 12 weeks of age received a sub-lethal dose (270 cGy) of radiation 4 h before intravenous injection of 1 × 10⁶ gene-modified human CD34⁺ cells. Mice were then followed for 12 weeks post transplant. At 12 weeks, animals were killed and lymphoid tissues were analysed for total human cell levels as well as frequency of genet modification. (b) Human CD45⁺ cell engraftment levels in PB of individual adult NSG mice receiving LV-transduced CD34⁺ cells from human apheresis (HPC-A) donors. (c) At 12 weeks after injection, BM was analysed for both total human CD45⁺ cell content and human CD34⁺ levels. (d) The level of gene-modified human cells in both PB and BM of each recipient was determined by quantitative PCR.