Reconstitution of Dual sh1005/sh516-transduced HSPCs in humanized BLT mice.

<p><b>A.</b> Schematic of generating vector-transduced HSPC-transplanted hu-BLT mouse. NSG mouse is treated with Busulfan 24 hours pre-transplantation. CD34⁺ and CD34⁻ cells are isolated from human fetal liver (FL). CD34⁺ cells are transduced with either therapeutic (EGFP-marked) or control (mCherry-marked) vectors. Therapeutic vector- and control vector- transduced CD34⁺ cells are mixed at a 50 50 ratio. The cell mixture is then 1) combined with CD34⁻ cells, solidified with Matrigel, and implanted under the kidney capsule with a human fetal thymus segment (FT) and also 2) intravenously injected. <b>B.</b> EGFP and mCherry reporter gene expression was monitored in human CD45⁺, CD3⁺CD45⁺, and CD19⁺CD45⁺ cells within a gated lymphocyte population in peripheral blood at twelve weeks post-transplantation. CD4/CD8 ratios were analyzed in mCherry- and EGFP- marked CD3⁺CD45⁺ cells. Data were generated from n = 13 mice for both Mono sh1005- and Dual sh1005/sh516- HSPC-transplanted animals from an aggregate of three donors. Error bars: mean + standard error of mean (SEM) in n = 13 per group.