Figure 2.

VCN and chimerism in bone marrow after gene therapy

(A) Chimerism, expressed as the percentage of reconstituted male donor cells in bone marrow of female recipients treated with gene therapy, determined by qPCR on Sry and Gapdh loci. (B) VCN measured in bone marrow by qPCR on HIV and Gapdh loci. VCN is not normalized for chimerism. MOI, multiplicity of infection; Gy, gray. Data represent means ± SEM and are analyzed by two-way ANOVA followed by Bonferroni’s multiple testing correction, using vector (LV-GAAco or LV-IGF2.GAAco) and gene therapy dose as categorical variables. Significant results are indicated by brackets. Results of multiple comparison analysis are reported in Table S3. LV-GAAco and LV-IGF2.GAAco, n = 7 per group; KO, n = 5; WT, n = 5. ns, not significant; ∗p ≤ 0.05, ∗∗∗∗p ≤ 0.0001.