Fig. 4: Gene correction of PB mature lineages, BM CD34+ cells and HSPC subsets in BM-GT and MPB-GT patients. | Nature Communications

Fig. 4: Gene correction of PB mature lineages, BM CD34+ cells and HSPC subsets in BM-GT and MPB-GT patients.

From: Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy

Fig. 4: Gene correction of PB mature lineages, BM CD34+ cells and HSPC subsets in BM-GT and MPB-GT patients.The alternative text for this image may have been generated using AI.

All data are shown as mean +/- standard error mean (SEM). Vector copy number (VCN) measurement up to 3 years after GT in (a) Peripheral Blood Mononuclear Cells (PBMC) and purified (b) CD3 PB, (c) CD56 PB, (d) CD15 PB, (e) CD14 PB, (f) CD19 PB and (g) CD34 BM cells in BM-GT (n = 5 biologically independent samples), MPB-GT (n = 7 biologically independent samples) and Pt1. h–k Violin plots displaying VCN of sorted HSPC subpopulations from BM-GT or MPB-GT patients at early (<90 days) and late (>2 years) time points after GT. Dots with the same color in all the subsets show the VCN values for the same patient. Lines within the violin show the median value, while dashed lines show quartile ranges. lPercentage and (m) mean VCN of gene-corrected colonies derived from BM CD34+ cells isolated from BM-GT patients, MPB-GT patients and Pt1 at early (30 days after GT) and late (>1 year after GT) time points. Lines within the violin show the median value, while dashed lines show quartile ranges. Dot plots showing correlation between the percentage of transduced colonies at 2 years after GT and (n) number of CD34+ cells infused, (o) number of HSC infused in GT patients and (p) number of platelets at 2 years after GT. Exact p values are shown. Blue dots: MPB-GT patients (n = 6); Red dots: BM-GT patients (n = 5); Purple dot: Pt1 (BM Bone Marrow; MPB Mobilized Peripheral Blood; GT Gene Therapy; PB Peripheral Blood; HSC Hematopoietic Stem Cells; MPP Multi-Potent Progenitors; MLP Multi-lymphoid Progenitors; ETP Early T cell Progenitors; Pre B/NK = B cells and natural killer cell precursors; CMP Common Myeloid Progenitors; GMP Granulocytes-Monocytes Progenitors; MEP Megakaryocytes Erythrocytes Progenitors; CFC Colony Forming Cell) (Statistical tests on VCN collected over time: two-sided tests from longitudinal models on CD34+ cell dose, no adjustment for multiple comparisons were made *p < 0.05; **p < 0.01; exact p values were reported in Supplementary Table 1. Statistical tests for groups’ comparisons: Two-sided Mann–Whitney test, only statistically significant p values are reported within the graphs. Statistical test for correlations: Two-sided Spearman r. Tendency lines and 95% of confidence intervals were drawn only if residuals passed normality tests). Source Data for panels h–k are provided as a Source Data file.

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