Extended Data Fig. 5: Bone Marrow Hematopoiesis at Screening and Recovery with Restored Hematopoiesis and DNA-Damage Resistance Post αβdepleted-HSCT.

a) Minimal in vitro colony forming counts (CFCs) at screening (red) indicative of reduced HSPC activity due to BMF in each individual with FA pre-treatment compared to healthy adult and pediatric controls (black) with increased CFC activity post allo-HSCT in all patients assessed (grey, gold and teal). Data are presented as mean ± SEM. Each bar represents an independent sample, with values averaged from three technical replicates. b) No mitomycin C (MMC)-resistance was observed in BM cells on research testing at patient enrollment in each of the treated individuals, indicative of FA disease with no somatic mosaicism at screening that subsequently increased post-HSCT with normalized 10 nM and 50 nM MMC-resistance in all patients due to engraftment of donor HSPCs without FA defects. Data are presented as mean ± SEM. Each data point represents an independent sample, with values averaged from three technical replicates. Parametric unpaired t-test (two-tailed) was used for statistical analysis.