Integration site number, Diversity index and hematopoietic output measured in WAS BM-GT and MPB-GT patients overtime.
Violin plots showing We then estimated the minimum number of engrafted HSPC clones by capture-and-recapture approach in myeloid cells, which are short-lived and provide a readout of stem cell output, as previously describedFinally, to assess the in vivo output of the engrafted primitive HSPC derived from the two sources at steady state, we measured the frequency of IS shared between sorted HSC+MPP subsets, BM progenitors and PB mature populations (Supplementary Tables Overall, IS analyses showed that the infusion of higher number of gene-corrected primitive cells resulted in increased amount of total engrafting clones in the bone marrow of MPB-GT patients. Nevertheless, once engrafted, primitive HSPC deriving from either BM or MPB source display similar multilineage output in patients after GT at steady state.