Characterization of CD34+ cells infused in WAS BM-GT and MPB-GT patients.
MPB, MPB CMP
PROLIFERATION
Violin plots showing: However, the characterization of purified BM or MPB CD34+ cells employed for transductionTo address whether the culture conditions during ex vivo gene-correction could have induced distinct differentiation and/or proliferation of the two stem cell sources, we reproduced the in vitro clinical transduction protocol on BM-derived (The direct comparison of BM- or MPB-derived CD34+ cell composition before transduction by using either the conventional or the alternative gating strategy, revealed that the alternative approach was not able to measure the differences in CD34+ cell composition between the two sources (Supplementary Fig. Given these analyses, we estimated the number of infused cells for each HSPC subpopulation in BM- and MPB-GT patients based on the CD34+ cell composition assessed before transduction. We found that MPB-GT patients, were infused with higher numbers of primitive (BM vs. MPB HSC 10^6/Kg median values: 0.06 vs. 0.5; BM vs. MPB HSC+MPP 10^6/Kg median values: 0.09 vs. 1.36) and myeloid progenitors (BM vs. MPB CMP + GMP 10^6/Kg median values: 1.37 vs. 5.26) with respect to BM-GT patients (Fig. 