Gene correction of PB mature lineages, BM CD34+ cells and HSPC subsets in BM-GT and MPB-GT patients.
WAS
All data are shown as mean +/- standard error mean (SEM). Vector copy number (VCN) measurement up to 3 years after GT in (Since both groups of patients received similar sub-myeloablative conditioningFinally, since WAS patients are characterized also by platelet defectsOur molecular characterization indicated that the number of primitive HSPC in the CD34+ cells before transduction plays a key role in the transplantation outcome. The higher number of infused HSC in MPB-GT patients was associated with higher transduced BM chimerism in vivo, ultimately leading to higher gene-correction level in HSPC, myeloid and B cell compartment and to increased platelet counts in the long-term.