Introduction
MPB, hematological disorders
HEMATOLOGICAL DISORDERS, DISORDERS
Hematopoietic Stem/Progenitor Cells (HSPC) are widely exploited in the clinic for hematopoietic stem cell transplantation (HSCT) and autologous ex vivo gene therapy (GT) for treating genetic and acquired disorders, following a preparatory conditioning regimenSince a substantial fraction of the engrafting HSPC and their differentiated progeny are marked by the vector in a unique genomic site (insertion site, IS), IS analyses have been used to assess the in vivo hematopoietic dynamics after GT, unveiling common features of hematopoietic reconstitution after transplantationHSPC mobilized from the BM into the PB and harvested through leukapheresis, mobilized peripheral blood (MPB), has become in most cases the preferred method for isolating HSPC for patients undergoing HSCTNo study has evaluated so far the capability of engineered G+P MPB CD34+ cells to reconstitute the hematopoietic compartment, to survive and to maintain long-term polyclonal gene-corrected progeny, in comparison with BM-derived CD34+ cells. Given the broad application of HSPC-GT, not only in the treatment of hematological disorders