Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy

In vitro differentiation and transplantation studies in mice confirm that primitive hematopoietic stem/progenitor cells from both sources have comparable engraftment and multilineage differentiation potential. Altogether, our analyses reveal that the differential behavior after gene therapy of hematopoietic stem/progenitor cells derived from either bone marrow or mobilized peripheral blood is mainly due to the distinct cell composition rather than functional differences of the infused cell products, providing new frames of references for clinical interpretation of hematopoietic stem/progenitor cell transplantation outcome.

Data supporting the current study are part of a registered clinical trial (NCT01515462). These data are available under restricted access for the sensitive nature of the clinical data, access can be obtained by request to the corresponding author (Prof. Alessandro Aiuti, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, 20132 Italy.).

We intend to reply to any requests within two weeks and we will share the deposited data only for research purposes. The non-clinical data generated in this study are also provided as the Source Data file associated to the published work.