Hematopoietic Reconstitution Dynamics of Mobilized- and Bone Marrow-Derived Human Hematopoietic Stem Cells After Gene Therapy

Investigators presented an unplanned exploratory analysis evaluating the hematopoietic reconstitution kinetics, engraftment, and clonality in 13 pediatric Wiskott-Aldrich syndrome patients treated with autologous lentiviral-vector transduced hematopoietic stem/progenitor cells derived from mobilized peripheral blood, bone marrow or the combination of the two sources