Lineage Specific Expression of a Human β-Globin Gene in Murine Bone Marrow Transplant Recipients

Abstract

Introduction of foreign genes into the hematopoietic system of adult animals has been accomplished by recombinant retroviral infection of the pluripotent bone marrow stem cell^1–3. Retroviral vectors are highly efficient for gene transduction of this rare cell type which is present in bone marrow at a frequency of approximately 1 in 10⁵. Engraftment of lethally irradiated recipients with infected bone marrow results in the reconstitution of all mature blood cell lineages from the transduced stem cell(s). The unique integration property of the provirus allows for the lineage mapping of individually transduced stem cell(s) and the assessment of the developmental and dynamic nature of hematopoiesis in vivo ³. While retroviruses have been demonstrated to serve as efficient vectors of the tranduction of exogenous genes into the hematopoietic system, there have been only limited demonstrations of expression of such inserted sequences ^1,2,3,4,5.