Hematopoietic stem cell gene therapy: selecting only the best
J. Clin. Invest. Arthur Bank, et al. 112:1478 doi:10.1172/JCI20336 [Go to this article.]

Figure 1
Gene therapy using HSCs. (a) Transduction of cells with lentiviral supernatants containing the transgene MGMT that integrates into chromosomal DNA. (b) Gene transfer into HSCs with subsequent injection of populations of hematopoietic cells containing gene-corrected HSCs into animals, either dogs or, for human HSCs, NOD/SCID mice. Cells given by vein home to the marrow. (c) In vivo selection. Before: Small numbers of MGMT-expressing gene-corrected cells. After: Almost complete killing of untransduced HSCs with BCNU and O⁶BG therapy. Self-renewal and expansion of MGMT gene-corrected HSCs repopulate all hematopoietic lineages.