Transduction of human CD34(+) cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors

Efficient gene transfer into human hematopoietic stem cells (HSCs) is an im portant goal in the study of the hematopoietic system as well as for gene t herapy of hematopoietic disorders. A Lentiviral vector based on the human i mmunodeficiency virus (HIV) was able to transduce human CD34(+) cells capab le of stable, Long-term reconstitution of nonobese diabetic/severe combined immunodeficient (NOD/SCID) mice. High-efficiency transduction occurred in the absence of cytokine stimulation and resulted in transgene expression in multiple Lineages of human hematopoietic cells for up to 22 weeks after tr ansplantation.