GaLV pseudotyped vectors and cationic lipids transduce human CD34+ cells

High transduction frequency of hematopoietic stem/progenitor cells is essential to derive clinical benefits for treating certain inherited and acquired diseases. We demonstrate here stable gene transfer into human bone marrow-derived CD34+ progenitors using cationic lipids to facilitate GaLV and amphotroic pseudotyped retroviral-mediated transductions. Furthermore, the transgene was detected only in the progeny of flow cytometer sorted CD34+ population transduced by the LAPSN (PG13) viral vector in the presence of cationic lipids but not when transduction was facilitated with conventional polycations Polybrene or protamine sulfate. Thus, a combination of GaLV pseudotyped vectors and cationic lipids results in increased transduction frequencies of the CD34+ cells without a requirement of extended in vitro culture, or co-cultivation with producer cell lines. These improvements may result in the production of therapeutically significant quantities of genetically modified hematopoietic cells.