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 a nd acquired diseases. We demonstrate here stable gene transfer into hu man bone marrow-derived CD34(+) progenitors using cationic lipids to f acilitate GaLV and amphotroic pseudotyped retroviral-mediated transduc tions. 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 transdu ction was facilitated with conventional polycations Polybrene or prota mine sulfate. Thus, a combination of GaLV pseudotyped vectors and cati onic lipids results in increased transduction frequencies of the CD34( +) cells without a requirement of extended in vitro culture, or co-cul tivation with producer cell lines. These improvements may result in th e production of therapeutically significant quantities of genetically modified hematopoietic cells.