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Related Collections Hematopoiesis and Stem Cells Transplantation Plenary Papers Gene Therapy Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  Blood, 15 August 2000, Vol. 96, No. 4, pp. 1206-1214 PLENARY PAPER Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein Patrick F. Kelly, Jody Vandergriff, Amit Nathwani, Arthur W. Nienhuis, and Elio F. Vanin From the Division of Experimental Hematology, Department of Hematology/ Oncology, St Jude Children's Research Hospital, Memphis, TN. Limited expression of the amphotropic envelope receptor is a recognized barrier to efficient oncoretroviral vector-mediated gene transfer. Human hematopoietic cell lines and cord blood-derived CD34+ and CD34+, CD38 cell populations and the progenitors contained therein were transduced far more efficiently with oncoretroviral particles pseudotyped with the envelope protein of feline endogenous virus (RD114) than with conventional amphotropic vector particles. Similarly, human repopulating cells from umbilical cord blood capable of establishing hematopoiesis in immunodeficient mice were efficiently transduced with RD114-pseudotyped particles, whereas amphotropic particles were ineffective at introducing the proviral genome. After only a single exposure of CD34+ cord blood cells to RD114-pseudotyped particles, all engrafted nonobese diabetic/severe combined immunodeficiency mice (15 of 15) contained genetically modified human bone marrow cells. Human cells that were positive for enhanced green fluorescent protein represented as much as 90% of the graft. 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