biostudies-literatureUnknown23(1)Brendel CGene therapy for hematological disorders relies on the genetic modification of CD34(+) cells, a heterogeneous cell population containing about 0.01% long-term repopulating cells. Here, we show that the lentiviral vector CD133-LV, which uses a surface marker on human primitive hematopoietic stem cells (HSCs) as entry receptor, transfers genes preferentially into cells with high engraftment capability. Transduction of unstimulated CD34(+) cells with CD133-LV resulted in gene marking of cells with competitive proliferative advantage in vitro and in immunodeficient mice. The CD133-LV-transduced population contained significantly more cells with repopulating capacity than cells transduced with vesicular stomatitis virus (VSV)-LV, a lentiviral vector pseudotyped with the vesicular stomatitis virMolecular therapy : the journal of the American Society of Gene Therapy63-70https://www.ebi.ac.uk/biostudies/studies/S-EPMC4426797biostudies-literatureCD133-targeted gene transfer into long-term repopulating hematopoietic stem cells.PMC4426797Kneissl SAbel TSchwable JBuchholz CJServe HMuller-Kuller UBystrykh LKunkel HBrendel CBrugman MGrez MDaniela AChen-Wichmann LGoebel BKaufmann KBfalseCD133-targeted gene transfer into long-term repopulating hematopoietic stem cells.Gene therapy for hematological disorders relies on the genetic modification of CD34(+) cells, a heterogeneous cell population containing about 0.01% long-term repopulating cells. Here, we show that the lentiviral vector CD133-LV, which uses a surface marker on human primitive hematopoietic stem cells (HSCs) as entry receptor, transfers genes preferentially into cells with high engraftment capability. Transduction of unstimulated CD34(+) cells with CD133-LV resulted in gene marking of cells with competitive proliferative advantage in vitro and in immunodeficient mice. The CD133-LV-transduced population contained significantly more cells with repopulating capacity than cells transduced with vesicular stomatitis virus (VSV)-LV, a lentiviral vector pseudotyped with the vesicular stomatitis vir2015-01-01T00:00:00Z2015 Jan2026-04-29T07:43:39.155Z2019-03-27T01:51:21ZS-EPMC44267972518974210.1038/mt.2014.173