{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["23(1)"],"submitter":["Brendel C"],"pubmed_abstract":["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 vir"],"journal":["Molecular therapy : the journal of the American Society of Gene Therapy"],"pagination":["63-70"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC4426797"],"repository":["biostudies-literature"],"pubmed_title":["CD133-targeted gene transfer into long-term repopulating hematopoietic stem cells."],"pmcid":["PMC4426797"],"pubmed_authors":["Kneissl S","Abel T","Schwable J","Buchholz CJ","Serve H","Muller-Kuller U","Bystrykh L","Kunkel H","Brendel C","Brugman M","Grez M","Daniela A","Chen-Wichmann L","Goebel B","Kaufmann KB"],"additional_accession":[]},"is_claimable":false,"name":"CD133-targeted gene transfer into long-term repopulating hematopoietic stem cells.","description":"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 vir","dates":{"release":"2015-01-01T00:00:00Z","publication":"2015 Jan","modification":"2026-04-29T07:43:39.155Z","creation":"2019-03-27T01:51:21Z"},"accession":"S-EPMC4426797","cross_references":{"pubmed":["25189742"],"doi":["10.1038/mt.2014.173"]}}