Transduction of human NOD/SCID-repopulating cells with both lymphoid and myeloid potential by foamy virus vectors.

ABSTRACT: The efficiency of gene transfer into human hematopoietic stem cells by oncoretroviral vectors is too low for effective gene therapy of most hematologic diseases. Retroviral vectors based on the nonpathogenic foamy viruses (FV) are an alternative gene-transfer system. In this study, human umbilical cord blood CD34(+) cells were transduced with FV vectors by a single 10-h exposure to vector stocks and then injected into sublethally irradiated nonobese diabetic-severe combined immunodeficiency (NOD/SCID) mice. At 5-7 weeks after transplantation, high transgene expression rates were observed in engrafted human hematopoietic cells, including over 60% of clonogenic progenitors. Significant transgene silencing did not occur. We developed an approach for expanding human cell populations derived fr

SUBMITTER: Josephson NC 

PROVIDER: S-EPMC123061 | biostudies-literature | 2002 Jun

REPOSITORIES: biostudies-literature