Abstract
SPECIFIC AIMSIn this study we examined the transduction efficiency and cell cycle status of circulating human fetal blood stem cells. We reveal that retroviral transduction of unique repopulating stem cells in human fetal circulation is superior to full gestation cord blood or adult sources and is enriched for actively cycling stem cells. Our findings indicate that active, transducible hematopoietic reconstituting cells are present in the circulation of the human fetus and represent novel target cells for future in utero gene therapy trials using autologous transplantation.PRINCIPAL FINDINGS1. Circulating human fetal blood repopulating stem cells can be transduced at a higher efficiency than full-term cord bloodIn utero gene therapy has been proposed as a method for permanent correction of somatic disorders affecting the hematopoietic system before disease initiation. However, clinical trials using transplantation of allogenic fetal liver, bone marrow, or adult stem cells have been unsuccessful largely du...
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