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Blood, 1 December 2000, Vol. 96, No. 12, pp. 3725-3733
GENE THERAPY
Lentiviral gene transfer into primary and secondary
NOD/SCID repopulating cells
Niels-Bjarne Woods,
Cecilia Fahlman,
Hanna Mikkola,
Isao Hamaguchi,
Karin Olsson,
Romain Zufferey,
Sten Eirik Jacobsen,
Didier Trono, and
Stefan Karlsson
From the Department for Molecular Medicine and Gene
Therapy, Institute of Laboratory Medicine, Lund University, Lund,
Sweden; The Stem Cell Laboratory, Institute of Laboratory Medicine,
Lund University Hospital, Lund, Sweden; Department of Genetics and
Microbiology, University of Geneva Medical School, Geneva, Switzerland.
The ability of lentiviral vectors to transfer genes into human
hematopoietic stem cells was studied, using a human immunodeficiency virus 1 (HIV-1)-derived vector expressing the green fluorescence protein (GFP) downstream of the phosphoglycerate kinase (PGK) promoter
and pseudotyped with the G protein of vesicular stomatitis virus (VSV).
High-efficiency transduction of human cord blood CD34+
cells was achieved after overnight incubation with vector particles. Sixteen to 28 percent of individual colony-forming units
granulocyte-macrophage (CFU-GM) colonies derived from cord blood
CD34+ cells were positive by polymerase chain reaction
(PCR) for the GFP gene. The transduction efficiency of
SCID-repopulating cells (SRC) within the cord blood CD34+
population was assessed by serial transplantation into nonobese diabetic/severe combined immunodeficient (NOD/SCID) mice. When 400 000
cord blood CD34+ cells were transplanted into primary
recipients, all primary and secondary recipients contained and
expressed the transgene. Over 50% of CFU-GM colonies derived from the
bone marrow of these primary and secondary recipients contained the
vector on average as determined by PCR. Transplantation of transduced
cells in limiting dilution generated GFP+ lymphoid and
myeloid progeny cells that may have arisen from a single SRC. Inverse
PCR analysis was used to amplify vector-chromosomal junctional
fragments in colonies derived from SRC and confirmed that the vector
was integrated. These results show that lentiviral vectors can
efficiently transduce very primitive human hematopoietic progenitor and
stem cells.
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