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Blood, Vol. 92 No. 9 (November 1), 1998:
pp. 3163-3171
High Efficiency Gene Transfer to Human Hematopoietic
SCID-Repopulating Cells Under Serum-Free Conditions
Andrea J. Schilz,
Gaby Brouns,
Heike Knö ,
Oliver G. Ottmann,
Dieter Hoelzer,
Axel A. Fauser,
Adrian J. Thrasher, and
Manuel Grez
From the Molecular Virology Lab, Georg-Speyer-Haus, Frankfurt,
Germany; the Department of Hematology/Oncology, Bone Marrow
Transplantation Hospital, Idar-Oberstein, Germany; the Molecular
Immunology Unit, Institute of Child Health, London, UK; and the
Department of Hematology/Oncology, School of Medicine, University of
Frankfurt, Frankfurt, Germany.
Stable gene transfer to human pluripotent hematopoietic stem cells
(PHSCs) is an attractive strategy for the curative treatment of many
genetic hematologic disorders. In clinical trials, the levels of gene
transfer to this cell population have generally been low, reflecting
deficiencies in both the vector systems and transduction conditions. In
this study, we have used a pseudotyped murine retroviral vector to
transduce human CD34+ cells purified from bone marrow
(BM) and umbilical cord blood (CB) under optimized conditions. After
transduction, 71% to 97% of the hematopoietic cells were found to
express a low-affinity nerve growth factor receptor (LNGFR) marker
gene. Six weeks after transplantation into immunodeficient
NOD/LtSz-scid/scid (NOD/SCID) mice, LNGFR expression was
detected in 6% to 57% of CD45+ cells in eight of nine
engrafted animals. Moreover, proviral DNA was detected in 8.3% to 45%
of secondary colonies derived from BM cells of engrafted NOD/SCID mice.
Our data show consistent transduction of SCID-repopulating cells (SRCs)
and suggest that the efficiency of gene transfer to human hematopoietic
repopulating cells can be improved using existing retroviral vector
systems and carefully optimized transduction conditions.
© 1998 by The American Society of Hematology.
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