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Blood, 1 October 2001, Vol. 98, No. 7, pp. 2065-2070
GENE THERAPY
Sustained multilineage gene persistence and expression in dogs
transplanted with CD34+ marrow cells transduced by
RD114-pseudotype oncoretrovirus vectors
Martin Goerner,
Peter A. Horn,
Laura Peterson,
Peter Kurre,
Rainer Storb,
John E. J. Rasko, and
Hans-Peter Kiem
From the Divisions of Clinical Research and Human
Biology, Fred Hutchinson Cancer Research Center; and the Department of
Medicine, University of Washington School of Medicine, Seattle, WA.
Previous studies have shown that the choice of envelope
protein (pseudotype) can have a significant effect on the efficiency of
retroviral gene transfer into hematopoietic stem cells. This study used
a competitive repopulation assay in the dog model to evaluate
oncoretroviral vectors carrying the envelope protein of the endogenous
feline virus, RD114. CD34-enriched marrow cells were divided into equal
aliquots and transduced with vectors produced by the RD114-pseudotype
packaging cells FLYRD (LgGLSN and LNX) or by the gibbon ape leukemia
virus (GALV)-pseudotype packaging cells PG13 (LNY). A total of 5 dogs
were studied. One dog died because of infection before sustained
engraftment could be achieved, and monitoring was discontinued after 9 months in another animal that had very low overall gene-marking levels.
The 3 remaining animals are alive with follow-ups at 11, 22, and 23 months. Analyses of gene marking frequencies in peripheral blood and
marrow by polymerase chain reaction revealed no significant differences between the RD114 and GALV-pseudotype vectors. The LgGLSN vector also
contained the enhanced green fluorescent protein (GFP), enabling us to
monitor proviral expression by flow cytometry. Up to 10% of peripheral
blood cells expressed GFP shortly after transplantation and
approximately 6% after the longest follow-up of 23 months. Flow
cytometric analysis of hematopoietic subpopulations showed that most of
the GFP-expressing cells were granulocytes, although GFP-positive
lymphocytes and monocytes were also detected. In summary, these results
show that RD114-pseudotype oncoretroviral vectors are able to transduce
hematopoietic long-term repopulating cells and, thus, may be useful for
human stem cell gene therapy.
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