In utero gene transfer and expression: a sheep transplantation model
PW Kantoff, AW Flake, MA Eglitis, S Scharf, S Bond, E Gilboa, H Erlich, MR Harrison, ED Zanjani and WF Anderson
Laboratory of Molecular Hematology, National Heart, Lung, and Blood
Institute, Bethesda, MD 20892.
Retroviral-mediated gene transfer was used to insert a Neo R gene into
fetal sheep hematopoietic cells obtained by exchange transfusion from lambs
in utero. After gene transfer the cells were returned to the donor fetus.
The lambs were examined after birth for the presence of a functioning Neo R
gene. Of ten analyzable animals, six were positive for G418 resistant
progenitor cells (CFU-Mix, CFU-C, BFU-E, CFU-E). Two animals were studied
for extended periods of time: 8 and 24 months. Each has demonstrated a
pattern wherein positive periods are interspersed with times when there
were no detectable G418-resistant cells. We conclude that
retroviral-mediated gene transfer can be used to insert genes into early
progenitor cells of fetal sheep in utero and that the animals can continue
to demonstrate blood cells expressing the gene for more than 2 years after
birth. This is a US government work. There are no restrictions on its use.
Volume 73,
Issue 4,
pp. 1066-1073,
03/01/1989
Copyright © 1989 by The American Society of Hematology
