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Targeted gene transfer to human hematopoietic progenitor cell lines through
the c-kit receptor
P Schwarzenberger, SE Spence, JM Gooya, D Michiel, DT Curiel, FW Ruscetti and JR Keller
Laboratory of Leukocyte Biology, SAIC-Frederick, MD, USA.
In this report, we describe a novel gene therapy approach for hematopoietic
stem/progenitor cells using a specific receptor-mediated gene transfection
procedure to target c-kit+ cell lines. The vector consists of plasmid DNA
containing a luciferase reporter gene that is condensed by electrostatic
forces with polylysine (PL) covalently linked to streptavidin (binds
biotinylated ligand) and PL covalently linked to adenovirus (AD; to achieve
endosomal lysis) with the final addition of biotinylated steel factor
(SLF-biotin). Targeted transfection of growth factor-dependent
hematopoietic progenitor cell lines that express c-kit showed specific
luciferase gene expression over cell lines that did not express c-kit. This
effect was dependent on the dose of SLF-biotin and was competed by excess
SLF or with monoclonal antibodies that recognize c-kit and block the
binding of SLF to its receptor. Maximum transfection efficiency (> 90%)
requires a 2- hour incubation period of the vector with the cells, and
maximum gene expression occurred 30 hours later. Removal of the
endosomalytic agent, AD, from the vector resulted in the loss of gene
expression. Vector targeting was versatile and could be changed by the
addition of other biotinylated ligands. In principle, this vector should be
broadly applicable to deliver genes to hematopoietic stem/progenitor cells
in vitro and in vivo.
Volume 87,
Issue 2,
pp. 472-478,
01/15/1996
Copyright © 1996 by The American Society of Hematology
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