Blood, 1 March 2003, Vol. 101, No. 5, pp. 1798-1800
HEMATOPOIESIS
Brief report
BVL-1-like VL30 promoter sustains long-term expression in
erythroid progenitor cells
William R. Staplin and
Joseph A. Knezetic
From the Department of Biomedical Sciences, Creighton
University, Omaha, NE.
Congenital blood disorders are common and yet clinically
challenging globin disorders. Gene therapy continues to serve as a
potential therapeutic method to treat these disorders. While tremendous
advances have been made in vivo, gene delivery protocols and vector
prototypes still require optimization. Alternative cis-acting promoter elements derived from VL30
retroelements have been effective in expressing tissue-specific
transgene expression in vivo in nonerythroid cells. VL30 promoter
elements were isolated from ELM-I-1 erythroid progenitor cells upon
erythropoietin (epo) treatment. These promoters were inserted into a
VL30-derived expression vector and reintroduced into the ELM-I-1 cells.
-Galactosidase reporter gene activity from the ELM 5 clone, a
BVL-1-like VL30 promoter, was capable of expressing sustained levels
of the transgene expression over a 16-week assay period. These findings
delineate the potential utility of these retroelement promoters as
transcriptionally active, erythroid-specific, long terminal repeat
(LTR) components for current globin vector constructs.
