Submitted July 15, 2002
Accepted October 4, 2002
BVL-1 like VL30 promoter sustains long-term expression in erythroid progenitor cells
William R Staplin and Joseph A Knezetic*
Department of Biomedical Sciences, Creighton University, Omaha, NE, USA
* Corresponding author; email: joek{at}creighton.edu.
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 non-erythroid 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. ß-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 sixteen week assay period. These findings delineate the potential utility of these retroelement promoters as transcriptionally active, erythroid specific, LTR components, for current globin vector constructs.
