Human β-Globin Gene Expression after Gene Transfer Using Retroviral Vectors

Abstract
A retroviral vector cntaining a 4.4-kb Pst I human βS-globin gene and a neomycin resistance gene was used to infect NIH-3T3 and mouse erythroleukemia cells (MELC). In MELC, human (β-globin mRNA transcripts are transcribed and properly initiated and spliced. In some cases, there is an appropriate increase in β-globin mRNA on addition of dimethylsulfoxide (DMSO), an inducer of hemoglobin synthesis and erythroid differentiation in these cells. When NIH-3T3 cells are infected with the same retroviral vector, there is less globin mRNA accumulation and no evidence for appropriate regulation. Human β-globin gene expression in MELC clones induced with DMSO is 2–3% that of endogenous mouse β-globin gene expression. These results indicate that retroviral vectors can be used to transfer and appropriately express human β-globin genes in erythroid cells.