Adeno-associated virus-mediated integration and expression of human β- globin gene with the core fragment of locus control region in erythroid cells

摘要

To improve the integration stability and expression of the transferred human β-globin gene, the two recombinant adeno-associated virus (AAV)vectors containing the human β-globin gene with a single or multiple Dnase I hypersensitive site (HS) core fragment of the LCR were constructed. These recombinants were respectively introduced Into MEL cells via AAV-mediated gene transfer to investigate their integration and expression. The results suggested that Following AAV vector-mediated gene transfer, the human β-globin gene with the multiple HS core fragment of the LCR could steadily integrate into MEL cells and confer an expression level comparable with endogenous mouse α-globin gene.