Adeno-associated virus-mediated integration and expression of humanβ-globin gene with the core fragm

• 作 者: 李斌 刘德培 王晶 董文吉 郭志晨 夏薇 梁植权
• 机 构: State Key Laboratory of Medical Molecular Biology, Institute of Basic Medical Sciences, Chinese Acad
• 出 处: Progress in Natural Science
• 发表日期: 2000年06期
• 关键词: globin hypersensitive confer comparable locus endogenous deletion DNase integrat

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 a-globin gene.

To improve the integration stability and expression of the 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 the 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 a-globin gene.