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为提高人β-珠蛋白基因在基因转移中的稳定性与在红系细胞中的表达水平,构建了带单个和多个基因座控制区核心序列的人β-珠蛋白基因腺相关病毒(ade-no-associated virus,AAV)载体AV53HS2Δβ2Neo和AV53HS432ΔβNeo。利用AAV载体介导的基因转移将两种重组体导入红系细胞(MEL),分析了它们在MEL细胞中的整合与表达。结果表明,AAV载体介导带多个基因座控制区核心序列的人β-珠蛋白基因在MEL细胞中较稳定整合,呈现与内源α-珠蛋白基因可比的表达水平。
To improve the stability of human β-globin gene in gene transfer and the expression level in erythroid cells, a human β-globin gene adeno-associated virus -no-associated virus (AAV) vectors AV53HS2Δβ2Neo and AV53HS432ΔβNeo. Both recombinants were introduced into erythroid cells (MEL) using AAV vector-mediated gene transfer and their integration and expression in MEL cells was analyzed. The results showed that the AAV vector mediates the more stable integration of human beta-globin gene in the MEL cells with a core sequence with multiple locus control regions and comparable expression levels to the endogenous alpha-globin gene.