【摘要】 采用回体（ｅｘ ｖｉｖｏ） 基因转移策略， 分析了人β－珠蛋白基因在小鼠体内的整合和表达。研究发现， 在小鼠ＣＦＵ－Ｓ１２中检测到转移的人β－珠蛋白基因的整合， 其表达水平约为内源性鼠α－珠蛋白基因的０．５％ ～５％ 。在两只长期造血重建小鼠的骨髓、脾及胸腺和另一只小鼠的骨髓及脾中整合有人β－珠蛋白基因， 人β－珠蛋白基因在一只重建小鼠的骨髓和脾中表达，表达水平约为鼠内源α－珠蛋白基因的７％ ，表明反转录病毒载体介导的人β－珠蛋白基因可稳定整合在小鼠骨髓造血干细胞中， 并呈现红系组织特异性表达更多还原

【Abstract】 To examine the in vivo properties of retroviral recombinant carrying partially deleted human β globin gene(Δβ) and truncated erythroid enhancer (342 bp of 5HS2) at the mRNA levels following short and long term reconstitution in mice with infected marrow cells, the human β globin gene was transferred into murine hematopoietic progenitor cells and the integration and expression of transferred gene were analyzed by soughern blot and RNase protection assay or RT PCR. Results showed that the transferred human β globin gene was detected in murine CFU S 12 and the expression level was about 0.5%～5% of the endogenous mouse α globin gene. In mice surviving long term transplanted with bone marrow cells transduced with high titer virus, bone marrow, spleen and thymus from two mice and bone marrow and spleen from another mouse contained the intact proviral genome. Long term expression of the transferred gene was seen in one mouse at the level of 7% of endogenous murine α globin gene.更多还原