Dzierzak E A, Papayannopoulou T, Mulligan R C
Whitehead Institute for Biomedical Research, Nine Cambridge Center, Massachusetts 02142.
Nature. 1988 Jan 7;331(6151):35-41. doi: 10.1038/331035a0.
Recombinant retroviral genomes encoding a chromosomal human beta-globin gene have been used to transduce murine haematopoietic stem cells in vitro. After permanent engraftment of lethally irradiated recipients with the transduced cells, the human beta-globin gene is expressed at significant levels only within the erythroid lineage. These results indicate that it is possible to obtain stable expression of exogenous chromosomal DNA sequences introduced into mature haematopoietic cells in vivo via stem cell infection, and that human disorders of haemoglobin production may be more feasible candidates for somatic cell gene therapy than previously suspected.
编码染色体人β-珠蛋白基因的重组逆转录病毒基因组已被用于体外转导小鼠造血干细胞。在用转导细胞对致死性照射的受体进行永久植入后，人β-珠蛋白基因仅在红系谱系中以显著水平表达。这些结果表明，通过干细胞感染将外源染色体DNA序列引入体内成熟造血细胞中以获得稳定表达是可能的，并且人类血红蛋白生成障碍可能比以前认为的更适合作为体细胞基因治疗的候选对象。
