LINEAGE-SPECIFIC EXPRESSION OF A HUMAN BETA-GLOBIN GENE IN MURINE BONE-MARROW TRANSPLANT RECIPIENTS RECONSTITUTED WITH RETROVIRUS-TRANSDUCED STEM-CELLS
LINEAGE-SPECIFIC EXPRESSION OF A HUMAN BETA-GLOBIN GENE IN MURINE BONE-MARROW TRANSPLANT RECIPIENTS RECONSTITUTED WITH RETROVIRUS-TRANSDUCED STEM-CELLS
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DOI：
10.1038/331035a0
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发表时间：
1988-01-07
期刊：
影响因子：
64.8
通讯作者：
MULLIGAN, RC
中科院分区：
文献类型：
--
作者：
DZIERZAK, EA;PAPAYANNOPOULOU, T;MULLIGAN, RC
重组逆转录病毒基因组编码染色体人β-珠蛋白基因已被用于体外转导小鼠造血干细胞。在将转染的细胞永久植入受致死性辐射的受体后，人β-珠蛋白基因仅在红系中显著表达。这些结果表明，通过干细胞感染将外源染色体DNA序列引入成熟的造血细胞中，在体内获得稳定的表达是可能的，并且人类血红蛋白产生障碍可能比以前怀疑的更可行的体细胞基因治疗候选者。
Recombinant retroviral genomes encoding a chromosomal humanβ-globin gene have been used to transduce murine haematopoietic stem cells in vitro. After permanent engraftment oflethally irradiated recipients with the transduced cells, the humanβ-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.