High-level erythroid lineage-directed gene expression using globin gene regulatory elements after lentiviral vector-mediated gene transfer into primitive human and murine hematopoietic cells.

High-level erythroid lineage-directed gene expression using globin gene regulatory elements after lentiviral vector-mediated gene transfer into primitive human and murine hematopoietic cells.
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慢病毒载体介导的基因转移至原始人类和小鼠造血细胞后,使用珠蛋白基因调控元件进行高水平红细胞谱系定向基因表达。

DOI:
10.1089/10430340260395866
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发表时间:
2002
期刊:
Human gene therapy.
影响因子:
--
通讯作者:
Nienhuis,ArthurW
Nienhuis,ArthurW
中科院分区:
--
文献类型:
--
作者:
Hanawa,Hideki;Persons,DerekA;Nienhuis,ArthurW

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Lentiviral vectors efficiently transduce primitive human hematopoietic cells and are capable of transferring complex genomes. Vectors were designed with hypersensitive sites containing regulatory elements from theβ-globin locus control region linked to theβ-globin gene promoter to drive expression of the enhanced green fluorescent protein marker to facilitate analysis of the pattern of gene expression in various hematopoietic lineages. Such vectors gave higher level, induced expression in mouse erythroleukemia cells than a previously described vector that utilized an enhancer from the α locus and the ankyrin-1 promoter [Moreau-Gaudry, F., Xia, P., Jiang, G., Perelman, N.P., Bauer, G., Ellis, J., Surinya, K.H., Mavilio, F., Shen, C.K., and Malik, P. (2001). Blood 98, 2664-2672]. The addition ofγ-globin intron sequences further augmented vector expression. Expression was also effectively targeted to the erythroid lineage in cultured human cells from peripheral blood and in mouse red blood cellsin vivo, although lower levels of expression were also observed in other lineages. Thus, these newly described vectors provide a means to achieve high-level gene expression, predominantly in erythroid cells, an outcome that may have potential therapeutic application.
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发表时间: 2001
期刊: Human gene therapy.
影响因子: --
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