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reconstituting retroviral vector (recon vector) for selective gene expression

机译:重组逆转录病毒载体(recon载体)进行选择性基因表达

摘要

The present invention relates to a retroviral vector which is especially applicable as a safe gene transfer vehicle for targeted gene therapy. Said retroviral vector comprises one or more promoters inserted in antisense orientation within the 5'LTR region and one or more coding sequences inserted in antisense orientation within the 3'LTR region. Both, the promoter as well as the coding sequence, are additionally inserted in such a way as to ensure that the promoter and the coding sequence become duplicated during the process of reverse transcription in a target cell and thus appear in the 3' as well as in the 5'LTR region of the resulting provirus in a fashion where the promoter is located upstream of the coding sequence allowing it to drive gene expression. This system avoids any leakiness of gene expression in the packaging cells, and allows expression of transferred genes in the target cell without the necessity for external stimuli.
机译:逆转录病毒载体技术领域本发明涉及逆转录病毒载体,其特别适合用作用于靶向基因治疗的安全基因转移载体。所述逆转录病毒载体包含以反义方向插入在5'LTR区域内的一个或多个启动子和以反义方向插入在3'LTR区域内的一个或多个编码序列。另外,以确保启动子和编码序列在靶细胞的逆转录过程中重复并因此出现在3'以及5'中的方式另外插入启动子和编码序列。在启动子位于编码序列上游以允许其驱动基因表达的方式中,在所得原病毒的5'LTR区域中存在“启动子”。该系统避免了包装细胞中基因表达的任何泄漏,并允许转移的基因在靶细胞中表达而无需外部刺激。

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