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A trial of somatic gene targeting in vivo with an adenovirus vector

机译:用腺病毒载体体内靶向体细胞基因的试验

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摘要

BackgroundGene targeting in vivo provides a potentially powerful method for gene analysis and gene therapy. In order to sensitively detect and accurately measure designed sequence changes, we have used a transgenic mouse system, MutaMouse, which has been developed for detection of mutation in vivo. It carries bacteriophage lambda genome with lacZ+ gene, whose change to lacZ-negative allele is detected after in vitro packaging into bacteriophage particles. We have also demonstrated that gene transfer with a replication-defective adenovirus vector can achieve efficient and accurate gene targeting in vitro.
机译:背景技术体内靶向基因为基因分析和基因治疗提供了潜在强大的方法。为了灵敏地检测和准确测量设计的序列变化,我们使用了转基因小鼠系统MutaMouse,该系统已开发用于体内突变的检测。它携带带有lacZ + 基因的噬菌体λ基因组,在体外包装入噬菌体颗粒后,可检测到其向lacZ阴性等位基因的变化。我们还证明了利用复制缺陷型腺病毒载体进行基因转移可以在体外实现有效且准确的基因靶向。

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