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AAV-ITR基因表达微载体在小鼠体内表达

Gene Expression of AAV-ITR Mini Vector in Mice vivo
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摘要 旨在比较AAV-ITR基因表达微载体及其单体在小鼠不同部位的表达差异,将相同拷贝数的AAV-ITR基因表达微载体和p UC57-minivector-EGFP分别转入小鼠脑组织,另外将相同拷贝数的AAV-ITR基因表达微载体单体,AAV-ITR基因表达微载体和p UC57-minivector-EGFP转入小鼠骨骼肌中,比较不同时期组织中的残余分子数量。荧光定量PCR、显微荧光观察以及荧光平均光密度和荧光灰度值分析表明,相同拷贝数的AAV-ITR基因表达微载体单体比AAV-ITR基因表达微载体和p UC57-minivectorEGFP具有更高的转染效率,其稳定性也优于AAV-ITR基因表达微载体。结果显示,AAV-ITR基因表达微载体单体在动物体内具有高效表达安全稳定的特点,有望成为基因治疗中一种安全可靠,稳定高效的新型载体。 In order to compare the expression difference between AAV-ITR expression mini vector and its monomers in different parts of mice,the same copy number of AAV-ITR mini vector and p UC57-minivector-EGFP were separately transferred into the brain of mice. In addition,the same copy number of monomers of AAV-ITR mini vector and p UC57-minivector-EGFP were transferred into mouse skeletal muscle,and the number of residual molecules were compared in the different tissues at different periods. Analysis by florescence quantitative PCR,microscope observation,average optical density,and gray value showed that monomers of AAV-ITR mini vector in the same copy number had higher transfection efficiency and more stable than AAV-ITR mini vector and p UC57-minivector-EGFP. The comprehensive results suggest that the monomers of AAV-ITR mini vector present the feature of efficient,secure and stable expression,which may be expected to become a novel,secure,reliable,stable,and efficient vector in gene therapy.
出处 《生物技术通报》 CAS CSCD 北大核心 2016年第5期246-254,共9页 Biotechnology Bulletin
基金 国家自然科学基金项目(81371670) 江苏省自然科学基金项目(BK20140381)
关键词 基因治疗 基因表达微载体 腺相关病毒(AAV) RT-PCR 组织切片 gene therapy gene expression mini vector adeno-associated virus(AAV) RT-PCR skeletal muscle section
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