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    • 5. 发明申请
    • LONG-TERM IN VIVO TRANSGENE EXPRESSION
    • VIVO TRANSGENE EXPRESSION的长期使用
    • WO2009036280A1
    • 2009-03-19
    • PCT/US2008/076177
    • 2008-09-12
    • COPERNICUS THERAPEUTICS, INC.COOPER, Mark J.PADEGIMAS, Linas
    • COOPER, Mark J.PADEGIMAS, Linas
    • C12N15/85
    • C07K14/4712A61K48/0066C12N15/85C12N2799/04C12N2830/42C12N2830/50C12N2830/85C12N2840/00C12N2840/85
    • Efficient and prolonged hCFTR expression is one of the major obstacles for cystic fibrosis lung therapy. hCFTR mRNA expression levels depend on eukaryotic expression cassette components, prokaryotic backbone elements, and the gene transfer method may also influence transcriptional silencing mechanisms. A codon-optimized and CpG-reduced human CFTR gene (CO-CFTR) was made. Various vector modifications were tested to facilitate extended duration of CO-CFTR expression. Insertion of an extended 3'BGH transcribed sequence (712 bp) in an inverted orientation produced prolonged expression of CO-CFTR expression at biologically relevant levels. Further studies revealed that prolonged CO-CFTR expression is dependant on the orientation of the extended BGH 3' BGH transcribed sequence and its transcription, is not specific to the UbC promoter, and is less dependent on other vector backbone elements.
    • 有效和长期的hCFTR表达是囊性纤维化肺部治疗的主要障碍之一。 hCFTR mRNA表达水平取决于真核表达盒组分,原核骨架元素,基因转移方法也可能影响转录沉默机制。 制备了密码子优化和CpG还原的人CFTR基因(CO-CFTR)。 测试各种载体修饰以促进CO-CFTR表达的延长的持续时间。 以倒置方向插入延伸的3'BGH转录序列(712bp)产生CO-CFTR表达在生物相关水平上的延长表达。 进一步的研究表明,延长的CO-CFTR表达依赖于扩展的BGH 3'BGH转录序列的方向及其转录,对UbC启动子不是特异性的,并且较少依赖于其它载体骨架元素。