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    • 3. 发明申请
    • NOVEL SIRNA GENE LIBRARIES AND METHODS FOR THEIR PRODUCTION AND USE
    • 新的SIRNA基因库及其生产和使用方法
    • WO2004009794A2
    • 2004-01-29
    • PCT/US0323157
    • 2003-07-23
    • IMMUSOL INCLI HENRYCHATTERTON JON EKE NINGWONG-STAAL FLOSSIE
    • LI HENRYCHATTERTON JON EKE NINGWONG-STAAL FLOSSIE
    • C12N15/09C12N15/10C12N15/11C12N
    • C12N15/111C12N15/1075C12N2310/111C12N2310/14C12N2310/53C12N2330/31
    • The present invention relates to methods and compositions for the elucidation of gene function and the identification of novel genes. Specifically, the present invention relates to methods and compositions for improved functional genomic screening, functional inactivation of specific essential or non-essential genes, and identification of genes that are modulated in response to specific stimuli or encode recognizable phenotypic traits. In particular, the compositions of the present invention include, but are not limited to, expression cassettes comprising a novel dual promoter transcription system, that utilizes modified promoters, preferably containing complementary termination sequences, positioned across a coding sequence and in opposite orientation to each other. In addition, the present invention includes libraries comprising the expression cassettes of the invention, including vectors for transforming cells, such as replication-deficient retroviral vectors. The invention also includes methods for the production and screening of dsRNA/siRNA libraries, as well as therapeutic uses for the siRNAs expressed in accordance with the invention.
    • 本发明涉及阐明基因功能和鉴定新基因的方法和组合物。 具体而言,本发明涉及用于改进功能基因组筛选,特异性必需或非必需基因的功能失活以及根据特定刺激或编码可识别的表型性状调节的基因的鉴定的方法和组合物。 特别地,本发明的组合物包括但不限于包含新颖的双重启动子转录系统的表达盒,其利用修饰的启动子,优选含有互补终止序列,跨越编码序列并且彼此相反取向 。 此外,本发明包括包含本发明的表达盒的文库,包括用于转化细胞的载体,例如复制缺陷型逆转录病毒载体。 本发明还包括用于生产和筛选dsRNA / siRNA文库的方法以及根据本发明表达的siRNA的治疗用途。