发明名称 BLOCKING OF GENE EXPRESSION IN EUKARYOTIC CELLS
摘要 The present invention provides methods for designing a sequence for efficient short interference RNA molecules. In particular, the present invention defines a universal target for siRNA derived from a poly A sequence, optionally in conjunction with unique sequences for gene silencing and inhibition of viral replication in a eukaryotic host cell. The present invention further provides methods for the treatment and prevention of diseases and disorders by silencing a gene of a virus, an oncogene, genes encoding transcription factors and many other diseases related genes. The present invention describes antisense nucleic acids compositions comprising sequences complementary to a target nucleic acid. The antisense sequences are designed to hybridize to complementary nucleic acid target regions in a target RNA, and inhibit translation, processing, transport, or binding by proteins or riboproteins. Target regions include, and are limited to a poly-A tail, and exclude, AUG, 5' non-translated sequences, translation initiation factor binding sites, ribosome subunit binding sites, Shine Dalgarno sequence, 3' nontranslated sequences, poly- addition site, 3' cleavage site, coding region, intron, intron branch site, intron/exon junction, and splice sequence.
申请公布号 WO2008039937(A3) 申请公布日期 2008.08.07
申请号 WO2007US79763 申请日期 2007.09.27
申请人 NOVARX;FAKHRAI, HABIB;FAKHRAI, FARIDEH 发明人 FAKHRAI, HABIB;FAKHRAI, FARIDEH
分类号 A61K31/7088;A61K31/713;A61P35/00;C12N15/11;C12N15/113 主分类号 A61K31/7088
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