发明名称 RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
摘要 This invention relates to compounds, compositions, and methods useful for modulating gene expression using short interfering nucleic acid (siNA) molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of genes, such as expressed pseudogenes associated with the maintenance or development of diseases, disorders, traits, and conditions in a subject or organism. The invention also provides small nucleic acid molecules with reduced or attenuated immunostimulatory properties and methods for designing and synthesizing such small nucleic acid molecules having improved toxicologic properties while retaining RNAi activity.
申请公布号 US2008161256(A1) 申请公布日期 2008.07.03
申请号 US20050205646 申请日期 2005.08.17
申请人 发明人 MORRISEY DAVID;MCSWIGGEN JAMES;BEIGELMAN LEONID
分类号 A61K31/70;A61P43/00 主分类号 A61K31/70
代理机构 代理人
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