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发明申请
US20080161256A1 RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA) 审中-公开
RNA干扰介导使用短干扰核酸（siNA）抑制基因表达

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.
摘要（中）：

本发明涉及可用于使用短干扰核酸（siNA）分子调节基因表达的化合物，组合物和方法。 特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA） ）分子和用于调节基因表达的方法，例如与受试者或生物中的疾病，病症，性状和病症的维持或发展相关的表达的假基因。 本发明还提供具有降低或减弱的免疫刺激性质的小核酸分子以及用于设计和合成具有改善的毒理学特性同时保留RNAi活性的这种小核酸分子的方法。

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