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RNA INTERFERENCE MEDIATED INHIBITION OF RETINOBLASTOMA (RB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)

机译:使用短干扰核酸(siNA)的RNA干扰介导的视网膜母细胞瘤(RB1)基因表达的抑制。

摘要

This invention relates to compounds, compositions, and methods useful for modulating retinoblastoma (RBl) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of retinoblastoma gene expression and/or activity by RNA interference (RNAi) using small nucleic acid 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 retinoblastoma genes. Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing hearing loss, deafness, tinnitus, motion and balance disorders, cancer and proliferative diseases and any other disease, condition, trait or indication that can respond to the level of retinoblastoma in a cell or tissue, alone or in combination with other treatments or therapies.
机译:本发明涉及用于使用短干扰核酸(siNA)分子调节成视网膜细胞瘤(RB1)基因表达的化合物,组合物和方法。本发明还涉及化合物,组合物和方法,所述化合物,组合物和方法可用于利用小核酸分子通过RNA干扰(RNAi)来调节参与成视网膜细胞瘤基因表达和/或活性的途径的其他基因的表达和活性。特别地,本发明的特征在于小的核酸分子,例如短干扰核酸(siNA),短干扰RNA(siRNA),双链RNA(dsRNA),微小RNA(miRNA)和短发夹RNA(shRNA)。 )分子和调节视网膜母细胞瘤基因表达的方法。这样的小核酸分子可用于例如治疗,预防,抑制或减少听力损失,耳聋,耳鸣,运动和平衡障碍,癌症和增生性疾病以及可对之作出反应的任何其他疾病,病症,性状或适应症单独或与其他疗法或疗法组合使用时,细胞或组织中视网膜母细胞瘤的水平。

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