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NUCLEIC ACID COMPOUND FOR SUPPRESSING EXPRESSION OF AKT GENE AND USE THEREOF

机译:用于抑制AKT基因表达的核酸化合物及其用途

摘要

The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing AKT gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to an AKT mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside replaced with a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of an AKT gene in a cell or in a subject to treat an AKT-related disease.
机译:本公开提供了能够降低或沉默AKT基因表达的双链核糖核酸分子(mdRNA)。本公开的mdRNA包含至少三条链,其结合以形成至少两个被切口或缺口隔开的非重叠双链区,其中一条链与AKT mRNA互补。另外,所述铁双链体可具有至少一个被5-甲基尿苷取代的尿苷,被锁核酸取代的核苷,或任选地其他修饰,及其任意组合。还提供了降低细胞或受试者中AKT基因表达以治疗AKT相关疾病的方法。

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