申请人:Marina Biotech, Inc.
公开号:EP2471920A2
公开(公告)日:2012-07-04
The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing WNT1, WNT2, or WNT3A 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 a WNT1, WNT2, or WNT3A mRNA. In addition, the meroduplex may have at least one uridine is a 5-methyluridine, a nucleoside is a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a WNT1, WNT2, or WNT3A gene in a cell or in a subject to treat a WNT1, WNT2, or WNT3A-related disease.
本公开提供了能够降低或沉默 WNT1、WNT2 或 WNT3A 基因表达的经双链核糖核酸分子(mdRNA)。本公开的 mdRNA 包括至少三条链,它们结合形成至少两个非重叠的双链区域,由缺口或间隙隔开,其中一条链与 WNT1、WNT2 或 WNT3A mRNA 互补。此外,经双链可具有至少一个尿苷,即 5-甲基尿苷、一个核苷,即锁定核酸,或可选的其他修饰,以及它们的任意组合。还提供了降低细胞或受试者中 WNT1、WNT2 或 WNT3A 基因表达以治疗 WNT1、WNT2 或 WNT3A 相关疾病的方法。