NUCLEIC ACID COMPOUNDS FOR INHIBITING CTNNB1 GENE EXPRESSION AND USES THEREOF
摘要
The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing CTNNB1 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 CTNNB1 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 a CTNNBl gene in a cell or in a subject to treat a CTNNB1 -related disease.
申请公布号
WO2008109460(A4)
申请公布日期
2009.03.12
申请号
WO2008US55532
申请日期
2008.02.29
申请人
NASTECH PHARMACEUTICAL COMPANY INC.;QUAY, STEVEN, C.;MCSWIGGEN, JAMES;VAISH, NARENDRA, K.;AHMADIAN, MOHAMMAD
发明人
QUAY, STEVEN, C.;MCSWIGGEN, JAMES;VAISH, NARENDRA, K.;AHMADIAN, MOHAMMAD