发明名称 RNA INTERFERENCE MEDIATED INHIBITION OF CATENIN (CADHERIN-ASSOCIATED PROTEIN), BETA 1 (CTNNB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
摘要 The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of CTNNB1 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including 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 that are capable of mediating or that medium RNA interference (RNAi) against CTNNB1 gene expression.
申请公布号 US2017029824(A1) 申请公布日期 2017.02.02
申请号 US201615224822 申请日期 2016.08.01
申请人 Sirna Therapeutics, Inc. 发明人 Brown Duncan;Cunningham James J.;Gindy Marian;Pickering Victoria;Stanton Matthew G.;Stirdivant Steven M.;Strapps Walter R.
分类号 C12N15/113 主分类号 C12N15/113
代理机构 代理人
主权项 1. An isolated double-stranded short interfering nucleic acid (siNA) molecule that inhibits the expression of cadherin-associated protein, beta 1 (CTNNB1), wherein (a) the siNA comprises a sense strand and an antisense strand; (b) each strand is independently 15 to 30 nucleotides in length; and (c) at least one strand comprises at least a 15 nucleotide sequence of SEQ ID NO:5.
地址 Cambridge MA US