发明公开
EP1891217A2 RNA INTERFERENCE MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR-1 (SDF-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
审中-公开
通过RNA干扰介导的来源的间质的基因的表达抑制因子1使用短干扰核酸（SDF-1）（新浪网）
- 专利标题： RNA INTERFERENCE MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR-1 (SDF-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
- 专利标题（中）： 通过RNA干扰介导的来源的间质的基因的表达抑制因子1使用短干扰核酸（SDF-1）（新浪网）
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申请号： EP06784507.3申请日： 2006-05-26
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公开(公告)号： EP1891217A2公开(公告)日： 2008-02-27
- 发明人: GUERCIOLINI, Roberto , MCSWIGGEN, James , JADHAV, Vasant , VARGEESE, Chandra
- 申请人： Sirna Therapeutics, Inc.
- 申请人地址： Suite 6504 185 Berry Street San Francisco, CA 94107 US
- 专利权人： Sirna Therapeutics, Inc.
- 当前专利权人： Sirna Therapeutics, Inc.
- 当前专利权人地址： Suite 6504 185 Berry Street San Francisco, CA 94107 US
- 代理机构： Hussain, Deeba
- 优先权： US140328 20050527; US703946P 20050729; US205646 20050817; US234730 20050923; US737024P 20051115; US299254 20051208; US353630 20060214; US369108 20060306
- 国际公布： WO2006128141 20061130
- 主分类号： C12N15/11
- IPC分类号： C12N15/11 ; C07K14/52
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 stromal cell-derived factor- 1 (SDF-I) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in stromal cell-derived factor- 1 (SDF-I) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. 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 capable of mediating RNA interference (RNAi) against stromal cell- derived factor- 1 (SDF-I) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate SDF-I gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce angiogenesis related diseases traits and conditions, including but not limited to ocular disease, cancer and proliferative diseases, traits, and conditions, and/or other disease states, conditions, or traits associated with SDF-I gene expression or activity in a subject or organism.
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