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    • 10. 发明申请
    • COMPOUNDS, COMPOSITIONS, AND METHODS USEFUL FOR MODULATING THE EXPRESSION OF HISTONE DEACETYLASE (HDAC) GENE EXPRESSION AND/OR ACTIVITY
    • 化合物,组合物和用于调节表达甲基脱乙酰酶(HDAC)基因表达和/或活性的表达的方法
    • WO2009156853A2
    • 2009-12-30
    • PCT/IB2009/006274
    • 2009-06-26
    • ISTITUTO DI RICERCHE DI BIOLOGIA MOLECOLARE P. ANGELETTI SPASTEINKUHLER, ChristianFILOCAMO, GessicaCADOT, Bruno
    • STEINKUHLER, ChristianFILOCAMO, GessicaCADOT, Bruno
    • C12N15/11A61K31/713A61P35/00C07H21/00
    • C12N15/1137C12N2310/14C12N2310/317C12N2310/321C12N2310/322C12Y305/01098C12N2310/3521
    • 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 histone deacetylase, particularly histone deacetylase 4 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 HDAC4 genes or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Exemplarity diseases or traits associated with HDAC4 expression or activity include cellular proliferative disorders such as oncogenic disorders exemplified by cancer and cardiovascular diseases. 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 HDAC4 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 endogenou
    • 本发明涉及用于研究,诊断和治疗响应于组蛋白脱乙酰酶的调节,特别是组蛋白脱乙酰酶4表达和/或活性的性状,疾病和病症的化合物,组合物和方法。 本发明还涉及与调节HDAC4基因的表达和/或活性调节的性状,疾病和病症相关的化合物,组合物和方法,所述HDAC4基因或其它细胞过程介导这些性状,疾病的维持或发展, 条件。 与HDAC4表达或活性相关的示例性疾病或性状包括细胞增殖性疾病,例如癌症和心血管疾病所示的致癌性疾病。 具体地说,本发明涉及包括小核酸分子如短干扰核酸(siNA),短干扰RNA(siRNA),双链RNA(dsRNA),微RNA(miRNA)和双链RNA 能够介导针对HDAC4基因表达的RNA干扰(RNAi)的短发夹RNA(shRNA)分子,包括这种小核酸分子的这种小核酸分子的混合物和这种小核酸分子的脂质纳米颗粒(LNP)制剂。 本发明还涉及可以抑制内源性RNA分子如内源性微RNA(miRNA)(例如miRNA抑制剂)或内源性内源性RNA分子的功能的小核酸分子,例如siNA,siRNA和其他