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公开(公告)号：US20090306184A1
公开(公告)日：2009-12-10
申请号：US12418477
申请日：2009-04-03
IPC分类号： A61K31/7105 , C07H21/02
CPC分类号： C12N15/1131 , A61K47/6911 , C12N2310/14 , C12N2770/24211
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 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 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 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 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 diseases, traits and conditions that are associated with gene expression or activity in a subject or organism.
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2.发明授权RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA) 有权使用短干扰核酸（siNA）的RNA干扰介导的丙型肝炎病毒（HCV）表达的抑制公开(公告)号：US07935812B2
公开(公告)日：2011-05-03
申请号：US12418477
申请日：2009-04-03
CPC分类号： C12N15/1131 , A61K47/6911 , C12N2310/14 , C12N2770/24211
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 hepatitis C virus (HCV) 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 hepatitis C virus (HCV) 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 capable of mediating or that mediate RNA interference (RNAi) against hepatitis C virus (HCV) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules.
摘要翻译： 本发明涉及用于研究，诊断和治疗对丙型肝炎病毒（HCV）基因表达和/或活性的调节作用的性状，疾病和病症的化合物，组合物和方法。 本发明还涉及与涉及丙型肝炎病毒（HCV）基因表达途径或介导的其他细胞过程的基因的表达和/或活性的调节作用的性状，疾病和病症有关的化合物，组合物和方法 维持或发展这些性状，疾病和状况。 具体地，本发明涉及能够介导或介导针对丙型肝炎病毒（HCV）基因表达的RNA干扰（RNAi）的双链核酸分子，包括这种小核酸分子的混合物和这种小的核酸分子的脂质纳米颗粒（LNP）制剂 核酸分子。
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3.发明申请RNA interference mediated inhibition of stromal cell-derived factor-1 (SDF-1) gene expression using short interfering nucleic acid (siNA) 审中-公开使用短干扰核酸（siNA）的RNA干扰介导的基质细胞衍生因子-1（SDF-1）基因表达的抑制公开(公告)号：US20060019917A1
公开(公告)日：2006-01-26
申请号：US11140328
申请日：2005-05-27
CPC分类号： C07H21/02 , A61K48/00 , C12N15/1137 , C12N2310/14 , C12N2310/318 , C12N2310/321 , C12N2310/53 , C12N2310/3521
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-1) 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 SDF-1 gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to 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 or that mediate RNA interference (RNAi) against SDF-1 gene expression. Such small nucleic acid molecules are useful, for example, in providing compositions for treatment of traits, diseases and conditions that can respond to modulation of SDF-1 expression in a subject, such as ocular disease, cancer and proliferative diseases and any other disease, condition, trait or indication that can respond to the level of SDF-1 in a cell or tissue.
摘要翻译： 本发明涉及用于研究，诊断和治疗对基质细胞衍生因子-1（SDF-1）基因表达和/或活性的调节作用的性状，疾病和病症的化合物，组合物和方法。 本发明还涉及与涉及SDF-1基因表达途径的基因的表达和/或活性的调节作用的性状，疾病和病症相关的化合物，组合物和方法，或调节维持的其他细胞过程 发展这种性状，疾病和状况。 具体而言，本发明涉及小核酸分子，如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA） 能够介导或介导针对SDF-1基因表达的RNA干扰（RNAi）的分子。 这样的小核酸分子可用于例如提供用于治疗能够对受试者的SDF-1表达的调节作用的性状，疾病和病症的组合物，例如眼部疾病，癌症和增殖性疾病以及任何其它疾病， 条件，特征或指示，可以响应于细胞或组织中SDF-1的水平。
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4.发明申请RNA interference mediated inhibtion of vitamin D receptor gene expression using short interfering nucleic acid (siNA) 审中-公开使用短干扰核酸（siNA）的RNA干扰介导的维生素D受体基因表达的抑制公开(公告)号：US20050277608A1
公开(公告)日：2005-12-15
申请号：US10898311
申请日：2004-07-23
IPC分类号： A61K48/00 , C07H21/02 , C12N15/113
CPC分类号： C12N15/1138 , C12N2310/14
摘要： This invention relates to compounds, compositions, and methods useful for modulating Vitamin D Receptor (VDR) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of Vitamin D Receptor gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features 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 and methods used to modulate the expression of Vitamin D Receptor.
摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节维生素D受体（VDR）基因表达的化合物，组合物和方法。 本发明还涉及可用于通过使用小核酸分子调节涉及维生素D受体基因表达和/或通过RNA干扰（RNAi）的活性的其它基因的其他基因的表达和活性的方法。 特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA） ）分子和用于调节维生素D受体表达的方法。
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公开(公告)号：US20070173473A1
公开(公告)日：2007-07-26
申请号：US11487788
申请日：2006-07-17
CPC分类号： C12N15/1131 , C12N2310/14
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 Proprotein Convertase Subtilisin Kexin 9 (PCSK9) 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 Proprotein Convertase Subtilisin Kexin 9 (PCSK9) 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 Proprotein Convertase Subtilisin Kexin 9 (PCSK9) 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 PCSK9 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 metabolic diseases traits and conditions, including but not limited to hyperlipidemia, hypercholesterolemia, cardiovascular disease, atherosclerosis, hypertension, diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity and/or other disease states, conditions, or traits associated with PCSK9 gene expression or activity in a subject or organism.
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6.发明申请RNA interference mediated inhibition of CXCR4 gene expression using short interfering nucleic acid (siNA) 审中-公开RNA干扰介导的使用短干扰核酸（siNA）的CXCR4基因表达的抑制公开(公告)号：US20050124569A1
公开(公告)日：2005-06-09
申请号：US10892922
申请日：2004-07-16
IPC分类号： A61K38/00 , A61K45/06 , A61K47/48 , C12N15/11 , C12N15/113 , C12N15/115 , C12N15/87 , A61K48/00 , C07H21/02
CPC分类号： C12N15/87 , A61K38/00 , A61K45/06 , A61K49/0008 , C12N15/111 , C12N15/113 , C12N15/1132 , C12N15/1135 , C12N15/1137 , C12N15/1138 , C12N15/115 , C12N2310/111 , C12N2310/12 , C12N2310/121 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2320/32 , C12N2330/30 , C12Y103/01022 , C12Y104/03003 , C12Y114/19001 , C12Y207/07049 , C12Y207/11001 , C12Y207/11013 , C12Y301/03048 , C12Y604/01002 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating chemokine receptor (CXCR) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of CXCR gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features 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 and methods used to modulate the expression of CXCR genes such as CXCR4 and CXCR7A.
摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节趋化因子受体（CXCR）基因表达的化合物，组合物和方法。 本发明还涉及可用于通过使用小核酸分子调节涉及CXCR基因表达和/或通过RNA干扰（RNAi）的活性的其它基因的其他基因的表达和活性的化合物，组合物和方法。 特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA） ）分子和用于调节CXCR基因如CXCR4和CXCR7A的表达的方法。
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7.发明申请RNA interference mediated inhibition of myostatin gene expression using short interfering nucleic acid (siNA) 审中-公开RNA干扰介导使用短干扰核酸（siNA）抑制肌生成抑制素基因表达公开(公告)号：US20050124566A1
公开(公告)日：2005-06-09
申请号：US10879867
申请日：2004-06-28
IPC分类号： A61K38/00 , A61K47/48 , C07H21/02 , C12N15/11 , C12N15/113 , C12N15/115 , C12N15/87 , A61K48/00 , C12N15/85
CPC分类号： A61K49/0008 , A61K38/00 , A61K48/00 , C07H21/02 , C12N15/111 , C12N15/113 , C12N15/1132 , C12N15/1137 , C12N15/1138 , C12N15/115 , C12N15/87 , C12N2310/111 , C12N2310/12 , C12N2310/121 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2320/32 , C12N2330/30 , C12Y103/01022 , C12Y104/03003 , C12Y114/19001 , C12Y207/07049 , C12Y207/11001 , C12Y207/11013 , C12Y301/03048 , C12Y604/01002 , Y02A50/465 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating myostatin (GDF8) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of myostatin gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features 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 and methods used to modulate the expression of myostatin genes.
摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节肌生成抑制素（GDF8）基因表达的化合物，组合物和方法。 本发明还涉及可用于通过使用小核酸分子调节涉及肌肉生长抑制素基因表达和/或通过RNA干扰（RNAi）的活性的其它基因的其它基因的表达和活性的化合物，组合物和方法。 特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA） ）分子和用于调节肌生成抑制素基因表达的方法。
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公开(公告)号：US20080249040A1
公开(公告)日：2008-10-09
申请号：US11488374
申请日：2006-07-18
CPC分类号： C12N15/1136 , C12N15/113 , C12N2310/14 , C12P19/34
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 Sterol Regulatory Element-Binding Protein 1 (SREBP1) 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 Sterol Regulatory Element-Binding Protein 1 (SREBP1) 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 Sterol Regulatory Element-Binding Protein 1 (SREBP1) 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 SREBP1 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 metabolic diseases traits and conditions, including but not limited to hyperlipidemia, hypercholesterolemia, cardiovascular disease, atherosclerosis, hypertension, diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity and/or other disease states, conditions, or traits associated with SREBP1 gene expression or activity in a subject or organism.
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9.发明申请公开(公告)号：US20080207542A1
公开(公告)日：2008-08-28
申请号：US11510872
申请日：2006-08-25
IPC分类号： A61K31/713
CPC分类号： C12N15/1131 , A61K31/713 , A61K47/6911 , C12N15/111 , C12N2310/14 , C12N2310/318 , C12N2310/51 , C12N2770/24211
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 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 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 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 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 diseases, traits and conditions that are associated with gene expression or activity in a subject or organism.
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10.发明申请RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA) 审中-公开RNA干扰介导的使用短干扰核酸（siNA）的丙型肝炎病毒（HVC）基因表达的抑制公开(公告)号：US20060211642A1
公开(公告)日：2006-09-21
申请号：US11311826
申请日：2005-12-19
CPC分类号： C12N15/1131 , A61K47/6911 , C12N2310/14 , C12N2770/24211
摘要： This invention relates to compounds, compositions, and methods useful for modulating Hepatitis C Virus (HCV) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of HCV gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features 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 and methods used to modulate the expression of HCV genes, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such such small nucleic acid molecules cocktails thereof. The application also relates to methods of treating diseases and conditions associated with HCV gene expression, such as HCV infection, hepatocellular carcinoma, cirrhosis, liver failure, as well as providing dosing regimens and treatment protocols.
摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节丙型肝炎病毒（HCV）基因表达的化合物，组合物和方法。 本发明还涉及可用于通过使用小核酸分子调节参与通过RNA干扰（RNAi）的HCV基因表达和/或活性的途径的其它基因的表达和活性的化合物，组合物和方法。 特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA） ）分子和用于调节HCV基因表达的方法，包括这种小核酸分子的混合物和这种这种小核酸分子的脂质纳米颗粒制剂的混合物。 该应用还涉及治疗与HCV基因表达相关的疾病和病症的方法，例如HCV感染，肝细胞癌，肝硬化，肝功能衰竭，以及提供给药方案和治疗方案。
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