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1.发明申请RNA interference mediated of inhibition of influenza virus gene expression using short interfering nucleic acid (siNA) 审中-公开使用短干扰核酸（siNA）介导的流感病毒基因表达抑制的RNA干扰公开(公告)号：US20070099858A1
公开(公告)日：2007-05-03
申请号：US11242567
申请日：2005-10-03
申请人： Vasant Jadhav , Howard Robin , James McSwiggen
发明人： Vasant Jadhav , Howard Robin , James McSwiggen
CPC分类号： C12N15/1131 , C12N15/115 , C12N2310/14
摘要： This invention relates to compounds, compositions, and methods useful for modulating influenza virus gene expression using short interfering nucleic acid (siNA) 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 influenza virus genes, such as viral and host genes associated with the maintenance or development of influenza virus infection and related diseases and conditions in a subject or organism.
摘要翻译： 本发明涉及可用于使用短干扰核酸（siNA）分子调节流感病毒基因表达的化合物，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） ）分子和用于调节流感病毒基因表达的方法，例如与维持或发展流感病毒感染以及受试者或生物体中的相关疾病和病症相关的病毒和宿主基因。
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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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3.发明申请RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor 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）抑制血管内皮生长因子和血管内皮生长因子受体基因表达公开(公告)号：US20050233998A1
公开(公告)日：2005-10-20
申请号：US10944611
申请日：2004-09-16
申请人： Vasant Jadhav , Karl Kossen , Shawn Zinnen , Narendra Vaish , James McSwiggen
发明人： Vasant Jadhav , Karl Kossen , Shawn Zinnen , Narendra Vaish , James McSwiggen
CPC分类号： C12N15/113 , C12N2310/14
摘要： This invention relates to compounds, compositions, and methods useful for modulating VEGF and/or VEGFR 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 VEGF and/or VEGFR 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 (mRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of VEGF and/or VEGFR genes.
摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节VEGF和/或VEGFR基因表达的化合物，组合物和方法。 本发明还涉及使用小核酸分子调节参与VEGF和/或VEGFR基因表达和/或活性通过RNA干扰（RNAi）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（mRNA）和短发夹RNA（shRNA） ）分子和用于调节VEGF和/或VEGFR基因表达的方法。
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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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公开(公告)号：US20070049543A1
公开(公告)日：2007-03-01
申请号：US11455205
申请日：2006-06-16
申请人： James McSwiggen , Chandra Vargeese , Vasant Jadhav
发明人： James McSwiggen , Chandra Vargeese , Vasant Jadhav
CPC分类号： C12N15/1137 , C12N15/1131 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/33 , C12N2310/52 , C12Y101/01146 , 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 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-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 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-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 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 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) 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 11 beta-HSD-1 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 diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity, hypercholesterolemia, cardiovascular disease and/or other disease states, conditions, or traits associated with 11 beta-HSD-1 gene expression or activity in a subject or organism.
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6.发明授权公开(公告)号：US07521184B2
公开(公告)日：2009-04-21
申请号：US10568134
申请日：2004-08-17
申请人： Susan Radka , Zinnen Shawn , Vasant Jadhav , James McSwiggen , Narendra Vaish
发明人： Susan Radka , Zinnen Shawn , Vasant Jadhav , James McSwiggen , Narendra Vaish
CPC分类号： C12Q1/6813 , Y10T436/143333 , C12Q2545/114
摘要： The present invention concerns processes for the detection and quantitation of nucleic acid molecules, polynucleotides, and/or oligonucleotides in a sample using hybridizationdetection assays, antibody-mediated recognition assays, nucleic acid sensor molecules, chromatographic assays, and/or electrophoresis assays. The present invention specifically concerns processes for the detection and quantitation of double stranded nucleic acid molecules, polynucleotides, and/or oligonucleotides in a sample using hybridization-detection assays. The nucleic acid molecules, polynucleotides, and/or oligonucleotides can include molecules that mediate RNA interference, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules. The nucleic acid molecules, polynucleotides, and/or oligonucleotides can include nucleic acid aptamers, enzymatic nucleic acid molecules, decoys, antisense, 2′,5′-oligoadenylate molecules, triplex forming oligonucleotides or any other nucleic acid molecule of interest. The present invention also concerns kits that allow for the detection and quantitation of nucleic acid molecules, polynucleotides, and/or oligonucleotides in a sample.
摘要翻译： 本发明涉及使用杂交检测测定，抗体介导的识别测定，核酸传感器分子，色谱测定和/或电泳测定来检测和定量样品中核酸分子，多核苷酸和/或寡核苷酸的方法。 本发明具体涉及使用杂交检测试验检测和定量样品中双链核酸分子，多核苷酸和/或寡核苷酸的方法。 核酸分子，多核苷酸和/或寡核苷酸可以包括介导RNA干扰的分子，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。 核酸分子，多核苷酸和/或寡核苷酸可以包括核酸适配体，酶核酸分子，诱饵，反义，2'，5'-寡聚腺苷酸分子，形成三链体的寡核苷酸或任何其它感兴趣的核酸分子。 本发明还涉及允许在样品中检测和定量核酸分子，多核苷酸和/或寡核苷酸的试剂盒。
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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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8.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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9.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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10.发明申请Rna interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (sina) 审中-公开Rna干扰介导使用化学修饰的短干扰核酸（新生）的基因表达抑制公开(公告)号：US20070032441A1
公开(公告)日：2007-02-08
申请号：US10557542
申请日：2004-05-24
CPC分类号： C12N15/87 , A61K38/00 , C07H21/02 , C12N15/111 , C12N15/113 , C12N15/1131 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/332 , C12N2310/344 , C12N2310/346 , C12N2310/3515 , C12N2320/32 , C12N2320/51 , C12Y103/01022 , C12Y104/03003 , C12Y114/19001 , C12Y207/07049 , C12Y207/11001 , C12Y207/11013 , C12Y301/03048 , C12Y604/01002 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, cosmetic, cosmeceutical, prophylactic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified 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 target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease (e.g., cancer, proliferative, inflammatory, metabolic, autoimmune, neurologic, ocular diseases), condition, trait (e.g., hair growth and removal), genotype or phenotype that responds to modulation of gene expression or activity in a cell, tissue, or organism. Such small nucleic acid molecules can be administered systemically, locally, or topically.
摘要翻译： 本发明涉及可用于调节多种应用中的基因表达的方法和试剂，包括用于治疗，化妆品，药妆，预防，诊断，靶标验证和基因组发现应用。 具体地说，本发明涉及合成的化学修饰的小核酸分子，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）分子，其能够介导针对靶核酸序列的RNA干扰（RNAi）。 小核酸分子可用于治疗任何疾病（例如癌症，增殖性，炎症，代谢，自身免疫，神经系统，眼部疾病），病症，性状（例如毛发生长和去除），基因型或表型， 调节细胞，组织或生物体中的基因表达或活性。 这样的小核酸分子可以全身，局部或局部施用。
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