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    • 41. 发明申请
    • RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS
    • RNA干扰介导的HEPATITIS C病毒的抑制
    • WO2007076328A3
    • 2008-08-14
    • PCT/US2006062252
    • 2006-12-18
    • SIRNA THERAPEUTICS INCMCSWIGGEN JAMESMORRISSEY DAVIDGUERCIOLINI ROBERTOVARGEESE CHANDRAJADHAV VASANT
    • MCSWIGGEN JAMESMORRISSEY DAVIDGUERCIOLINI ROBERTOVARGEESE CHANDRAJADHAV VASANT
    • A61K9/127A61K31/713C12N15/11C12N15/113
    • C12N15/1131A61K31/713A61K47/6911C12N15/111C12N2310/14C12N2310/318C12N2310/51C12N2770/24211
    • 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 nanoparticlc (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.
    • 本发明涉及用于研究,诊断和治疗对基因表达和/或活性调节作出反应的性状,疾病和病症的化合物,组合物和方法。 本发明还涉及与涉及参与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,或调节其维持或发展的其他细胞过程 性状,疾病和状况。 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微RNA(miRNA)(例如miRNA抑制剂)或内源性短干扰RNA(例如miRNA抑制剂)的内源性RNA分子的功能的小核酸分子,例如siNA,siRNA等, siRNA)(例如siRNA抑制剂)或可以通过干扰与这些内源性RNA(例如RISC)相关的内源性RNA或蛋白质的调节功能来调节基因表达来调节基因表达(例如,RISC抑制剂) ,包括这种小核酸分子的鸡尾酒和这种小核酸分子的脂质纳米颗粒(LNP)制剂。 这样的小核酸分子并且可用于例如提供组合物以预防,抑制或减少与受试者或生物体中的基因表达或活性相关的疾病,性状和病症。
    • 42. 发明申请
    • RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
    • RNA干扰介导的抑制丙糖转化酶底物KEXIN 9(PCSK9)使用短暂干扰核酸(SINA)的基因表达
    • WO2008011431A3
    • 2008-07-17
    • PCT/US2007073723
    • 2007-07-17
    • SIRNA THERAPEUTICS INCMCSWIGGEN JAMESJADHAV VASANTVAISH NARENDRA KGUERCIOLINI ROBERTOVARGEESE CHANDRA
    • MCSWIGGEN JAMESJADHAV VASANTVAISH NARENDRA KGUERCIOLINI ROBERTOVARGEESE CHANDRA
    • A61K48/00C12N15/113
    • C12N15/1137C12N2310/14C12N2310/321C12N2310/322C12N2310/3521
    • 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 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 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 that are associated with gene expression or activity in a subject or organism.
    • 本发明涉及用于研究,诊断和治疗对Proprotein转化酶枯草杆菌蛋白酶Kexin 9(PCSK9)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Proprotein转化酶枯草杆菌蛋白酶Kexin 9(PCSK9)基因表达途径或其他细胞过程的基因的表达和/或活性的调节相关的性状,疾病和病症的化合物,组合物和方法 调节这种性状,疾病和状况的维持或发展。 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Proprotein转化酶枯草杆菌蛋白酶Kexin 9(PCSK9)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和脂质纳米颗粒(LNP)制剂的混合物。 本发明还涉及可以抑制内源性微RNA(miRNA)(例如miRNA抑制剂)或内源性短干扰RNA(例如miRNA抑制剂)的内源性RNA分子的功能的小核酸分子,例如siNA,siRNA等, siRNA)(例如siRNA抑制剂)或可以通过干扰与这些内源性RNA(例如RISC)相关的内源性RNA或蛋白质的调节功能来调节基因表达来调节基因表达(例如,RISC抑制剂) ,包括这种小核酸分子的鸡尾酒和这种小核酸分子的脂质纳米颗粒(LNP)制剂。 这样的小核酸分子并且可用于例如提供预防,抑制或降低代谢疾病,性状和病症的组合物,包括但不限于高脂血症,高胆固醇血症,心血管疾病,动脉粥样硬化,高血压,糖尿病(例如,类型 I和/或II型糖尿病),胰岛素抵抗,肥胖症和/或与受试者或生物体中的基因表达或活性相关的其它疾病状态,病症或性状。
    • 43. 发明申请
    • RNA INTERFERENCE MEDIATED INHIBITION OF 11 BETA-HYDROXYSTEROID DEHYDROGENASE-1 (11 BETA-HSD-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
    • RNA干扰介导的使用短暂干扰核酸(SINA)的11个羟基脱氢酶脱氢酶-1(11-BET-HSD-1)基因表达的抑制
    • WO2007147143A2
    • 2007-12-21
    • PCT/US2007071388
    • 2007-06-15
    • SIRNA THERAPEUTICS INCMCSWIGGEN JAMESVARGEESE CHANDRAJADHAV VASANT
    • MCSWIGGEN JAMESVARGEESE CHANDRAJADHAV VASANT
    • C12N15/113
    • C12N15/1137C12N15/1131C12N2310/14C12N2310/315C12N2310/317C12N2310/321C12N2310/322C12N2310/33C12N2310/52C12Y101/01146C12N2310/3521
    • 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 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.
    • 本发明涉及用于研究,诊断和治疗对11β-羟类固醇脱氢酶-1(11β-HSD-1)基因表达的调节作用的性状,疾病和病症的化合物,组合物和方法和/ 或活动。 本发明还涉及与涉及11β-羟类固醇脱氢酶-1(11β-HSD-1)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, 基因表达途径或介导维持或发展这些性状,疾病和病症的其他细胞过程。 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11β-羟类固醇脱氢酶-1(11β-HSD-1)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混合物和脂质纳米颗粒(LNP)制剂 这样的小核酸分子。 本发明还涉及可以抑制内源性微RNA(miRNA)(例如miRNA抑制剂)或内源性短干扰RNA(例如miRNA抑制剂)的内源性RNA分子的功能的小核酸分子,例如siNA,siRNA等, siRNA)(例如siRNA抑制剂)或可以抑制RISC功能(如RISC抑制剂)调节11β-HSD-1基因表达的方法是通过干扰与内源性RNA相关的内源性RNA或蛋白质的调控功能 RNA(例如RISC),包括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。 这样的小核酸分子并且可用于例如提供预防,抑制或降低代谢性疾病性状和病症的组合物,包括但不限于糖尿病(例如I型和/或II型糖尿病),胰岛素抵抗, 肥胖症,高胆固醇血症,心血管疾病和/或与受试者或生物中的11β-HSD-1基因表达或活性相关的其他疾病状态,病症或性状。
    • 48. 发明申请
    • DETECTION AND QUANTITATION OF NUCLEIC ACID MOLECULES IN BIOLOGICAL SAMPLES
    • 生物样品中核酸分子的检测和定量
    • WO2005021800A8
    • 2005-06-23
    • PCT/US2004026857
    • 2004-08-17
    • SIRNA THERAPEUTICS INCRADKA SUSANZINNEN SHAWNJADHAV VASANTMCSWIGGEN JAMESVAISH NARENDRA K
    • RADKA SUSANZINNEN SHAWNJADHAV VASANTMCSWIGGEN JAMESVAISH NARENDRA K
    • C12Q1/68
    • C12Q1/6813Y10T436/143333C12Q2545/114
    • 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.
    • 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的核酸分子,多核苷酸和/或寡核苷酸的方法。 本发明具体涉及使用杂交 - 检测测定法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体,酶促核酸分子,诱饵,反义,2',5'-寡聚腺苷酸分子,三链形成寡核苷酸或任何其他感兴趣的核酸分子。 本发明还涉及允许检测和定量样品中的核酸分子,多核苷酸和/或寡核苷酸的试剂盒。
    • 49. 发明申请
    • DETECTION AND QUANTITATION OF NUCLEIC ACID MOLECULES IN BIOLOGICAL SAMPLES
    • 核酸分子在生物样品中的检测和定量
    • WO2005021800A3
    • 2005-04-21
    • PCT/US2004026857
    • 2004-08-17
    • SIRNA THERAPEUTICS INCRADKA SUSANZINNEN SHAWNJADHAV VASANTMCSWIGGEN JAMESVAISH NARENDRA K
    • RADKA SUSANZINNEN SHAWNJADHAV VASANTMCSWIGGEN JAMESVAISH NARENDRA K
    • C12Q1/68
    • C12Q1/6813Y10T436/143333C12Q2545/114
    • 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.
    • 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。