专利快速检索-快速检索全球专利，免费商用专利数据库-IPRDB

1. 发明申请

WO2005021800A8 DETECTION AND QUANTITATION OF NUCLEIC ACID MOLECULES IN BIOLOGICAL SAMPLES 审中-公开
标题翻译：生物样品中核酸分子的检测和定量
公开(公告)号：WO2005021800A8
公开(公告)日：2005-06-23
申请号：PCT/US2004026857
申请日：2004-08-17
申请人： SIRNA THERAPEUTICS INC , RADKA SUSAN , ZINNEN SHAWN , JADHAV VASANT , MCSWIGGEN JAMES , VAISH NARENDRA K
发明人： RADKA SUSAN , ZINNEN SHAWN , JADHAV VASANT , MCSWIGGEN JAMES , VAISH NARENDRA K
IPC分类号： C12Q1/68
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。

2. 发明申请

WO2005021800A3 DETECTION AND QUANTITATION OF NUCLEIC ACID MOLECULES IN BIOLOGICAL SAMPLES 审中-公开
标题翻译：核酸分子在生物样品中的检测和定量
公开(公告)号：WO2005021800A3
公开(公告)日：2005-04-21
申请号：PCT/US2004026857
申请日：2004-08-17
申请人： SIRNA THERAPEUTICS INC , RADKA SUSAN , ZINNEN SHAWN , JADHAV VASANT , MCSWIGGEN JAMES , VAISH NARENDRA K
发明人： RADKA SUSAN , ZINNEN SHAWN , JADHAV VASANT , MCSWIGGEN JAMES , VAISH NARENDRA K
IPC分类号： C12Q1/68
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。

3. 发明申请

WO2008011431A3 RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： 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
公开(公告)号：WO2008011431A3
公开(公告)日：2008-07-17
申请号：PCT/US2007073723
申请日：2007-07-17
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , JADHAV VASANT , VAISH NARENDRA K , GUERCIOLINI ROBERTO , VARGEESE CHANDRA
发明人： MCSWIGGEN JAMES , JADHAV VASANT , VAISH NARENDRA K , GUERCIOLINI ROBERTO , VARGEESE CHANDRA
IPC分类号： A61K48/00 , C12N15/113
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/321 , C12N2310/322 , 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 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.
摘要翻译：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。

4. 发明申请

WO2005019453A3 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的化学修饰短介导核酸（siNA）抑制基因表达
公开(公告)号：WO2005019453A3
公开(公告)日：2005-06-23
申请号：PCT/US2004016390
申请日：2004-05-24
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , MORRISSEY DAVID , ZINNEN SHAWN , JADHAV VASANT , VAISH NARENDRA
发明人： MCSWIGGEN JAMES , MORRISSEY DAVID , ZINNEN SHAWN , JADHAV VASANT , VAISH NARENDRA
IPC分类号： A01N43/04 , A61K48/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12P19/34 , C12N15/11
CPC分类号： C12N15/113 , C12N2310/14
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, nuerologic, 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：本发明涉及用于调节各种应用中的基因表达的方法和试剂，包括用于治疗，美容，药妆，预防，诊断，靶标验证和基因组发现应用。具体而言，本发明涉及合成的化学修饰的小核酸分子，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，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，炎性，代谢性，自身免疫性，神经性，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。

5. 发明申请

WO2005028650A2 RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS (HCV) EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制HEPATITIS C病毒（HCV）表达使用短暂的干扰核酸（siNA）
公开(公告)号：WO2005028650A2
公开(公告)日：2005-03-31
申请号：PCT/US2004031012
申请日：2004-09-15
申请人： SIRNA THERAPEUTICS INC , JADHAV VASANT , KOSSEN KARL , ZINNEN SHAWN , VAISH NARENDRA , MCSWIGGEN JAMES
发明人： JADHAV VASANT , KOSSEN KARL , ZINNEN SHAWN , VAISH NARENDRA , MCSWIGGEN JAMES
IPC分类号： A61K38/00 , C12N15/11 , C12N15/113 , C12N15/87
CPC分类号： C12N15/113 , A61K38/00 , C12N15/111 , C12N15/1131 , C12N15/1137 , C12N15/1138 , C12N15/87 , C12N2310/111 , C12N2310/12 , C12N2310/121 , C12N2310/14 , C12N2310/315 , C12N2310/316 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/333 , C12N2310/346 , C12N2310/53 , C12N2330/30 , C12Y103/01022 , C12N2310/3521 , C12N2310/3533
摘要： This invention relates to compounds, compositions, and methods useful for modulating HCVgene 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.
摘要翻译：本发明涉及可用于使用短干扰核酸（siNA）分子调节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。

6. 发明申请

WO2007120527A2 RNA INTERFERENCE MEDIATED INHIBITION OF RESPIRATORY SYNCYTIAL VIRUS (RSV) EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的使用短暂干扰核酸（SINA）的呼吸道同源性病毒（RSV）表达的抑制
公开(公告)号：WO2007120527A2
公开(公告)日：2007-10-25
申请号：PCT/US2007008186
申请日：2007-03-30
申请人： SIRNA THERAPEUTICS INC , VAISH NARENDRA K , VARGEESE CHANDRA , MCSWIGGEN JAMES
发明人： VAISH NARENDRA K , VARGEESE CHANDRA , MCSWIGGEN JAMES
IPC分类号： C12N15/113
CPC分类号： C12N15/1131 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/346 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating sespiratory syncytial virus (RSV) 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 RSV 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 RSV 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 RSV gene expression, such as RSV infection, respiratory failure, bronchiolitis and pneumonia, 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）分子调节呼吸道合胞病毒（RSV）基因表达的化合物，组合物和方法。本发明还涉及用于通过使用小核酸分子调节参与RSV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））分子和方法用于调节RSV基因的表达，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RSV基因表达相关的疾病和病症的方法，例如RSV感染，呼吸衰竭，细支气管炎和肺炎，以及提供给药方案和治疗方案。

7. 发明申请

WO2008011467A3 RNA INTERFERENCE MEDIATED INHIBITION OF STEROL REGULATORY ELEMENT-BINDING PROTEIN 1 (SREBP1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译：利用短干扰核酸（SINA）RNA干扰介导的甾醇调节元件结合蛋白1（SREBP1）基因表达抑制
公开(公告)号：WO2008011467A3
公开(公告)日：2008-08-07
申请号：PCT/US2007073792
申请日：2007-07-18
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , VASANT JADHAV , VAISH NARENDRA K , GUERCIOLINI ROBERTO , VARGEESE CHANDRA
发明人： MCSWIGGEN JAMES , VASANT JADHAV , VAISH NARENDRA K , GUERCIOLINI ROBERTO , VARGEESE CHANDRA
IPC分类号： C12N15/113
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.
摘要翻译：本发明涉及用于研究，诊断和治疗对固醇调节元件结合蛋白1（SREBP1）基因表达和/或活性的调节作出反应的性状，疾病和病症的化合物，组合物和方法。本发明还涉及涉及对Sterol调控元件结合蛋白1（SREBP1）基因表达途径或其他基因参与的基因的表达和/或活性的调节作出响应的性状，疾病和病症的化合物，组合物和方法调解这些特征，疾病和病症的维持或发展的细胞过程。具体而言，本发明涉及双链核酸分子，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）和能够介导针对固醇调节元件结合蛋白1（SREBP1）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鸡尾酒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RNA分子（例如内源性微RNA（miRNA）（例如，miRNA抑制剂）或内源性短干扰RNA（例如，miRNA））的功能的小核酸分子，例如siNA，siRNA， siRNA）（例如siRNA抑制剂）或可以抑制RISC功能（例如RISC抑制剂），通过干扰与内源RNA（例如RISC）相关的内源RNA或蛋白质的调节功能来调节SREBP1基因表达），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。这样的小核酸分子可用于例如提供组合物以预防，抑制或减少代谢疾病特征和病症，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I型和/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SREBP1基因表达或活性相关的其他疾病状态，病症或性状。

8. 发明申请

WO2006128141A3 RNA INTERFERENCE MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR-1 (SDF-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的使用短暂干扰核酸（SINA）的细胞衍生因子-1（SDF-1）基因表达的抑制作用
公开(公告)号：WO2006128141A3
公开(公告)日：2007-07-12
申请号：PCT/US2006020846
申请日：2006-05-26
申请人： SIRNA THERAPEUTICS INC , GUERCIOLINI ROBERTO , MCSWIGGEN JAMES , JADHAV VASANT , VARGEESE CHANDRA
发明人： GUERCIOLINI ROBERTO , MCSWIGGEN JAMES , JADHAV VASANT , VARGEESE CHANDRA
IPC分类号： C07K14/52 , C12N15/113
CPC分类号： C12N15/1136 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , 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-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.
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。本发明还涉及涉及对参与基质细胞衍生的因子-1（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，疾病和病症有关的化合物，组合物和方法或介导这些性状，疾病和状况的维持或发展的其他细胞过程。具体地说，本发明涉及包括小核酸分子的双链核酸分子，如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和能够介导针对基质细胞衍生因子-1（SDF-1）基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括这种小核酸的这种小核酸分子和脂质纳米颗粒（LNP）制剂的混合物酸分子。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（例如RISC抑制剂）通过干扰与这些内源性RNA相关的内源性RNA或蛋白质的调节功能来调节SDF-1基因表达（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，抑制或减少血管生成相关疾病性状和病症的组合物，包括但不限于眼部疾病，癌症和增殖性疾病，性状和病症，和/或与受试者或生物体内的SDF-1基因表达或活性相关的其它疾病状态，病症或性状。

9. 发明申请

WO2008011431A8 RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： 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
公开(公告)号：WO2008011431A8
公开(公告)日：2009-11-05
申请号：PCT/US2007073723
申请日：2007-07-17
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , JADHAV VASANT , VARGEESE CHANDRA
发明人： MCSWIGGEN JAMES , JADHAV VASANT , VARGEESE CHANDRA
IPC分类号： A61K48/00 , C12N15/113
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/321 , C12N2310/322 , 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 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.
摘要翻译：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。

10. 发明申请

WO2007076328A3 RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS 审中-公开
标题翻译： RNA干扰介导的HEPATITIS C病毒的抑制
公开(公告)号：WO2007076328A3
公开(公告)日：2008-08-14
申请号：PCT/US2006062252
申请日：2006-12-18
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , MORRISSEY DAVID , GUERCIOLINI ROBERTO , VARGEESE CHANDRA , JADHAV VASANT
发明人： MCSWIGGEN JAMES , MORRISSEY DAVID , GUERCIOLINI ROBERTO , VARGEESE CHANDRA , JADHAV VASANT
IPC分类号： A61K9/127 , A61K31/713 , C12N15/11 , C12N15/113
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 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.
摘要翻译：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。

你已经成功收藏专利！

检索式保存成功!

IPRDB

热门服务

关于我们

友情链接

联系方式