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21. 发明申请

WO03102131A2 NUCLEIC ACID MEDIATED DISRUPTION OF HIV FUSOGENIC PEPTIDE INTERACTIONS 审中-公开
标题翻译：核酸介导的艾滋病毒纤维蛋白肽相互作用的破坏
公开(公告)号：WO03102131A2
公开(公告)日：2003-12-11
申请号：PCT/US0312626
申请日：2003-04-22
申请人： SIRNA THERAPEUTICS INC , MACEJAK DENNIS , BLATT LAWRENCE , MCSWIGGEN JAMES
发明人： MACEJAK DENNIS , BLATT LAWRENCE , MCSWIGGEN JAMES
IPC分类号： A61K38/00 , A61K48/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12N15/115 , C12N15/85 , C12N15/86 , C12P19/34 , C12N
CPC分类号： C12N15/115 , C12N15/1132 , C12N2310/111 , C12N2310/12 , C12N2310/121 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2310/3521
摘要： The present invention relates to nucleic acid aptamers that bind to HIV envelope glycoprotein, gp120 and/or gp41 and methods for their use alone or in combination with other therapies, such as HIV RT inhibitors and HIV protease inhibitors. Also disclosed are nucleic acids such as siRNA, antisense, and enzymatic nucleic acid molecules that can modulate the expression of HIV env genes and HIV viral replication. The compounds and methods of the invention are expected to inhibit HIV viral fusion, cell entry, gene expression, and replication.
摘要翻译：本发明涉及结合HIV包膜糖蛋白，gp120和/或gp41的核酸适配子及其单独使用或与其它疗法如HIV RT抑制剂和HIV蛋白酶抑制剂组合使用的方法。还公开了可以调节HIV env基因表达和HIV病毒复制的核酸，例如siRNA，反义和酶核酸分子。预期本发明的化合物和方法可以抑制HIV病毒融合，细胞进入，基因表达和复制。

22. 发明申请

WO2007084865A3 RNA INTERFERENCE MEDIATED INHIBITION OF B7-H1 GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的B7-H1基因表达的抑制使用短暂的干扰核酸（siNA）
公开(公告)号：WO2007084865A3
公开(公告)日：2008-04-24
申请号：PCT/US2007060522
申请日：2007-01-12
申请人： SIRNA THERAPEUTICS INC , USMAN NASSIM , MCSWIGGEN JAMES
发明人： USMAN NASSIM , MCSWIGGEN JAMES
IPC分类号： A61K31/713 , C12N15/113
CPC分类号： C12N15/1138 , 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 B7-H1 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 that respond to the moduclation of expression and/or activity of genes involved in B7-H1 gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases, and conditions, including but not limited to PD-1, 1L-2, IL-10, IL- 12 and IFN-?. 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.
摘要翻译：本发明涉及用于研究，诊断和治疗对B7-H1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对参与B7-H1基因表达途径的基因的表达和/或活性的调节作用的反应的性状，疾病和病症，或调节维持或发展这些性状，疾病和病症的其他细胞过程，包括但不限于 PD-1，1L-2，IL-10，IL-12和IFN-γ。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）或可以抑制RISC功能（例如RISC抑制剂）调节基因表达，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（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。

23. 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。

24. 发明申请

WO2007022369A3 CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID MOLECULES THAT MEDIATE RNA INTERFERENCE 审中-公开
标题翻译：化学修饰的短暂干扰RNA干扰的核酸分子
公开(公告)号：WO2007022369A3
公开(公告)日：2007-07-26
申请号：PCT/US2006032168
申请日：2006-08-17
申请人： SIRNA THERAPEUTICS INC , MORRISEY DAVID , MCSWIGGEN JAMES , BEIGELMAN LEONID
发明人： MORRISEY DAVID , MCSWIGGEN JAMES , BEIGELMAN LEONID
IPC分类号： C07K14/02 , C12N15/11 , C12N15/113
CPC分类号： C12N15/1131 , C12N15/111 , C12N2310/14 , 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 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.
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。

25. 发明申请

WO2006060598A2 RNA INTERFERENCE MEDIATED INHIBITION OF INTERLEUKIN AND INTERLEUKIN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制干扰素和白介素受体基因表达使用短暂的核酸（siNA）
公开(公告)号：WO2006060598A2
公开(公告)日：2006-06-08
申请号：PCT/US2005043510
申请日：2005-12-01
申请人： SIRNA THERAPEUTICS INC , RICHARDS IVAN , POLISKY BARRY , MCSWIGGEN JAMES
发明人： RICHARDS IVAN , POLISKY BARRY , MCSWIGGEN JAMES
IPC分类号： A61K48/00 , C12N15/113 , C12N15/85 , C12Q1/68
CPC分类号： A61K49/0008 , A61K38/00 , C12N15/1136 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/3519 , C12N2310/53 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating interleukin and/or interleukin receptor 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 interleukin and/or interleukin 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 interleukin and/or interleukin receptor genes, such as IL-1, IL-2, IL-3, IL-4, IL-5, IL-6, IL-7, IL-8, IL-9, IL-10, IL-11, IL-12, IL-13, IL-14, IL-15, IL-16, IL-17, IL-18, IL-19, IL-20, IL-21, IL-22, IL-23, IL24, IL-25, IL-26, and IL-27 genes and IL-1R, IL-2R, IL-3R, IL-4R, IL-5R, IL-6R, IL-7R, IL8R, IL-9R, IL-10R, IL-11R, IL-12R, IL-13R, IL-14R, IL-15R, IL-16R, IL-17R, IL-18R, IL19R, IL-20R, IL-21R, IL-22R, IL-23R, IL-24R, IL-25R, IL-26R, and IL-27R. Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing cancer, inflammatory, respiratory, autoimmune, cardiovascular, neurological, and/or proliferative diseases, disorders, or conditions in a subject or organism, and for any other disease, trait, or condition that is related to or will respond to the levels of interleukin and/or interleukin receptor in a cell or tissue, alone or in combination with other treatments or therapies.
摘要翻译：本发明涉及可用于使用短干扰核酸（siNA）分子调节白介素和/或白细胞介素受体基因表达的化合物，组合物和方法。本发明还涉及可用于通过使用小核酸分子通过RNA干扰（RNAi）调节参与白细胞介素和/或白细胞介素受体基因表达和/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））分子和用于调节白介素和/或白介素受体基因如IL-1，IL-2，IL-3，IL-4，IL-5，IL-6，IL-7，IL- 8，IL-9，IL-10，IL-11，IL-12，IL-13，IL-14，IL-15，IL-16，IL-17，IL-18，IL-19， IL-21，IL-22，IL-23，IL24，IL-25，IL-26和IL-27基因和IL-1R，IL-2R，IL-3R，IL-4R，IL-5R， 6R，IL-7R，IL8R，IL-9R，IL-10R，IL-11R，IL-12R，IL-13R，IL-14R，IL-15R，IL-16R，IL-17R，IL-18R， IL-20R，IL-21R，IL-22R，IL-23R，IL-24R，IL-25R，IL-26R和IL-27R。这样的小核酸分子可用于例如治疗，预防，抑制或减少受试者或生物体内的癌症，炎症，呼吸，自身免疫，心血管，神经和/或增殖性疾病，病症或病症，与细胞或组织中白细胞介素和/或白细胞介素受体水平相关或将会对其单独或与其它治疗或疗法结合的任何其它疾病，性状或病症。

26. 发明申请

WO2005045032A3 RNA INTERFERENCE MEDIATED INHIBITION OF EARLY GROWTH RESPONSE GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制使用短暂的核酸（siNA）的早期生长反应基因表达
公开(公告)号：WO2005045032A3
公开(公告)日：2006-03-02
申请号：PCT/US2004026941
申请日：2004-08-19
申请人： SIRNA THERAPEUTICS INC , USMAN NASSIM , MCSWIGGEN JAMES
发明人： USMAN NASSIM , MCSWIGGEN JAMES
IPC分类号： A01N43/04 , A61K31/713 , A61K38/00 , A61K49/00 , C07H21/02 , C07H21/04 , C12N15/11 , C12N15/113 , C12P19/34
CPC分类号： C12N15/1136 , A61K38/00 , A61K49/0008 , C12N15/111 , C12N15/113 , C12N2310/111 , C12N2310/12 , C12N2310/121 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2330/30 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating early growth response (Egr-1) 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 Egr-1 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 Egr-1 genes.
摘要翻译：本发明涉及可用于使用短干扰核酸（siNA）分子调节早期生长反应（Egr-1）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涉及Egr-1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Egr-1基因表达的方法。

27. 发明申请

WO2005105995A3 RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的治疗聚氨酯（POLYQ）重复扩张疾病使用短暂的干细胞核酸（siNA）
公开(公告)号：WO2005105995A3
公开(公告)日：2005-12-29
申请号：PCT/US2005006661
申请日：2005-03-01
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES
发明人： MCSWIGGEN JAMES
IPC分类号： A61K31/713 , A61K38/00 , A61P25/28 , C07H21/00 , C12N15/113 , C12N15/11
CPC分类号： C12N15/113 , A61K38/00 , C12N2310/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/53 , C12N2310/3521
摘要： The present invention concerns compounds, compositions, and methods for the study, diagnosis, and treatment of diseases and conditions associated with polyglutamine repeat (polyQ) allelic variants that respond to the modulation of gene expression and/or activity. The present invention also concerns compounds, compositions, and methods relating to diseases and conditions associated with polyglutamine repeat (polyQ) allelic variants that respond to the modulation of expression and/or activity of genes involved in polyQ repeat gene expression pathways. or other cellular processes that mediate the maintenance or development of polyQ repeat diseases and conditions such as Huntington disease and related conditions such as progressive chorea, rigidity, dementia, and seizures, spinocerebellar ataxia, Spinal and bulbar muscular dystrophy (SBMA), dentatorubropallidoluysian atrophy (DIZPLA), and any other diseases or conditions that are related to or will respond to the levels of a repeat expansion (RE) protein in a cell or tissue, alone or in combination with other therapies. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), douse-stranded RN (dsRNA), micro-RNA (miRNA), and short hairpin RN (shRNA) molecules capable of mediating RN interference (RNAi) against the expression disease related genes or alleles having polyQ repeat sequences.
摘要翻译：本发明涉及用于研究，诊断和治疗与对应于基因表达和/或活性的调节的多聚谷氨酰胺重复（polyQ）等位变体相关的疾病和病症的化合物，组合物和方法。本发明还涉及与多聚谷氨酰胺重复（polyQ）等位变体相关的疾病和病症相关的化合物，组合物和方法，所述多聚谷氨酰胺重复（polyQ）等位基因变体响应于参与polyQ重复基因表达途径的基因的表达和/或活性的调节。或调节维持或发展polyQ重复疾病和病症的其他细胞过程，例如亨廷顿病和相关病症如进行性舞蹈病，僵硬，痴呆和癫痫发作，脊髓小脑性共济失调，脊髓和延髓肌营养不良症（SBMA），牙周性尿道粘膜炎萎缩症 DIZPLA）以及与细胞或组织中的重复扩展（RE）蛋白的水平相关或将会对其单独或与其它疗法组合的水平相关的任何其它疾病或病症。具体地说，本发明涉及小核酸分子，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）能够介导RN干扰（RNAi）对具有polyQ重复序列的疾病相关基因或等位基因表达的分子。

28. 发明申请

WO2005014811A3 RNA INTERFERENCE MEDIATED INHIBITION OF XIAP GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的XIAP基因表达抑制短干扰核酸（siNA）
公开(公告)号：WO2005014811A3
公开(公告)日：2005-12-22
申请号：PCT/US2004025589
申请日：2004-08-06
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , CHOWRIRA BHARAT M
发明人： MCSWIGGEN JAMES , CHOWRIRA BHARAT M
IPC分类号： A61K38/00 , C12N15/11 , C12N15/113 , C12N15/87 , A01N43/04 , A61K31/713 , C07H21/02 , C07H21/04 , C12P19/34
CPC分类号： C12N15/113 , A61K38/00 , C12N15/111 , C12N15/1137 , C12N15/1138 , C12N15/87 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2330/30 , C12Y103/01022 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating XIAP 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 XIAP 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 XIAP genes.
摘要翻译：本发明涉及用于使用短干扰核酸（siNA）分子调节XIAP基因表达的化合物，组合物和方法。本发明还涉及用于使用小核酸分子调节参与XIAP基因表达和/或通过RNA干扰（RNAi）的活性的途径中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XIAP基因表达的方法。

29. 发明申请

WO2005007859A3 RNA INTERFERENCE MEDIATED INHIBITION OF ACETYL-COA CARBOXYLASE GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的乙酰辅酶C羧酸酶基因表达的抑制使用短暂的核酸（SINA）
公开(公告)号：WO2005007859A3
公开(公告)日：2005-12-01
申请号：PCT/US2004022247
申请日：2004-07-09
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , USMAN NASSIM
发明人： MCSWIGGEN JAMES , USMAN NASSIM
IPC分类号： A61K38/00 , C12N15/11 , C12N15/113 , C12N15/87 , C12N15/63 , A01N43/04 , C07H21/02 , C07H21/04 , C12P19/34 , C12Q1/68
CPC分类号： C12N15/113 , A61K38/00 , C12N15/111 , C12N15/1137 , C12N15/1138 , C12N15/87 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2330/30 , C12Y103/01022 , C12Y604/01002 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating acetyl-CoA carboxylase 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 acetyl-CoA carboxylase 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 acetyl-CoA carboxylase genes.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节乙酰辅酶A羧化酶基因表达的化合物，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乙酰辅酶A羧化酶基因表达和/或通过RNA干扰（RNAi）的活性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））分子和用于调节乙酰辅酶A羧化酶基因表达的方法。

30. 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。

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