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1. 发明申请

WO2009045457A2 TRIPARTITE RNAI CONSTRUCTS 审中-公开
标题翻译： TRIPARTITE RNAI构建体
公开(公告)号：WO2009045457A2
公开(公告)日：2009-04-09
申请号：PCT/US2008011394
申请日：2008-10-02
申请人： RXI PHARMACEUTICALS CORP , WOOLF TOD M , WAGNER RICK , PAVCO PAM , SALOMON WILLIAM , USMAN NASSIM , SAMARSKY DMITRY
发明人： WOOLF TOD M , WAGNER RICK , PAVCO PAM , SALOMON WILLIAM , USMAN NASSIM , SAMARSKY DMITRY
IPC分类号： A61K31/713 , A61K47/48 , C07H21/00 , C12N15/11
CPC分类号： C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/351 , C12N2310/3519 , C12N2310/3521
摘要： The present invention provides compositions and methods for inhibiting expression of a target gene in a cell. The process comprises introduction of double-stranded tripartite RNAi constructs into the cells and reducing the expression of the corresponding messenger RNA in the cells. The constructs, which may be packaged in or delivered as sequestered RNAi constructs, differ from the canonical siRNA in that they comprise a tripartite structure which follows the general formula of having (1) an RNAi core (either native or abbreviated), (2) one or more terminal moieties attached to the RNAi core and optionally (3) a linker between the RNAi core and the terminal moiety. Once packaged into sequestration vehicles, the constructs are activated for gene regulation by the application of certain forms of energy.
摘要翻译：本发明提供抑制细胞中靶基因表达的组合物和方法。该方法包括将双链三方RNAi构建体引入细胞中并降低细胞中相应的信使RNA的表达。可以包封或作为隔离RNAi构建体递送的构建体不同于规范siRNA，因为它们包含三分子结构，其遵循具有（1）RNAi核心（天然或缩写）的通式，（2）连接到RNAi核的一个或多个末端部分和任选地（3）RNAi核心和末端部分之间的接头。一旦包装成隔离载体，通过应用某些形式的能量，构建体被激活用于基因调控。

2. 发明申请

WO2005007855A3 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核酸（siNA）
公开(公告)号：WO2005007855A3
公开(公告)日：2005-03-24
申请号：PCT/US2004022658
申请日：2004-07-14
申请人： SIRNA THERAPEUTICS INC , USMAN NASSIM , MCSWIGGEN JAMES
发明人： USMAN NASSIM , MCSWIGGEN JAMES
IPC分类号： A61K38/00 , C12N15/11 , C12N15/113 , C12N15/87 , A61K31/713
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 B7-H1 (also known as PD-L1) 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 B7-H1 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 B7-H1 genes.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节B7-H1（也称为PD-L1）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B7-H1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，本发明具有小的核酸分子，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B7-H1基因表达的方法。

3. 发明申请

WO2004097020A3 RNA INTERFERENCE MEDIATED INHIBITION OF MAP KINASE GENE EXPRESSION 审中-公开
标题翻译： RNA干扰介导的MAP激酶基因表达的抑制
公开(公告)号：WO2004097020A3
公开(公告)日：2006-01-05
申请号：PCT/US2004012517
申请日：2004-04-23
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , BEIGELMAN LEONID , USMAN NASSIM , HAEBERLI PETER , CHOWRIRA BHARAT , POLISKY BARRY
发明人： MCSWIGGEN JAMES , BEIGELMAN LEONID , USMAN NASSIM , HAEBERLI PETER , CHOWRIRA BHARAT , POLISKY BARRY
IPC分类号： A61K38/00 , C12N15/11 , C12N15/113 , C12N15/87 , A61K31/713
CPC分类号： C12N15/111 , A61K38/00 , C12N15/1137 , C12N15/87 , C12N2310/14 , C12N2310/315 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2320/11 , C12N2320/32 , C12N2330/30 , C12Y207/11024 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating mitogen activated protein kinase (MAP kinase ) 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 MAP kinase 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 mitogen activated protein kinase (MAP kinase ) genes such as c-JUN, JNK1, JNK2, p38, ERK1, or ERK2.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节有丝分裂原活化蛋白激酶（MAP激酶）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涉及MAP激酶和/或活性通过RNA干扰（RNAi）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））分子和用于调节丝裂原活化蛋白激酶（MAP激酶）基因如c-JUN，JNK1，JNK2，p38，ERK1或ERK2的表达的方法。

4. 发明申请

WO0166721A3 NUCLEIC ACID SENSOR MOLECULES 审中-公开
标题翻译：核酸传感器分子
公开(公告)号：WO0166721A3
公开(公告)日：2002-07-25
申请号：PCT/US0107163
申请日：2001-03-06
申请人： RIBOZYME PHARM INC , USMAN NASSIM , MCSWIGGEN JAMES A , ZINNEN SHAWN , SEIWERT SCOTT , HAEBERLI PETER , CHOWRIRA BHARAT , BLATT LAWRENCE
发明人： USMAN NASSIM , MCSWIGGEN JAMES A , ZINNEN SHAWN , SEIWERT SCOTT , HAEBERLI PETER , CHOWRIRA BHARAT , BLATT LAWRENCE
IPC分类号： C12N15/09 , A61K31/7088 , A61K38/00 , C12N15/113 , C12Q1/68 , C12N15/11 , C07H21/00 , C12N9/00
CPC分类号： C12N15/1138 , A61K31/7088 , A61K38/00 , C12N2310/111 , C12N2310/12 , C12N2310/121 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/332 , C12N2310/3517 , C12N2310/3521
摘要： Nucleic acid sensor molecules and methods are disclosed for the detection and amplification of signaling agents using enzymatic nucleic acid constructs, including hammerhead enzymatic nucleic acid molecules, inozymes, G-cleaver enzymatic nucleic acid molecules, zinzymes, amberzymes and DNAzymes; kits for detection and amplification; use in diagnostics, nucleic acid circuits, nucleic acid computers, and other uses are disclosed.
摘要翻译：公开了核酸传感器分子和方法，用于使用酶核酸构建体（包括锤头酶核酸分子，酶切，G切割酶酶核酸分子，酶，琥珀酶和DNA酶）检测和扩增信号传导。用于检测和扩增的试剂盒; 公开了用于诊断，核酸回路，核酸计算机和其它用途。

5. 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。

6. 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。

7. 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。

8. 发明申请

WO2005035759A3 RNA interference mediated inhibition of hypoxia inducible factor 1 (HIF1) gene expression using sho rt interfering nucleic acid (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制低氧诱导因子1（HIF1）基因表达使用干扰核酸（siNA）
公开(公告)号：WO2005035759A3
公开(公告)日：2006-03-16
申请号：PCT/US2004027294
申请日：2004-08-20
申请人： USMAN NASSIM , MCSWIGGEN JAMES , SIRNA THERAPEUTICS INC
发明人： USMAN NASSIM , MCSWIGGEN JAMES
IPC分类号： A61K38/00 , C12N15/11 , C12N15/113 , C12N15/115 , C12N15/87 , A01N43/04 , C07H21/02 , C07H21/04 , C12P19/34
CPC分类号： C12N15/1137 , A61K38/00 , C12N15/111 , C12N15/113 , 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 , C12N2330/30 , C12Y103/01022 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating hyproxia inducable factor (e.g., HIF1) 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 HIF1 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 HIF1 genes.

9. 发明申请

WO2005035759A8 RNA INTERFERENCE MEDIATED INHIBITION OF HYPOXIA INDUCIBLE FACTOR 1 (HIF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制HYPOXIA诱导因子1（HIF1）使用短暂的核酸（siNA）的基因表达
公开(公告)号：WO2005035759A8
公开(公告)日：2006-02-02
申请号：PCT/US2004027294
申请日：2004-08-20
申请人： SIRNA THERAPEUTICS INC , USMAN NASSIM , MCSWIGGEN JAMES
发明人： USMAN NASSIM , MCSWIGGEN JAMES
IPC分类号： A61K38/00 , C12N15/11 , C12N15/113 , C12N15/115 , C12N15/87 , A01N43/04 , C07H21/02 , C07H21/04 , C12P19/34
CPC分类号： C12N15/1137 , A61K38/00 , C12N15/111 , C12N15/113 , 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 , C12N2330/30 , C12Y103/01022 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating hyproxia inducable factor (e.g., HIF1) 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 HIF1 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 HIF1 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hypoxia诱导因子（例如，HIF1）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HIF1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HIF1基因表达的方法。

10. 发明申请

WO0116312A3 NUCLEIC ACID BASED MODULATORS OF GENE EXPRESSION 审中-公开
标题翻译：基于核酸的基因表达调控因子
公开(公告)号：WO0116312A3
公开(公告)日：2001-08-09
申请号：PCT/US0023998
申请日：2000-08-30
申请人： RIBOZYME PHARM INC , MCSWIGGEN JAMES , USMAN NASSIM , BLATT LAWRENCE , BEIGELMAN LEONID , BURGIN ALEX , KARPEISKY ALEXANDER , MATULIC ADAMIC JASENKA , SWEEDLER DAVID , DRAPER KENNETH , CHOWRIRA BHARAT , STINCHCOMB DAN , BEAUDRY AMBER , ZINNEN SHAWN , LUGWIG JANOS , SPROAT BRIAN S
发明人： MCSWIGGEN JAMES , USMAN NASSIM , BLATT LAWRENCE , BEIGELMAN LEONID , BURGIN ALEX , KARPEISKY ALEXANDER , MATULIC-ADAMIC JASENKA , SWEEDLER DAVID , DRAPER KENNETH , CHOWRIRA BHARAT , STINCHCOMB DAN , BEAUDRY AMBER , ZINNEN SHAWN , LUGWIG JANOS , SPROAT BRIAN S
IPC分类号： C12N15/09 , A61K31/7115 , A61K38/00 , A61K48/00 , A61P1/16 , A61P3/04 , A61P3/10 , A61P9/00 , A61P9/04 , A61P25/28 , A61P35/00 , A61P43/00 , C07H19/10 , C07H19/20 , C07H19/22 , C07H21/00 , C07H21/04 , C12N5/10 , C12N15/113 , C12P19/30 , C12N15/11 , A61K31/7088 , C07H19/00 , C12N9/00 , C12P19/34
CPC分类号： C12N15/113 , A61K38/00 , C07H19/10 , C07H19/20 , C07H21/00 , C12N15/1131 , C12N15/1137 , C12N15/1138 , C12N2310/12 , C12N2310/18 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/346 , C12P19/30 , C12P19/305 , C12Y207/07049 , C12Y301/03048 , C12N2310/3523
摘要： Nucleic acid molecules (antisenses or ribozymes) useful as inhibitors of gene expression, especially of HER2, BACE, TERT, PTP-1B, MetAP-2, HBV, phospholamban, presenilin-2 and PKC-alpha. The nucleic acid molecules can be modified in various ways on the sugar and/or base moieties and/or on the phosphate backbone. They are used in pharmaceutical formulations for the treatment of diseases involving increased expression of the target genes. Also disclosed is a method for the synthesis of a modified pyrimidine nucleotide triphosphate and its incorporation into an oligonucleotide.
摘要翻译：用作基因表达抑制剂，特别是HER2，BACE，TERT，PTP-1B，MetAP-2，HBV，受磷蛋白，早老素-2和PKC-α的核酸分子（反义物或核酶）。核酸分子可以在糖和/或碱部分和/或磷酸骨架上以各种方式修饰。它们用于药物制剂用于治疗涉及靶基因表达增加的疾病。还公开了合成修饰的嘧啶核苷酸三磷酸并将其掺入寡核苷酸的方法。

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