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1. 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。

2. 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。

3. 发明申请

WO9955857A3 NUCLEOSIDE TRIPHOSPHATES AND THEIR INCORPORATION INTO RIBOZYMES 审中-公开
标题翻译：核苷酸二磷酸酯及其与RIBOZYMES的合成
公开(公告)号：WO9955857A3
公开(公告)日：2000-02-24
申请号：PCT/US9909348
申请日：1999-04-28
申请人： RIBOZYME PHARM INC , BEIGELMAN LEONID , BURGIN ALEX , BEAUDRY AMBER , KARPEISKY ALEXANDER , MATULIC ADAMIC JASENKA , SWEEDLER DAVID , ZINNEN SHAWN
发明人： BEIGELMAN LEONID , BURGIN ALEX , BEAUDRY AMBER , KARPEISKY ALEXANDER , MATULIC-ADAMIC JASENKA , SWEEDLER DAVID , ZINNEN SHAWN
IPC分类号： C12N15/09 , C07H19/10 , C07H19/20 , C07H21/00 , C12N9/00 , C12N15/113 , C12P19/34 , C12N15/11 , C07H19/04
CPC分类号： C12N15/113 , C07H19/10 , C07H19/20 , C07H21/00 , C12N2310/12 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/33 , C12N2310/345
摘要： Novel nucleotide triphosphates, methods of synthesis and process of incorporating these nucleotide triphosphates into oligonucleotides, and isolation of novel nucleic acid catalysts (e.g., ribozymes) are disclosed.
摘要翻译：公开了新型核苷酸三磷酸酯，合成方法和将这些核苷酸三磷酸引入寡核苷酸的方法，以及分离新型核酸催化剂（例如核酶）。

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

WO2005078097A3 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING MULTIFUNCTIONAL SHORT INTERFERING NUCLEIC ACID (Multifunctional siNA) 审中-公开
标题翻译： RNA干扰介导的使用多功能短暂干扰核酸（多功能siNA）进行基因表达的抑制
公开(公告)号：WO2005078097A3
公开(公告)日：2005-10-27
申请号：PCT/US2005004270
申请日：2005-02-09
申请人： SIRNA THERAPEUTICS INC , JADHAV VASANT , ZINNEN SHAWN
发明人： JADHAV VASANT , ZINNEN SHAWN
IPC分类号： A61K38/00 , C12N15/113 , C12N15/11 , A01N43/04 , A61K31/713 , C07H21/02 , C07H21/04 , C12P19/34
CPC分类号： C12N15/1131 , A61K38/00 , C12N15/1136 , C12N15/1138 , C12N2310/111 , C12N2310/14 , C12N2310/3519 , C12N2310/51 , C12N2310/53
摘要： [00101] The present invention concerns methods and nucleic acid based reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, veterinary, agricultural, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to multifunctional short interfering nucleic acid (multifunctional siNA) molecules that modulate the expression of one or more genes in a biologic system, such as a cell, tissue, or organism via RNA interference (RNAi). The bifuntional short interfering nucleic acid (multifunctional siNA) molecules of the invention can target more than one regions of nucleic acid sequence in a single target nucleic acid molecule or can target regions of nucleic acid sequence in differing target nucleic acid molecules. The self multifunctional siNA molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
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基于核酸的试剂，包括在治疗，兽医，农业，诊断，靶标验证和基因组发现应用中的用途。具体地，本发明涉及通过RNA干扰（RNAi）调节生物系统例如细胞，组织或生物体中的一种或多种基因的表达的多功能短干扰核酸（多功能siNA）分子。本发明的双功能短干扰核酸（多功能siNA）分子可以靶向单个靶核酸分子中的多于一个核酸序列区域，或者靶向不同靶核酸分子中核酸序列的区域。自身多功能siNA分子可用于治疗对细胞，组织或生物中基因表达或活性的调节作出反应的任何疾病或病症。

7. 发明申请

WO2013019874A1 VITAMIN B6 DERIVATIVES OF NUCLEOTIDES, ACYCLONUCLEOTIDES AND ACYCLONUCLEOSIDE PHOSPHONATES 审中-公开
标题翻译：维生素B6衍生物，核苷酸和环磷酰胺磷酸酯
公开(公告)号：WO2013019874A1
公开(公告)日：2013-02-07
申请号：PCT/US2012049181
申请日：2012-08-01
申请人： MBC PHARMA INC , KARPEISKY ALEXANDER , ZINNEN SHAWN , URLAM MURALI , VVEDENSKY VLADIMIR Y , GUZAEV ANDREI P
发明人： KARPEISKY ALEXANDER , ZINNEN SHAWN , URLAM MURALI , VVEDENSKY VLADIMIR Y , GUZAEV ANDREI P
IPC分类号： A01N57/00 , A61K31/675
CPC分类号： A61K31/7068 , C07D213/65 , C07D213/76 , C07H19/10 , C07H19/12 , C07H19/19 , C07H19/20 , C07H19/23
摘要： Compounds, compositions, and methods of using them to treat infections, neoplastic disease, inflammatory disease, and pain. Such compounds are nucleotides, acydonucleotides, and acydic nucleoside phosphonates (ANP) phosphonates conjugated with forms and/or moieties of Vitamin B6 for delivery past the cell membrane and into the cell. Methods of delivering nucleotides, acydonucleotide and ANPs to the inside of the cell by conjugating said compounds with forms and/or moieties of Vitamin B6.
摘要翻译：使用它们治疗感染，肿瘤疾病，炎性疾病和疼痛的化合物，组合物和方法。这些化合物是与维生素B6的形式和/或部分缀合的核苷酸，酰基二核苷酸和酰基核苷膦酸盐（ANP）膦酸盐，用于递送通过细胞膜并进入细胞。通过将所述化合物与维生素B6的形式和/或部分缀合，将核苷酸，半胱氨酸核苷酸和ANPs递送至细胞内部的方法。

8. 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。

9. 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。

10. 发明申请

WO2004014312A3 SMALL-MER COMPOSITIONS AND METHODS OF USE 审中-公开
标题翻译：小型组合物和使用方法
公开(公告)号：WO2004014312A3
公开(公告)日：2005-02-17
申请号：PCT/US0325031
申请日：2003-08-08
申请人： SIRNA THERAPEUTICS INC , ZINNEN SHAWN
发明人： ZINNEN SHAWN
IPC分类号： A61K48/00 , C07H21/02 , C07H21/04 , C12Q1/68
CPC分类号： C07H21/04
摘要： The present invention concerns small-mer compositions and methods useful in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small-mer compositions capable of inhibiting viral replication, useful in treating diseases and conditions related to viral infection, and/or capable of inhibiting cellular proliferation, useful in treating diseases and conditions related to cellular proliferation.
摘要翻译：本发明涉及可用于各种应用的小分子组合物和方法，包括用于治疗，诊断，靶标验证和基因组发现应用。具体地，本发明涉及能够抑制病毒复制的小分子组合物，其可用于治疗与病毒感染相关的疾病和病症，和/或能够抑制细胞增殖，可用于治疗与细胞增殖相关的疾病和病症。

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