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1. 发明申请

WO2005078097A2 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING MULTIFUNCTIONAL SHORT INTERFERING NUCLEIC ACID (Multifunctional siNA) 审中-公开
标题翻译： RNA干扰介导的基因表达抑制多功能短干扰核酸（多功能siNA）
公开(公告)号：WO2005078097A2
公开(公告)日：2005-08-25
申请号：PCT/US2005/004270
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： C12N15/11
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：本发明涉及用于调节各种应用中的基因表达的方法和基于核酸的试剂，包括用于治疗，兽医，农业，诊断，靶标验证和基因组发现应用。具体而言，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，或者可以靶向不同靶核酸分子中的核酸序列区域。自我多功能siNA分子可用于治疗对细胞，组织或生物体中的基因表达或活性的调节作出响应的任何疾病或病症。

2. 发明申请

WO2005028649A1 RNA INTERFERENCE MEDIATED INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR AND VASCULAR ENDOTHELIAL GROWTH FACTOR RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的血管内皮生长因子和血管内皮生长因子受体基因表达的抑制使用短暂的核酸（siNA）
公开(公告)号：WO2005028649A1
公开(公告)日：2005-03-31
申请号：PCT/US2004/030488
申请日：2004-09-16
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： C12N15/11
CPC分类号： C12N15/111 , C12N15/1136 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/51 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： This invention relates to compounds, compositions, and methods useful for modulating VEGF and/or VEGFR 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 VEGF and/or VEGFR 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 VEGF and/or VEGFR genes.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节VEGF和/或VEGFR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VEGF和/或VEGFR基因表达和/或活性的其它基因的表达和活性的化合物，组合物和方法，所述基因表达和/或活性通过RNA干扰（RNAi）。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VEGF和/或VEGFR基因表达的方法。

3. 发明申请

WO2004014312A2 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：WO2004014312A2
公开(公告)日：2004-02-19
申请号：PCT/US2003/025031
申请日：2003-08-08
申请人： SIRNA THERAPEUTICS, INC. , ZINNEN, Shawn
发明人： ZINNEN, Shawn
IPC分类号： A61K
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。

4. 发明申请

WO2005028650A3 RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS (HCV) EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
公开(公告)号：WO2005028650A3
公开(公告)日：2005-03-31
申请号：PCT/US2004/031012
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： C12N15/11
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.

5. 发明申请

WO2005019453A2 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的基因表达的抑制使用化学修饰的短暂干扰核酸（siNA）
公开(公告)号：WO2005019453A2
公开(公告)日：2005-03-03
申请号：PCT/US2004/016390
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： 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：本发明涉及可用于调节多种应用中的基因表达的方法和试剂，包括在治疗，化妆品，药妆，预防，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，炎症性，代谢性，自身免疫性，炎性，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。

6. 发明申请

WO2003089650A3 ALLOSTERIC NUCLEIC ACID SENSOR MOLECULES 审中-公开
公开(公告)号：WO2003089650A3
公开(公告)日：2003-10-30
申请号：PCT/US2002/035529
申请日：2002-11-05
申请人： SIRNA THERAPEUTICS, INC. , SEIWERT, Scott , ZINNEN, Shawn , USMAN, Nassim , MCSWIGGEN, James , VAISH, Narendra , HAEBERLI, Peter , CHOWRIRA, Bharat , BLATT, Lawrence , JADHAV, Vasant , KOSSEN, Karl
发明人： SEIWERT, Scott , ZINNEN, Shawn , USMAN, Nassim , MCSWIGGEN, James , VAISH, Narendra , HAEBERLI, Peter , CHOWRIRA, Bharat , BLATT, Lawrence , JADHAV, Vasant , KOSSEN, Karl
IPC分类号： C12Q1/68
摘要： Nucleic acid sensor molecules and methods are provided for the detection and amplification of signaling agents using enzymatic nucleic acid constructs, including Halfzymes, multicomponent nucleic acid sensor molecules, hammerhead enzymatic nucleic acid molecules, inozymes, G-cleaver enzymatic nucleic acid molecules, zinzymes, amberzymes and DNAzymes. Also provided are kits for detection and amplification. The nucleic acid sensor molecules, methods and kits provided herein can be used in diagnostics, nucleic acid circuits, nucleic acid computers, therapeutics, target validation, target discovery, drug optimization, single nucleotide polymorphism (SNP) detection, single nucleotide polymorphism (SNP) scoring, and proteome scoring as well as other uses described herein.

7. 发明申请

WO2013019874A9 VITAMIN B6 DERIVATIVES OF NUCLEOTIDES, ACYCLONUCLEOTIDES AND ACYCLONUCLEOSIDE PHOSPHONATES 审中-公开
公开(公告)号：WO2013019874A9
公开(公告)日：2013-02-07
申请号：PCT/US2012/049181
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
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.

8. 发明申请

WO2003089650A2 ALLOSTERIC NUCLEIC ACID SENSOR MOLECULES 审中-公开
标题翻译：多功能核酸传感器分子
公开(公告)号：WO2003089650A2
公开(公告)日：2003-10-30
申请号：PCT/US2002/035529
申请日：2002-11-05
申请人： RIBOZYME PHARMACEUTICALS, INCORPORATED , SEIWERT, Scott , ZINNEN, Shawn , USMAN, Nassim , MCSWIGGEN, James , VAISH, Narendra , HAEBERLI, Peter , CHOWRIRA, Bharat , BLATT, Lawrence , JADHAV, Vasant , KOSSEN, Karl
发明人： SEIWERT, Scott , ZINNEN, Shawn , USMAN, Nassim , MCSWIGGEN, James , VAISH, Narendra , HAEBERLI, Peter , CHOWRIRA, Bharat , BLATT, Lawrence , JADHAV, Vasant , KOSSEN, Karl
IPC分类号： C12Q
CPC分类号： C12N15/113 , C12N15/1131 , C12N2310/12 , C12N2310/121
摘要： Nucleic acid sensor molecules and methods are provided for the detection and amplification of signaling agents using enzymatic nucleic acid constructs, including Halfzymes, multicomponent nucleic acid sensor molecules, hammerhead enzymatic nucleic acid molecules, inozymes, G-cleaver enzymatic nucleic acid molecules, zinzymes, amberzymes and DNAzymes. Also provided are kits for detection and amplification. The nucleic acid sensor molecules, methods and kits provided herein can be used in diagnostics, nucleic acid circuits, nucleic acid computers, therapeutics, target validation, target discovery, drug optimization, single nucleotide polymorphism (SNP) detection, single nucleotide polymorphism (SNP) scoring, and proteome scoring as well as other uses described herein.
摘要翻译：提供了核酸传感器分子和方法，用于使用酶核酸构建体检测和扩增信号试剂，包括半酶，多组分核酸传感器分子，锤头酶核酸分子，酶，G切割酶酶核酸分子，酶，琥珀酸和DNAzymes。还提供了用于检测和扩增的试剂盒。本文提供的核酸传感器分子，方法和试剂盒可用于诊断，核酸回路，核酸计算机，治疗学，靶标验证，靶发现，药物优化，单核苷酸多态性（SNP）检测，单核苷酸多态性（SNP）评分和蛋白质组评分以及本文所述的其它用途。

9. 发明申请

WO2003070918A3 RNA INTERFERENCE BY MODIFIED SHORT INTERFERING NUCLEIC ACID 审中-公开
公开(公告)号：WO2003070918A3
公开(公告)日：2003-08-28
申请号：PCT/US2003/005346
申请日：2003-02-20
申请人： RIBOZYME PHARMACEUTICALS, INCORPORATED , McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , ZINNEN, Shawn , PAVCO, Pamela , MORRISSEY, David , FOSNAUGH, Kathy , MOKLER, Victor , JAMISON, Sharon
发明人： McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , ZINNEN, Shawn , PAVCO, Pamela , MORRISSEY, David , FOSNAUGH, Kathy , MOKLER, Victor , JAMISON, Sharon
IPC分类号： C07H21/04
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, 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 or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

10. 发明申请

WO2003070918A2 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID 审中-公开
标题翻译： RNA干扰介导的基因表达的抑制使用化学修饰的短暂干扰核酸
公开(公告)号：WO2003070918A2
公开(公告)日：2003-08-28
申请号：PCT/US2003/005346
申请日：2003-02-20
申请人： RIBOZYME PHARMACEUTICALS, INCORPORATED , McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , ZINNEN, Shawn , PAVCO, Pamela , MORRISSEY, David , FOSNAUGH, Kathy , MOKLER, Victor , JAMISON, Sharon
发明人： McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , ZINNEN, Shawn , PAVCO, Pamela , MORRISSEY, David , FOSNAUGH, Kathy , MOKLER, Victor , JAMISON, Sharon
IPC分类号： C12N
CPC分类号： C12N15/8218 , A61K38/00 , A61K47/54 , C07H21/02 , C12N15/111 , C12N15/1131 , C12N15/1138 , C12N2310/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2320/51 , C12N2330/30 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, 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 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各种应用中的基因表达的方法和试剂，包括在治疗，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对细胞，组织或生物中的基因表达或活性的调节作出反应的任何疾病或病症。

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