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1. 发明申请

WO2003070912A3 RNA INTERFERENCE MEDIATED INHIBITION OF EPIDERMAL GROWTH FACTOR RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
公开(公告)号：WO2003070912A3
公开(公告)日：2003-08-28
申请号：PCT/US2003/005045
申请日：2003-02-20
申请人： SIRNA THERAPEUTICS, INC. , McSWIGGEN, James , PAVCO, Pamela , BEIGELMAN, Leonid , FOSNAUGH, Kathy , JAMISON, Sharon
发明人： McSWIGGEN, James , PAVCO, Pamela , BEIGELMAN, Leonid , FOSNAUGH, Kathy , JAMISON, Sharon
IPC分类号： C07H21/02
摘要： The present invention concerns methods and reagents useful in modulating EGFR gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to 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 HER1, HER2, HER3 and/or HER4. The small nucleic acid molecules are useful in the treatment and diagnosis of cancer.

2. 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.

3. 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。

4. 发明申请

WO2003070910A2 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：WO2003070910A2
公开(公告)日：2003-08-28
申请号：PCT/US2003/005022
申请日：2003-02-20
申请人： RIBOZYME PHARMACEUTICALS, INCORPORATED , McSWIGGEN, James , BEIGELMAN, Leonid , PAVCO, Pamela
发明人： McSWIGGEN, James , BEIGELMAN, Leonid , PAVCO, Pamela
IPC分类号： C12N
CPC分类号： C12N15/115 , A61K38/00 , A61K47/54 , C07H21/00 , C07H21/02 , C12N15/1132 , C12N15/1136 , C12N15/1137 , C12N15/1138 , C12N2310/111 , C12N2310/12 , C12N2310/121 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/351 , C12N2310/53 , C12Y104/03003 , C12Y114/19001 , C12Y207/07049 , C12Y207/11001 , C12Y207/11013 , C12Y301/03048 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating vascular endothelial growth factor (VEGF, VEGF-B, VEGF-C, VEGF-D) and/or vascular endothelial growth factor receptor (e.g., VEGFr1, VEGFr2, and/or VEGFr3) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to 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 VEGF and/or VEGFr gene expression and/or activity. The small nucleic acid molecules are useful in the diagnosis and treatment of cancer, proliferative diseases, and any other disease or condition that responds to modulation of VEGF and/or VEGFr expression or activity.
摘要翻译：本发明涉及可用于调节血管内皮生长因子（VEGF，VEGF-B，VEGF-C，VEGF-D）和/或血管内皮生长因子受体（例如VEGFr1，VEGFr2和/或VEGFr3）基因的方法和试剂在各种应用中表达，包括用于治疗，诊断，靶标验证和基因组发现应用。具体而言，本发明涉及小核酸分子，如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA）能够介导针对VEGF和/或VEGFr基因表达和/或活性的RNA干扰（RNAi）的分子。小核酸分子可用于诊断和治疗癌症，增殖性疾病以及对VEGF和/或VEGFr表达或活性的调节作出响应的任何其它疾病或病症。

5. 发明申请

WO2003070910A3 INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR (VEGF) AND VEGF RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
公开(公告)号：WO2003070910A3
公开(公告)日：2003-08-28
申请号：PCT/US2003/005022
申请日：2003-02-20
申请人： RIBOZYME PHARMACEUTICALS, INCORPORATED , McSWIGGEN, James , BEIGELMAN, Leonid , PAVCO, Pamela
发明人： McSWIGGEN, James , BEIGELMAN, Leonid , PAVCO, Pamela
IPC分类号： C07H21/04
摘要： The present invention concerns methods and reagents useful in modulating vascular endothelial growth factor (VEGF, VEGF-B, VEGF-C, VEGF-D) and/or vascular endothelial growth factor receptor (e.g., VEGFr1, VEGFr2, and/or VEGFr3) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to 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 VEGF and/or VEGFr gene expression and/or activity. The small nucleic acid molecules are useful in the diagnosis and treatment of cancer, proliferative diseases, and any other disease or condition that responds to modulation of VEGF and/or VEGFr expression or activity.

6. 发明申请

WO2003070896A3 RNA INTERFERENCE MEDIATED INHIBITION OF PROLIFERATING CELL NUCLEAR ANTIGEN (PCNA) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
公开(公告)号：WO2003070896A3
公开(公告)日：2003-08-28
申请号：PCT/US2003/004738
申请日：2003-02-18
申请人： SIRNA THERAPEUTICS, INC , McSWIGGEN, James , CHOWRIRA, Bharat , BEIGELMAN, Leonid
发明人： McSWIGGEN, James , CHOWRIRA, Bharat , BEIGELMAN, Leonid
IPC分类号： C07H21/00
摘要： The present invention concerns methods and reagents useful in modulating proliferating cell nuclear antigen (PCNA) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to 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 PCNA gene expression. The small nucleic acid molecules are useful in the treatment of cancer or restenosis.

7. 发明申请

WO2003070885A3 SHORT INTERFERING NUCLEIC ACID INHIBITION OF STEAROYL-CoA DESATURASE (SCD) GENE 审中-公开
公开(公告)号：WO2003070885A3
公开(公告)日：2003-08-28
申请号：PCT/US2003/004317
申请日：2003-02-13
申请人： SIRNA THERAPEUTICS, INC. , McSWIGGEN, James , BEIGELMAN, Leonid , THOMPSON, James
发明人： McSWIGGEN, James , BEIGELMAN, Leonid , THOMPSON, James
IPC分类号： C07H21/02
摘要： The present invention concerns methods and reagents useful in modulating Stearoyl-CoA desaturase (SCD) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to 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 Steraroyl-CoA desaturase (SCD) gene expression and/or activity. The siNA molecules are useful in the treatment of diabetes (type I and/or type II), atherosclerosis, cancer, obesity, and viral infection, and any other condition that responds to modulation of SCD expression or activity.

8. 发明申请

WO2003070750A3 RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS 审中-公开
公开(公告)号：WO2003070750A3
公开(公告)日：2003-08-28
申请号：PCT/US2003/005043
申请日：2003-02-20
申请人： SIRNA THERAPEUTICS, INC , McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , MORRISSEY, David
发明人： McSWIGGEN, James , BEIGELMAN, Leonid , MACEJAK, Dennis , MORRISSEY, David
IPC分类号： C12Q1/68
摘要： The present invention concerns methods and reagents useful in modulating hepatitis C virus (HCV) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to 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 (RNA/) against hepatitis C virus (HCV) gene expression and/or activity. The small nucleic acid molecules are useful in the treatment and diagnosis of HCV infection, liver failure, hepatocellular carcinoma, cirrhosis and any other disease or condition that responds to modulation of HCV expression or activity.

9. 发明申请

WO2007022369A2 CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID MOLECULES THAT MEDIATE RNA INTERFERENCE 审中-公开
标题翻译：化学修饰短介导核酸介导RNA干扰的核酸分子
公开(公告)号：WO2007022369A2
公开(公告)日：2007-02-22
申请号：PCT/US2006/032168
申请日：2006-08-17
申请人： SIRNA THERAPEUTICS, INC. , MORISEY, David , McSWIGGEN, James , BEIGELMAN, Leonid
发明人： MORISEY, David , McSWIGGEN, James , BEIGELMAN, Leonid
IPC分类号： C12N15/11
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，诊断和治疗响应基因表达和/或活性调节的性状，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因表达途径的基因的表达和/或活性的调节或介导这种维持或发展的其他细胞过程的性状，疾病和病症的化合物，组合物和方法性状，疾病和状况。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）和能够介导针对基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括此类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。

10. 发明申请

WO2003070884A3 RNA INTERFERENCE MEDIATED INHIBITION OF MDR P-GLYCOPROTEIN GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
公开(公告)号：WO2003070884A3
公开(公告)日：2003-08-28
申请号：PCT/US2003/004250
申请日：2003-02-13
申请人： SIRNA THERAPEUTICS, INC. , McSWIGGEN, James , BEIGELMAN, Leonid , THOMPSON, James
发明人： McSWIGGEN, James , BEIGELMAN, Leonid , THOMPSON, James
IPC分类号： C07H21/04
摘要： The present invention concerns methods and reagents useful in modulating MDR P-glycoprotein (MDR) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to 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 MDR gene expression and/or activity. The small nucleic acid molecules are useful in the diagnosis and treatment of cancer, proliferative diseases, and any other disease or condition that responds to modulation of MDR expression or activity.

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