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1. 发明申请

WO2007076328A3 RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS 审中-公开
标题翻译： RNA干扰介导的HEPATITIS C病毒的抑制
公开(公告)号：WO2007076328A3
公开(公告)日：2008-08-14
申请号：PCT/US2006062252
申请日：2006-12-18
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , MORRISSEY DAVID , GUERCIOLINI ROBERTO , VARGEESE CHANDRA , JADHAV VASANT
发明人： MCSWIGGEN JAMES , MORRISSEY DAVID , GUERCIOLINI ROBERTO , VARGEESE CHANDRA , JADHAV VASANT
IPC分类号： A61K9/127 , A61K31/713 , C12N15/11 , C12N15/113
CPC分类号： C12N15/1131 , A61K31/713 , A61K47/6911 , C12N15/111 , C12N2310/14 , C12N2310/318 , C12N2310/51 , C12N2770/24211
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 nanoparticlc (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 能够介导针对基因表达的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。

2. 发明专利

AU2006228026A1 RNA interference mediated inhibition of hepatitis C virus (HCV) gene expression using short interfering nucleic acid (siNA) 未知
公开(公告)号：AU2006228026A1
公开(公告)日：2006-11-02
申请号：AU2006228026
申请日：2006-10-11
申请人： SIRNA THERAPEUTICS INC
发明人： MACEJAK DENNIS , MORRISSEY DAVID , BEIGELMAN LEONID , MCSWIGGEN JAMES
IPC分类号： C12N15/11 , A61K38/00

3. 发明专利

AU2003219817B2 RNA interference mediated inhibition of hepatitis C virus 未知
公开(公告)号：AU2003219817B2
公开(公告)日：2006-08-31
申请号：AU2003219817
申请日：2003-02-20
申请人： SIRNA THERAPEUTICS INC
发明人： BEIGELMAN LEONID , MORRISSEY DAVID , MACEJAK DENNIS , MCSWIGGEN JAMES
IPC分类号： C12N15/09 , A61K31/7105 , A61K38/00 , A61K47/48 , A61K48/00 , A61P1/16 , A61P31/14 , A61P35/00 , A61P43/00 , C07H21/02 , C12N15/113 , C12N15/115
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 (RNAi) 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.

5. 发明专利

GB2413557A RNA interference mediated by short nucleic acids containing modified nucleotides 未知
公开(公告)号：GB2413557A
公开(公告)日：2005-11-02
申请号：GB0511689
申请日：2003-02-20
申请人： SIRNA THERAPEUTICS INC
发明人： MCSWIGGEN JAMES , BEIGELMAN LEONID , PAVCO PAMELA , MACEJAK DENNIS , ZINNEN SHAWN , MORRISSEY DAVID , FOSNAUGH KATHY , MOKLER VICTOR , JAMISON SHARON
IPC分类号： A61K48/00 , C07H21/02 , C12N15/11 , C12N15/113 , C12P19/34 , C12Q1/68
摘要： A chemically synthesised double stranded short interfering nucleic acid (siNA) that directs RNA interference (RNAi)wherein at least 10 % of the internal nucleotide positions on at least one strand comprise modified nucleotides. Preferably the molecule is from 19 to 27 nucleotides in length and may or may not include ribonucleotides. The chemical modifications may be to one or more pyrimidines which are 2'-deoxy-2'-fluro nucleotides or 2'-trifluoromethoyy nucleotides or to one or more purine nucleotides which are 2'-O-methyl or 2' deoxy nucleotides. The molecule may be synthesised as two fragments joined by a linker or as a single molecule comprising both sense and antisense regions. Further modified nucleotides may be incorporated into the molecule in the sense as well as the antisense region. Modified nucleotides at the 3' end are also claimed. Pharmaceutical compositions comprising such molecules are also claimed. In preferred embodiments 20, 30 40 or 50 % of the residues may be modified.

6. 发明专利

CA2526831C RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (SINA) 未知
公开(公告)号：CA2526831C
公开(公告)日：2012-07-31
申请号：CA2526831
申请日：2004-05-24
申请人： SIRNA THERAPEUTICS INC
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
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.

7. 发明专利

AU2004266311B2 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) 未知
公开(公告)号：AU2004266311B2
公开(公告)日：2009-07-23
申请号：AU2004266311
申请日：2004-05-24
申请人： SIRNA THERAPEUTICS INC
发明人： MORRISSEY DAVID , JADHAV VASANT , MCSWIGGEN JAMES , ZINNEN SHAWN , VAISH NARENDRA
IPC分类号： A01N43/04 , A61K48/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12P19/34
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 (mRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of VEGF and/or VEGFR genes.

8. 发明专利

CA2633684A1 RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS (HCV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 未知
公开(公告)号：CA2633684A1
公开(公告)日：2007-07-05
申请号：CA2633684
申请日：2006-12-18
申请人： SIRNA THERAPEUTICS INC
发明人： JADHAV VASANT , MCSWIGGEN JAMES , MORRISSEY DAVID , GUERCIOLINI ROBERTO , VARGEESE CHANDRA
IPC分类号： A61K9/127 , A61K31/713 , C12N15/11 , C12N15/113
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.

9. 发明专利

AU2006203725A1 RNA interference by modified short interfering nucleic acid 未知
公开(公告)号：AU2006203725A1
公开(公告)日：2006-09-14
申请号：AU2006203725
申请日：2006-08-25
申请人： SIRNA THERAPEUTICS INC
发明人： ZINNEN SHAWN , MACEJAK DENNIS , JAMISON SHARON , HAEBERLI PETER , BEIGELMAN LEONID , PAVCO PAMELA , MCSWIGGEN JAMES , FOSNAUGH KATHY , MORRISSEY DAVID , MOKLER VICTOR
IPC分类号： C12N15/11 , A61K38/00 , A61K47/48 , C07H21/02

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