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1. 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。

2. 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.

3. 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。

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

WO2005021800A2 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：WO2005021800A2
公开(公告)日：2005-03-10
申请号：PCT/US2004/026857
申请日：2004-08-17
申请人： SIMA 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，色谱测定等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。和/或电泳测定。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。

6. 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。

7. 发明申请

WO2008011431A2 RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译：干扰素介导的枯草杆菌蛋白酶KEXIN 9（PCSK9）基因表达的短干扰核酸（SINA）抑制作用
公开(公告)号：WO2008011431A2
公开(公告)日：2008-01-24
申请号：PCT/US2007/073723
申请日：2007-07-17
申请人： SIRNA THERAPEUTICS INC. , MCSWIGGEN, James , JADHAV, Vasant , VAISH, Narendra, K. , GUERCIOLINI, Roberto , VARGEESE, Chandra
发明人： MCSWIGGEN, James , JADHAV, Vasant , VAISH, Narendra, K. , GUERCIOLINI, Roberto , VARGEESE, Chandra
IPC分类号： C12N15/11
CPC分类号： C12N15/1137 , C12N2310/14 , 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 Proprotein Convertase Subtilisin Kexin 9 (PCSK9) 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 Proprotein Convertase Subtilisin Kexin 9 (PCSK9) 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 Proprotein Convertase Subtilisin Kexin 9 (PCSK9) 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 metabolic diseases, traits and conditions, including but not limited to hyperlipidemia, hypercholesterolemia, cardiovascular disease, atherosclerosis, hypertension, diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity and/or other disease states, conditions, or traits 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Prop蛋白转化酶枯草杆菌蛋白酶9（PCSK9）基因表达的调节作出响应的性状，疾病和病症的化合物，组合物和方法，和 /或活动。本发明还涉及涉及对Prop蛋白转化酶枯草杆菌蛋白酶Kexin 9（PCSK9）基因表达途径或其他细胞过程中涉及的基因的表达和/或活性的调节作出响应的性状，疾病和病症的化合物，组合物和方法调解这些特征，疾病和状况的维持或发展。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Proprotein转化酶枯草杆菌蛋白酶Kexin 9（PCSK9）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混合物和此类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，抑制或减少代谢疾病，性状和病症，包括但不限于高脂血症，高胆固醇血症，心血管疾病，动脉粥样硬化，高血压，糖尿病（例如类型 I型和/或II型糖尿病），胰岛素抵抗，肥胖和/或与受试者或生物体中的基因表达或活性相关的其他疾病状态，病症或性状。

8. 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。

9. 发明申请

WO2007147143A3 RNA INTERFERENCE MEDIATED INHIBITION OF 11 BETA-HYDROXYSTEROID DEHYDROGENASE-1 (11 BETA-HSD-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
公开(公告)号：WO2007147143A3
公开(公告)日：2007-12-21
申请号：PCT/US2007/071388
申请日：2007-06-15
申请人： SIRNA THERAPEUTICS, INC. , MCSWIGGEN, James , VARGEESE, Chandra , JADHAV, Vasant
发明人： MCSWIGGEN, James , VARGEESE, Chandra , JADHAV, Vasant
IPC分类号： C12N15/11 , C12P19/34 , C07H21/02 , C07H21/04 , A01N43/04
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 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) 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 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) 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 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) 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 11 beta-HSD-1 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 metabolic diseases traits and conditions, including but not limited to diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity, hypercholesterolemia, cardiovascular disease and/or other disease states, conditions, or traits associated with 11 beta-HSD-1 gene expression or activity in a subject or organism.

10. 发明申请

WO2007076328A2 RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS (HCV) GENE 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）基因表达使用短暂的干扰核酸（siNA）
公开(公告)号：WO2007076328A2
公开(公告)日：2007-07-05
申请号：PCT/US2006/062252
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： C12N15/11 , A61K31/713 , A61K9/127
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。

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