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1. 发明专利

AU2003215345A1 RNA INTERFERENCE MEDIATED INHIBITION OF HIV GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 未知
公开(公告)号：AU2003215345A1
公开(公告)日：2003-09-09
申请号：AU2003215345
申请日：2003-02-20
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES
发明人： BEIGELMAN LEONID , MACEJAK DENNIS , MCSWIGGEN JAMES
IPC分类号： A61K31/7088 , A61K31/712 , A61K31/713 , A61K38/00 , A61K45/06 , A61P31/18 , C07H21/00 , C12N15/11 , C12N15/113
摘要： The present invention concerns methods and reagents useful in modulating HIV gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small interfering RNA (siRNA) molecules capable of mediating RNA interference (RNAi) against HIV polypeptide and polynucleotide targets.

2. 发明申请

WO2005045036A3 RNA INTERFERENCE MEDIATED INHIBITION OF HAIRLESS (HR) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的使用短暂干扰核酸（SINA）的无毛（HR）基因表达的抑制
公开(公告)号：WO2005045036A3
公开(公告)日：2006-03-02
申请号：PCT/US2004027042
申请日：2004-08-18
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES
发明人： MCSWIGGEN JAMES
IPC分类号： A01N43/04 , A61K31/713 , A61K48/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12P19/34
CPC分类号： C12N15/113 , C12N2310/14
摘要： This invention relates to compounds, compositions, and methods useful for modulating Hairless (HR) 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 Hairless 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 Hairless.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节无毛（HR）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涉及无毛基因表达和/或通过RNA干扰（RNAi）的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））分子和方法用于调节无毛的表达。

3. 发明申请

WO2005045038A8 RNA INTERFERENCE MEDIATED INHIBITION OF GPRA AND AAA1 GENE EXPRESSION USING SHORT NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的GPRA和AAA1基因表达的抑制使用短核酸（siNA）
公开(公告)号：WO2005045038A8
公开(公告)日：2005-07-14
申请号：PCT/US2004027231
申请日：2004-08-20
申请人： SIRNA THERAPEUTICS INC , RICHARDS IVAN , POLISKY BARRY , MCSWIGGEN JAMES
发明人： RICHARDS IVAN , POLISKY BARRY , MCSWIGGEN JAMES
IPC分类号： A01N43/04 , A61K31/713 , A61K38/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12N15/87 , C12P19/34 , C12N15/11
CPC分类号： C12N15/87 , A61K38/00 , A61K49/0008 , C12N15/113 , C12N15/1138 , C12N2310/111 , C12N2310/12 , C12N2310/121 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating G protein-coupled receptor for asthma susceptibility (GPRA) and asthma-associated alternatively spliced gene 1 (AAA1) 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 GPRA and/or AAA1 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 GPRA and/or AAA1 genes.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节G蛋白偶联受体哮喘易感性（GPRA）和哮喘相关性剪接基因1（AAA1）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涉及GPRA和/或AAA1基因表达和/或活性通过RNA干扰（RNAi）的途径的其它基因的表达和活性的方法。特别地，本发明具有小的核酸分子，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GPRA和/或AAA1基因表达的方法。

4. 发明申请

WO03070914A2 RNA INTERFERENCE MEDIATED INHIBITION OF FOS GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID siNA 审中-公开
标题翻译： RNA干扰介导的抑制FOS基因表达使用短暂的干扰核酸siNA
公开(公告)号：WO03070914A2
公开(公告)日：2003-08-28
申请号：PCT/US0305162
申请日：2003-02-20
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , BEIGELMAN LEONID
发明人： MCSWIGGEN JAMES , BEIGELMAN LEONID
IPC分类号： C12N15/09 , A61K31/7088 , A61K31/7105 , A61K31/711 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61K31/713 , A61K38/00 , A61K47/48 , A61K48/00 , A61P1/00 , A61P1/04 , A61P1/16 , A61P3/00 , A61P3/10 , A61P11/06 , A61P13/08 , A61P13/10 , A61P13/12 , A61P17/00 , A61P17/02 , A61P19/00 , A61P19/02 , A61P21/00 , A61P25/00 , A61P25/02 , A61P25/28 , A61P27/02 , A61P27/16 , A61P29/00 , A61P31/00 , A61P31/04 , A61P31/10 , A61P31/12 , A61P31/14 , A61P31/16 , A61P31/18 , A61P31/20 , A61P31/22 , A61P35/00 , A61P35/02 , A61P37/00 , A61P37/04 , A61P37/06 , A61P37/08 , A61P43/00 , C07H21/02 , C07H21/04 , C12N15/11 , C12N15/113 , C12N15/82 , 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 , C12N2310/344 , C12N2310/346 , C12N2310/53 , C12N2320/51 , C12N2330/30 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating c-fos gene expression in a variety of applications, including use of 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 c-fos gene expression and/ 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 c-fos expression or activity.
摘要翻译：本发明涉及可用于调节各种应用中的c-fos基因表达的方法和试剂，包括使用治疗，诊断，靶标验证和基因组发现应用。具体地说，本发明涉及能够介导RNA干扰RNAi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， fos基因表达和/活性。小核酸分子可用于诊断和治疗癌症，增殖性疾病以及对c-fos表达或活性调节作出反应的任何其它疾病或病症。

5. 发明申请

WO2006128141A3 RNA INTERFERENCE MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR-1 (SDF-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的使用短暂干扰核酸（SINA）的细胞衍生因子-1（SDF-1）基因表达的抑制作用
公开(公告)号：WO2006128141A3
公开(公告)日：2007-07-12
申请号：PCT/US2006020846
申请日：2006-05-26
申请人： SIRNA THERAPEUTICS INC , GUERCIOLINI ROBERTO , MCSWIGGEN JAMES , JADHAV VASANT , VARGEESE CHANDRA
发明人： GUERCIOLINI ROBERTO , MCSWIGGEN JAMES , JADHAV VASANT , VARGEESE CHANDRA
IPC分类号： C07K14/52 , C12N15/113
CPC分类号： C12N15/1136 , C12N2310/14 , C12N2310/315 , 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 stromal cell-derived factor- 1 (SDF-I) 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 stromal cell-derived factor- 1 (SDF-I) 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 stromal cell- derived factor- 1 (SDF-I) 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 SDF-I 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 angiogenesis related diseases traits and conditions, including but not limited to ocular disease, cancer and proliferative diseases, traits, and conditions, and/or other disease states, conditions, or traits associated with SDF-I gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗对基质细胞衍生因子-1（SDF-1）基因表达和/或活性的调节作用的性状，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质细胞衍生的因子-1（SDF-1）基因表达途径的基因的表达和/或活性的调节作用的性状，疾病和病症有关的化合物，组合物和方法或介导这些性状，疾病和状况的维持或发展的其他细胞过程。具体地说，本发明涉及包括小核酸分子的双链核酸分子，如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和能够介导针对基质细胞衍生因子-1（SDF-1）基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括这种小核酸的这种小核酸分子和脂质纳米颗粒（LNP）制剂的混合物酸分子。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）或可以抑制RISC功能（例如RISC抑制剂）通过干扰与这些内源性RNA相关的内源性RNA或蛋白质的调节功能来调节SDF-1基因表达（例如，，RISC），包括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。这样的小核酸分子并且可用于例如提供预防，抑制或减少血管生成相关疾病性状和病症的组合物，包括但不限于眼部疾病，癌症和增殖性疾病，性状和病症，和/或与受试者或生物体内的SDF-1基因表达或活性相关的其它疾病状态，病症或性状。

6. 发明申请

WO2006078798A2 RNA INTERFERENCE MEDIATED INHIBITION OF RETINOBLASTOMA (RBI) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的使用短暂干扰核酸（siNA）的雷公藤内酯（RBI）基因表达的抑制
公开(公告)号：WO2006078798A2
公开(公告)日：2006-07-27
申请号：PCT/US2006001863
申请日：2006-01-18
申请人： SIRNA THERAPEUTICS INC , CHRISTIANO ANGELA , MCSWIGGEN JAMES
发明人： CHRISTIANO ANGELA , MCSWIGGEN JAMES
IPC分类号： C12N15/113
CPC分类号： C12N15/1135 , C12N2310/14
摘要： This invention relates to compounds, compositions, and methods useful for modulating retinoblastoma (RBl) 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 retinoblastoma 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 retinoblastoma genes. Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing hearing loss, deafness, tinnitus, motion and balance disorders, cancer and proliferative diseases and any other disease, condition, trait or indication that can respond to the level of retinoblastoma in a cell or tissue, alone or in combination with other treatments or therapies.
摘要翻译：本发明涉及使用短干扰核酸（siNA）分子调节视网膜母细胞瘤（RBI）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视网膜母细胞瘤基因表达和/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））分子和用于调节视网膜母细胞瘤基因表达的方法。这样的小核酸分子可用于例如治疗，预防，抑制或降低听力损失，耳聋，耳鸣，运动和平衡障碍，癌症和增殖性疾病以及任何其他可以响应的疾病，病症，特征或指征视网膜母细胞瘤在细胞或组织中的水平，单独或与其他治疗或疗法组合。

7. 发明申请

WO2005035759A3 RNA interference mediated inhibition of hypoxia inducible factor 1 (HIF1) gene expression using sho rt interfering nucleic acid (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制低氧诱导因子1（HIF1）基因表达使用干扰核酸（siNA）
公开(公告)号：WO2005035759A3
公开(公告)日：2006-03-16
申请号：PCT/US2004027294
申请日：2004-08-20
申请人： USMAN NASSIM , MCSWIGGEN JAMES , SIRNA THERAPEUTICS INC
发明人： USMAN NASSIM , MCSWIGGEN JAMES
IPC分类号： A61K38/00 , C12N15/11 , C12N15/113 , C12N15/115 , C12N15/87 , A01N43/04 , C07H21/02 , C07H21/04 , C12P19/34
CPC分类号： C12N15/1137 , A61K38/00 , C12N15/111 , C12N15/113 , C12N15/1138 , C12N15/115 , C12N15/87 , C12N2310/111 , C12N2310/12 , C12N2310/121 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2330/30 , C12Y103/01022 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating hyproxia inducable factor (e.g., HIF1) 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 HIF1 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 HIF1 genes.

8. 发明申请

WO2005040379A3 RNA INTERFERENCE MEDIATED INHIBITION OF RAS GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的RAS基因表达的抑制使用短暂的干扰核酸（siNA）
公开(公告)号：WO2005040379A3
公开(公告)日：2006-03-02
申请号：PCT/US2004027333
申请日：2004-08-20
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES
发明人： MCSWIGGEN JAMES
IPC分类号： A61K38/00 , C12N5/02 , C12N5/22 , C12N15/11 , C12N15/113 , C12N15/87 , A01N43/04 , A61K31/713 , C07H21/02 , C07H21/04 , C12P19/34
CPC分类号： A61K49/0008 , C12N15/111 , C12N15/1135 , C12N15/87 , C12N2310/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2320/32 , C12N2330/30 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating RAS, e.g. K-RAS, H-RAS, and/or N-RAS 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 RAS, e.g. K-RAS, H-RAS, and/or N-RAS 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 RAS genes, such as K-RAS, H-RAS, and/or N-RAS.
摘要翻译：本发明涉及可用于调节RAS的化合物，组合物和方法，例如。使用短干扰核酸（siNA）分子的K-RAS，H-RAS和/或N-RAS基因表达。本发明还涉及可用于调节参与RAS途径的其它基因的表达和活性的化合物，组合物和方法，例如。使用小核酸分子的RNA干扰（RNAi）的K-RAS，H-RAS和/或N-RAS基因表达和/或活性。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RAS基因如K-RAS，H-RAS和/或N-RAS表达的方法。

9. 发明申请

WO2005035759A8 RNA INTERFERENCE MEDIATED INHIBITION OF HYPOXIA INDUCIBLE FACTOR 1 (HIF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制HYPOXIA诱导因子1（HIF1）使用短暂的核酸（siNA）的基因表达
公开(公告)号：WO2005035759A8
公开(公告)日：2006-02-02
申请号：PCT/US2004027294
申请日：2004-08-20
申请人： SIRNA THERAPEUTICS INC , USMAN NASSIM , MCSWIGGEN JAMES
发明人： USMAN NASSIM , MCSWIGGEN JAMES
IPC分类号： A61K38/00 , C12N15/11 , C12N15/113 , C12N15/115 , C12N15/87 , A01N43/04 , C07H21/02 , C07H21/04 , C12P19/34
CPC分类号： C12N15/1137 , A61K38/00 , C12N15/111 , C12N15/113 , C12N15/1138 , C12N15/115 , C12N15/87 , C12N2310/111 , C12N2310/12 , C12N2310/121 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/346 , C12N2310/53 , C12N2330/30 , C12Y103/01022 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating hyproxia inducable factor (e.g., HIF1) 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 HIF1 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 HIF1 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hypoxia诱导因子（例如，HIF1）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HIF1基因表达和/或通过RNA干扰（RNAi）的活性的其它基因的其他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HIF1基因表达的方法。

10. 发明申请

WO2005045037B1 RNA INTERFERENCE MEDIATED INHIBITION OF 5-ALPHA REDUCTASE AND ANDROGEN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA介导的介导5-α还原酶和雄激素受体基因表达的短期干扰核酸（siNA）抑制作用
公开(公告)号：WO2005045037B1
公开(公告)日：2005-12-29
申请号：PCT/US2004027169
申请日：2004-08-20
申请人： SIRNA THERAPEUTICS INC , MCSWIGGEN JAMES , VAISH NARENDRA
发明人： MCSWIGGEN JAMES , VAISH NARENDRA
IPC分类号： A61K38/00 , C12N15/113 , C12N15/11 , A01N43/04 , A61K31/713 , C07H21/02 , C07H21/04 , C12P19/34
CPC分类号： C12N15/113 , A61K38/00 , 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 , C12N2310/53 , C12Y103/01022 , C12N2310/3521
摘要： This invention relates to compounds, compositions, and methods useful for modulating 5-alpha reductase (SRD5A-1, SRD5A-2) and androgen receptor (AR) 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 5-alpha reductase and androgen receptor (AR) 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 5-alpha reductase and androgen receptor (AR) 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5-α还原酶（SRD5A-1，SRD5A-2）和雄激素受体（AR）基因表达的化合物，组合物和方法。本发明还涉及用于使用小核酸调节参与5-α还原酶和雄激素受体（AR）基因表达和/或RNA干扰活性（RNAi）的活性的其他基因的表达和活性的化合物，组合物和方法酸分子。特别地，本发明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5-α还原酶和雄激素受体（AR）基因表达的方法。

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