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1. 发明申请

WO2010111468A3 RNA INTERFERENCE MEDIATED INHIBITION OF THE NERVE GROWTH FACTOR BETA CHAIN (NGFß) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的神经生长因子β链（NGFß）基因表达抑制短时干扰核酸（SINA）
公开(公告)号：WO2010111468A3
公开(公告)日：2010-11-18
申请号：PCT/US2010028629
申请日：2010-03-25
申请人： MERCK SHARP & DOHME , STRAPPS WALTER , JADHAV VASANT , PICKERING VICTORIA
发明人： STRAPPS WALTER , JADHAV VASANT , PICKERING VICTORIA
IPC分类号： A61K31/712 , A61K31/713 , A61P11/00 , C12N15/113
CPC分类号： C12N15/1136 , A61K31/712 , A61K31/713 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3521 , C12N2310/3533
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 NGFß gene expression and/or activity, and/or modulate a NGFß gene expression pathway. 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 that are capable of mediating or that mediate RNA interference (RNAi) against NGFß gene expression.
摘要翻译：本发明涉及用于研究，诊断和治疗对NGFß基因表达和/或活性的调节和/或调节NGFβ基因表达途径的性状，疾病和病症的化合物，组合物和方法。具体而言，本发明涉及包括小核酸分子（例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA））的双链核酸分子，和能够介导或介导针对NGFβ基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子。

2. 发明申请

WO2010111464A1 RNA INTERFERENCE MEDIATED INHIBITION OF APOPTOSIS SIGNAL-REGULATING KINASE 1 (ASK1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的抑制信号调节激酶1（ASK1）基因表达使用短暂的干扰核酸（siNA）
公开(公告)号：WO2010111464A1
公开(公告)日：2010-09-30
申请号：PCT/US2010/028618
申请日：2010-03-25
申请人： MERCK SHARP & DOHME CORP. , STRAPPS, Walter , JADHAV, Vasant , PICKERING, Victoria
发明人： STRAPPS, Walter , JADHAV, Vasant , PICKERING, Victoria
IPC分类号： C12N15/113 , A61K31/713 , C07H21/00 , A61P11/00
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/344 , C12N2320/32 , C12N2320/51 , C12N2310/3521 , C12N2310/3533 , C12N2310/3531
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 ASK1 gene expression and/or activity, and/or modulate a ASK1 gene expression pathway. 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 that are capable of mediating or that mediate RNA interference (RNAi) against ASK1 gene expression.
摘要翻译：本发明涉及用于研究，诊断和治疗对ASK1基因表达和/或活性的调节作用和/或调节ASK1基因表达途径的性状，疾病和病症的化合物，组合物和方法。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）等双链核酸分子，和能够介导或介导针对ASK1基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子。

3. 发明申请

WO2008137775A3 RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE (PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
公开(公告)号：WO2008137775A3
公开(公告)日：2008-11-13
申请号：PCT/US2008/062548
申请日：2008-05-02
申请人： SIRNA THERAPEUTICS, INC. , STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
发明人： STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
IPC分类号： C12N15/11
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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-7, IL-8, IL- 15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-I, MMP -2, MMP-3, MMP-9 and MMP- 12. 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), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) 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, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), 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 PDE4B 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 are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.

4. 发明申请

WO2010111490A3 RNA INTERFERENCE MEDIATED INHIBITION OF THE THYMIC STROMAL LYMPHOPOIETIN (TSLP) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译：用干扰核酸介导的胸腺基质淋巴因子（TSLP）基因表达的RNA干扰介导的抑制（siNA）
公开(公告)号：WO2010111490A3
公开(公告)日：2010-12-29
申请号：PCT/US2010028664
申请日：2010-03-25
申请人： MERCK SHARP & DOHME , STRAPPS WALTER , JADHAV VASANT , PICKERING VICTORIA
发明人： STRAPPS WALTER , JADHAV VASANT , PICKERING VICTORIA
IPC分类号： C12N15/113
CPC分类号： C12N15/1136 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3521 , C12N2310/3533
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 TSLP gene expression and/or activity, and/or modulate a TSLP gene expression pathway. 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 that are capable of mediating or that mediate RNA interference (RNAi) against TSLP gene expression.
摘要翻译：本发明涉及用于研究，诊断和治疗响应TSLP基因表达和/或活性的调节和/或调节TSLP基因表达途径的性状，疾病和病症的化合物，组合物和方法。具体而言，本发明涉及包括小核酸分子（例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA））的双链核酸分子，和能够介导或介导针对TSLP基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子。

5. 发明申请

WO2010107952A3 RNA INTERFERENCE MEDIATED INHIBITION OF CONNECTIVE TISSUE GROWTH FACTOR (CTGF) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的连接性组织生长因子（CTGF）基因表达的抑制使用短暂的核酸（siNA）
公开(公告)号：WO2010107952A3
公开(公告)日：2010-11-11
申请号：PCT/US2010027721
申请日：2010-03-17
申请人： MERCK SHARP & DOHME , STRAPPS WALTER , PICKERING VICTORIA , SHAH JYOTI
发明人： STRAPPS WALTER , PICKERING VICTORIA
IPC分类号： C12N15/113 , A61K31/713 , A61P11/00 , C07H21/00
CPC分类号： C12N15/1136 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/344 , C12N2320/51 , C12N2310/3521 , C12N2310/3531 , C12N2310/3533
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 CTGF gene expression and/or activity, and/or modulate a CTGF gene expression pathway. 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 that are capable of mediating or that mediate RNA interference (RNAi) against CTGF gene expression.
摘要翻译：本发明涉及用于研究，诊断和治疗对CTGF基因表达和/或活性的调节作用和/或调节CTGF基因表达途径的性状，疾病和病症的化合物，组合物和方法。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）等双链核酸分子，和能够介导或介导针对CTGF基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子。

6. 发明申请

WO2010111503A2 RNA INTERFERENCE MEDIATED INHIBITION OF THE HIGH AFFINITY IGE RECEPTOR ALPHA CHAIN (FCεRLα) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的抑制高亲和力受体ALPHA链（FCeRLa）使用短暂的核酸（SINA）的基因表达
公开(公告)号：WO2010111503A2
公开(公告)日：2010-09-30
申请号：PCT/US2010/028687
申请日：2010-03-25
申请人： MERCK SHARP & DOHME CORP. , STRAPPS, Walter , JADHAV, Vasant , PICKERING, Victoria
发明人： STRAPPS, Walter , JADHAV, Vasant , PICKERING, Victoria
IPC分类号： C12N15/113 , A61K31/713 , C07H21/00 , A61P11/00
CPC分类号： C12N15/1138 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/344 , C12N2310/3515 , C12N2320/51 , C12N2310/3521 , C12N2310/3533 , C12N2310/3531
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 FCεR1α gene expression and/or activity, and/or modulate a FCεR1α gene expression pathway. Specifically, the invention relates to doublestranded 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 that are capable of mediating or that mediate RNA interference (RNAi) against FCεR1α gene expression.
摘要翻译：本发明涉及用于研究，诊断和治疗对FCeR1a基因表达和/或活性的调节和/或调节FCeR1a基因表达途径的性状，疾病和病症的化合物，组合物和方法。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）和短链的双链核酸分子能够介导或介导针对FCeR1a基因表达的RNA干扰（RNAi）的发夹RNA（shRNA）分子。

7. 发明申请

WO2010062817A1 RNA INTERFERENCE MEDIATED INHIBITION OF EPITHELIAL SODIUM CHANNEL (ENaC) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译： RNA干扰介导的上皮干细胞通道（ENaC）基因表达的抑制使用短暂的核酸（siNA）
公开(公告)号：WO2010062817A1
公开(公告)日：2010-06-03
申请号：PCT/US2009/064994
申请日：2009-11-18
申请人： MERCK SHARP & DOHME CORP. , PICKERING, Victoria , STRAPPS, Walter
发明人： PICKERING, Victoria , STRAPPS, Walter
IPC分类号： C12N15/113 , A61K31/713
CPC分类号： C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , 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 ENaC gene expression and/or activity, and/or modulate a ENaC gene expression pathway. 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 that are capable of mediating or that mediate RNA interference (RNAi) against ENaC gene expression.
摘要翻译：本发明涉及用于研究，诊断和治疗响应ENaC基因表达和/或活性的调节和/或调节ENaC基因表达途径的性状，疾病和病症的化合物，组合物和方法。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）等双链核酸分子，和能够介导或介导针对ENaC基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子。

8. 发明申请

WO2008137776A2 RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE (PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的抑制循环核型4型磷酸酯酶（PDE4B）基因表达使用短暂的核酸（SINA）
公开(公告)号：WO2008137776A2
公开(公告)日：2008-11-13
申请号：PCT/US2008/062549
申请日：2008-05-02
申请人： SIRNA THERAPEUTICS, INC. , STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
发明人： STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
IPC分类号： C12N15/09
CPC分类号： C12N15/1137 , C12N2310/14 , C12Y301/04017
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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-I, IL-8, IL- 15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-I, MMP -2, MMP-3, MMP-9 and MMP- 12. 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), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) 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, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), 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 PDE4B 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 are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗响应于环核苷酸4型磷酸二酯酶（PDE4B）基因表达和/或活性的调节的性状，疾病和病症的化合物，组合物和方法，包括PDE4B1，PDE4B2 ，和PDE4B3基因表达和/或活性。本发明还涉及涉及与参与环核苷酸4型磷酸二酯酶（PDE4B）基因表达途径或其他细胞过程的基因的表达和/或活性的调节相关的性状，疾病和病症的化合物，组合物和方法包括但不限于IL-6，IL-1，IL-8，IL-15，TNF-α和基质金属蛋白酶（MMP），如MMP-1 ，MMP-2，MMP-3，MMP-9和MMP-12。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）和短发夹RNA（shRNA），以及能够介导针对环核苷酸4型磷酸二酯酶（PDE4B）基因表达的RNA干扰（RNAi）的多功能siNA分子，包括鸡尾酒的这种小核酸分子的这种小核酸分子和脂质纳米颗粒（LNP）制剂。本发明还涉及可以抑制内源性RNA分子或RNAi途径组分（RNAi抑制剂）如内源性微RNA（miRNA）的功能的小核酸分子，例如siNA，siRNA，反义寡核苷酸和其它， miRNA抑制剂）或内源性短干扰RNA（siRNA）（例如siRNA抑制剂）或可以抑制RISC功能（如RISC抑制剂）调节PDE4B基因表达的干扰该内源性RNA或蛋白质的调控功能与这样的内源性RNA（例如，RISC）相关联，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。这样的小核酸分子可用于例如提供预防，抑制或减少与受试者中PDE4B基因表达或活性相关的炎症，呼吸和自身免疫性疾病，性状和病症和/或其它疾病状态的组合物或生物体。

9. 发明申请

WO2008137751A2 RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE (PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译： RNA干扰介导的环核苷酸4型磷酸二酯酶（PDE4B）基因表达的短期干扰核酸（SINA）抑制作用
公开(公告)号：WO2008137751A2
公开(公告)日：2008-11-13
申请号：PCT/US2008/062512
申请日：2008-05-02
申请人： SIRNA THERAPEUTICS, INC. , STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
发明人： STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
IPC分类号： C12N15/09
CPC分类号： C12N15/1137 , A01K2207/05 , A01K2227/105 , A01K2267/0368 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/332 , 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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 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 cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-I, IL-8, IL- 15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-I, MMP -2, MMP-3, MMP-9 and MMP- 12. 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), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) 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, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), 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 PDE4B 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 are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗响应环核苷酸4型磷酸二酯酶（PDE4B）基因表达的调节的性状，疾病和病症的化合物，组合物和方法，和 /或活性，包括PDE4B1，PDE4B2和PDE4B3基因的表达和/或活性。本发明还涉及与响应参与环核苷酸4型磷酸二酯酶（PDE4B）基因表达途径或其他细胞过程的基因的表达和/或活性的调节有关的性状，疾病和病症的化合物，组合物和方法其介导了包括但不限于IL-6，IL-1，IL-8，IL-15，TNF-α和基质金属蛋白酶（MMP）如MMP-1在内的这些性状，疾病和病症的维持或发展，MMP-2，MMP-3，MMP-9和MMP-12。特别地，本发明涉及双链核酸分子，包括小核酸分子，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），以及能够介导针对环核苷酸4型磷酸二酯酶（PDE4B）基因表达的RNA干扰（RNAi）的多功能siNA分子，包括鸡尾酒的这种小核酸分子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RNA分子或RNAi途径组分（RNAi抑制剂）的功能的小核酸分子，例如siNA，siRNA，反义和其它，例如内源性微RNA（miRNA）（例如， miRNA抑制剂）或内源性短干扰RNA（siRNA）（例如siRNA抑制剂）或可以抑制RISC功能（例如RISC抑制剂）通过干扰这种内源性RNA或蛋白质的调节功能来调节PDE4B基因表达与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）制剂的内源RNA（例如RISC）相关。例如，这种小核酸分子可用于提供组合物以预防，抑制或减轻与受试者中的PDE4B基因表达或活性相关的炎症，呼吸和自身免疫疾病，性状和/或病症和/或其他疾病状态或有机体。

10. 发明申请

WO2012018754A2 RNA INTERFERENCE MEDIATED INHIBITION OF CATENIN (CADHERIN-ASSOCIATED PROTEIN), BETA 1 (CTNNB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
标题翻译：利用短干扰核酸（siNA）RNA干扰介导的抑制素（钙依敏蛋白相关蛋白），β1（CTNNB1）基因表达
公开(公告)号：WO2012018754A2
公开(公告)日：2012-02-09
申请号：PCT/US2011046178
申请日：2011-08-02
申请人： MERCK SHARP & DOHME , BROWN DUNCAN , CUNNINGHAM JAMES J , GINDY MARIAN , PICKERING VICTORIA , STANTON MATTHEW G , STIRDIVANT STEVEN M , STRAPPS WALTER
发明人： BROWN DUNCAN , CUNNINGHAM JAMES J , GINDY MARIAN , PICKERING VICTORIA , STANTON MATTHEW G , STIRDIVANT STEVEN M , STRAPPS WALTER
IPC分类号： A61K48/00
CPC分类号： C12N15/1138 , C12N15/113 , C12N2310/14 , C12N2320/32
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 CTNNB1 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. 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 that are capable of mediating or that mediate RNA interference (RNAi) against CTNNB1 gene expression.
摘要翻译：本发明涉及用于研究，诊断和治疗对CTNNB1基因表达和/或活性的调节和/或调节β-连环蛋白基因表达途径的性状，疾病和病症的化合物，组合物和方法。具体而言，本发明涉及包括小核酸分子（例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA））的双链核酸分子，和能够介导或介导针对CTNNB1基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子。

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