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1.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING BIRC5 GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制BIRC5基因表达的核酸化合物及其用途公开(公告)号：US20110236972A1
公开(公告)日：2011-09-29
申请号：US13057741
申请日：2009-08-05
CPC分类号： C12N15/1135 , C12N15/113 , C12N2310/11 , C12N2310/14
摘要： The present disclosure provides RNA molecules, for example, meroduplex ribonucleic acid molecules (mdRNA), capable of decreasing or silencing BIRC5 gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a BIRC5 mRNA. Also provided are methods of decreasing expression of a BIRC5 gene in a cell or in a subject to treat a BIRC5-related disease.
摘要翻译： 本公开提供了能够降低或沉默BIRC5基因表达的RNA分子，例如meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其组合形成由切口或间隙隔开的至少两个不重叠的双链区域，其中一条链与BIRC5 mRNA互补。 还提供减少细胞或受试者中BIRC5基因表达以治疗BIRC5相关疾病的方法。
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2.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING MYC GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制MYC基因表达的核酸化合物及其用途公开(公告)号：US20100055782A1
公开(公告)日：2010-03-04
申请号：US12529138
申请日：2008-03-03
CPC分类号： C12N15/1135 , C12N2310/14
摘要： The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing MYC gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-over-lapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a MYC mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside replaced with a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a MYC gene in a cell or in a subject to treat a MYC-related disease.
摘要翻译： 本公开提供能够降低或沉默MYC基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其组合形成由切口或间隙分开的至少两个非重叠双链区域，其中一条链与MYC mRNA互补。 此外，meroduplex可以具有至少一个用5-甲基尿苷取代的尿苷，被锁定的核酸替代的核苷，或任选的其它修饰，以及它们的任何组合。 还提供减少细胞或受试者中MYC基因表达以治疗MYC相关疾病的方法。
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3.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING BCL2 GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制BCL2基因表达的核酸化合物及其用途公开(公告)号：US20100041140A1
公开(公告)日：2010-02-18
申请号：US12529011
申请日：2008-02-29
CPC分类号： C12N15/1135 , C12N2310/14 , C12N2310/53
摘要： The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing BCL2 gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-over-lapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a BCL2 mRNA. In addition, the meroduplex may have at least one uridine is a 5-methyluridine, a nucleoside is a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a BCL2 gene in a cell or in a subject to treat a BCL2-related disease.
摘要翻译： 本公开提供能够降低或沉默BCL2基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其组合形成由切口或间隙分开的至少两个非重叠双链区域，其中一条链与BCL2 mRNA互补。 此外，meroduplex可以具有至少一个尿苷是5-甲基尿苷，核苷是锁定的核酸或任选的其它修饰，以及它们的任何组合。 还提供减少细胞或受试者中BCL2基因表达以治疗BCL2相关疾病的方法。
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4.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING RAS GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制RAS基因表达的核酸化合物及其用途公开(公告)号：US20100055783A1
公开(公告)日：2010-03-04
申请号：US12529302
申请日：2008-03-03
CPC分类号： C12N15/1135 , C12N2310/14 , C12N2310/321 , C12N2310/3231 , C12N2310/33 , C12N2310/53 , C12N2310/3521
摘要： The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing RAS (e.g., HRAS, KRAS, NRAS) gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to an HRAS, KRAS, or NRAS mRNA. In addition, the meroduplex may have at least one uridine is a 5-methyluridine, a nucleoside is a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a RAS gene in a cell or in a subject to treat a RAS-related disease.
摘要翻译： 本公开提供能够降低或沉默RAS（例如，HRAS，KRAS，NRAS）基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其组合形成由切口或间隙隔开的至少两个不重叠的双链区域，其中一条链与HRAS，KRAS或NRAS mRNA互补。 此外，meroduplex可以具有至少一个尿苷是5-甲基尿苷，核苷是锁定的核酸或任选的其它修饰，以及它们的任何组合。 还提供减少细胞或受试者中RAS基因表达以治疗RAS相关疾病的方法。
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5.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING APOB GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制APOB基因表达的核酸化合物及其用途公开(公告)号：US20080299659A1
公开(公告)日：2008-12-04
申请号：US12039668
申请日：2008-02-28
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/3231
摘要： The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing ApoB gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to an ApoB mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside replaced with a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of an ApoB gene in a cell or in a subject to treat an ApoB-related disease.
摘要翻译： 本公开提供能够降低或沉默ApoB基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其组合形成由切口或间隙分开的至少两个不重叠的双链区域，其中一条链与ApoB mRNA互补。 此外，meroduplex可以具有至少一个用5-甲基尿苷取代的尿苷，被锁定的核酸替代的核苷，或任选的其它修饰，以及它们的任何组合。 还提供减少细胞或受试者中ApoB基因表达以治疗ApoB相关疾病的方法。
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6.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING AKT GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制AKT基因表达的核酸化合物及其用途公开(公告)号：US20080293136A1
公开(公告)日：2008-11-27
申请号：US12039662
申请日：2008-02-28
CPC分类号： C12N15/1137 , C12N2310/111 , C12N2310/14 , C12N2310/321 , C12N2310/3231 , C12N2310/53 , C12N2310/3521
摘要： The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing AKT gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to an AKT mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside replaced with a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of an AKT gene in a cell or in a subject to treat an AKT-related disease.
摘要翻译： 本公开提供能够降低或沉默AKT基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其结合形成由切口或间隙分开的至少两个不重叠的双链区域，其中一条链与AKT mRNA互补。 此外，meroduplex可以具有至少一个用5-甲基尿苷取代的尿苷，被锁定的核酸替代的核苷，或任选的其它修饰，以及它们的任何组合。 还提供减少细胞或受试者中AKT基因表达以治疗AKT相关疾病的方法。
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7.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING VEGF GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制VEGF基因表达的核酸化合物及其用途公开(公告)号：US20080286866A1
公开(公告)日：2008-11-20
申请号：US12039650
申请日：2008-02-28
CPC分类号： C12N15/1136 , C12N2310/14
摘要： The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing VEGFA, VEGFB, VEGFC, FIGF, or PGF gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a VEGF mRNA. In addition, the meroduplex may have at least one 5-methyluridine, locked nucleic acid, 2′-O-methyl, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a VEGF gene in a cell or in a subject to treat a VEGF-related disease.
摘要翻译： 本公开提供能够降低或沉默VEGFA，VEGFB，VEGFC，FIGF或PGF基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其组合形成由切口或间隙隔开的至少两个不重叠的双链区域，其中一条链与VEGF mRNA互补。 此外，meroduplex可以具有至少一个5-甲基尿苷，锁定核酸，2'-O-甲基或任选的其它修饰及其任何组合。 还提供减少细胞或受试者中VEGF基因表达以治疗VEGF相关疾病的方法。
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8.发明申请公开(公告)号：US20100105134A1
公开(公告)日：2010-04-29
申请号：US12552082
申请日：2009-09-01
CPC分类号： C12N15/111 , C12N2310/14 , C12N2310/533
摘要： The present disclosure provides RNA molecules, for example, meroduplex ribonucleic acid molecules (mdRNA), and blunt ended double-stranded ribonucleic acid molecules capable of decreasing or silencing expression of a target gene. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a target mRNA. Also provided are methods of decreasing expression of a target gene in a cell or in a subject to treat a disease or condition associated with the target gene.
摘要翻译： 本公开提供了RNA分子，例如，meroduplex核糖核酸分子（mdRNA）和能够降低或沉默靶基因表达的平端双链核糖核酸分子。 本公开的mdRNA包含至少三条链，其结合形成由切口或间隙分开的至少两个不重叠的双链区域，其中一条链与靶mRNA互补。 还提供减少细胞或受试者中靶基因表达以治疗与靶基因相关的疾病或病症的方法。
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9.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING HIF1A GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制HIF1A基因表达的核酸化合物及其用途公开(公告)号：US20100015706A1
公开(公告)日：2010-01-21
申请号：US12528786
申请日：2008-02-28
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/3231 , C12N2310/53
摘要： The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing HIF1A gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a HIF1A mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside is a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a HIF1A gene in a cell or in a subject to treat a HIF1A-related disease.
摘要翻译： 本公开提供能够降低或沉默HIF1A基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其组合形成由切口或间隙分开的至少两个不重叠的双链区域，其中一条链与HIF1A mRNA互补。 此外，meroduplex可以具有至少一个被5-甲基尿苷取代的尿苷，核苷是锁定的核酸，或任选的其它修饰及其任何组合。 还提供减少细胞或受试者中HIF1A基因表达以治疗HIF1A相关疾病的方法。
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10.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING ERBB FAMILY GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制ERBB家族基因表达的核酸化合物及其用途公开(公告)号：US20100112687A1
公开(公告)日：2010-05-06
申请号：US12528619
申请日：2008-02-28
CPC分类号： C12N15/1138 , C12N2310/14 , C12N2310/321 , C12N2310/3231 , C12N2310/3521
摘要： The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing one or more ERBB family gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to one or more ERBB family mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine and optionally other modifications or combinations thereof. Also provided are methods of decreasing expression of one or more ERBB family gene in a cell or in a subject to treat one or more ERBB family-related disease.
摘要翻译： 本公开提供能够降低或沉默一种或多种ERBB家族基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其结合形成由切口或间隙分开的至少两个不重叠的双链区域，其中一条链与一个或多个ERBB家族mRNA互补。 此外，meroduplex可以具有至少一个被5-甲基尿苷取代的尿苷和任选的其它修饰或组合。 还提供了降低细胞或受试者中一种或多种ERBB家族基因表达以治疗一种或多种ERBB家族相关疾病的方法。
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