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1.发明申请NUCLEIC ACID COMPOUNDS WITH CONFORMATIONALLY RESTRICTED MONOMERS AND USES THEREOF 审中-公开具有一致限制性单体的核酸化合物及其用途公开(公告)号：US20130190383A1
公开(公告)日：2013-07-25
申请号：US13643180
申请日：2011-04-26
申请人： Narendra K. Vaish , Kathy L. Fosnaugh , Shaguna Seth , Michael E. Houston, JR. , Michael V. Templin
发明人： Narendra K. Vaish , Kathy L. Fosnaugh , Shaguna Seth , Michael E. Houston, JR. , Michael V. Templin
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1135 , C12N15/1136 , C12N15/1137 , C12N15/1138 , C12N2310/11 , C12N2310/14 , C12N2310/3231 , C12N2310/533 , C12N2320/30 , C12N2320/51 , C12Y103/99005 , C12Y203/0102 , C12Y207/01153 , C12Y207/11001 , C12Y207/11021 , C12Y207/11024 , C12Y304/21021
摘要： This disclosure provides single-stranded and multi-stranded nucleic acid compounds having one or more double-stranded regions that regulate the function or expression of nucleic acid molecules expressed in a cell or a cell regulatory system dependent upon a nucleic acid. The disclosure provides a range of nucleic acid compounds having one or more conformationally restricted nucleomonomers (CRN). Certain nucleic acid compounds may have one or more conformationally restricted nucleomonomers and one or more hydroxymethyl substituted nucleomonomers (UNA). The nucleic acid compounds are useful in various therapeutic modalities.
摘要翻译： 本公开提供具有调节在细胞中表达的核酸分子的功能或表达的一个或多个双链区域的单链和多链核酸化合物或依赖于核酸的细胞调节系统。 本公开提供了一系列具有一个或多个构象限制性核单体（CRN）的核酸化合物。 某些核酸化合物可以具有一个或多个构象限制的核小体单体和一个或多个羟甲基取代的核子单体（UNA）。 核酸化合物可用于各种治疗方式。
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2.发明申请NUCLEIC ACID COMPOUNDS FOR INHIBITING PLK1 GENE EXPRESSION AND USES THEREOF 审中-公开用于抑制PLK1基因表达的核酸化合物及其用途公开(公告)号：US20110136233A1
公开(公告)日：2011-06-09
申请号：US13057743
申请日：2009-08-05
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/32 , C12N2310/533
摘要： The present disclosure provides RNA molecules, for example, meroduplex ribonucleic acid molecules (mdRNA), capable of decreasing or silencing gene expression of PLK1 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 PLK1 mRNA. Also provided are methods of decreasing expression of a PLK1 gene in a cell or in a subject to treat a PLK family member-related disease.
摘要翻译： 本公开提供了能够降低或沉默PLK1基因的基因表达的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分开的至少两个不重叠的双链区域，其中一条链与PLK1mRNA互补。 还提供减少细胞或受试者中PLK1基因表达以治疗PLK家族成员相关疾病的方法。
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3.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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公开(公告)号：US09340789B2
公开(公告)日：2016-05-17
申请号：US13130032
申请日：2009-12-03
IPC分类号： C07H21/04 , C12N15/113 , C07H21/00 , C12N15/11
CPC分类号： C12N15/1138 , C12N15/113 , C12N15/1131 , C12N2310/14 , C12N2310/32 , C12N2310/323 , C12N2320/30
摘要： This disclosure provides double-stranded RNA complexes having one or more hydroxymethyl substituted nucleomonomer(s) in the passenger strand (or sense strand) of an RNA complex. RNA complexes of the disclosure may be useful for therapeutic applications, diagnostic applications or research applications. RNA complexes include short interfering RNA complexes (siRNA) capable of modulating gene expression comprising an antisense strand and a continuous or a discontinuous passenger strand (“sense strand”). Further, one or more hydroxymethyl substituted nucleomonomer(s) of this disclosure may be positioned at the 3′-end, at the 5′-end, at both the 3′-end and 5′end.
摘要翻译： 本公开提供了在RNA复合物的乘客链（或有义链）中具有一个或多个羟甲基取代的核小体单体的双链RNA复合物。 本公开的RNA复合物可用于治疗应用，诊断应用或研究应用。 RNA复合物包括能够调节包含反义链和连续或不连续乘客链（“有义链”）的基因表达的短干扰RNA复合物（siRNA）。 此外，本公开的一个或多个羟甲基取代的核小体单体可以位于5'末端的3'末端和5'末端的3'末端。
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公开(公告)号：US20110313020A1
公开(公告)日：2011-12-22
申请号：US13130032
申请日：2009-12-03
IPC分类号： A61K31/713 , C07H21/00 , A61P29/00 , A61P3/06 , A61P25/00 , A61P19/08 , A61P9/00 , A61P31/16 , A61P31/12 , A61P35/00 , C12N5/071 , A61P1/16
CPC分类号： C12N15/1138 , C12N15/113 , C12N15/1131 , C12N2310/14 , C12N2310/32 , C12N2310/323 , C12N2320/30
摘要： This disclosure provides double-stranded RNA complexes having one or more hydroxymethyl substituted nucleomonomer(s) in the passenger strand (or sense strand) of an RNA complex. RNA complexes of the disclosure may be useful for therapeutic applications, diagnostic applications or research applications. RNA complexes include short interfering RNA complexes (siRNA) capable of modulating gene expression comprising an antisense strand and a continuous or a discontinuous passenger strand (“sense strand”). Further, one or more hydroxymethyl substituted nucleomonomer(s) of this disclosure may be positioned at the 3′-end, at the 5′-end, at both the 3′-end and 5′end.
摘要翻译： 本公开提供了在RNA复合物的乘客链（或有义链）中具有一个或多个羟甲基取代的核小体单体的双链RNA复合物。 本公开的RNA复合物可用于治疗应用，诊断应用或研究应用。 RNA复合物包括能够调节包含反义链和连续或不连续乘客链（“有义链”）的基因表达的短干扰RNA复合物（siRNA）。 此外，本公开的一个或多个羟甲基取代的核小体单体可以位于5'末端的3'末端和5'末端的3'末端。
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6.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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7.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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8.发明申请公开(公告)号：US20100015708A1
公开(公告)日：2010-01-21
申请号：US12486889
申请日：2009-06-18
申请人： Steven C. Quay , Narendra K. Vaish
发明人： Steven C. Quay , Narendra K. Vaish
CPC分类号： C07H21/02
摘要： The present disclosure provides a ribonucleic acid comprising a double-stranded region having at least one base pair comprising a 5-methyluridine base paired with a 2,6-diaminopurine and methods for preparing the same. Also provided are methods for treating or preventing a disease or disorder by inducing RNAi.
摘要翻译： 本公开内容提供了包含具有至少一个碱基对的双链区域的核糖核酸，所述碱基对包含与2,6-二氨基嘌呤配对的5-甲基尿苷碱基及其制备方法。 还提供了通过诱导RNAi治疗或预防疾病或病症的方法。
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公开(公告)号：US20100209487A1
公开(公告)日：2010-08-19
申请号：US12445868
申请日：2007-10-18
IPC分类号： A61K31/713 , C07H21/02 , A61K9/127 , A61K9/51 , C12N5/071 , A61P35/00 , A61P29/00 , A61P31/00
CPC分类号： A61K31/713 , A61K38/00 , A61K47/645 , A61K47/66 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/15 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/3513 , C12N2310/533 , C12N2320/30 , C12N2320/51 , C12N2330/30 , C12N2310/3521
摘要： The present disclosure provides meroduplex (nicked or gapped) ribonucleic acid molecules (mdRNA) that decreases or silences target 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 target gene RNA. In addition, the meroduplex may have one or more modifications or substitutions, such as nucleotide base, sugar, terminal cap structure, internucleotide linkage, or any combination of such modifications. Also provided are methods of decreasing expression of a target gene in a cell or in a subject to treat a disease related to altered expression of a target gene.
摘要翻译： 本公开提供降低或沉默靶基因表达的meroduplex（切口或间隙）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的双链区域，其中一条链与靶基因RNA互补。 此外，meroduplex可以具有一个或多个修饰或取代，例如核苷酸碱基，糖，末端帽结构，核苷酸间连接，或这些修饰的任何组合。 还提供减少细胞或受试者中靶基因表达以治疗与靶基因表达改变相关的疾病的方法。
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10.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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