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公开(公告)号：US20090203896A1
公开(公告)日：2009-08-13
申请号：US12430465
申请日：2009-04-27
IPC分类号： A61K31/713 , C07H21/04 , A61P35/00
CPC分类号： C12N15/1135 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322
摘要： Compounds and compositions are provided for modulating the expression of survivin. The compounds, exemplified by those acting through an RNAi antisense mechanism of action, include double-stranded and single-stranded constructs, as well as siRNAs, canonical siRNAs, blunt-ended siRNAs and single-stranded antisense RNA compounds. Methods of using these compounds for modulation of survivin expression and for treatment of diseases associated with expression of survivin are provided.
摘要翻译： 提供化合物和组合物用于调节存活蛋白的表达。 由通过RNAi反义作用机制起作用的那些实例所列举的化合物包括双链和单链构建体，以及siRNA，典型siRNA，平端siRNA和单链反义RNA化合物。 提供了使用这些化合物调节存活蛋白表达和治疗与存活蛋白表达相关的疾病的方法。
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公开(公告)号：US07541344B2
公开(公告)日：2009-06-02
申请号：US10559636
申请日：2004-06-03
CPC分类号： C12N15/1135 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322
摘要： Compounds and compositions are provided for modulating the expression of survivin. The compounds, exemplified by those acting through an RNAi antisense mechanism of action, include double-stranded and single-stranded constructs, as well as siRNAs, canonical siRNAs, blunt-ended siRNAs and single-stranded antisense RNA compounds. Methods of using these compounds for modulation of survivin expression and for treatment of diseases associated with expression of survivin are provided.
摘要翻译： 提供化合物和组合物用于调节存活蛋白的表达。 由通过RNAi反义作用机制起作用的那些实例所列举的化合物包括双链和单链构建体，以及siRNA，典型siRNA，平端siRNA和单链反义RNA化合物。 提供了使用这些化合物调节存活蛋白表达和治疗与存活蛋白表达相关的疾病的方法。
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公开(公告)号：US20070275921A1
公开(公告)日：2007-11-29
申请号：US11569929
申请日：2005-06-02
IPC分类号： A61K31/7052 , A61P43/00 , C07H21/00
CPC分类号： A61K31/7052
摘要： The present invention provides double stranded compositions wherein one of the strands is useful in, for example, influencing the preferential loading the opposite strand into the RISC (or cleavage) complex. In particular, the present invention provides oligomeric compounds that comprise chemical modifications in at least one of the strands to drive loading of the opposite strand into the RISC (or cleavage) complex. Such modifications can be used to increase potency of duplex constructs that have been modified to enhance stability. Examples of chemical modifications that drive loading of the second strand include, but are not limited to, MOE (2′-O(CH2)2OCH3), 2′-O-methyl, -ethyl, -propyl, and —N-methylacetamide. Such modifications can be distributed throughout the strand, or placed at the 5′ and/or 3′ ends to make a gapmer motif on the sense strand. The activity of the 4′-thio gapmer RNA antisense strand can be improved by incorporating alternating MOE or MOE gapmer motif into the sense strand.
摘要翻译： 本发明提供双链组合物，其中一条链可用于例如影响相对链优先加载到RISC（或切割）复合物中。 特别地，本发明提供了在至少一个链中包含化学修饰以驱动相反链加载到RISC（或切割）复合物中的低聚化合物。 可以使用这些修饰来增加经修饰以增强稳定性的双链构建体的效力。 驱动第二链的负载的化学修饰的实例包括但不限于MOE（2'-O（CH 2）2 OCH 3） ，2'-O-甲基， - 乙基， - 丙基和-N-甲基乙酰胺。 这样的修饰可以分布在整个链上，或者放置在5'和/或3'末端以在有义链上形成gapmer基序。 通过将交替的MOE或MOE gapmer基序并入有义链中，可以改善4'-硫代间隔RNA反义链的活性。
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4.发明申请公开(公告)号：US20070185047A1
公开(公告)日：2007-08-09
申请号：US11565785
申请日：2006-12-01
申请人： Balkrishen Bhat , Thazha Prakash , Prasad Dande , Charles Allerson , Richard Griffey , Eric Swayze
发明人： Balkrishen Bhat , Thazha Prakash , Prasad Dande , Charles Allerson , Richard Griffey , Eric Swayze
IPC分类号： A61K48/00
CPC分类号： C12N15/113 , C07H21/02 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/346 , C12N2320/30 , C12N2320/51 , C12N2310/3521
摘要： The present invention provides double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and sugar modified nucleosides. More particularly, the present compositions comprise one strand having an alternating motif and another strand having a hemimer motif, a blockmer motif, a fully modified motif or a positionally modified motif. At least one of the strands has complementarity to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.
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5.发明申请DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION 审中-公开包含用于基因调控的差异化修饰条纹的双重条纹组合公开(公告)号：US20070179107A1
公开(公告)日：2007-08-02
申请号：US11565816
申请日：2006-12-01
申请人： Balkrishen Bhat , Thazha Parkash , Prasad Dande , Charles Allerson , Richard Griffey , Eric Swayze
发明人： Balkrishen Bhat , Thazha Parkash , Prasad Dande , Charles Allerson , Richard Griffey , Eric Swayze
CPC分类号： C12N15/113 , C07H21/02 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/346 , C12N2320/30 , C12N2320/51 , C12N2310/3521
摘要： The present invention provides double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and sugar modified nucleosides. More particularly, the present compositions comprise one strand having a gapped motif and another strand having a gapped motif, a hemimer motif, a blockmer motif, a fully modified motif, a positionally modified motif or an alternating motif. At least one of the strands has complementarity to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In some embodiments, the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.
摘要翻译： 本发明提供双链组合物，其中每条链被修饰以具有通过定位β-D-核糖核苷和糖修饰核苷定义的基序。 更具体地，本发明的组合物包含一个具有间隙基序的链，另一个链具有间隙基序，半缩体基序，封闭基序，完全修饰的基序，位置修饰的基序或交替基序。 至少一条链与核酸靶具有互补性。 该组合物可用于靶向选定的核酸分子并调节一个或多个基因的表达。 在一些实施方案中，本发明的组合物与靶RNA的一部分杂交导致靶RNA的正常功能丧失。 本发明还提供了调节基因表达的方法。
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6.发明申请DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION 审中-公开包含用于基因调控的差异化修饰条纹的双重条纹组合公开(公告)号：US20070166734A1
公开(公告)日：2007-07-19
申请号：US11565770
申请日：2006-12-01
CPC分类号： C12N15/113 , C07H21/02 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/346 , C12N2320/30 , C12N2320/51 , C12N2310/3521
摘要： The present invention provides double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and sugar modified nucleosides. More particularly, the present compositions comprise one strand having an alternating motif and another strand having a hemimer motif, a blockmer motif, a fully modified motif or a positionally modified motif. At least one of the strands has complementarity to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.
摘要翻译： 本发明提供双链组合物，其中每条链被修饰以具有通过定位β-D-核糖核苷和糖修饰核苷定义的基序。 更具体地说，本发明组合物包含一条具有交替基序的链，另一条链具有半缩体基序，封闭基序，完全修饰的基序或位置修饰的基序。 至少一条链与核酸靶具有互补性。 该组合物可用于靶向选定的核酸分子并调节一个或多个基因的表达。 在优选的实施方案中，本发明的组合物与靶RNA的一部分杂交导致靶RNA的正常功能丧失。 本发明还提供了调节基因表达的方法。
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公开(公告)号：US20070161547A1
公开(公告)日：2007-07-12
申请号：US10559636
申请日：2004-06-03
申请人： Balkrishen Bhat , Bharvin Patel , Eric Swayze
发明人： Balkrishen Bhat , Bharvin Patel , Eric Swayze
CPC分类号： C12N15/1135 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322
摘要： Compounds and compositions are provided for modulating the expression of survivin. The compounds, exemplified by those acting through an RNAi antisense mechanism of action, include double-stranded and single-stranded constructs, as well as siRNAs, canonical siRNAs, blunt-ended siRNAs and single-stranded antisense RNA compounds. Methods of using these compounds for modulation of survivin expression and for treatment of diseases associated with expression of survivin are provided.
摘要翻译： 提供化合物和组合物用于调节存活蛋白的表达。 由通过RNAi反义作用机制起作用的那些实例所列举的化合物包括双链和单链构建体，以及siRNA，典型siRNA，平端siRNA和单链反义RNA化合物。 提供了使用这些化合物调节存活蛋白表达和治疗与存活蛋白表达相关的疾病的方法。
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8.发明申请Compositions comprising alternating 2'-modified nucleosides for use in gene modulation 有权包含交替的2'-修饰核苷的组合物用于基因调控公开(公告)号：US20050080246A1
公开(公告)日：2005-04-14
申请号：US10860265
申请日：2004-06-03
申请人： Charles Allerson , Balkrishen Bhat , Anne Eldrup , Muthiah Manoharan , Richard Griffey , Brenda Baker , Eric Swayze
发明人： Charles Allerson , Balkrishen Bhat , Anne Eldrup , Muthiah Manoharan , Richard Griffey , Brenda Baker , Eric Swayze
CPC分类号： C12N15/113 , C07H21/00 , C07H21/02 , C07H21/04 , C12N15/111 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2320/30 , C12N2320/51
摘要： The present invention provides compositions comprising at least one oligomeric compound comprising an alternating motif and further include a region that is complementary to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.
摘要翻译： 本发明提供包含至少一种包含交替基序的低聚化合物并且还包括与核酸靶互补的区域的组合物。 该组合物可用于靶向选定的核酸分子并调节一个或多个基因的表达。 在优选的实施方案中，本发明的组合物与靶RNA的一部分杂交导致靶RNA的正常功能丧失。 本发明还提供了调节基因表达的方法。
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公开(公告)号：US20050026160A1
公开(公告)日：2005-02-03
申请号：US10701007
申请日：2003-11-04
申请人： Charles Allerson , Balkrishen Bhat , Anne Eldrup , Muthiah Manoharan , Richard Griffey , Brenda Baker , Eric Swayze
发明人： Charles Allerson , Balkrishen Bhat , Anne Eldrup , Muthiah Manoharan , Richard Griffey , Brenda Baker , Eric Swayze
IPC分类号： C07H21/00 , C07H21/04 , C12N15/113 , C12Q1/68
CPC分类号： C07H21/00 , C07H21/04 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/343 , C12N2310/3521 , C12N2310/3533 , C12N2320/51
摘要： The present invention provides compositions comprising at least one oligomeric compound comprising an alternating motif and further include a region that is complementary to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.
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10.发明申请公开(公告)号：US20070167392A1
公开(公告)日：2007-07-19
申请号：US11565858
申请日：2006-12-01
申请人： Balkrishen Bhat , Thazha Prakash , Prasad Dande , Charles Allerson , Richard Griffey , Eric Swayze
发明人： Balkrishen Bhat , Thazha Prakash , Prasad Dande , Charles Allerson , Richard Griffey , Eric Swayze
IPC分类号： A61K48/00 , C07H21/02 , C07F9/6512
CPC分类号： C12N15/113 , C07H21/02 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/346 , C12N2320/30 , C12N2320/51 , C12N2310/3521
摘要： The present invention provides double stranded compositions wherein each strand is modified to have a motif defined by positioning of β-D-ribonucleosides and sugar modified nucleosides. More particularly, the present compositions comprise one strand having a gapped motif and another strand having a gapped motif, a hemimer motif, a blockmer motif, a fully modified motif, a positionally modified motif or an alternating motif. At least one of the strands has complementarity to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In some embodiments, the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.
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