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公开(公告)号：EP2850190A1
公开(公告)日：2015-03-25
申请号：EP13791546.8
申请日：2013-05-16
IPC分类号： C12N15/11 , C07H21/00 , A61K31/7088 , A61P25/00
摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of MECP2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of MECP2. Methods for modulating expression of MECP2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of MECP2.
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公开(公告)号：EP2850189A1
公开(公告)日：2015-03-25
申请号：EP13791343.0
申请日：2013-05-16
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/3231 , C12N2310/343 , C12N2310/3517 , C12N2310/322 , C12N2310/3533 , C12N2310/321 , C12N2310/3521
摘要： Aspects of the invention provide methods for selecting a candidate oligonucleotide for activating expression of a target gene. Further aspects of the invention provide methods of selecting a set of oligonucleotides that is enriched in oligonucleotides that activate expression of a target gene. Further aspects provide single stranded oligonucleotides that modulate gene expression and compositions and kits comprising the same. Methods for modulating gene expression using the single stranded oligonucleotides are also provided.
摘要翻译： 本发明的方面提供了用于选择用于激活靶基因表达的候选寡核苷酸的方法。 本发明的其他方面提供了选择一组富集激活靶基因表达的寡核苷酸的寡核苷酸的方法。 其他方面提供调节基因表达的单链寡核苷酸和组合物以及包含它们的试剂盒。 还提供了使用单链寡核苷酸调节基因表达的方法。
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3.发明公开
公开(公告)号：EP2850188A1
公开(公告)日：2015-03-25
申请号：EP13790998.2
申请日：2013-05-16
IPC分类号： C12N15/11 , C07H21/00 , A61K31/7125 , A61P7/06
CPC分类号： C12N15/113 , A61K31/7088 , A61K31/712 , A61K31/7125 , A61K47/28 , A61K47/64 , C07H21/00 , C07H21/02 , C07H21/04 , C12N2310/11 , C12N2310/315 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of hemoglobin genes (HBB, HBD, HBE1, HBG1 or HBG2). Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of hemoglobin genes. Methods for modulating expression of hemoglobin genes using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of hemoglobin genes.
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4.发明公开
公开(公告)号：EP2849801A1
公开(公告)日：2015-03-25
申请号：EP13790940.4
申请日：2013-05-16
CPC分类号： C12N15/113 , A61K31/7088 , A61K47/543 , A61K47/62 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/346 , C12N2310/351 , C12N2310/3513 , C12N2310/3515
摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of APOA1 or ABCA1. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of APOA1 or ABCA1. Methods for modulating expression of APOA1 or ABCA1 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of APOA1 or ABCA1.
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公开(公告)号：EP2850187A1
公开(公告)日：2015-03-25
申请号：EP13790838.0
申请日：2013-05-16
IPC分类号： C12N15/11 , C07H21/00 , C12N15/63 , A61K31/7088 , A61P35/00
CPC分类号： C12N15/1137 , A61K31/7115 , A61K31/712 , A61K31/7125 , C07H21/00 , C07H21/02 , C07H21/04 , C12N15/1135 , C12N2310/11 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/346 , C12N2310/351 , C12Y301/03016 , C12Y301/03048 , C12Y301/03067 , C12N2310/3521
摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of PTEN. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of PTEN. Methods for modulating expression of PTEN using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of PTEN.
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公开(公告)号：EP2850184A1
公开(公告)日：2015-03-25
申请号：EP13790575.8
申请日：2013-05-16
IPC分类号： C12N15/11 , C07H21/00 , C12N15/63 , A61K31/7088
CPC分类号： C12N15/113 , A61K31/7088 , A61K47/54 , A61K47/64 , A61K48/00 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/343 , C12N2310/3517 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of a target gene. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of a target gene. Methods for modulating expression of a target gene using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of a target gene.
摘要翻译： 本发明的方面提供了用于激活或增强靶基因表达的单链寡核苷酸。 其它方面提供了包含用于激活或增强靶基因表达的单链寡核苷酸的组合物和试剂盒。 还提供了使用单链寡核苷酸调节靶基因表达的方法。 本发明的其他方面提供了用于选择用于激活或增强靶基因表达的候选寡核苷酸的方法。
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公开(公告)号：EP2850182A1
公开(公告)日：2015-03-25
申请号：EP13790212.8
申请日：2013-05-16
IPC分类号： C12N15/11 , C07H21/00 , C12N15/63 , A61K31/7088
CPC分类号： C12N15/1137 , C07H21/00 , C07H21/02 , C07H21/04 , C12N15/52 , C12N2310/11 , C12N2310/321 , C12N2310/3231 , C12N2310/351 , C12N2310/3513 , C12N2310/3515 , C12N2310/3521 , C12Y306/03008
摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of ATP2A2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of ATP2A2. Methods for modulating expression of ATP2A2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of ATP2A2.
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公开(公告)号：EP2850183A1
公开(公告)日：2015-03-25
申请号：EP13790349.8
申请日：2013-05-16
IPC分类号： C12N15/11 , C07H21/00 , A61K31/7088
CPC分类号： A61K31/7088 , C07H21/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12N2310/11 , C12N2310/34 , C12N2310/343
摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of a target gene. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of a target gene. Methods for modulating expression of a target gene using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of a target gene.
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9.发明公开COMPOSITIONS AND METHODS FOR MODULATING BDNF EXPRESSION 审中-公开ZUSAMMENSETZUNGEN UND VERFAHREN ZUR MODULATION DER BDNF-EXPRESSION
公开(公告)号：EP2849800A1
公开(公告)日：2015-03-25
申请号：EP13790378.7
申请日：2013-05-16
CPC分类号： C12N15/1136 , A61K31/712 , A61K48/00 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/351 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of BDNF. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of BDNF. Methods for modulating expression of BDNF using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of BDNF.
摘要翻译： 本发明的方面提供用于激活或增强BDNF表达的单链寡核苷酸。 其它方面提供包含用于激活或增强BDNF表达的单链寡核苷酸的组合物和试剂盒。 还提供了使用单链寡核苷酸调节BDNF表达的方法。 本发明的其它方面提供了选择用于激活或增强BDNF表达的候选寡核苷酸的方法。
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公开(公告)号：EP2756080A1
公开(公告)日：2014-07-23
申请号：EP12830969.7
申请日：2012-09-14
发明人： UHLMANN, Eugen , WEBER, Markus , SUBRAMANIAN, Romesh , ROCKEL, Thomas, Dino , KRIEG, Arthur, M.
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/3231 , C12N2310/341 , C12N2310/346 , C12N2310/51 , C12N2310/52
摘要： The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided.
摘要翻译： 本公开提供了包含两个或多个靶特异性寡核苷酸（例如反义寡核苷酸（ASO））的多聚寡核苷酸化合物，其各自对切割具有抗性，并通过可切割连接子连接在一起。 特别地，两个或更多个连接的靶特异性寡核苷酸，各自到不同的靶标，允许同时抑制多种基因的表达水平，同时显示出有利的药代动力学和药效学性质。 还提供了制备和使用所述化合物的方法。
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