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91.发明授权Duplex oligonucleotide complexes and methods for gene silencing by RNA interference 有权双链寡核苷酸复合物和通过RNA干扰进行基因沉默的方法公开(公告)号：US08252755B2
公开(公告)日：2012-08-28
申请号：US11858829
申请日：2007-09-20
IPC分类号： A61K48/00
CPC分类号： C12N15/111 , C07H19/00 , C12N15/11 , C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/346 , C12N2310/3515 , C12N2320/32 , C12N2330/30 , C12N2310/3521
摘要： Provided herein are duplex oligonucleotide complexes which can be administered to a cell, tissue or organism to silence a target gene without the aid of a transfection reagent(s). The duplex oligonucleotide complexes of the disclosure include a conjugate moiety that facilitates delivery to a cell, tissue or organism.
摘要翻译： 本文提供了可以在不借助转染试剂的情况下向细胞，组织或生物体施用以沉默靶基因的双链寡核苷酸复合物。 本公开的双链寡核苷酸复合物包括有助于递送至细胞，组织或生物体的缀合物部分。
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公开(公告)号：US08232386B2
公开(公告)日：2012-07-31
申请号：US13317752
申请日：2011-10-27
申请人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
发明人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1135 , C12N2310/11 , C12N2310/14 , C12N2320/11 , G06F19/18
摘要： Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for APOB.
摘要翻译： 通过使用siRNA技术，有效的序列特异性基因沉默是可能的。 通过合理设计选择特定的siRNA，可以最大限度地产生有效的基因沉默试剂，以及沉默基因的方法。 公开了通过合理设计siRNA产生的方法，组合物和试剂盒，包括针对APOB的核苷酸序列的那些。
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公开(公告)号：US20120184463A1
公开(公告)日：2012-07-19
申请号：US13385320
申请日：2012-02-14
申请人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
发明人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1135 , C12N2310/11 , C12N2310/14 , C12N2320/11 , G06F19/18
摘要： Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to CTGF.
摘要翻译： 通过使用siRNA技术，有效的序列特异性基因沉默是可能的。 通过合理设计选择特定的siRNA，可以最大限度地产生有效的基因沉默试剂，以及沉默基因的方法。 公开了通过合理设计siRNA产生的方法，组合物和试剂盒，包括针对CTGF的那些。
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公开(公告)号：US20120136144A1
公开(公告)日：2012-05-31
申请号：US11980210
申请日：2007-10-30
申请人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
发明人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
IPC分类号： C07H21/02
CPC分类号： A61K31/713 , C07K14/705 , C12N9/90 , C12N15/111 , C12N15/113 , C12N15/1135 , C12N15/1136 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2320/11 , C12Y304/21047 , C12Y502/01008 , G06F19/18
摘要： Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for CTNNB1.
摘要翻译： 通过使用siRNA技术，有效的序列特异性基因沉默是可能的。 通过合理设计选择特定的siRNA，可以最大限度地产生有效的基因沉默试剂，以及沉默基因的方法。 公开了通过合理设计siRNA产生的方法，组合物和试剂盒，包括针对CTNNB1的核苷酸序列的那些。
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公开(公告)号：US20120059046A1
公开(公告)日：2012-03-08
申请号：US13130194
申请日：2009-11-19
IPC分类号： A61K31/7088 , C12N5/071 , A61P35/00 , A61P27/02 , A61P37/06 , A61P29/00 , A61P31/18 , A61P31/14 , A61P9/00 , A61P17/06 , A61P3/00 , A61P3/08 , A61P3/10 , A61P9/12 , A61P3/04 , A61P3/06 , C12N15/113
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/321 , C12N2320/11 , C12N2320/30 , C12Y207/01037
摘要： RNAi constructs directed to MAP4K4 that demonstrate unexpectedly high gene silencing activities, and uses thereof are disclosed. The blunt-ended constructs have a double-stranded region of 19-49 nucleotides. The constructs have selective minimal modifications to confer an optimal balance of biological activity, toxicity, stability, and target gene specificity. For example, the strands may be modified (e.g., one or both ends of the sense strand is modified by 2′-O-methyl groups), such that the construct is not cleaved by Dicer or other RNAse III, the antisense strand may also be modified by a 2′-O-methyl group at the penultimate 5′-end nucleotide to greatly reduce off-target silencing.
摘要翻译： 公开了针对表达意想不到的高基因沉默活性的MAP4K4的RNAi构建体及其用途。 平端构建体具有19-49个核苷酸的双链区域。 该构建体具有选择性的最小修饰以赋予生物活性，毒性，稳定性和靶基因特异性的最佳平衡。 例如，可以修饰链（例如，有义链的一端或两端被2'-O-甲基修饰），使得构建体不被切割酶或其它RNA酶III切割，反义链也可以 在倒数第二个5'-末端核苷酸处被2'-O-甲基修饰，以大大减少脱靶沉默。
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公开(公告)号：US08090542B2
公开(公告)日：2012-01-03
申请号：US10714333
申请日：2003-11-14
CPC分类号： C12Y113/12007 , A61K31/713 , C12N15/1048 , C12N15/111 , C12N15/113 , C12N15/1135 , C12N15/1136 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2320/10 , C12N2320/11 , C12Y502/01008 , G06F19/18
摘要： Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rationale design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes.
摘要翻译： 通过使用siRNA技术，有效的序列特异性基因沉默是可能的。 通过基本设计选择特定的siRNA，可以最大限度地产生有效的基因沉默试剂，以及沉默基因的方法。
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公开(公告)号：US20110319474A1
公开(公告)日：2011-12-29
申请号：US13199001
申请日：2011-08-17
申请人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
发明人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
IPC分类号： A61K31/713 , C12N5/00 , C07H21/02
CPC分类号： C12Y113/12007 , A61K31/713 , C12N15/1048 , C12N15/111 , C12N15/113 , C12N15/1135 , C12N15/1136 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2320/10 , C12N2320/11 , C12Y502/01008 , G16B20/00
摘要： Efficient sequence specific gene silencing for cyclin-dependent kinase 4 is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed.
摘要翻译： 通过使用siRNA技术，细胞周期蛋白依赖性激酶4的有效序列特异性基因沉默是可能的。 通过合理设计选择特定的siRNA，可以最大限度地产生有效的基因沉默试剂，以及沉默基因的方法。 公开了通过合理设计siRNA产生的方法，组合物和试剂盒。
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公开(公告)号：US20110281769A1
公开(公告)日：2011-11-17
申请号：US13135336
申请日：2011-07-01
申请人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
发明人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1135 , C12N2310/11 , C12N2310/14 , C12N2320/11 , G06F19/18
摘要： Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for BACE.
摘要翻译： 通过使用siRNA技术，有效的序列特异性基因沉默是可能的。 通过合理设计选择特定的siRNA，可以最大限度地产生有效的基因沉默试剂，以及沉默基因的方法。 公开了通过合理设计siRNA产生的方法，组合物和试剂盒，包括针对BACE的核苷酸序列的那些。
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99.发明授权公开(公告)号：US08000902B2
公开(公告)日：2011-08-16
申请号：US12802647
申请日：2010-06-11
申请人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
发明人： Anastasia Khvorova , Angela Reynolds , Devin Leake , William Marshall , Steven Read , Stephen Scaringe
CPC分类号： C12Y113/12007 , A61K31/713 , C12N15/1048 , C12N15/111 , C12N15/113 , C12N15/1135 , C12N15/1136 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2320/10 , C12N2320/11 , C12Y502/01008 , G06F19/18
摘要： Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed.
摘要翻译： 通过使用siRNA技术，有效的序列特异性基因沉默是可能的。 通过合理设计选择特定的siRNA，可以最大限度地产生有效的基因沉默试剂，以及沉默基因的方法。 公开了通过合理设计siRNA产生的方法，组合物和试剂盒。
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100.发明申请IDENTIFICATION OF MIRNA PROFILES THAT ARE DIAGNOSTIC OF HYPERTROPHIC CARDIOMYOPATHY 审中-公开鉴别诊断超声心动图的MIRNA轮廓公开(公告)号：US20110160285A1
公开(公告)日：2011-06-30
申请号：US12921827
申请日：2009-03-12
申请人： Emily Anderson , Anastasia Khvorova , Anita G. Seto , Scott Baskerville , Leslie Leinwand , Kevin G. Sullivan
发明人： Emily Anderson , Anastasia Khvorova , Anita G. Seto , Scott Baskerville , Leslie Leinwand , Kevin G. Sullivan
IPC分类号： A61K31/7105 , C40B30/04 , A61P9/00
CPC分类号： C12Q1/6883 , A61K48/00 , C12Q2600/178
摘要： Disclosed herein are a collection of miRNAs and genes whose expression is altered in hypertrophic cardiomyopathy. Accordingly, these miRNAs and genes, singly or in combination, are useful as molecular markers for diagnosis or prognosis of hypertrophic cardiomyopathy. The miRNAs and genes disclosed can also be therapeutic targets for cardiac hypertrophy. For example, agents such as miRNA mimics, miRNA inhibitors or siRNAs for a given miRNA or gene can be used to modulate the level of these molecules thereby inhibiting or preventing hypertrophic cardiomyopathy.
摘要翻译： 本文公开了在增生性心肌病中表达改变的miRNA和基因的集合。 因此，这些miRNA和基因单独或组合可用作增生性心肌病的诊断或预后的分子标记物。 所公开的miRNA和基因也可以是心脏肥大的治疗靶点。 例如，可以使用诸如miRNA模拟物，miRNA抑制剂或用于给定miRNA或基因的siRNA的试剂来调节这些分子的水平，从而抑制或预防肥大性心肌病。
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