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11. 发明申请

US20080085869A1 DUPLEX OLIGONUCLEOTIDE COMPLEXES AND METHODS FOR GENE SILENCING BY RNA INTERFERENCE 有权
标题翻译：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：US20080085869A1
公开(公告)日：2008-04-10
申请号：US11858829
申请日：2007-09-20
申请人： Christina Yamada , Anastasia Khvorova , Robert Kaiser , Emily Anderson , Devin Leake
发明人： Christina Yamada , Anastasia Khvorova , Robert Kaiser , Emily Anderson , Devin Leake
IPC分类号： A61K31/711 , A61P43/00 , C07H19/00 , C12N5/00
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。

12. 发明申请

US20070218495A1 Methods, libraries and computer program products for gene silencing with reduced off-target effects 审中-公开
标题翻译：用于基因沉默的方法，图书馆和计算机程序产品，减少脱靶效应
公开(公告)号：US20070218495A1
公开(公告)日：2007-09-20
申请号：US11724346
申请日：2007-03-15
申请人： Amanda Birmingham , Emily Anderson , Angela Reynolds , Devin Leake , Scott Baskerville , Yuriy Fedorov , Jon Karpilow , William Marshall , Anastasia Khvorova
发明人： Amanda Birmingham , Emily Anderson , Angela Reynolds , Devin Leake , Scott Baskerville , Yuriy Fedorov , Jon Karpilow , William Marshall , Anastasia Khvorova
IPC分类号： C40B40/08 , C07H21/02
CPC分类号： C12N15/111 , C12N2310/14 , C12N2320/50 , C12N2330/31
摘要： The present invention provides methods, libraries and computer program products for selecting siRNA that reduce off-target effects and methods for gene silencing using these siRNAs. By comparing nucleotide sequences at positions 2-7 or 2-8 of the sense and/or antisense regions of candidate siRNAs to the 3′ UTR region of mRNAs, one can select siRNAs that have reduced off-target effects.
摘要翻译：本发明提供用于选择减少脱靶效应的siRNA的方法，文库和计算机程序产品以及使用这些siRNA的基因沉默方法。通过将候选siRNAs的有义和/或反义区的2-7位或2-8位的核苷酸序列与mRNA的3'UTR区进行比较，可以选择具有降低的脱靶效应的siRNA。

13. 发明授权

US09095504B2 RNA interference in ocular indications 有权
标题翻译：眼睛指征中的RNA干扰
公开(公告)号：US09095504B2
公开(公告)日：2015-08-04
申请号：US13636748
申请日：2011-03-24
申请人： Lyn Libertine , Anastasia Khvorova , William Salomon , Joanne Kamens , Dmitry Samarsky , Tod M. Woolf , James Cardia
发明人： Lyn Libertine , Anastasia Khvorova , William Salomon , Joanne Kamens , Dmitry Samarsky , Tod M. Woolf , James Cardia
IPC分类号： C12N15/11 , A61K9/00 , A61K31/713 , A61K45/06
CPC分类号： C12N15/1136 , A61K9/0048 , A61K9/0051 , A61K31/713 , A61K45/06 , A61K47/554 , C12N2310/14 , C12N2310/313 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2320/31 , C12N2320/32 , A61K2300/00
摘要： The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.
摘要翻译：本发明涉及sd-rxRNA和rxRNAori分子的眼部给药。

14. 发明授权

US09080171B2 Reduced size self-delivering RNAi compounds 有权
标题翻译：减小大小的自我传递RNAi化合物
公开(公告)号：US09080171B2
公开(公告)日：2015-07-14
申请号：US13636728
申请日：2011-03-24
申请人： Anastasia Khvorova , William Salomon , Joanne Kamens , Dmitry Samarsky , Tod M. Woolf , James Cardia
发明人： Anastasia Khvorova , William Salomon , Joanne Kamens , Dmitry Samarsky , Tod M. Woolf , James Cardia
IPC分类号： C07H21/04 , C12N15/113 , C07H21/02 , C12N15/11
CPC分类号： C12N15/113 , C07H21/02 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/322 , C12N2310/334 , C12N2310/335 , C12N2310/344 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , C12N2320/51 , C12N2310/3521 , C12N2310/3533
摘要： The present invention relates to methods for in vivo administration of sd-rxRNA molecules.
摘要翻译：本发明涉及体内施用sd-rxRNA分子的方法。

15. 发明授权

US08664189B2 RNA interference in skin indications 有权
标题翻译： RNA干扰皮肤适应症
公开(公告)号：US08664189B2
公开(公告)日：2014-03-04
申请号：US13120315
申请日：2009-09-22
申请人： Anastasia Khvorova , William Salomon , Joanne Kamens , Dmitry Samarsky , Tod M. Woolf , Pamela A. Pavco , James Cardia
发明人： Anastasia Khvorova , William Salomon , Joanne Kamens , Dmitry Samarsky , Tod M. Woolf , Pamela A. Pavco , James Cardia
IPC分类号： A61K48/00
CPC分类号： C12N15/1136 , C12N15/111 , C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/3341 , C12N2310/3515 , C12N2310/3519 , C12N2310/3521 , C12N2320/32 , C12N2320/51 , C12N2320/53
摘要： The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal applications.
摘要翻译：本发明涉及具有改善的组织和细胞摄取特性的RNAi构建体以及在真皮应用中使用这些化合物的方法。

16. 发明授权

US08575329B2 siRNA targeting kinase insert domain receptor (KDR) 有权
公开(公告)号：US08575329B2
公开(公告)日：2013-11-05
申请号：US13524015
申请日：2012-06-15
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 , C07H21/04 , C12Q1/68
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 KDR.

17. 发明申请

US20130197055A1 INHIBITION OF PCSK9 THROUGH RNAI 有权
标题翻译：通过RNAi抑制PCSK9
公开(公告)号：US20130197055A1
公开(公告)日：2013-08-01
申请号：US13143275
申请日：2010-01-05
申请人： Joanne Kamens , Anastasia Khvorova
发明人： Joanne Kamens , Anastasia Khvorova
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/344 , C12N2310/3515 , C12N2310/531 , C12N2320/11 , C12Y304/21061 , C12Y304/21112 , C12N2310/3521 , C12N2310/3533
摘要： The invention relates to various PCSK9 RNAi constructs with gene silencing activities, and uses thereof The construct has a double-stranded region of 19-49 nucleotides, preferably 25, 26, or 27 nucleotides, and preferably blunt-ended The construct has selective minimal modifications to confer an optimal balance of biological activity, toxicity, stability, and target gene specificity The sense strand may be modified such that the construct is not cleaved by Dicer or other RNAse III, and the entire length of the antisense strand is loaded into RISC In addition, the antisense strand may also be modified by 2′-O-methyl groups at the 2nd 5′-end nucleotide to greatly reduce off-target silencing The constructs of the invention largely avoid the interferon response and sequence-independent apoptosis in mammalian cells, exhibits better serum stability, and enhanced target specificity.
摘要翻译：本发明涉及具有基因沉默活性的各种PCSK9 RNAi构建体及其用途。该构建体具有19-49个核苷酸，优选25,26或27个核苷酸的双链区域，优选为平端。该构建体具有选择性最小修饰赋予生物活性，毒性，稳定性和靶基因特异性的最佳平衡有义链可以被修饰，使得构建体不被切丝虫或其他RNA酶III切割，并且反义链的整个长度被加载到RISC In 此外，反义链也可以在第二个5'-末端核苷酸处被2'-O-甲基修饰，以大大减少脱靶沉默本发明的构建体大大地避免了哺乳动物细胞中的干扰素应答和序列无关的凋亡，表现出更好的血清稳定性和增强的靶特异性。

18. 发明授权

US08445668B2 SiRNA targeting apolipoprotein (APOB) 有权
标题翻译：靶向载脂蛋白（APOB）的SiRNA
公开(公告)号：US08445668B2
公开(公告)日：2013-05-21
申请号：US13536005
申请日：2012-06-28
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： C07H21/04 , C07H21/02
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。

19. 发明授权

US08426579B2 SiRNA targeting myeloid differentiation primary response gene (88) (MYD88) 有权
标题翻译：靶向骨髓分化原发性反应基因（88）（MYD88）
公开(公告)号：US08426579B2
公开(公告)日：2013-04-23
申请号：US13136812
申请日：2011-08-11
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
IPC分类号： C07H21/00
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 MYD88.
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MYD88的核苷酸序列的那些。

20. 发明授权

US08415466B2 Duplex oligonucleotides with enhanced functionality in gene regulation 有权
标题翻译：具有增强基因调控功能的双链寡核苷酸
公开(公告)号：US08415466B2
公开(公告)日：2013-04-09
申请号：US13460026
申请日：2012-04-30
申请人： Anastasia Khvorova , Devin Leake , Barbara Robertson , Annaleen Vermeulen , Christina Yamada
发明人： Anastasia Khvorova , Devin Leake , Barbara Robertson , Annaleen Vermeulen , Christina Yamada
IPC分类号： C07H21/04
CPC分类号： C12N15/111 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/344 , C12N2310/3515 , C12N2310/533 , C12N2320/52 , C12N2310/3521 , C12N2310/3533
摘要： Disclosed are methods of enhancing functionality of duplex oligonucleotides and compositions made by the methods. The duplex oligonucleotides include siRNAs, miRNA mimics, and piRNA mimics which contain modified nucleotides and mismatches between the two strands of the molecule at specific nucleotide positions.
摘要翻译：公开的是增强双功能寡核苷酸和通过该方法制备的组合物的功能的方法。双链寡核苷酸包括siRNA，miRNA模拟物和piRNA模拟物，其在特定核苷酸位置含有修饰的核苷酸和分子的两条链之间的错配。

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