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1. 发明专利

ES2426918T3 Polinucleótidos modificados para reducir los efectos no específicos en la interferencia con ARN 未知
公开(公告)号：ES2426918T3
公开(公告)日：2013-10-25
申请号：ES05734330
申请日：2005-03-31
申请人： DHARMACON INC , MERCK SHARP & DOHME
发明人： LEAKE DEVIN , REYNOLDS ANGELA , KHVOROVA ANASTASIA , MARSHALL WILLIAM , LINSLEY PETER S
IPC分类号： C12N15/11
摘要： Un ARNip de doble hebra que comprende: i. a hebra efectora que comprende a. un primer nucleótido efector terminal 5', en donde dicho primer nucleótido efector terminal 5' comprende una primera modificación 2'-O-alquilo, y b. un segundo nucleótido efector terminal 5', en donde dicho segundo nucleótido efector terminal 5' comprende una segunda modificación 2'-O-alquilo; y ii. una hebra antisentido que comprende a. un primer nucleótido antisentido terminal 5', en donde dicho primer nucleótido antisentido terminal 5' tiene uno o más grupos fosfato anclados al carbono 5' de su radical azúcar, y b. un segundo nucleótido antisentido terminal 5', en donde dicho segundo nucleótido antisentido terminal 5' comprende una tercera modificación 2'-O-alquilo, en donde dicha hebra efectora y dicha hebra antisentido son capaces de formar un dúplex de 18-19 pares de bases de nucleótidos que tiene una complementariedad de 100% a lo largo del intervalo del dúplex, y dentro de dicho dúplex dicho primer nucleótido efector terminal 5' es el nucleótido más 5' de la hebra efectora, dicho segundo nucleótido efector terminal 5' es inmediatamente adyacente y está aguas abajo del primer nucleótido efector terminal 5', dicho primer nucleótido antisentido terminal 5' es el nucleótido más 5' de la hebra antisentido y dicho segundo nucleótido antisentido terminal 5' es inmediatamente adyacente y está aguas abajo del primer nucleótido antisentido terminal 5', en donde todos los nucleótidos de la hebra efectora y la hebra antisentido distintos de dicho primer nucleótido efector terminal 5', dicho segundo nucleótido efector terminal 5', y dicho segundo nucleótido antisentido terminal 5' comprenden un 2'-OH.

2. 发明专利

JP2010187668A FUNCTIONAL siRNA, AND SUPER FUNCTIONAL siRNA 审中-公开
标题翻译：功能性siRNA和超功能siRNA
公开(公告)号：JP2010187668A
公开(公告)日：2010-09-02
申请号：JP2010035279
申请日：2010-02-19
申请人： Dharmacon Inc , ダーマコン, インコーポレイテッド
发明人： KHVOROVA ANASTASIA , REYNOLDS ANGELA , LEAKE DEVIN , MARSHALL WILLIAM , SCARINGE STEPHEN
IPC分类号： C12N15/09 , A01H1/00 , A61B17/56 , A61K31/713 , A61K48/00 , A61P3/10 , A61P13/12 , A61P25/28 , A61P35/00 , A61P35/02 , A61P37/02 , C07H21/00 , C07H21/02 , C12N15/11 , C12N15/113 , C12N15/82 , C12Q1/68 , G01N33/48 , G01N33/50
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： PROBLEM TO BE SOLVED: To provide a kit, an si(small interfering)RNA and a method for increasing the effectiveness of the siRNA. SOLUTION: This kit consists of a pool of at least two siRNA double strands, each of which consists of a sequence complementary to a part of a sequence of one or more target messenger RNAs, and performing efficient sequence specific gene silencing. Especially, in a mammalian animal system, the effectiveness of the RNAi is increased. COPYRIGHT: (C)2010,JPO&INPIT
摘要翻译：待解决的问题：提供试剂盒，si（小干扰）RNA和提高siRNA有效性的方法。解决方案：该试剂盒由至少两个siRNA双链库组成，每个RNA双链由与一个或多个靶信使RNA的序列的一部分互补的序列组成，并执行有效的序列特异性基因沉默。特别是在哺乳动物系统中，RNAi的有效性增加。版权所有（C）2010，JPO＆INPIT

3. 发明专利

AU2005322468B2 Apparatus and system having dry gene silencing compositions 未知
公开(公告)号：AU2005322468B2
公开(公告)日：2011-03-10
申请号：AU2005322468
申请日：2005-11-21
申请人： DHARMACON INC
发明人： KHVOROVA ANASTASIA , MARSHALL WILLIAM S , ROBERTSON BARBARA , ROBINSON KATHRYN , LEAKE DEVIN
IPC分类号： C12N15/113 , A61K48/00 , C07H21/02 , C07H21/04 , C12N15/00 , C12N15/11 , C12N15/63 , C12N15/85 , C12N15/87 , C12Q1/68
摘要： A reverse transfection apparatus can be used for introducing siRNA into a cell to effect gene silencing. Such an apparatus can include a well plate having a well configured for transfecting cells. The well can include a substantially dry gene silencing composition that has at least a first siRNA which silences a first target gene. The gene silencing composition can be configured such that the at least first siRNA is capable of being solubilized or suspended in an aqueous medium in an amount sufficient for transfecting cells in the well. Additionally, the at least first siRNA can include a modification or a conjugate. The reverse transfection apparatus can be provided as a kit or system that additionally includes cells, polynucleotide carriers, reverse transfection reagents, and the like.

4. 发明专利

CA2587786A1 APPARATUS AND SYSTEM HAVING DRY GENE SILENCING COMPOSITIONS 未知
公开(公告)号：CA2587786A1
公开(公告)日：2006-07-06
申请号：CA2587786
申请日：2005-11-21
申请人： DHARMACON INC
发明人： LEAKE DEVIN , ROBINSON KATHRYN , ROBERTSON BARBARA , MARSHALL WILLIAM S , KHVOROVA ANASTASIA
IPC分类号： C12N15/63 , C07H21/02 , C07H21/04 , C12N15/00 , C12N15/85 , C12N15/87 , C12Q1/68
摘要： A reverse transfection apparatus can be used for introducing siRNA into a ce ll to effect gene silencing. Such an apparatus can include a well plate having a well configured for transfecting cells. The well can include a substantially dry gene silencing composition that has at least a first siRNA which silence s a first target gene. The gene silencing composition can be configured such that the at least first siRNA is capable of being solubilized or suspended i n an aqueous medium in an amount sufficient for transfecting cells in the well . Additionally, the at least first siRNA can include a modification or a conjugate. The reverse transfection apparatus can be provided as a kit or system that additionally includes cells, polynucleotide carriers, reverse transfection reagents, and the like.

5. 发明专利

ES2574625T3 Antagonistas de miARN de oligonucleótido conjugado con aglutinante del surco menor (MGB) 未知
公开(公告)号：ES2574625T3
公开(公告)日：2016-06-21
申请号：ES10788176
申请日：2010-11-23
申请人： ELITECHGROUP B V , DHARMACON INC
发明人： KHVOROVA ANASTASIA , VERMEULEN ANNALEEN , KAISER ROB , KARPILOW JON , VERMEULEN NICOLAAS , MAHONEY WALT
IPC分类号： C12N15/113
摘要： Una composición inhibidora para inhibir ARN no codificantes que comprende: un oligonucleótido; y un aglutinante del surco menor (MGB), en la que el oligonucleótido comprende nucleótidos, en la que todos los nucleótidos del oligonucleótido comprenden ribofuranosa que tiene la siguiente estructura:**Fórmula** en la que Rz es -OCH3, en la que B es una base normal o una base modificada; y en la que el MGB está conjugado con el extremo 5' o con el extremo 3' del oligonucleótido; y en la que el oligonucleótido es esencialmente complementario a una secuencia de miARN o ARNpi madura endógena.

6. 发明专利

CA2687336C MICRO-RNA SCAFFOLDS AND NON-NATURALLY OCCURRING MICRO-RNAS 未知
公开(公告)号：CA2687336C
公开(公告)日：2014-12-23
申请号：CA2687336
申请日：2008-05-22
申请人： DHARMACON INC
发明人： KELLEY MELISSA , BIRMINGHAM AMANDA , KARPILOW JON , KHVOROVA ANASTASIA , SULLIVAN KEVIN
IPC分类号： C12N15/113 , A61K31/7105 , A61K48/00 , C07H21/02 , C12N5/10 , C12N15/63 , C12Q1/68
摘要： The present disclosure provides a non-naturally occurring miRNA having a stem-loop structure comprising a scaffold derived from a first endogenous miRNA (e.g., miR-196a-2 or miR-204), a mature strand derived from a second endogenous miRNA, and a star strand sequence that is at least partially complementary to the mature strand sequence. The present disclosure also provides a non-naturally occurring miRNA having a stem-loop structure comprising a scaffold derived from an endogenous miRNA (e.g., miR-196a-2 or miR-204), a mature strand designed t be at least partially complementary to a target RNA, and a star strand sequence that is at least partially complementary to the mature strand sequence. The methods and compositions of the disclosure may be used to mediate gene silencing via the RNAi pathway.

7. 发明专利

ES2440284T3 ARNip dirigido a tp53 未知
公开(公告)号：ES2440284T3
公开(公告)日：2014-01-28
申请号：ES10012132
申请日：2003-11-14
申请人： DHARMACON INC , THERMO FISHER SCIENT BIOSCIENCES INC
发明人： KHVOROVA ANASTASIA , REYNOLDS ANGELA , LEAKE DEVIN , MARSHALL WILLIAM , SCARINGE STEPHEN
IPC分类号： C12N15/113 , A01H1/00 , A61B17/56 , A61K31/713 , A61K48/00 , C07H21/00 , C07H21/02 , C12N15/11 , C12N15/82 , C12Q1/68 , G01N33/48 , G01N33/50
摘要： Una molécula de ARNip que comprende una región bicatenaria que consiste de una cadena codificante y unacadena no codificante, en donde dicha cadena codificante comprende la secuencia GAGAAUAUUUCACCCUUCA(sec. con núm. de ident. 512,246).

8. 发明专利

CA2561868C MODIFIED POLYNUCLEOTIDES FOR REDUCING OFF-TARGET EFFECTS IN RNA INTERFERENCE 未知
公开(公告)号：CA2561868C
公开(公告)日：2012-11-06
申请号：CA2561868
申请日：2005-03-31
申请人： DHARMACON INC , ROSETTA INPHARMATICS LLC
发明人： LEAKE DEVIN , FEDOROV YURIY , REYNOLDS ANGELA , KHVOROVA ANASTASIA , MARSHALL WILLIAM , LINSLEY PETER S
IPC分类号： C12N15/11 , A01H1/00 , A61K31/713 , C07H21/02 , C12N15/113 , C12N15/82
摘要： Methods and compositions for performing RNA interference with decreased off-target effects are provided. The methods and compositions permit effective and efficient applications of RNA interference to applications such as diagnostics and therapeutics through the use of modifications to the siRNA. Uniquely modified siRNAs have been developed that reduce off-target effects incurred in gene-silencing. The modifications comprise 2'-O-alkyl or mismatch modification(s) at specific positions on the sense and/or antisense strands.

9. 发明专利

AT536408T 未知
公开(公告)号：AT536408T
公开(公告)日：2011-12-15
申请号：AT04749718
申请日：2004-04-01
申请人： DHARMACON INC
发明人： LEAKE DEVIN , REYNOLDS ANGELA , KHVOROVA ANASTASIA , MARSHALL WILLIAM , FEDOROV YURIY , NICHOLS KIMBERLY
IPC分类号： C12N15/113 , A61K31/713

10. 发明专利

AT519772T 未知
公开(公告)号：AT519772T
公开(公告)日：2011-08-15
申请号：AT05852038
申请日：2005-11-21
申请人： DHARMACON INC
发明人： ROBERTSON BARBARA , LEAKE DEVIN , ROBINSON KATHRYN , MARSHALL WILLIAM , KHVOROVA ANASTASIA
IPC分类号： C07H19/00 , A61K31/70 , C12N15/11 , C12N15/87 , C12Q1/68
摘要： A reverse transfection apparatus can be used for introducing siRNA into a cell to effect gene silencing. Such an apparatus can include a well plate having a well configured for transfecting cells. The well can include a substantially dry gene silencing composition that has at least a first siRNA which silences a first target gene. The gene silencing composition can be configured such that the at least first siRNA is capable of being solubilized or suspended in an aqueous medium in an amount sufficient for transfecting cells in the well. Additionally, the at least first siRNA can include a modification or a conjugate. The reverse transfection apparatus can be provided as a kit or system that additionally includes cells, polynucleotide carriers, reverse transfection reagents, and the like.

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