JP2013511990A5 - - Google Patents

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Publication number
JP2013511990A5
JP2013511990A5 JP2012541211A JP2012541211A JP2013511990A5 JP 2013511990 A5 JP2013511990 A5 JP 2013511990A5 JP 2012541211 A JP2012541211 A JP 2012541211A JP 2012541211 A JP2012541211 A JP 2012541211A JP 2013511990 A5 JP2013511990 A5 JP 2013511990A5
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JP
Japan
Prior art keywords
double
nucleic acid
stranded nucleic
acid molecule
modified
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
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JP2012541211A
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English (en)
Japanese (ja)
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JP2013511990A (ja
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Priority claimed from PCT/US2010/058123 external-priority patent/WO2011066475A1/en
Publication of JP2013511990A publication Critical patent/JP2013511990A/ja
Publication of JP2013511990A5 publication Critical patent/JP2013511990A5/ja
Pending legal-status Critical Current

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JP2012541211A 2009-11-26 2010-11-25 末端置換を含むsiRNA化合物 Pending JP2013511990A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US26466809P 2009-11-26 2009-11-26
US61/264,668 2009-11-26
US29572110P 2010-01-17 2010-01-17
US61/295,721 2010-01-17
US37207210P 2010-08-09 2010-08-09
US61/372,072 2010-08-09
PCT/US2010/058123 WO2011066475A1 (en) 2009-11-26 2010-11-25 Sirna compounds comprising terminal substitutions

Publications (2)

Publication Number Publication Date
JP2013511990A JP2013511990A (ja) 2013-04-11
JP2013511990A5 true JP2013511990A5 (https=) 2014-01-09

Family

ID=43589717

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2012541211A Pending JP2013511990A (ja) 2009-11-26 2010-11-25 末端置換を含むsiRNA化合物

Country Status (13)

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US (3) US8796239B2 (https=)
EP (1) EP2504435B1 (https=)
JP (1) JP2013511990A (https=)
KR (1) KR20120102630A (https=)
CN (1) CN102597239A (https=)
AU (1) AU2010324658A1 (https=)
CA (1) CA2776568A1 (https=)
DK (1) DK2504435T3 (https=)
ES (1) ES2759003T3 (https=)
IL (1) IL218965A0 (https=)
NZ (1) NZ599237A (https=)
TW (1) TW201124160A (https=)
WO (1) WO2011066475A1 (https=)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2411413B1 (en) 2009-03-23 2016-05-11 Quark Pharmaceuticals, Inc. Compounds compositions and methods of treating cancer and fibrotic diseases
AU2010324658A1 (en) * 2009-11-26 2012-05-03 Quark Pharmaceuticals, Inc. siRNA compounds comprising terminal substitutions
WO2011072082A2 (en) 2009-12-09 2011-06-16 Nitto Denko Corporation Modulation of hsp47 expression
WO2011084193A1 (en) 2010-01-07 2011-07-14 Quark Pharmaceuticals, Inc. Oligonucleotide compounds comprising non-nucleotide overhangs
AU2011338682B2 (en) 2010-12-06 2017-04-27 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising threose modifications
CA2828544A1 (en) 2011-03-03 2012-09-07 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
US9796979B2 (en) 2011-03-03 2017-10-24 Quark Pharmaceuticals Inc. Oligonucleotide modulators of the toll-like receptor pathway
EP2681314B1 (en) 2011-03-03 2017-11-01 Quark Pharmaceuticals, Inc. Compositions and methods for treating lung disease and injury
AU2012332517B9 (en) 2011-11-03 2017-08-10 Quark Pharmaceuticals, Inc. Methods and compositions for neuroprotection
US20140323549A1 (en) 2011-11-08 2014-10-30 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the nervous system
JP2015502365A (ja) * 2011-12-12 2015-01-22 オンコイミューニン,インコーポレイティド オリゴヌクレオチドのイン−ビボ送達
JP2015509085A (ja) 2012-01-01 2015-03-26 キュービーアイ エンタープライゼズ リミテッドQbi Enterprises Ltd. 治療剤および診断剤の選択的送達のためのendo180を標的とする粒子
EP2895607B1 (en) 2012-09-12 2021-05-05 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide molecules to ddit4 and methods of use thereof
US9611473B2 (en) 2012-09-12 2017-04-04 Quark Pharmaceuticals, Inc. Double-stranded nucleic acid compounds
ES2704855T3 (es) 2012-09-12 2019-03-20 Quark Pharmaceuticals Inc Moléculas de oligonucleótido de doble cadena para p53 y métodos de uso de las mismas
CN105452465B (zh) 2013-07-31 2019-06-21 奇比艾企业有限公司 鞘脂-聚烷基胺-寡核苷酸化合物
EP3027223A1 (en) 2013-07-31 2016-06-08 QBI Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
KR101696704B1 (ko) * 2013-12-17 2017-01-16 주식회사 인코드젠 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도
KR101627404B1 (ko) * 2014-04-08 2016-06-03 성균관대학교산학협력단 Dab2 유전자가 넉 다운된 수지상 세포를 포함하는 암 예방 또는 치료용 약학적 조성물
CA2949109A1 (en) 2014-05-29 2015-12-03 Quark Pharmaceuticals, Inc. Methods and compositions for preventing ischemia reperfusion injury in organs
US20160089391A1 (en) * 2014-09-30 2016-03-31 Temple University-Of The Commonwealth System Of Higher Education Molecular Targets for Cardiovascular Disease
WO2017173297A1 (en) * 2016-04-01 2017-10-05 Avidity Biosciences Llc Beta-catenin nucleic acids and uses thereof
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
TN2020000038A1 (en) * 2017-09-14 2021-10-04 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use
WO2019165267A1 (en) * 2018-02-22 2019-08-29 The Regents Of The University Of California Methods for treating brain injury
CN112331258B (zh) * 2020-11-06 2024-02-23 大连大学 基于dna笼状结构的人工神经元计算模型构建方法
KR102404883B1 (ko) * 2020-11-30 2022-06-07 (주)이노보테라퓨틱스 벤즈브로마론을 포함하는 켈로이드 또는 비대흉터 예방 또는 치료용 약학 조성물
AU2022315530A1 (en) 2021-07-20 2024-01-18 Ags Therapeutics Sas Extracellular vesicles from microalgae, their preparation, and uses
WO2023144127A1 (en) 2022-01-31 2023-08-03 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon administration, and uses
WO2023232976A1 (en) 2022-06-03 2023-12-07 Ags Therapeutics Sas Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses
WO2024088808A1 (en) 2022-10-24 2024-05-02 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon intranasal administration, and uses thereof
WO2025176843A1 (en) 2024-02-21 2025-08-28 Ags Therapeutics Sas Microalgae extracellular vesicle based gene therapy vectors (mev-gtvs), their preparation, and uses thereof
CN120574835B (zh) * 2025-07-31 2025-10-03 羿美诚健(上海)生物医药有限公司 一种靶向rankl基因的小核酸及其在治疗骨科疾病中的应用

Family Cites Families (85)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4475196A (en) 1981-03-06 1984-10-02 Zor Clair G Instrument for locating faults in aircraft passenger reading light and attendant call control system
US4447233A (en) 1981-04-10 1984-05-08 Parker-Hannifin Corporation Medication infusion pump
US4439196A (en) 1982-03-18 1984-03-27 Merck & Co., Inc. Osmotic drug delivery system
US4447224A (en) 1982-09-20 1984-05-08 Infusaid Corporation Variable flow implantable infusion apparatus
US4487603A (en) 1982-11-26 1984-12-11 Cordis Corporation Implantable microinfusion pump system
US4486194A (en) 1983-06-08 1984-12-04 James Ferrara Therapeutic device for administering medicaments through the skin
US4666828A (en) 1984-08-15 1987-05-19 The General Hospital Corporation Test for Huntington's disease
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4801531A (en) 1985-04-17 1989-01-31 Biotechnology Research Partners, Ltd. Apo AI/CIII genomic polymorphisms predictive of atherosclerosis
US4959217A (en) 1986-05-22 1990-09-25 Syntex (U.S.A.) Inc. Delayed/sustained release of macromolecules
US4925678A (en) 1987-04-01 1990-05-15 Ranney David F Endothelial envelopment drug carriers
US5080646A (en) 1988-10-03 1992-01-14 Alza Corporation Membrane for electrotransport transdermal drug delivery
US5272057A (en) 1988-10-14 1993-12-21 Georgetown University Method of detecting a predisposition to cancer by the use of restriction fragment length polymorphism of the gene for human poly (ADP-ribose) polymerase
US5270030A (en) 1988-12-29 1993-12-14 Bio-Technology General Corp. Fibrin binding domain polypeptide and method of producing
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5192659A (en) 1989-08-25 1993-03-09 Genetype Ag Intron sequence analysis method for detection of adjacent and remote locus alleles as haplotypes
US5167616A (en) 1989-12-14 1992-12-01 Alza Corporation Iontophoretic delivery method
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5225182A (en) 1991-10-31 1993-07-06 Sharma Yash P Vectored drug delivery system using a cephaloplastin linking agent and a methed of using the system
JP3131222B2 (ja) 1991-12-24 2001-01-31 アイシス・ファーマシューティカルス・インコーポレーテッド ギャップを有する2′修飾オリゴヌクレオチド
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION.
US6235886B1 (en) 1993-09-03 2001-05-22 Isis Pharmaceuticals, Inc. Methods of synthesis and use
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
ATE227342T1 (de) 1993-09-02 2002-11-15 Ribozyme Pharm Inc Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
US20050042647A1 (en) 1996-06-06 2005-02-24 Baker Brenda F. Phosphorous-linked oligomeric compounds and their use in gene modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US6251666B1 (en) 1997-03-31 2001-06-26 Ribozyme Pharmaceuticals, Inc. Nucleic acid catalysts comprising L-nucleotide analogs
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
CA2361201A1 (en) 1999-01-28 2000-08-03 Medical College Of Georgia Research Institute, Inc. Composition and method for in vivo and in vitro attenuation of gene expression using double stranded rna
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
WO2000049035A1 (en) 1999-02-19 2000-08-24 The General Hospital Corporation Gene silencing
KR20070118315A (ko) 1999-04-21 2007-12-14 와이어쓰 폴리뉴클레오티드 서열의 기능을 억제하기 위한 조성물
HK1047109A1 (zh) 1999-10-15 2003-02-07 University Of Massachusetts 作为指定基因干预工具的rna干预轨迹基因
GB9925459D0 (en) 1999-10-27 1999-12-29 Plant Bioscience Ltd Gene silencing
WO2001036641A2 (en) 1999-11-02 2001-05-25 Chiron Corporation DOUBLE-STRANDED RNA RECEPTOR (dsRNA-R) AND METHODS RELATING THERETO
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US20050020525A1 (en) 2002-02-20 2005-01-27 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
EP1272630A2 (en) 2000-03-16 2003-01-08 Genetica, Inc. Methods and compositions for rna interference
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
ES2467154T3 (es) 2000-05-18 2014-06-12 Commwell, Inc. Método para la monitorización médica remota que incorpora procesamiento de video
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
EP1386004A4 (en) * 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
US20070032441A1 (en) 2001-05-18 2007-02-08 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (sina)
US20040063654A1 (en) 2001-11-02 2004-04-01 Davis Mark E. Methods and compositions for therapeutic use of RNA interference
EP1445312B1 (en) 2001-11-21 2012-12-26 Astellas Pharma Inc. Method of inhibiting gene expression
EP1572902B1 (en) 2002-02-01 2014-06-11 Life Technologies Corporation HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc RNA inhibition mediated inhibition of MAP KINASE GENES
US20040254146A1 (en) 2002-05-21 2004-12-16 Nastech Pharmaceutical Company Inc. Carboxylate salts of galantamine and their pharmaceutical use
US20030225031A1 (en) 2002-05-21 2003-12-04 Quay Steven C. Administration of acetylcholinesterase inhibitors to the cerebral spinal fluid
DE60310944T3 (de) 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Weitere neue formen von interferierende rns moleküle
AU2003291755A1 (en) * 2002-11-05 2004-06-07 Isis Pharmaceuticals, Inc. Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use
US9150605B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US20050233342A1 (en) 2003-03-07 2005-10-20 Muthiah Manoharan Methods of preventing off-target gene silencing
PT1633767T (pt) 2003-06-02 2019-02-27 Univ Massachusetts Métodos e composições para controlar a eficácia do silenciamento de arn
EP1633890B2 (en) 2003-06-02 2020-11-18 University of Massachusetts METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF FNAi
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
EP1636342A4 (en) 2003-06-20 2008-10-08 Isis Pharmaceuticals Inc OLIGOMER COMPOUNDS FOR USE IN THE MODULATION OF GENES
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
EP1791567B1 (en) 2004-08-10 2015-07-29 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
EP2319925B1 (en) 2004-08-16 2018-07-25 Quark Pharmaceuticals, Inc. Therapeutic uses of inhibitors of RTP801
AU2005288522B2 (en) 2004-09-28 2012-06-28 Quark Pharmaceuticals, Inc. Oligoribonucleotides and methods of use thereof for treatment of alopecia, acute renal failure and other diseases
NL2000439C2 (nl) 2006-01-20 2009-03-16 Quark Biotech Therapeutische toepassingen van inhibitoren van RTP801.
US20070259827A1 (en) 2006-01-25 2007-11-08 University Of Massachusetts Compositions and methods for enhancing discriminatory RNA interference
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
WO2007141796A2 (en) 2006-06-09 2007-12-13 Quark Pharmaceuticals, Inc. Therapeutic uses of inhibitors of rtp801l
JP2010507387A (ja) 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のsiRNAおよびその使用方法
US7872119B2 (en) 2007-02-26 2011-01-18 Quark Pharmaceuticals, Inc. Inhibitors of RTP801 and their use in disease treatment
US20100292301A1 (en) 2007-02-28 2010-11-18 Elena Feinstein Novel sirna structures
US7812002B2 (en) 2007-03-21 2010-10-12 Quark Pharmaceuticals, Inc. Oligoribonucleotide inhibitors of NRF2 and methods of use thereof for treatment of cancer
WO2008152636A2 (en) 2007-06-15 2008-12-18 Quark Pharmaceuticals, Inc. Compositions and methods for inhibiting nadph oxidase expression
WO2009001359A2 (en) 2007-06-27 2008-12-31 Quark Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of pro-apoptotic genes
RU2487716C2 (ru) * 2007-10-03 2013-07-20 Кварк Фармасьютикалс, Инк. Новые структуры малых интерферирующих рнк (sirna)
EP2242854A4 (en) 2008-01-15 2012-08-15 Quark Pharmaceuticals Inc COMPOUNDS AND USES THEREOF
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
CA2962219C (en) 2008-10-22 2020-08-25 Quark Pharmaceuticals, Inc. Methods for treating eye disorders
AU2010324658A1 (en) * 2009-11-26 2012-05-03 Quark Pharmaceuticals, Inc. siRNA compounds comprising terminal substitutions
WO2011072082A2 (en) 2009-12-09 2011-06-16 Nitto Denko Corporation Modulation of hsp47 expression
AU2011338682B2 (en) 2010-12-06 2017-04-27 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising threose modifications
EP2681314B1 (en) * 2011-03-03 2017-11-01 Quark Pharmaceuticals, Inc. Compositions and methods for treating lung disease and injury

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