JP5723154B2 - ＲＮＡｉにおけるオフターゲット表現型の影響を減少させるためのＳｉＲＮＡ配列非依存性修飾フォーマットおよびその安定化型 - Google Patents

ＲＮＡｉにおけるオフターゲット表現型の影響を減少させるためのＳｉＲＮＡ配列非依存性修飾フォーマットおよびその安定化型 Download PDF

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JP5723154B2
JP5723154B2 JP2010525916A JP2010525916A JP5723154B2 JP 5723154 B2 JP5723154 B2 JP 5723154B2 JP 2010525916 A JP2010525916 A JP 2010525916A JP 2010525916 A JP2010525916 A JP 2010525916A JP 5723154 B2 JP5723154 B2 JP 5723154B2
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oligonucleotide
sirna
modified
passenger
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JP2010538677A (ja
JP2010538677A5 (enExample
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ニティン パリ，
ニティン パリ，
イルダヤ チャールズ，
イルダヤ チャールズ，
スーザン マグダレノ，
スーザン マグダレノ，
アレキサンダー ブラッソフ，
アレキサンダー ブラッソフ，
クリス バーネット，
クリス バーネット，
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アプライド バイオシステムズ リミテッド ライアビリティー カンパニー
アプライド バイオシステムズ リミテッド ライアビリティー カンパニー
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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JP2010525916A 2007-09-19 2008-09-17 ＲＮＡｉにおけるオフターゲット表現型の影響を減少させるためのＳｉＲＮＡ配列非依存性修飾フォーマットおよびその安定化型 Active JP5723154B2 (ja)

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US97354807P 2007-09-19 2007-09-19
US60/973,548 2007-09-19
PCT/US2008/076675 WO2009039173A2 (en) 2007-09-19 2008-09-17 SiRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAi, AND STABILIZED FORMS THEREOF

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JP2014035378A Division JP2014097073A (ja) 2007-09-19 2014-02-26 ＲＮＡｉにおけるオフターゲット表現型の影響を減少させるためのＳｉＲＮＡ配列非依存性修飾フォーマットおよびその安定化型

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JP2010538677A JP2010538677A (ja) 2010-12-16
JP2010538677A5 JP2010538677A5 (enExample) 2012-10-11
JP5723154B2 true JP5723154B2 (ja) 2015-05-27

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JP2014035378A Withdrawn JP2014097073A (ja) 2007-09-19 2014-02-26 ＲＮＡｉにおけるオフターゲット表現型の影響を減少させるためのＳｉＲＮＡ配列非依存性修飾フォーマットおよびその安定化型

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US (6) US8524681B2 (enExample)
EP (2) EP2548962B1 (enExample)
JP (2) JP5723154B2 (enExample)
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DK (2) DK2548962T3 (enExample)
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Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101849008A (zh) 2007-09-19 2010-09-29 应用生物系统有限公司 用于减少RNAi中的脱靶表型效应的siRNA的不依赖于序列的修饰形式和其稳定形式
JP5816556B2 (ja) 2008-12-03 2015-11-18 アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 治療剤のためのｕｎａオリゴマー構造
KR101224828B1 (ko) 2009-05-14 2013-01-22 (주)바이오니아 ｓｉＲＮＡ 접합체 및 그 제조방법
US20120207818A1 (en) * 2009-07-14 2012-08-16 Shinohara Fumikazu Composition for suppressing expression of target gene
US8877439B2 (en) * 2009-12-17 2014-11-04 Sirna Therapeutics, Inc. Method for rapidly evaluating performance of short interfering RNA with novel chemical modifications
EP2631291B1 (en) * 2010-10-22 2019-05-15 OliX Pharmaceuticals, Inc. Nucleic acid molecules inducing rna interference, and uses thereof
JP2014508102A (ja) 2010-11-15 2014-04-03 ライフ テクノロジーズ コーポレーション アミン含有トランスフェクション試薬ならびにそれを生成および使用するための方法
JP5952197B2 (ja) * 2011-01-19 2016-07-13 協和発酵キリン株式会社 標的遺伝子の発現を抑制する組成物
CN102719434A (zh) * 2011-03-31 2012-10-10 百奥迈科生物技术有限公司 抑制rna干扰脱靶效应的特异性修饰
DK2853597T3 (en) 2012-05-22 2019-04-08 Olix Pharmaceuticals Inc RNA INTERFERENCE-INducing NUCLEIC ACID MOLECULES WITH CELL PENETENING EQUIPMENT AND USE THEREOF
US10006024B2 (en) 2012-11-29 2018-06-26 Gifu University RNA interference agent, method for producing same, and use therefor
US9006200B2 (en) 2013-03-13 2015-04-14 Asbestos Diseases Research Foundation MicroRNA-based approach to treating malignant pleural mesothelioma
US9919002B2 (en) 2013-10-21 2018-03-20 North Carolina State University Methods and constructs for compound delivery
WO2016069717A1 (en) * 2014-10-28 2016-05-06 MiRagen Therapeutics, Inc. Inhibitors of mirnas in regulation of arterial stiffness and uses thereof
EP4710943A3 (en) 2015-08-28 2026-04-29 Molecular Transfer, Inc. Transfection complexes and methods of using the same
EP3377630A4 (en) 2015-11-16 2020-01-01 Olix Pharmaceuticals, Inc. Treatment of age-related macular degeneration using rna complexes that target myd88 or tlr3
EP3411481A4 (en) 2016-02-02 2020-02-26 Olix Pharmaceuticals, Inc. TREATMENT OF DISEASES ASSOCIATED WITH ANGIOGENESIS USING ANGPT2 AND PDGFB TARGETED RNA COMPLEXES
JP6944942B2 (ja) 2016-02-02 2021-10-06 オリックス ファーマシューティカルズ，インコーポレーテッド ＩＬ４Ｒα、ＴＲＰＡ１、またはＦ２ＲＬ１を標的とするＲＮＡ複合体を用いたアトピー性皮膚炎および喘息の治療
US10538784B2 (en) 2016-03-01 2020-01-21 Molecular Transfer, Inc. Plant virus movement proteins and methods of using the same
US10858656B2 (en) 2016-04-01 2020-12-08 Avidity Biosciences, Inc. KRAS nucleic acids and uses thereof
MA45349A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques egfr et leurs utilisations
MA45469A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de bêta-caténine et leurs utilisations
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
WO2017178883A2 (en) 2016-04-11 2017-10-19 Olix Pharmaceuticals, Inc. Treatment of idiopathic pulmonary fibrosis using rna complexes that target connective tissue growth factor
US10036024B2 (en) 2016-06-03 2018-07-31 Purdue Research Foundation siRNA compositions that specifically downregulate expression of a variant of the PNPLA3 gene and methods of use thereof for treating a chronic liver disease or alcoholic liver disease (ALD)
KR101916652B1 (ko) 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
WO2018055577A1 (en) * 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
CA3037663A1 (en) * 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified-rna oligonucleotide compositions and uses thereof
EP3580339A4 (en) 2017-02-10 2020-12-23 Research & Business Foundation Sungkyunkwan University LONG DOUBLE STRAND RNA FOR RNA INTERFERENCE
US12054711B2 (en) 2017-07-14 2024-08-06 City Of Hope Meta-stable oligonucleotides junctions for delivery of therapeutics
WO2019033079A1 (en) 2017-08-10 2019-02-14 City Of Hope CONDITIONAL RNAIDS AND THEIR USES IN THE TREATMENT OF CARDIAC HYPERTROPHY
CN118638788A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
CN113166750B (zh) 2018-08-10 2025-02-18 希望之城 可编程的条件性sirna及其用途
US20220195500A1 (en) * 2019-04-26 2022-06-23 Vanderbilt University Probes for detecting rna and methods of use thereof
EP4000638A4 (en) * 2019-07-16 2023-12-27 The University of Tokyo Rna molecule, chimeric na molecule, double-stranded rna molecule, and double-stranded chimeric na molecule
WO2021188390A1 (en) 2020-03-19 2021-09-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
AU2020450890B2 (en) * 2020-05-26 2025-04-10 Olix Pharmaceuticals, Inc. RNAi agent targeting MyD88 and use thereof
US20240296903A1 (en) * 2021-07-06 2024-09-05 Switch Therapeutics Inc. Methods of placing locked nucleic acids in small interfering rna strands

Family Cites Families (108)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5744101A (en) 1989-06-07 1998-04-28 Affymax Technologies N.V. Photolabile nucleoside protecting groups
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
DK0942000T3 (da) * 1989-10-24 2004-11-01 Isis Pharmaceuticals Inc 2'-modificerede oligonukleotider
US6005087A (en) * 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5412087A (en) 1992-04-24 1995-05-02 Affymax Technologies N.V. Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces
AU3728093A (en) 1992-02-19 1993-09-13 Public Health Research Institute Of The City Of New York, Inc., The Novel oligonucleotide arrays and their use for sorting, isolating, sequencing, and manipulating nucleic acids
US6309823B1 (en) 1993-10-26 2001-10-30 Affymetrix, Inc. Arrays of nucleic acid probes for analyzing biotransformation genes and methods of using the same
US20040203024A1 (en) * 1996-06-06 2004-10-14 Baker Brenda F. Modified oligonucleotides for use in RNA interference
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US20040171031A1 (en) * 1996-06-06 2004-09-02 Baker Brenda F. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US20040147022A1 (en) * 1996-06-06 2004-07-29 Baker Brenda F. 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations
WO1998012354A1 (en) 1996-09-19 1998-03-26 Affymetrix, Inc. Identification of molecular sequence signatures and methods involving the same
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
CA2303299C (en) 1997-09-12 2016-02-23 Exiqon A/S Oligonucleotide analogues
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US7045610B2 (en) * 1998-04-03 2006-05-16 Epoch Biosciences, Inc. Modified oligonucleotides for mismatch discrimination
US6324479B1 (en) 1998-05-08 2001-11-27 Rosetta Impharmatics, Inc. Methods of determining protein activity levels using gene expression profiles
US6406921B1 (en) 1998-07-14 2002-06-18 Zyomyx, Incorporated Protein arrays for high-throughput screening
US6316193B1 (en) 1998-10-06 2001-11-13 Origene Technologies, Inc. Rapid-screen cDNA library panels
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US6734291B2 (en) 1999-03-24 2004-05-11 Exiqon A/S Synthesis of [2.2.1]bicyclo nucleosides
DK1163250T3 (da) 1999-03-24 2006-11-13 Exiqon As Forbedret syntese af [2.2.1]bicyclonukleosider
NZ514348A (en) 1999-05-04 2004-05-28 Exiqon As L-ribo-LNA analogues
US6083482A (en) 1999-05-11 2000-07-04 Icn Pharmaceuticals, Inc. Conformationally locked nucleosides and oligonucleotides
WO2001025478A1 (en) 1999-10-04 2001-04-12 Exiqon A/S A method of increasing the specificity of oxy-lna oligonucleotides
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)
US20070026394A1 (en) * 2000-02-11 2007-02-01 Lawrence Blatt Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies
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
ATE450621T2 (de) * 2000-03-30 2009-12-15 Whitehead Biomedical Inst Mediatoren von rns-interferenz, die rns- sequenzspezifisch sind
EP1332150A1 (en) 2000-11-09 2003-08-06 Jyoti Chattopadhyaya Modified nucleosides and nucleotides and use thereof
KR100872437B1 (ko) * 2000-12-01 2008-12-05 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Ｒｎａ 간섭을 매개하는 작은 ｒｎａ 분자
US20030077609A1 (en) * 2001-03-25 2003-04-24 Jakobsen Mogens Havsteen Modified oligonucleotides and uses thereof
US20050148530A1 (en) * 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20070042983A1 (en) * 2001-05-18 2007-02-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US7053195B1 (en) * 2001-06-12 2006-05-30 Syngenta Participatious Ag Locked nucleic acid containing heteropolymers and related methods
GB0114719D0 (en) * 2001-06-15 2001-08-08 Glaxo Group Ltd Compound
WO2003020739A2 (en) 2001-09-04 2003-03-13 Exiqon A/S Novel lna compositions and uses thereof
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
SI2264172T1 (sl) * 2002-04-05 2017-12-29 Roche Innovation Center Copenhagen A/S Oligomerne spojine za modulacijo izražanja HIF-1alfa
US7569575B2 (en) * 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
JP4476802B2 (ja) 2002-05-08 2010-06-09 サンタリス ファーマ アー／エス ロックト核酸誘導体の製造
AU2003241621A1 (en) * 2002-05-24 2003-12-12 Isis Pharmaceuticals, Inc. Oligonucleotides having modified nucleoside units
US20050053942A1 (en) 2002-06-24 2005-03-10 Sakari Kauppinen Methods and systems for detection and isolation of a nucleotide sequence
US8101348B2 (en) * 2002-07-10 2012-01-24 Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. RNA-interference by single-stranded RNA molecules
EP1532248B1 (en) * 2002-07-26 2009-04-01 Novartis Vaccines and Diagnostics, Inc. Modified small interfering rna molecules and methods of use
ES2280826T5 (es) * 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Nuevas formas adicionales de moléculas de ARN de interferencia
WO2004014933A1 (en) * 2002-08-07 2004-02-19 University Of Massachusetts Compositions for rna interference and methods of use thereof
EP1556402B1 (en) * 2002-09-25 2011-06-22 University of Massachusetts In vivo gene silencing by chemically modified and stable sirna
US7422853B1 (en) * 2002-10-04 2008-09-09 Myriad Genetics, Inc. RNA interference using a universal target
US20040219565A1 (en) * 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
WO2004035819A2 (en) 2002-10-21 2004-04-29 Exiqon A/S Oligonucleotide analogues for detecting and analyzing nucleic acids
WO2004041889A2 (en) * 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
JP2006507841A (ja) * 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
US9045518B2 (en) * 2002-11-18 2015-06-02 Santaris Pharma A/S Amino-LNA, thio-LNA and alpha-L-oxy-LN
US20040248299A1 (en) * 2002-12-27 2004-12-09 Sumedha Jayasena RNA interference
ES2356910T3 (es) * 2003-01-17 2011-04-14 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Construcciones para la expresión inducible de arn de interferencia pequeño (arns) para el silenciamiento génico seleccionado.
US7713738B2 (en) * 2003-02-10 2010-05-11 Enzon Pharmaceuticals, Inc. Oligomeric compounds for the modulation of survivin expression
US20040241717A1 (en) * 2003-02-10 2004-12-02 Santaris Pharma A/S Oligomeric compounds for the modulation of thioredoxin expression
EP1592795A2 (en) * 2003-02-10 2005-11-09 Santaris Pharma A/S Oligomeric compounds for the modulation of ras expression
WO2004080406A2 (en) 2003-03-07 2004-09-23 Alnylam Pharmaceuticals Therapeutic compositions
US8653252B2 (en) * 2003-03-21 2014-02-18 Santaris Pharma A/S Short interfering RNA (siRNA) analogues
US20040198640A1 (en) * 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
JP4597976B2 (ja) * 2003-04-17 2010-12-15 アルナイラム ファーマシューティカルズ インコーポレイテッド 修飾ｉＲＮＡ剤
AU2004252442B2 (en) * 2003-06-02 2010-04-08 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of FNAi
ES2712695T3 (es) * 2003-06-02 2019-05-14 Univ Massachusetts Métodos y composiciones para controlar la eficacia de la silenciación del ARN
US7750144B2 (en) * 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
WO2005014782A2 (en) * 2003-06-13 2005-02-17 Alnylam Europe Ag., Double-stranded ribonucleic acid with increased effectiveness in an organism
US20060241072A1 (en) * 2003-06-20 2006-10-26 Isis Pharmaceuticals, Inc. Oligomeric compounds for use in gene modulation
US20050256071A1 (en) * 2003-07-15 2005-11-17 California Institute Of Technology Inhibitor nucleic acids
US20050136437A1 (en) * 2003-08-25 2005-06-23 Nastech Pharmaceutical Company Inc. Nanoparticles for delivery of nucleic acids and stable double-stranded RNA
US20050053981A1 (en) * 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20050086356A1 (en) * 2003-10-15 2005-04-21 Shah Mehul Y. Systems and methods for scheduled recording of multimedia content streams
US20060134787A1 (en) * 2004-12-22 2006-06-22 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
JP4642775B2 (ja) * 2003-12-23 2011-03-02 サンタリス ファーマ アー／エス Ｂｃｌ−２のモデュレーションのためのオリゴマー化合物
US20080249039A1 (en) * 2004-01-30 2008-10-09 Santaris Pharma A/S Modified Short Interfering Rna (Modified Sirna)
US8084599B2 (en) * 2004-03-15 2011-12-27 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
KR101147147B1 (ko) * 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
US7575863B2 (en) * 2004-05-28 2009-08-18 Applied Biosystems, Llc Methods, compositions, and kits comprising linker probes for quantifying polynucleotides
US8394947B2 (en) * 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
WO2006093526A2 (en) * 2004-07-21 2006-09-08 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a modified or non-natural nucleobase
EP1791982B1 (en) * 2004-09-21 2011-01-05 Life Technologies Corporation TWO-COLOR REAL-TIME/END-POINT QUANTITATION OF MICRORNAS (MIRNAs)
CA2586701C (en) * 2004-11-09 2014-06-03 Santaris Pharma A/S Lna oligonucleotides and the treatment of cancer
MX2007005558A (es) * 2004-11-09 2008-01-31 Santaris Pharma As Oligonucleotidos de lna potentes para la inhibicion dela expresion de hif-1a.
US20070065840A1 (en) * 2005-03-23 2007-03-22 Irena Naguibneva Novel oligonucleotide compositions and probe sequences useful for detection and analysis of microRNAS and their target mRNAS
US20060234277A1 (en) * 2005-03-31 2006-10-19 Amgen Inc. Method for selectively blocking hemoglobin RNA amplification
ATE532878T1 (de) * 2005-08-24 2011-11-15 Life Technologies Corp Verfahren zur quantifizierung von sirnas, mirnas und polymorphen mirnas
WO2007051303A1 (en) * 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. MODIFIED siRNA MOLECULES AND USES THEREOF
DK1984382T3 (da) * 2006-01-27 2012-09-03 Santaris Pharma As LNA modificerede fosforthiolerede oligonukleotider
CA2651453C (en) * 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
WO2009002944A1 (en) 2007-06-22 2008-12-31 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
CN101849008A (zh) 2007-09-19 2010-09-29 应用生物系统有限公司 用于减少RNAi中的脱靶表型效应的siRNA的不依赖于序列的修饰形式和其稳定形式
KR20100110298A (ko) * 2007-11-26 2010-10-12 산타리스 팔마 에이/에스 안드로겐 수용체를 표적화하는 ｌｎａ 길항제
KR100949791B1 (ko) 2007-12-18 2010-03-30 이동기 오프-타겟 효과를 최소화하고 ＲＮＡｉ 기구를 포화시키지않는 신규한 ｓｉＲＮＡ 구조 및 그 용도

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