JP5243789B2 - 二本鎖rnaによる遺伝子発現の特異的阻害のための方法及び組成物 - Google Patents

二本鎖rnaによる遺伝子発現の特異的阻害のための方法及び組成物 Download PDF

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JP5243789B2
JP5243789B2 JP2007504009A JP2007504009A JP5243789B2 JP 5243789 B2 JP5243789 B2 JP 5243789B2 JP 2007504009 A JP2007504009 A JP 2007504009A JP 2007504009 A JP2007504009 A JP 2007504009A JP 5243789 B2 JP5243789 B2 JP 5243789B2
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strand
stranded rna
double
nucleotides
rna
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JP2007529224A (ja
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ロッシ,ジョン・ジェイ
ベールケ,マーク・エイ
キム,ドンホ
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City of Hope
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JP2007504009A 2004-03-15 2005-03-15 二本鎖rnaによる遺伝子発現の特異的阻害のための方法及び組成物 Expired - Lifetime JP5243789B2 (ja)

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US55348704P 2004-03-15 2004-03-15
US60/553,487 2004-03-15
PCT/US2005/008477 WO2005089287A2 (en) 2004-03-15 2005-03-15 Methods and compositions for the specific inhibition of gene expression by double-stranded rna

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JP2007529224A JP2007529224A (ja) 2007-10-25
JP2007529224A5 JP2007529224A5 (enExample) 2008-05-08
JP5243789B2 true JP5243789B2 (ja) 2013-07-24

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US (26) US8084599B2 (enExample)
EP (2) EP1742958B1 (enExample)
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Families Citing this family (200)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
WO2003064621A2 (en) 2002-02-01 2003-08-07 Ambion, Inc. HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES
CA2474910A1 (en) 2002-02-01 2003-08-07 Sequitur, Inc. Oligonucleotide compositions with enhanced efficiency
US7892793B2 (en) * 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US9879266B2 (en) 2002-11-14 2018-01-30 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
AU2004248136B2 (en) 2003-06-02 2011-09-15 University Of Massachusetts Methods and compositions for controlling efficacy of RNA silencing
ATE485394T1 (de) * 2003-06-02 2010-11-15 Univ Massachusetts Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai
US7750144B2 (en) * 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
US8680063B2 (en) * 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
ES2969371T3 (es) * 2003-09-12 2024-05-17 Univ Massachusetts Interferencia por ARN para el tratamiento de trastornos de ganancia de función
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
US20070265220A1 (en) * 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2005089287A2 (en) * 2004-03-15 2005-09-29 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded rna
WO2006085987A2 (en) * 2004-07-09 2006-08-17 University Of Iowa Research Foundation Rna interference in respiratory epitheial cells
US20060142228A1 (en) 2004-12-23 2006-06-29 Ambion, Inc. Methods and compositions concerning siRNA's as mediators of RNA interference
GB2421948A (en) * 2004-12-30 2006-07-12 Ist Superiore Sanita Retrotransposon inhibition to treat cancer
US20070213293A1 (en) * 2005-04-08 2007-09-13 Nastech Pharmaceutical Company Inc. Rnai therapeutic for respiratory virus infection
WO2006138145A1 (en) 2005-06-14 2006-12-28 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
CA2619534A1 (en) * 2005-08-18 2007-02-22 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating neurological disease
US20080124365A1 (en) * 2006-05-24 2008-05-29 Genolution Pharmaceuticals, Inc. Innate immune response inducer
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
HUE037173T2 (hu) 2006-08-08 2018-08-28 Univ Bonn Rheinische Friedrich Wilhelms 5'-Foszfát-oligonukleotidok szerkezete és alkalmazása
CA2666657A1 (en) * 2006-10-18 2008-04-24 Nastech Pharmaceutical Company Inc. Nicked or gapped nucleic acid molecules and uses thereof
CA2691066C (en) 2007-02-09 2018-07-31 Northwestern University Particles for detecting intracellular targets
JP5311854B2 (ja) 2007-03-23 2013-10-09 キヤノン株式会社 電子写真画像形成装置、現像装置、及び、カップリング部材
WO2008143774A2 (en) * 2007-05-01 2008-11-27 University Of Massachusetts Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy
CN101679978B (zh) * 2007-05-22 2016-05-04 阿克丘勒斯治疗公司 羟甲基取代的rna寡核苷酸和rna复合物
AU2013273719B2 (en) * 2007-05-22 2016-04-14 Arcturus Therapeutics, Inc. UNA amidites for therapeutic oligomers
MX2009013046A (es) 2007-05-30 2010-02-17 Univ Northwestern Nanoparticulas con grupo funcional de acido nucleico para aplicaciones terapeuticas.
TW200916117A (en) 2007-08-03 2009-04-16 Alcon Res Ltd RNAi-related inhibition of TNF α signaling pathway for treatment of ocular angiogenesis
CN105018492B (zh) * 2007-08-27 2018-08-24 北京强新生物科技有限公司 不对称干扰rna的组合物及其用途
JP5723154B2 (ja) * 2007-09-19 2015-05-27 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 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
TW200927177A (en) * 2007-10-24 2009-07-01 Nat Inst Of Advanced Ind Scien Lipid-modified double-stranded RNA having potent RNA interference effect
US8030290B2 (en) * 2007-12-07 2011-10-04 City Of Hope Cell-type specific aptamer-siRNA delivery system for HIV-1 Therapy
US9738680B2 (en) 2008-05-21 2017-08-22 Rheinische Friedrich-Wilhelms-Universität Bonn 5′ triphosphate oligonucleotide with blunt end and uses thereof
JP2011530289A (ja) * 2008-08-05 2011-12-22 マリーナ バイオテック,インコーポレイテッド Plk1遺伝子の発現を抑制するための核酸化合物およびその使用
EP2323695B1 (en) 2008-08-19 2018-12-05 Nektar Therapeutics Complexes of small-interfering nucleic acids
AU2009293636A1 (en) * 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsRNA possessing modifications
EP2365803B1 (en) * 2008-11-24 2017-11-01 Northwestern University Polyvalent rna-nanoparticle compositions
WO2010065756A2 (en) 2008-12-03 2010-06-10 Mdrna, Inc. Usirna complexes
AU2009336191B2 (en) * 2008-12-18 2017-08-24 Novo Nordisk A/S Extended dicer substrate agents and methods for the specific inhibition of gene expression
US11408003B2 (en) 2008-12-18 2022-08-09 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
EP3199165B1 (en) * 2009-04-03 2022-06-08 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of kras by asymmetric double-stranded rna
EP2756845B1 (en) * 2009-04-03 2017-03-15 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA
EP2437752A2 (en) * 2009-06-03 2012-04-11 Dicerna Pharmaceuticals, Inc. Peptide dicer substrate agents and methods for their specific inhibition of gene expression
WO2010141933A1 (en) * 2009-06-05 2010-12-09 Dicerna Pharmaceuticals, Inc. Specific inhibition of gene expression by nucleic acid containing a dicer substrate
CN102803496A (zh) 2009-06-10 2012-11-28 淡马锡生命科学研究院有限公司 用于棉花中基因功能性分析的病毒诱导的基因沉默(vigs)
JP5766188B2 (ja) 2009-07-01 2015-08-19 プロチバ バイオセラピューティクス インコーポレイティッド 固形腫瘍に治療剤を送達するための脂質製剤
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
WO2011032100A1 (en) 2009-09-11 2011-03-17 Government Of The U.S.A., As Represented By The Secretary, Department Of Health And Human Services Inhibitors of kshv vil6 and human il6
US8916693B2 (en) 2009-09-17 2014-12-23 Nektar Therapeutics Monoconjugated chitosans as delivery agents for small interfering nucleic acids
WO2011038160A2 (en) 2009-09-23 2011-03-31 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
EP2494075B1 (en) 2009-10-30 2018-04-04 Northwestern University Templated nanoconjugates
TWI611021B (zh) 2009-12-09 2018-01-11 日東電工股份有限公司 Hsp47表現之調節
JP5257796B2 (ja) * 2009-12-28 2013-08-07 株式会社村田製作所 固体電解コンデンサ素子及びその製造方法
WO2011109294A1 (en) * 2010-03-01 2011-09-09 Dicerna Pharmaceuticals, Inc. Lipid delivery formulations
EP2554669B1 (en) 2010-03-26 2018-09-19 Kyowa Hakko Kirin Co., Ltd. Novel antibody having modification site introduced therein, and antibody fragment
US20130202578A1 (en) * 2010-04-22 2013-08-08 Noah C. Welker Compositions and methods for selectively producing sirna
WO2011136368A1 (ja) 2010-04-28 2011-11-03 協和発酵キリン株式会社 カチオン性脂質
US20130129811A1 (en) 2010-04-28 2013-05-23 Takeshi Kuboyama Cationic lipid
CA2804210A1 (en) 2010-07-06 2012-01-12 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of androgen receptor by double-stranded rna
ES2734743T3 (es) 2010-07-06 2019-12-11 Dicerna Pharmaceuticals Inc Métodos y composiciones para la inhibición específica de beta-catenina por RNA bicatenario
WO2012051491A1 (en) 2010-10-14 2012-04-19 The United States Of America, As Represented By The Secretary National Institutes Of Health Compositions and methods for controlling neurotropic viral pathogenesis by micro-rna targeting
CN103492572A (zh) 2011-03-03 2014-01-01 夸克医药公司 用于治疗肺疾病和损伤的组合物和方法
EP2508530A1 (en) 2011-03-28 2012-10-10 Rheinische Friedrich-Wilhelms-Universität Bonn Purification of triphosphorylated oligonucleotides using capture tags
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
TWI658830B (zh) 2011-06-08 2019-05-11 日東電工股份有限公司 Hsp47表現調控強化用類視色素脂質體
BR112013031723B1 (pt) 2011-06-10 2020-10-13 Temasek Life Sciences Laboratory Limited construção de polinucleotídeo, método para transformação de uma célula fúngica e promotor isolado
CN104023749B (zh) 2011-09-14 2019-12-17 西北大学 能穿过血脑屏障的纳米缀合物
CA2852917C (en) 2011-10-18 2020-07-07 Dicerna Pharmaceuticals, Inc. Amine cationic lipids and uses thereof
WO2013060855A1 (en) * 2011-10-27 2013-05-02 Occlutech Holding Ag A medical implant, a kit and a method of manufacturing a 3d fabric of strands for forming a medical implant
JP2013095755A (ja) 2011-11-02 2013-05-20 Kyowa Hakko Kirin Co Ltd カチオン性脂質
CA2858694A1 (en) 2011-12-12 2013-06-20 Kyowa Hakko Kirin Co., Ltd. Lipid nano particles comprising combination of cationic lipids
US9839616B2 (en) 2011-12-12 2017-12-12 Kyowa Hakko Kirin Co., Ltd. Lipid nano particles comprising cationic lipid for drug delivery system
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
PT2817287T (pt) 2012-02-24 2018-12-28 Arbutus Biopharma Corp Lípidos catiónicos trialquílicos e seus métodos de utilização
WO2013158032A1 (en) 2012-04-19 2013-10-24 Temasek Life Sciences Laboratory Limited Methods for increasing cotton fiber length
US9272016B2 (en) 2012-04-20 2016-03-01 University Of Iowa Research Foundation Methods to enhance RNAi oligonucleotide delivery to respiratory epithelial cells
TW201408625A (zh) 2012-07-06 2014-03-01 Kyowa Hakko Kirin Co Ltd 陽離子性脂質
EP2876162A4 (en) * 2012-07-17 2016-03-16 Stelic Institute&Co MUCOSA CURE PROMOTER
WO2014018375A1 (en) 2012-07-23 2014-01-30 Xenon Pharmaceuticals Inc. Cyp8b1 and uses thereof in therapeutic and diagnostic methods
JP6336390B2 (ja) 2012-08-31 2018-06-06 協和発酵キリン株式会社 オリゴヌクレオチド
CN104884065B (zh) 2012-09-21 2019-01-01 强烈治疗剂公司 治疗癌症的方法
EP2712870A1 (en) 2012-09-27 2014-04-02 Rheinische Friedrich-Wilhelms-Universität Bonn Novel RIG-I ligands and methods for producing them
WO2014055825A1 (en) 2012-10-04 2014-04-10 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services A formulation of mycobacterial components as an adjuvant for inducing th17 responses
EP2931746A4 (en) 2012-12-14 2016-08-24 Dicerna Pharmaceuticals Inc METHOD AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF CKAP5 BY DOUBLE-STRUCTURED RNA
US10258682B2 (en) 2013-01-16 2019-04-16 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Attenuated chlamydia vaccine
SG11201506805QA (en) 2013-02-28 2015-09-29 Arrowhead Res Corp Organic compositions to treat epas1-related diseases
KR102310921B1 (ko) 2013-03-14 2021-10-13 다이서나 파마수이티컬, 인크. 음이온성 약제를 제형화하는 방법
US9546364B2 (en) 2013-03-15 2017-01-17 Carnegie Mellon University Synthetic lariat RNA for RNA interference
ES2905257T3 (es) 2013-07-03 2022-04-07 Dicerna Pharmaceuticals Inc Métodos y composiciones para la inhibición específica de alfa-1 antitripsina mediante ARN bicatenario
CN105230024B (zh) * 2013-07-19 2019-05-24 华为技术有限公司 一种媒体表示自适应方法、装置及计算机存储介质
KR20160035081A (ko) 2013-08-07 2016-03-30 애로우헤드 리서치 코오포레이션 생체 내에서 RNAi 유발제를 종양 세포로 전달하기 위한 폴리콘주게이트
WO2015085158A1 (en) 2013-12-06 2015-06-11 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of transthyretin (ttr) by double-stranded rna
ES2875558T3 (es) 2013-12-27 2021-11-10 Dicerna Pharmaceuticals Inc Métodos y composiciones para la inhibición específica de glicolato oxidasa (HAO1) por ARN de doble cadena
CN105980560A (zh) 2014-01-17 2016-09-28 协和发酵麒麟株式会社 抑制β2GPI表达的核酸
JP6752151B2 (ja) 2014-03-25 2020-09-09 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. 遺伝子サイレンシングにおいて低下したoff−target効果を有するunaオリゴマー
US9856475B2 (en) 2014-03-25 2018-01-02 Arcturus Therapeutics, Inc. Formulations for treating amyloidosis
WO2015148582A1 (en) 2014-03-25 2015-10-01 Arcturus Therapeutics, Inc. Transthyretin allele selective una oligomers for gene silencing
US9987299B2 (en) 2014-05-05 2018-06-05 University Of Iowa Research Foundation Methods of improving RNAi in well-differentiated airway epithelia
US10517890B2 (en) 2014-05-06 2019-12-31 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Triggering RNA interference with RNA-DNA and DNA-RNA nanoparticles
JP6779866B2 (ja) 2014-06-13 2020-11-04 チルドレンズ メディカル センター コーポレイション ミトコンドリアを単離するための製品および方法
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α-1抗胰蛋白酶基因表現之組合物及方法
KR101670254B1 (ko) * 2014-07-09 2016-10-28 서울대학교산학협력단 목적 유전자의 발현 억제 특이성이 증가된 siRNA 분자
US12264344B2 (en) 2014-08-19 2025-04-01 Northwestern University Protein/oligonucleotide core-shell nanoparticle therapeutics
JP2016071140A (ja) 2014-09-30 2016-05-09 富士フイルム株式会社 ズームレンズおよび撮像装置
US10351854B2 (en) 2014-10-10 2019-07-16 Dicerna Pharmaceuticals, Inc. Therapeutic inhibition of lactate dehydrogenase and agents therefor
JOP20200115A1 (ar) * 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
US10266837B2 (en) 2014-10-22 2019-04-23 Temasek Life Sciences Laboratory Limited Terpene synthases from ylang ylang (Cananga odorata var. fruticosa)
EP3220895B1 (en) 2014-11-21 2022-08-31 Northwestern University The sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
EP3865576A1 (en) 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
US10264976B2 (en) 2014-12-26 2019-04-23 The University Of Akron Biocompatible flavonoid compounds for organelle and cell imaging
US10036017B2 (en) 2015-02-17 2018-07-31 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of complement component 5(C5) by double-stranded RNA
JP6773677B2 (ja) 2015-03-17 2020-10-21 アローヘッド ファーマシューティカルズ インコーポレイテッド 改善されたジスルフィド含有アルキン連結剤
WO2016149331A2 (en) 2015-03-17 2016-09-22 Arrowhead Research Corporation Compositions and methods for inhibiting gene expression of factor xii
JP6830441B2 (ja) 2015-04-01 2021-02-17 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. 治療上のunaオリゴマーおよびその使用
US10689647B2 (en) 2015-05-08 2020-06-23 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of antithrombin 3 (AT3) by double-stranded RNA
CN107614002B (zh) 2015-05-29 2022-01-04 箭头药业股份有限公司 生物学可切割四肽连接剂
TWI761305B (zh) 2015-05-29 2022-04-21 美商愛羅海德製藥公司 抑制Hif2α基因表現之組合物及方法
WO2017015671A1 (en) 2015-07-23 2017-01-26 Arcturus Therapeutics, Inc. Compositions for treating amyloidosis
AU2016306275A1 (en) 2015-08-07 2018-02-08 Arrowhead Pharmaceuticals, Inc. RNAi therapy for Hepatitis B virus infection
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
EP3397261B1 (en) 2015-11-30 2025-07-09 Sana Biotechnology, Inc. Sub-mitochondrial particles for use in treating cardiovascular diseases
KR102740699B1 (ko) 2015-12-13 2024-12-09 닛토덴코 가부시키가이샤 높은 활성 및 감소한 오프 타겟을 위한 sirna 구조
JP6774965B2 (ja) 2015-12-25 2020-10-28 協和キリン株式会社 カチオン性脂質としての化合物
CN105462932A (zh) * 2015-12-29 2016-04-06 同济大学苏州研究院 一种采用慢病毒技术构建的抗肿瘤迁移药物筛选细胞模型及其应用方法
WO2017124037A1 (en) 2016-01-15 2017-07-20 The Children's Medical Center Corporation Therapeutic use of mitochondria and combined mitochondrial agents
SG11201806868TA (en) 2016-02-25 2018-09-27 Applied Biological Laboratories Inc Compositions and methods for protecting against airborne pathogens and irritants
US12447166B2 (en) 2016-02-25 2025-10-21 Applied Biological Laboratories, Inc. Compositions and methods for protecting against airborne pathogens and irritants
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45469A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de bêta-caténine et leurs utilisations
MA45470A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques kras et leurs utilisations
MA45349A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques egfr et leurs utilisations
CN107333099B (zh) * 2016-04-28 2019-11-19 瑞昱半导体股份有限公司 具有无线中继功能的网络摄影机
US11091795B2 (en) 2016-07-11 2021-08-17 Arizona Board Of Regents On Behalf Of The University Of Arizona Compositions and methods for diagnosing and treating arrhythmias
JOP20170161A1 (ar) 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
TW202436621A (zh) 2017-01-10 2024-09-16 美商愛羅海德製藥公司 α-1抗胰蛋白酶 (AAT) RNAi 藥劑、包含AAT RNAi 藥劑之組合物及使用方法
WO2018140630A1 (en) 2017-01-25 2018-08-02 Northwestern University Autophagy inducers for treatment of cns conditions
EP3594345A4 (en) 2017-03-09 2021-05-19 Kyowa Kirin Co., Ltd. Nucleic acid capable of inhibiting expression of masp2
WO2018187373A1 (en) 2017-04-03 2018-10-11 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Functionally-interdependent shape switching nucleic acid nanoparticles
WO2018199340A1 (ja) * 2017-04-28 2018-11-01 協和発酵キリン株式会社 オリゴヌクレオチド誘導体またはその塩
WO2018209270A1 (en) 2017-05-11 2018-11-15 Northwestern University Adoptive cell therapy using spherical nucleic acids (snas)
US11466272B2 (en) 2017-05-31 2022-10-11 Kyowa Kirin Co., Ltd. Nucleic acid suppressing expression of APCS
KR20200038236A (ko) 2017-06-13 2020-04-10 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 큐론을 포함하는 조성물 및 그의 용도
US11261447B2 (en) 2017-07-13 2022-03-01 Alnylam Pharmaceuticals, Inc. Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
CA3071968A1 (en) 2017-08-04 2019-02-07 Kyowa Kirin Co., Ltd. Nucleic acid-containing lipid nanoparticle
KR102609396B1 (ko) 2017-10-13 2023-12-01 노보 노르디스크 헬스 케어 악티엔게젤샤프트 Ldha의 발현을 억제하기 위한 방법 및 조성물
AU2018372906B2 (en) 2017-11-22 2025-04-03 The Regents Of The University Of Michigan Compositions and methods for treating cancer
KR20250148679A (ko) 2017-12-06 2025-10-14 어비디티 바이오사이언시스 인크. 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법
BR112020011670A2 (pt) 2017-12-15 2020-11-17 Flagship Pioneering Innovations Vi, Llc. composições compreendendo polirribonucleotídeos circulares e seus usos
US11746151B2 (en) 2018-04-13 2023-09-05 The Regents Of The University Of Michigan Compositions and methods for treating cancer
TWI840449B (zh) 2018-11-16 2024-05-01 日商日東電工股份有限公司 用於惡性腫瘤之rna干擾輸送配方及方法
EP3888663A1 (en) 2018-11-30 2021-10-06 Kyowa Kirin Co., Ltd. Nucleic acid complex
CA3120580A1 (en) 2019-01-09 2020-07-16 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
KR20210142678A (ko) 2019-03-25 2021-11-25 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 변형된 원형 폴리리보뉴클레오티드를 포함하는 조성물 및 그의 용도
WO2020205473A1 (en) 2019-03-29 2020-10-08 Decerna Pharmaceuticals, Inc. Compositions and methods for the treatment of kras associated diseases or disorders
CA3149775A1 (en) 2019-08-30 2021-03-04 Weimin Wang Ligand-2'-modified nucleic acids, synthesis thereof and intermediate compounds thereof
AU2020355000A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
JP2022548399A (ja) 2019-09-23 2022-11-18 オメガ セラピューティクス, インコーポレイテッド 肝細胞核因子4-アルファ(HNF4α)遺伝子発現をモジュレートするための組成物および方法
EP4061945A1 (en) 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
US20230181620A1 (en) 2020-01-29 2023-06-15 Flagship Pioneering Innovations Vi, Llc Compositions for translation and methods of use thereof
WO2021183720A1 (en) 2020-03-11 2021-09-16 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
IL296387B2 (en) 2020-03-19 2024-08-01 Avidity Biosciences Inc Preparations and methods for the treatment of facial, back and arm muscle atrophy
JP7621461B2 (ja) 2020-08-20 2025-01-24 エー2 バイオセラピューティクス, インコーポレイテッド Egfr陽性がんを治療するための組成物及び方法
IL300500A (en) 2020-08-20 2023-04-01 A2 Biotherapeutics Inc Preparations and methods for the treatment of mesothelin positive cancer
MX2023002017A (es) 2020-08-20 2023-04-28 A2 Biotherapeutics Inc Composiciones y métodos para tratar cánceres positivos para ceacam.
US20240082272A1 (en) 2020-12-09 2024-03-14 The Regents Of The University Of Michigan Compositions and methods for treating wnt-driven cancer
BR112023012460A2 (pt) 2020-12-23 2023-11-07 Flagship Pioneering Innovations V Inc Conjunto in vitro de capsídeos de anellovirus que envolvem rna
AU2022221606A1 (en) 2021-02-16 2023-08-24 A2 Biotherapeutics, Inc. Compositions and methods for treating her2 positive cancers
WO2022215758A1 (ja) 2021-04-09 2022-10-13 相互薬工株式会社 脂質および組成物
US20240287525A1 (en) 2021-06-17 2024-08-29 Oslo Universitetssykehus Hf Compositions and methods for treating cancer
WO2023283359A2 (en) 2021-07-07 2023-01-12 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
JP2024532019A (ja) 2021-08-25 2024-09-05 ノボ ノルディスク エー/エス アルファ-1アンチトリプシン発現を阻害するための組成物及び方法
KR20240090727A (ko) 2021-10-22 2024-06-21 세일 바이오메디슨스, 인크. Mrna 백신 조성물
WO2023092102A1 (en) 2021-11-19 2023-05-25 Sanegene Bio Usa Inc. Double stranded rna targeting angiopoietin-like 3 (angptl-3) and methods of use thereof
EP4436588A1 (en) 2021-11-23 2024-10-02 Sail Biomedicines, Inc. A bacteria-derived lipid composition and use thereof
JP2025500373A (ja) 2021-12-20 2025-01-09 セイル バイオメディシンズ インコーポレイテッド Mrna治療用組成物
WO2023220561A1 (en) 2022-05-09 2023-11-16 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
TW202409283A (zh) 2022-05-13 2024-03-01 美商旗艦先鋒創新有限責任(Vii)公司 雙股dna組合物及相關方法
EP4565696A1 (en) 2022-08-05 2025-06-11 Sanegene Bio USA Inc. Double stranded rna targeting angiotensinogen (agt) and methods of use thereof
JP2025534701A (ja) 2022-10-14 2025-10-17 セーンジーン バイオ ユーエスエー インコーポレイティド C3を標的とする低分子干渉rnaおよびその使用
CN120359023A (zh) 2022-11-10 2025-07-22 赛欧生物医药股份有限公司 包含脂质纳米颗粒或脂质重构的天然信使包的rna组合物
EP4638743A2 (en) 2022-12-19 2025-10-29 Sanegene Bio USA Inc. Small interfering rna targeting cfb and uses thereof
EP4654952A1 (en) 2023-01-27 2025-12-03 Sail Biomedicines, Inc. A modified lipid composition and uses thereof
WO2024173828A1 (en) 2023-02-17 2024-08-22 Flagship Pioneering Innovations Vii, Llc Dna compositions comprising modified uracil
WO2024173836A2 (en) 2023-02-17 2024-08-22 Flagship Pioneering Innovations Vii, Llc Dna compositions comprising modified cytosine
US20240309383A1 (en) 2023-02-24 2024-09-19 Suzhou Sanegene Bio Inc. Small interfering rna targeting hbv and uses thereof
US20240309380A1 (en) 2023-03-03 2024-09-19 Sanegene Bio Usa Inc. Small interfering rna targeting apoc3 and uses thereof
WO2024220712A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Vaccine compositions
WO2024220625A1 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Delivery of polynucleotides from lipid nanoparticles comprising rna and ionizable lipids
WO2024220752A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Rna therapeutic compositions
WO2024243438A2 (en) 2023-05-23 2024-11-28 Omega Therapeutics, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
US20250027089A1 (en) 2023-06-21 2025-01-23 Sanegene Bio Usa Inc. Double stranded rna targeting proprotein convertase subtilisin kexin 9 (pcsk9) and methods of use thereof
WO2025096807A2 (en) 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Novel therapeutic dna forms
WO2025133951A1 (en) 2023-12-21 2025-06-26 Genevant Sciences Gmbh Ionizable lipids suitable for lipid nanoparticles
WO2025228441A2 (en) 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof

Family Cites Families (74)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5208149A (en) * 1983-10-20 1993-05-04 The Research Foundation Of State University Of New York Nucleic acid constructs containing stable stem and loop structures
ATE208813T1 (de) * 1988-02-16 2001-11-15 Greatbatch Gen Aid Ltd Modifizierte zellen mit resistenz gegen retroviralinfektionen
US20030206887A1 (en) * 1992-05-14 2003-11-06 David Morrissey RNA interference mediated inhibition of hepatitis B virus (HBV) using short interfering nucleic acid (siNA)
CA2156288C (en) 1993-02-19 2005-10-18 Junichi Yano Glycerol derivative, device and pharmaceutical composition
US5858988A (en) 1993-02-24 1999-01-12 Wang; Jui H. Poly-substituted-phenyl-oligoribo nucleotides having enhanced stability and membrane permeability and methods of use
US6291438B1 (en) 1993-02-24 2001-09-18 Jui H. Wang Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use
US5624803A (en) 1993-10-14 1997-04-29 The Regents Of The University Of California In vivo oligonucleotide generator, and methods of testing the binding affinity of triplex forming oligonucleotides derived therefrom
US5902880A (en) * 1994-08-19 1999-05-11 Ribozyme Pharmaceuticals, Inc. RNA polymerase III-based expression of therapeutic RNAs
US6146886A (en) * 1994-08-19 2000-11-14 Ribozyme Pharmaceuticals, Inc. RNA polymerase III-based expression of therapeutic RNAs
US5684143A (en) 1996-02-21 1997-11-04 Lynx Therapeutics, Inc. Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates
US5994316A (en) 1996-02-21 1999-11-30 The Immune Response Corporation Method of preparing polynucleotide-carrier complexes for delivery to cells
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
EP2314700A1 (en) 1999-01-28 2011-04-27 Medical College of Georgia Research Institute, Inc Composition and method for in vivo and in vitro attenuation of gene expression using double stranded RNA
KR20010112944A (ko) 1999-04-21 2001-12-22 이곤 이 버그 폴리뉴클레오티드 서열의 기능을 억제하기 위한 방법 및조성물
BR0012325A (pt) 1999-07-09 2002-05-21 American Home Prod Métodos e composições para prevenção da formação de rna anormal durante a transcrição de uma sequência de plasmìdeo
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
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
WO2002081628A2 (en) * 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
US20050233329A1 (en) 2002-02-20 2005-10-20 Sirna Therapeutics, Inc. Inhibition of gene expression using duplex forming 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)
US20050032733A1 (en) * 2001-05-18 2005-02-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA)
KR20080023768A (ko) * 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Rna 간섭의 rna 서열 특이적인 매개체
KR100909681B1 (ko) 2000-12-01 2009-07-29 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Rna 간섭을 매개하는 작은 rna 분자
CA2369944A1 (en) * 2001-01-31 2002-07-31 Nucleonics Inc. Use of post-transcriptional gene silencing for identifying nucleic acid sequences that modulate the function of a cell
US20020114764A1 (en) 2001-02-20 2002-08-22 Berryman Michelle S. Tattoo method and system for medical and surgical applications
CA2372450A1 (en) * 2001-05-10 2001-09-19 Pharmaceutical Partners Of Canada Inc. Liquid injectable formulation of disodium pamidronate
US20050054598A1 (en) 2002-02-20 2005-03-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition hairless (HR) gene expression using short interfering nucleic acid (siNA)
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
EP1627061B1 (en) 2001-05-18 2009-08-12 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
ES2606290T3 (es) 2001-07-12 2017-03-23 University Of Massachusetts Producción in vivo de ARN de interferencia pequeños que median el silenciamiento génico
US10590418B2 (en) 2001-07-23 2020-03-17 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for RNAi mediated inhibition of gene expression in mammals
WO2003016572A1 (en) 2001-08-17 2003-02-27 Eli Lilly And Company Oligonucleotide therapeutics for treating hepatitis c virus infections
GB0224341D0 (en) 2002-10-19 2002-11-27 Qinetiq Ltd Mobile radio base station
AU2002343792A1 (en) 2001-11-28 2003-06-10 Center For Advanced Science And Technology Incubation, Ltd. siRNA EXPRESSION SYSTEM AND PROCESS FOR PRODUCING FUNCTIONAL GENE-KNOCKDOWN CELLS AND THE LIKE USING THE SAME
DE10202419A1 (de) * 2002-01-22 2003-08-07 Ribopharma Ag Verfahren zur Hemmung der Expression eines durch eine Chromosomen-Aberration entstandenen Zielgens
CA2474910A1 (en) * 2002-02-01 2003-08-07 Sequitur, Inc. Oligonucleotide compositions with enhanced efficiency
US20060009409A1 (en) * 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
WO2003064621A2 (en) 2002-02-01 2003-08-07 Ambion, Inc. HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES
WO2003079757A2 (en) * 2002-03-20 2003-10-02 Massachusetts Institute Of Technology Hiv therapeutic
US20040180438A1 (en) 2002-04-26 2004-09-16 Pachuk Catherine J. Methods and compositions for silencing genes without inducing toxicity
US20040121353A1 (en) * 2002-05-23 2004-06-24 Ceptyr, Inc. Modulation of TCPTP signal transduction by RNA interference
GB0212302D0 (en) 2002-05-28 2002-07-10 Isis Innovation Method of selecting targets for gene silencing by RNA interference
EP1537227B1 (en) * 2002-05-31 2010-02-17 The Regents Of The University Of California Method for efficient rna interference in mammalian cells
US7148342B2 (en) * 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
ES2322145T3 (es) 2002-07-26 2009-06-17 Novartis Vaccines And Diagnostics, Inc. Moleculas pequeñas modificadas de adn inerferente y procedimiento de uso.
WO2004011624A2 (en) * 2002-07-31 2004-02-05 Nucleonics, Inc. Double stranded rna structures and constructs, and methods for generating and using the same
US20040204420A1 (en) * 2002-08-05 2004-10-14 Rana Tariq M. Compounds for modulating RNA interference
US20040023390A1 (en) * 2002-08-05 2004-02-05 Davidson Beverly L. SiRNA-mediated gene silencing with viral vectors
US20050042646A1 (en) * 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US8729036B2 (en) 2002-08-07 2014-05-20 University Of Massachusetts Compositions for RNA interference and methods of use thereof
WO2004029215A2 (en) 2002-09-26 2004-04-08 The Regents Of The University Of California Conditional gene expression using rnai
WO2004035765A2 (en) 2002-10-18 2004-04-29 Nucleonics, Inc. Double-stranded rna structures and constructs, and methods for generating and using the same
EP2213738B1 (en) 2002-11-14 2012-10-10 Dharmacon, Inc. siRNA molecules targeting Bcl-2
US6960568B2 (en) * 2002-12-17 2005-11-01 Yale University Nucleosides and related processes, pharmaceutical compositions and methods
US20040248299A1 (en) * 2002-12-27 2004-12-09 Sumedha Jayasena RNA interference
JP3813934B2 (ja) 2003-02-26 2006-08-23 共同カイテック株式会社 緑化体
US20060263764A1 (en) 2003-02-27 2006-11-23 Nucleonics Inc. Methods and constructs for evaluation of rnai targets and effector molecules
EP1623009A4 (en) * 2003-04-22 2008-02-13 Nucleonics Inc TRANSFECTION KINETICS AND STRUCTURAL PROMOTERS
US20040224405A1 (en) * 2003-05-06 2004-11-11 Dharmacon Inc. siRNA induced systemic gene silencing in mammalian systems
US20050042641A1 (en) 2003-05-27 2005-02-24 Cold Spring Harbor Laboratory In vivo high throughput selection of RNAi probes
US7750144B2 (en) * 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
HUE042261T2 (hu) 2003-06-12 2019-06-28 Alnylam Pharmaceuticals Inc Gén kikapcsolásra alkalmas konzervált HBV és HCV szekvenciák
TW200523265A (en) 2003-07-31 2005-07-16 Basf Ag A process for the preparation of acylphosphines
WO2005019463A1 (en) 2003-08-11 2005-03-03 Therapeutic Human Polyclonals, Inc. Improved transgenesis with humanized immunoglobulin loci
JP2007503205A (ja) 2003-08-22 2007-02-22 ニュークレオニクス・インコーポレイテッド 多コンパートメント真核生物発現系
AU2003265142A1 (en) 2003-08-26 2005-03-10 Scuola Superiore Di Studi Universitari E Di Perfezionamento Sant'anna A wearable mechatronic device for the analysis of joint biomechanics
WO2005068630A1 (ja) * 2003-12-16 2005-07-28 National Institute Of Advanced Industrial Science And Technology 干渉用二重鎖rna
WO2005089287A2 (en) 2004-03-15 2005-09-29 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded rna
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
JP5196900B2 (ja) 2007-07-30 2013-05-15 三洋電機株式会社 ラミネート外装電池
JP2010021493A (ja) 2008-07-14 2010-01-28 Toshiba Corp 半導体装置およびその製造方法

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