BRPI0313202A8 - Formas adicionais para interferir com as moléculas de rna - Google Patents

Formas adicionais para interferir com as moléculas de rna

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Publication number
BRPI0313202A8
BRPI0313202A8 BRPI0313202A BR0313202A BRPI0313202A8 BR PI0313202 A8 BRPI0313202 A8 BR PI0313202A8 BR PI0313202 A BRPI0313202 A BR PI0313202A BR 0313202 A BR0313202 A BR 0313202A BR PI0313202 A8 BRPI0313202 A8 BR PI0313202A8
Authority
BR
Brazil
Prior art keywords
strand
interfere
rna molecules
additional ways
stretch
Prior art date
Application number
BRPI0313202A
Other languages
English (en)
Other versions
BR0313202A (pt
Inventor
Giese Klaus
Kaufmann Jörg
Klippel-Giese Anke
Original Assignee
Atugen Ag
Silence Therapeutics Ag
Silence Therapeutics Gmbh
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=31720998&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=BRPI0313202(A8) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from EP02017601A external-priority patent/EP1389637B1/en
Application filed by Atugen Ag, Silence Therapeutics Ag, Silence Therapeutics Gmbh filed Critical Atugen Ag
Publication of BR0313202A publication Critical patent/BR0313202A/pt
Publication of BRPI0313202A8 publication Critical patent/BRPI0313202A8/pt

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Abstract

FORMAS ADICIONAIS PARA INTERFERIR COM AS MOLÉCULAS DE RNA. A presente invenção refere-se a um ácido ribonucléico compreendendo uma estrutura filamentosa dupla, pelo que, a estrutura filamentosa dupla compreende um primeiro filamento e um segundo filamento, pelo que o primeiro filamento compreende um primeiro estiramento de nucleotídeos contíguos e pelo que o dito primeiro estiramento é pelo menos parcialmente complementar em relação ao ácido nucléico alvo, e o segundo filamento compreende um segundo estiramento de nucleotídeos contíguos, pelo que o dito segundo estiramento é pelo menos parcialmente idêntico em relação ao ácido nucléico alvo e pelo que, a estrutura filamentosa dupla possui terminação cega.
BRPI0313202A 2002-08-05 2003-08-05 Formas adicionais para interferir com as moléculas de rna BRPI0313202A8 (pt)

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EP02017601A EP1389637B1 (en) 2002-08-05 2002-08-05 Blunt-ended interfering RNA molecules
US40254102P 2002-08-12 2002-08-12
EP03008383 2003-04-10
PCT/EP2003/008666 WO2004015107A2 (en) 2002-08-05 2003-08-05 Further novel forms of interfering rna molecules

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Families Citing this family (269)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US20080119427A1 (en) * 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
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)
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
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
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체
US20050054836A1 (en) * 2000-11-09 2005-03-10 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
CN100523215C (zh) 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
US7767802B2 (en) 2001-01-09 2010-08-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
US20050282188A1 (en) * 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
EP1478656B1 (en) 2002-02-01 2009-09-16 Life Technologies Corporation Oligonucleotide compositions with enhanced efficiency
US20030166282A1 (en) 2002-02-01 2003-09-04 David Brown High potency siRNAS for reducing the expression of target genes
US20060009409A1 (en) * 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
US7910724B2 (en) * 2002-02-20 2011-03-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Fos gene expression using short interfering nucleic acid (siNA)
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US8232383B2 (en) * 2002-02-20 2012-07-31 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US7928218B2 (en) * 2002-02-20 2011-04-19 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of polycomb group protein EZH2 gene expression using short interfering nucleic acid (siNA)
US20100240730A1 (en) * 2002-02-20 2010-09-23 Merck Sharp And Dohme Corp. RNA Interference Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid (siNA)
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc RNA inhibition mediated inhibition of MAP KINASE GENES
US20090099117A1 (en) * 2002-02-20 2009-04-16 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF MYOSTATIN GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US20090093439A1 (en) * 2002-02-20 2009-04-09 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
EP1857547B2 (en) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Further novel forms of interfering RNA molecules
AU2003261449A1 (en) 2002-08-07 2004-02-25 Compositions for rna interference and methods of use thereof
US7923547B2 (en) * 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
EP1546344A4 (en) * 2002-09-18 2007-10-03 Isis Pharmaceuticals Inc EFFICIENT PRODUCTION OF TARGET RNAS USING SINGLE AND DOUBLE-STRONG OLIGOMER COMPOUNDS
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
US8604183B2 (en) * 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
US9827263B2 (en) * 2002-11-05 2017-11-28 Ionis Pharmaceuticals, Inc. 2′-methoxy substituted oligomeric compounds and compositions for use in gene modulations
US9150606B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
AU2004221760B2 (en) 2003-03-21 2010-03-18 Roche Innovation Center Copenhagen A/S Short interfering RNA (siRNA) analogues
ATE536408T1 (de) * 2003-04-02 2011-12-15 Dharmacon Inc Modifizierte polynukleotide zur verwendung bei rna-interferenz
US20070167384A1 (en) * 2003-04-02 2007-07-19 Dharmacon, Inc. Modified polynucleotides for use in rna interference
CA2527958C (en) 2003-06-02 2014-04-08 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of rnai
CA2528012C (en) * 2003-06-02 2015-11-24 University Of Massachusetts Methods and compositions for controlling efficacy of rna silencing
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
US8575327B2 (en) 2003-06-12 2013-11-05 Alnylam Pharmaceuticals, Inc. Conserved HBV and HCV sequences useful for gene silencing
US7790691B2 (en) * 2003-06-20 2010-09-07 Isis Pharmaceuticals, Inc. Double stranded compositions comprising a 3′-endo modified strand for use in gene modulation
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
US20050136437A1 (en) * 2003-08-25 2005-06-23 Nastech Pharmaceutical Company Inc. Nanoparticles for delivery of nucleic acids and stable double-stranded RNA
CN1845993B (zh) 2003-08-28 2010-06-23 诺瓦提斯公司 具有平端和3＇修饰的干扰rna双链体
US20050074801A1 (en) * 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
WO2005027980A1 (en) 2003-09-12 2005-03-31 University Of Massachusetts Rna interference for the treatment of gain-of-function disorders
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
WO2005073378A1 (en) * 2004-01-30 2005-08-11 Santaris Pharma A/S MODIFIED SHORT INTERFERING RNA (MODIFIED siRNA)
WO2005072057A2 (en) * 2004-01-30 2005-08-11 Quark Biotech, Inc. Oligoribonucleotides and methods of use thereof for treatment of fibrotic conditions and other diseases
US8569474B2 (en) 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
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
US20160230185A1 (en) 2013-03-14 2016-08-11 Monsanto Technology Llc Compositions and methods for controlling diabrotica
EP2471924A1 (en) 2004-05-28 2012-07-04 Asuragen, INC. Methods and compositions involving microRNA
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
US20090048192A1 (en) * 2004-06-03 2009-02-19 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
EP1766052A4 (en) * 2004-06-03 2009-12-16 Isis Pharmaceuticals Inc OLIGOMERIC CHIMERIC COMPOSITIONS WITH BRECH
NZ553162A (en) * 2004-08-16 2010-03-26 Quark Biotech Inc Double stranded siRNA compounds that inhibit RRTP801
DK1781787T3 (en) 2004-08-23 2017-07-31 Sylentis Sau TREATMENT OF EYE DISORDERS FEATURED BY AN INCREASED INTRAOCULAR PRESSURE WITH SIRNAS
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
JP4991547B2 (ja) 2004-09-28 2012-08-01 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 脱毛症、急性腎不全および他の疾患の治療のためのオリゴリボヌクレオチドおよびその使用の方法
ES2534300T3 (es) 2004-11-12 2015-04-21 Asuragen, Inc. Procedimientos y composiciones que implican miARN y moléculas inhibidoras de miARN
US7923207B2 (en) 2004-11-22 2011-04-12 Dharmacon, Inc. Apparatus and system having dry gene silencing pools
US7935811B2 (en) 2004-11-22 2011-05-03 Dharmacon, Inc. Apparatus and system having dry gene silencing compositions
US20060166234A1 (en) 2004-11-22 2006-07-27 Barbara Robertson Apparatus and system having dry control gene silencing compositions
WO2006060454A2 (en) * 2004-12-02 2006-06-08 B-Bridge International, Inc. Methods of designing small interfering rnas, antisense polynucleotides, and other hybridizing polynucleotides
US9550990B2 (en) * 2004-12-10 2017-01-24 Ionis Pharmaceuticals, Inc. Regulation of epigenetic control of gene expression
US20100266574A1 (en) * 2005-06-10 2010-10-21 Orna Mor Oligoribonucleotides and Methods of Use Thereof for Treatment of Fibrotic Conditions and Other Diseases
EP2239328A3 (en) 2005-08-18 2011-01-05 Alnylam Pharmaceuticals Inc. Methods and compositions for treating neurological disease
GB0520063D0 (en) * 2005-10-03 2005-11-09 Novartis Forschungsstiftung Methods for regulating metabolism and diagnosing disease
GB0521351D0 (en) 2005-10-20 2005-11-30 Genomica Sau Modulation of TRPV expression levels
GB0521716D0 (en) 2005-10-25 2005-11-30 Genomica Sau Modulation of 11beta-hydroxysteriod dehydrogenase 1 expression for the treatment of ocular diseases
EP1942943A2 (en) * 2005-11-04 2008-07-16 Nastech Pharmaceutical Company Inc. Peptide-dicer substrate rna conjugates as delivery vehicles for sirna
JP5232990B2 (ja) * 2005-11-08 2013-07-10 国立大学法人愛媛大学 Ａｋｔ遺伝子に特異的なｓｉＲＮＡ
KR100877824B1 (ko) 2005-11-11 2009-01-12 한국생명공학연구원 Ｅ2ｅｐｆ ｕｃｐ-ｖｈｌ 상호작용 및 그 용도
US7825099B2 (en) 2006-01-20 2010-11-02 Quark Pharmaceuticals, Inc. Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes
UY30097A1 (es) 2006-01-20 2007-08-31 Atugen Ag Usos terapeuticos de inhibidores de rtp801
WO2007085485A2 (en) * 2006-01-27 2007-08-02 Santaris Pharma A/S Lna modified phosphorothiolated oligonucleotides
EP2388328A1 (en) * 2006-01-27 2011-11-23 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
US8362229B2 (en) * 2006-02-08 2013-01-29 Quark Pharmaceuticals, Inc. Tandem siRNAS
US7910566B2 (en) 2006-03-09 2011-03-22 Quark Pharmaceuticals Inc. Prevention and treatment of acute renal failure and other kidney diseases by inhibition of p53 by siRNA
ES2556128T3 (es) * 2006-03-23 2016-01-13 Roche Innovation Center Copenhagen A/S ARN interfiriente pequeño internamente segmentado
FR2898908A1 (fr) 2006-03-24 2007-09-28 Agronomique Inst Nat Rech Procede de preparation de cellules aviaires differenciees et genes impliques dans le maintien de la pluripotence
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
WO2007141796A2 (en) 2006-06-09 2007-12-13 Quark Pharmaceuticals, Inc. Therapeutic uses of inhibitors of rtp801l
BRPI0714875A2 (pt) 2006-07-21 2013-05-28 Silence Therapeutics Ag meios para inibir a expressço da proteÍna cinase 3
EP1897941A1 (en) * 2006-09-11 2008-03-12 Max-Delbrück-Centrum Für Molekulare Medizin Means for increasing intracellular levels of protoporphyrin IX and use thereof
WO2008036741A2 (en) * 2006-09-19 2008-03-27 Asuragen, Inc. Mir-200 regulated genes and pathways as targets for therapeutic intervention
EP2081949B1 (en) 2006-09-22 2014-12-10 GE Healthcare Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by rna interference
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
WO2008052774A2 (en) 2006-10-31 2008-05-08 Noxxon Pharma Ag Methods for detection of a single- or double-stranded nucleic acid molecule
WO2008106102A2 (en) * 2007-02-26 2008-09-04 Quark Pharmaceuticals, Inc. Inhibitors of rtp801 and their use in disease treatment
WO2008104978A2 (en) * 2007-02-28 2008-09-04 Quark Pharmaceuticals, Inc. 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
WO2008143774A2 (en) * 2007-05-01 2008-11-27 University Of Massachusetts Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy
AU2008250033A1 (en) * 2007-05-11 2008-11-20 Enzon Pharmaceuticals, Inc. RNA antagonist compounds for the modulation of HER3
US20100273854A1 (en) * 2007-06-15 2010-10-28 Hagar Kalinski Compositions and methods for inhibiting nadph oxidase expression
SI2170403T1 (sl) 2007-06-27 2014-07-31 Quark Pharmaceuticals, Inc. Sestavki in postopki za inhibicijo ekspresije pro-apoptotskih genov
US8361714B2 (en) 2007-09-14 2013-01-29 Asuragen, Inc. Micrornas differentially expressed in cervical cancer and uses thereof
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
CA2701845A1 (en) 2007-10-03 2009-04-09 Quark Pharmaceuticals, Inc. Novel sirna structures
US20100317714A1 (en) * 2007-11-29 2010-12-16 Suzhou Ribo Life Science Co., Ltd Complex molecule interfering the expression of target genes and its preparing methods
WO2009070805A2 (en) 2007-12-01 2009-06-04 Asuragen, Inc. Mir-124 regulated genes and pathways as targets for therapeutic intervention
US20110105584A1 (en) * 2007-12-12 2011-05-05 Elena Feinstein Rtp80il sirna compounds and methods of use thereof
US8614311B2 (en) 2007-12-12 2013-12-24 Quark Pharmaceuticals, Inc. RTP801L siRNA compounds and methods of use thereof
KR100949791B1 (ko) * 2007-12-18 2010-03-30 이동기 오프-타겟 효과를 최소화하고 ＲＮＡｉ 기구를 포화시키지않는 신규한 ｓｉＲＮＡ 구조 및 그 용도
WO2009090639A2 (en) * 2008-01-15 2009-07-23 Quark Pharmaceuticals, Inc. Sirna compounds and methods of use thereof
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
EP3643782A1 (en) 2008-02-11 2020-04-29 Phio Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
EP2256191A4 (en) * 2008-02-15 2011-07-06 Riken CYCLIC SINGLE NUCLEIC ACID COMPLEX AND METHOD FOR THE PRODUCTION THEREOF
RU2010138558A (ru) * 2008-03-20 2012-03-27 Кварк Фармасьютикалс. Инк, (Us) Новые соединения миphk для ингибирования rtp801
WO2009144704A2 (en) * 2008-04-15 2009-12-03 Quark Pharmaceuticals, Inc. siRNA COMPOUNDS FOR INHIBITING NRF2
US8258111B2 (en) 2008-05-08 2012-09-04 The Johns Hopkins University Compositions and methods related to miRNA modulation of neovascularization or angiogenesis
CA2635187A1 (en) * 2008-06-05 2009-12-05 The Royal Institution For The Advancement Of Learning/Mcgill University Oligonucleotide duplexes and uses thereof
US8431692B2 (en) * 2008-06-06 2013-04-30 Quark Pharmaceuticals, Inc. Compositions and methods for treatment of ear disorders
TWI455944B (zh) 2008-07-01 2014-10-11 Daiichi Sankyo Co Ltd 雙股多核苷酸
WO2010008562A2 (en) * 2008-07-16 2010-01-21 Recombinetics Methods and materials for producing transgenic animals
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
US9433684B2 (en) 2008-08-19 2016-09-06 Nektar Therapeutics Conjugates of small-interfering nucleic acids
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
WO2010033225A2 (en) * 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications
EP2937418B1 (en) 2008-10-20 2017-10-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
ES2563984T3 (es) * 2008-10-22 2016-03-17 Quark Pharmaceuticals, Inc. Métodos para el tratamiento de trastornos oculares
EP2358397B1 (en) 2008-10-24 2020-01-01 Ionis Pharmaceuticals, Inc. 5' and 2' bis-substituted nucleosides and oligomeric compounds prepared therefrom
EP2447274B1 (en) 2008-10-24 2017-10-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds and methods
US9074211B2 (en) 2008-11-19 2015-07-07 Rxi Pharmaceuticals Corporation Inhibition of MAP4K4 through RNAI
US20110288155A1 (en) 2008-12-18 2011-11-24 Elena Feinstein Sirna compounds and methods of use thereof
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
WO2010091878A2 (en) 2009-02-13 2010-08-19 Silence Therapeutics Ag Means for inhibiting the expression of opa1
WO2010094491A1 (en) 2009-02-18 2010-08-26 Silence Therapeutics Ag Means for inhibiting the expression of ang2
JP2012520683A (ja) * 2009-03-19 2012-09-10 メルク・シャープ・エンド・ドーム・コーポレイション 低分子干渉核酸（ｓｉＮＡ）を用いた結合組織増殖因子（ＣＴＧＦ）遺伝子発現のＲＮＡ干渉媒介性阻害
US8444983B2 (en) 2009-03-23 2013-05-21 Quark Pharmaceuticals, Inc. Composition of anti-ENDO180 antibodies and methods of use for the treatment of cancer and fibrotic diseases
EP2440214A4 (en) 2009-06-08 2013-07-31 Quark Pharmaceuticals Inc METHOD FOR THE TREATMENT OF CHRONIC KIDNEY DISEASES
GB0910723D0 (en) 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
US8796238B2 (en) 2009-09-17 2014-08-05 Sigma-Aldrich Co. Llc Short RNA mimetics
WO2011035065A1 (en) 2009-09-17 2011-03-24 Nektar Therapeutics Monoconjugated chitosans as delivery agents for small interfering nucleic acids
CN102666856B (zh) 2009-11-08 2016-04-06 夸克制药公司 定向至RhoA靶基因的双链RNA化合物在制造治疗神经性疼痛的药物中的用途
TW201124160A (en) 2009-11-26 2011-07-16 Quark Pharmaceuticals Inc SiRNA compounds comprising terminal substitutions
CA2781896C (en) 2009-12-09 2021-03-30 Nitto Denko Corporation Modulation of hsp47 expression
WO2011072091A1 (en) 2009-12-09 2011-06-16 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the cns
WO2011084193A1 (en) 2010-01-07 2011-07-14 Quark Pharmaceuticals, Inc. Oligonucleotide compounds comprising non-nucleotide overhangs
US9095504B2 (en) 2010-03-24 2015-08-04 Rxi Pharmaceuticals Corporation RNA interference in ocular indications
CA2794189C (en) 2010-03-24 2022-01-11 Rxi Pharmaceuticals Corporation Rna interference in dermal and fibrotic indications
WO2011119852A1 (en) 2010-03-24 2011-09-29 Rxi Pharmaceuticals Corporation Reduced size self-delivering rnai compounds
WO2011139699A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
WO2011139702A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
US20110306653A1 (en) 2010-05-14 2011-12-15 Tagcyx Biotechnologies Stabilization method of functional nucleic acid
EP2390327A1 (en) 2010-05-27 2011-11-30 Sylentis S.A. siRNA and their use in methods and compositions for the treatment and/or prevention of eye conditions
AU2011270896B9 (en) 2010-06-24 2015-05-07 Quark Pharmaceuticals, Inc. Double stranded RNA compounds to RhoA and use thereof
SG186899A1 (en) * 2010-07-08 2013-02-28 Bonac Corp Single-strand nucleic acid molecule for controlling gene expression
EP2412724A1 (en) 2010-07-29 2012-02-01 Centre National de la Recherche Scientifique (C.N.R.S) Regulation of Glypican 4 activity to modulate the fate of stem cells and uses thereof
ES2533129T3 (es) 2010-08-03 2015-04-07 Bonac Corporation Molécula de ARN monocatenario que tiene esqueleto alicíclico que contiene nitrógeno
JP6106085B2 (ja) 2010-08-24 2017-03-29 サーナ・セラピューティクス・インコーポレイテッドＳｉｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 内部非核酸スペーサーを含む一本鎖ＲＮＡｉ剤
US9290760B2 (en) 2010-09-15 2016-03-22 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
CN103328633B (zh) 2010-10-22 2018-07-10 成均馆大学校产学协力团 诱导rna干扰的核酸分子及其用途
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
CA2818024C (en) 2010-12-06 2019-09-24 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising positional modifications
AU2012223366B2 (en) 2011-03-03 2017-02-23 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
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
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
PT2726613T (pt) 2011-06-30 2018-10-26 Arrowhead Pharmaceuticals Inc Composições e métodos para inibição da expressão de genes do vírus da hepatite b
US9644241B2 (en) 2011-09-13 2017-05-09 Interpace Diagnostics, Llc Methods and compositions involving miR-135B for distinguishing pancreatic cancer from benign pancreatic disease
AU2012325997C1 (en) 2011-10-18 2018-07-05 Dicerna Pharmaceuticals, Inc. Amine cationic lipids and uses thereof
US20140323549A1 (en) 2011-11-08 2014-10-30 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the nervous system
UA118649C2 (uk) 2011-11-18 2019-02-25 Елнілем Фармасьютікалз, Інк. ДВОЛАНЦЮГОВИЙ ЗАСІБ ДЛЯ PHKі ТА ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ТРАНСТИРЕТИНУ (TTR)
US9796974B2 (en) 2011-11-18 2017-10-24 Alnylam Pharmaceuticals, Inc. Modified RNAi agents
WO2013098813A1 (en) 2012-01-01 2013-07-04 Qbi Enterprises Ltd. Endo180-targeted particles for selective delivery of therapeutic and diagnostic agents
EP2802657B1 (en) 2012-01-12 2018-05-02 Quark Pharmaceuticals, Inc. Combination therapy for treating hearing and balance disorders
WO2013123996A1 (en) 2012-02-24 2013-08-29 Astrazeneca Uk Limited Novel sirna inhibitors of human icam-1
US9980942B2 (en) 2012-05-02 2018-05-29 Children's Hospital Medical Center Rejuvenation of precursor cells
CN108148838B (zh) * 2012-05-22 2026-07-31 奥利克斯医药有限公司 具有细胞内穿透能力的诱导rna干扰的核酸分子及用途
US9663784B2 (en) 2012-05-26 2017-05-30 Bonac Corporation Single-stranded nucleic acid molecule for regulating expression of gene having delivering function
WO2014018375A1 (en) 2012-07-23 2014-01-30 Xenon Pharmaceuticals Inc. Cyp8b1 and uses thereof in therapeutic and diagnostic methods
AU2012389270B2 (en) 2012-09-05 2018-11-08 Sylentis S.A.U. siRNA and their use in methods and compositions for the treatment and/or prevention of eye conditions
GB201215857D0 (en) 2012-09-05 2012-10-24 Sylentis Sau siRNA and their use in methods and compositions for the treatment and/or prevention of eye conditions
WO2014043289A2 (en) 2012-09-12 2014-03-20 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide molecules to ddit4 and methods of use thereof
EP2895608B1 (en) 2012-09-12 2018-12-05 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide molecules to p53 and methods of use thereof
CA3120574A1 (en) 2013-03-14 2014-09-25 Dicerna Pharmaceuticals, Inc. Process for formulating an anionic agent
US9822418B2 (en) 2013-04-22 2017-11-21 Icahn School Of Medicine At Mount Sinai Mutations in PDGFRB and NOTCH3 as causes of autosomal dominant infantile myofibromatosis
TW201534578A (zh) 2013-07-08 2015-09-16 Daiichi Sankyo Co Ltd 新穎脂質
EP3027223A1 (en) 2013-07-31 2016-06-08 QBI Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
EP3027222A1 (en) 2013-07-31 2016-06-08 QBI Enterprises Ltd. Sphingolipid-polyalkylamine-oligonucleotide compounds
EP2853595A1 (en) 2013-09-30 2015-04-01 Soluventis GmbH NOTCH 1 specific siRNA molecules
WO2015051366A2 (en) * 2013-10-04 2015-04-09 Novartis Ag Novel formats for organic compounds for use in rna interference
EP2865756A1 (en) * 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA and their use in methods and compositions for inhibiting the expression of the FLAP gene.
EP2865757A1 (en) 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA and their use in methods and compositions for inhibiting the expression of the PDK1 gene.
EP2865758A1 (en) * 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA and their use in methods and compositions for inhibiting the expression of the ORAI1 gene
JP6772062B2 (ja) 2013-12-02 2020-10-21 フィオ ファーマシューティカルズ コーポレーションＰｈｉｏ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｃｏｒｐ． 癌の免疫療法
WO2015099122A1 (ja) 2013-12-26 2015-07-02 学校法人東京医科大学 遺伝子発現制御のための人工ミミックｍｉＲＮＡおよびその用途
SG10201805087VA (en) 2013-12-27 2018-07-30 Bonac Corp Artificial match-type mirna for controlling gene expression and use therefor
US20160333346A1 (en) * 2014-01-17 2016-11-17 Kyowa Hakko Kirin Co., Ltd. Nucleic acid capable of inhibiting expression of beta-2gpi
EP3105331B1 (en) 2014-02-11 2021-06-23 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
WO2015132303A1 (en) 2014-03-04 2015-09-11 Sylentis Sau Sirnas and their use in methods and compositions for the treatment and/or prevention of eye conditions
CA2947270A1 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic acids targeting mdm2 or mycn
KR101995142B1 (ko) * 2014-08-07 2019-07-02 인더스트리얼 테크놀로지 리서치 인스티튜트 작은 간섭 rna, 이를 함유하는, 갈렉틴-12 발현을 억제하고/하거나 지방분해를 향상시키는 약학 조성물 및 이의 용도
EP3188799B1 (en) 2014-09-05 2022-07-06 Phio Pharmaceuticals Corp. Methods for treating aging and skin disorders using nucleic acids targeting tyr or mmp1
FR3026409B1 (fr) * 2014-09-26 2018-03-30 Centre National De La Recherche Scientifique (Cnrs) Procede de criblage de molecules interferentes
TWI769565B (zh) 2014-11-10 2022-07-01 美商阿尼拉製藥公司 Ｂ型肝炎病毒（ＨＢＶ）ｉＲＮＡ組成物及其用途方法
WO2016083624A1 (en) 2014-11-28 2016-06-02 Silence Therapeutics Gmbh Means for inhibiting the expression of edn1
CN107532162A (zh) 2014-12-12 2018-01-02 托德·M·伍尔夫 用于利用寡核苷酸编辑细胞中核酸的组合物和方法
WO2016104775A1 (ja) 2014-12-27 2016-06-30 株式会社ボナック 遺伝子発現制御のための天然型ｍｉＲＮＡおよびその用途
US20160272970A1 (en) * 2015-03-17 2016-09-22 Arrowhead Madison Inc. RNA Interference Agents
JP6602847B2 (ja) 2015-03-27 2019-11-06 株式会社ボナック デリバリー機能と遺伝子発現制御能を有する一本鎖核酸分子
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
US11001845B2 (en) 2015-07-06 2021-05-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (SOD1)
LT3329002T (lt) 2015-07-31 2021-01-11 Alnylam Pharmaceuticals, Inc. Transtiretino (ttr) irnr kompozicijos ir jų naudojimo būdai, skirti naudoti su ttr susijusių ligų gydymui arba prevencijai
CN115957337A (zh) 2015-08-07 2023-04-14 箭头药业股份有限公司 乙型肝炎病毒感染的RNAi疗法
WO2017042238A1 (en) 2015-09-08 2017-03-16 Sylentis Sau Sirna and their use in methods and compositions for inhibiting the expression of the nrarp gene
US11021707B2 (en) 2015-10-19 2021-06-01 Phio Pharmaceuticals Corp. Reduced size self-delivering nucleic acid compounds targeting long non-coding RNA
EP3371211B1 (en) 2015-11-04 2025-01-01 Icahn School of Medicine at Mount Sinai Rho-associated protein kinase ("rock") inhibitor for treating tumors and cancer, and identifying candidate subjects for such treatment
JP7027311B2 (ja) 2015-11-16 2022-03-01 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 ＭｙＤ８８又はＴＬＲ３を標的とするＲＮＡ複合体を使用した加齢黄斑変性の治療
EP3409779A4 (en) 2016-01-26 2019-07-03 Nissan Chemical Corporation Single-stranded oligonucleotide
KR102825945B1 (ko) 2016-02-02 2025-06-30 올릭스 주식회사 Angpt2 및 pdgfb를 표적화하는 rna 복합체를 사용하는 혈관신생 관련 질환의 치료
WO2017134525A1 (en) 2016-02-02 2017-08-10 Olix Pharmaceuticals, Inc. TREATMENT OF ATOPIC DERMATITIS AND ASTHMA USING RNA COMPLEXES THAT TARGET lL4Rα, TRPA1, OR F2RL1
WO2023150553A1 (en) 2022-02-01 2023-08-10 University Of Rochester Gpr17 promoter-based targeting and transduction of glial progenitor cells
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
JP7049262B2 (ja) 2016-04-11 2022-04-06 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 結合組織成長因子を標的とするｒｎａ複合体を用いた特発性肺胞線維症の治療
US11365415B2 (en) 2016-04-14 2022-06-21 University Of Florida Research Foundation, Inc. Use of miR-223-3p as a cancer therapeutic and method for treating cancer using the same
EP3448881B1 (en) 2016-04-26 2023-06-07 Icahn School of Medicine at Mount Sinai Treatment of hippo pathway mutant tumors and methods of identifying subjects as candidates for treatment
CA3022319A1 (en) 2016-05-06 2017-11-09 Tod M. Woolf Improved methods for genome editing with and without programmable nucleases
KR101916652B1 (ko) 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
JOP20190015A1 (ar) 2016-08-04 2019-02-04 Arrowhead Pharmaceuticals Inc عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب
EP3315125A1 (en) 2016-10-31 2018-05-02 Silence Therapeutics (London) Ltd Lipid nanoparticle formulation
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
KR20240172236A (ko) 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
JP7188087B2 (ja) * 2017-02-06 2022-12-13 日産化学株式会社 一本鎖オリゴヌクレオチド
WO2018146557A2 (en) 2017-02-10 2018-08-16 Dong Ki Lee Long double-stranded rna for rna interference
US11324820B2 (en) 2017-04-18 2022-05-10 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (HBV) infection
JP7167059B2 (ja) 2017-04-19 2022-11-08 フィオ ファーマシューティカルズ コーポレーション 核酸化合物の局所送達
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
WO2019048645A1 (en) * 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
WO2019048632A1 (en) * 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATORY RNA (PARNA) OF HNF4A AND METHODS OF USE
EP3684931A1 (en) 2017-09-19 2020-07-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
SG11202003082UA (en) * 2017-11-13 2020-05-28 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of a target gene comprising phosphorodithioate linkages
CN118638787A (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
KR102995657B1 (ko) 2018-03-29 2026-07-28 테크니온 리서치 엔드 디벨로프먼트 화운데이션 엘티디. Pten 억제제를 포함하는 소포 및 이의 용도
CN112566640A (zh) 2018-06-18 2021-03-26 罗切斯特大学 治疗精神分裂症和其他神经精神病症的方法
US12303549B2 (en) 2018-06-21 2025-05-20 University Of Rochester Methods of treating or inhibiting onset of Huntington's disease
WO2020036862A1 (en) 2018-08-13 2020-02-20 Alnylam Pharmaceuticals, Inc. HEPATITIS B VIRUS (HBV) dsRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
AU2019396450B2 (en) 2018-12-11 2025-08-28 University Of Rochester Methods of treating schizophrenia and other neuropsychiatric disorders
IL312067B2 (en) 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
EP3923962A2 (en) 2019-02-13 2021-12-22 University of Rochester Gene networks that mediate remyelination of the human brain
WO2020219820A1 (en) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
WO2021011936A2 (en) 2019-07-18 2021-01-21 University Of Rochester Cell-type selective immunoprotection of cells
CN114450027A (zh) 2019-08-16 2022-05-06 儿童医院医疗中心 用cdc42特异性抑制剂治疗受试者的方法
IL291251B2 (en) 2019-09-10 2026-06-01 Daiichi Sankyo Co Limited CONJUGATE OF GalNAC-OLIGONUCLEOTIDE FOR DELIVERY TO LIVER AND MANUFACTURING METHOD THEREOF
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法
KR102927945B1 (ko) 2020-03-27 2026-02-23 어비디티 바이오사이언시스 인크. 근이영양증의 치료용 조성물 및 방법
WO2021255262A1 (en) 2020-06-19 2021-12-23 Sylentis Sau siRNA AND COMPOSITIONS FOR PROPHYLACTIC AND THERAPEUTIC TREATMENT OF VIRUS DISEASES
US11459567B2 (en) 2020-06-24 2022-10-04 Patricia Virginia Elizalde Specific siRNA molecules, composition and use thereof for the treatment of triple negative breast cancer
EP4015634A1 (en) 2020-12-15 2022-06-22 Sylentis, S.A.U. Sirna and compositions for prophylactic and therapeutic treatment of virus diseases
WO2022162157A1 (en) 2021-01-30 2022-08-04 E-Therapeutics Plc Conjugated oligonucleotide compounds, methods of making and uses thereof
EP4176061A1 (en) 2021-01-30 2023-05-10 E-Therapeutics plc Nucleic acids containing abasic nucleotides
CN117355534B (zh) 2021-01-30 2026-05-01 唐格拉姆治疗公司 缀合寡核苷酸化合物、其制备方法和用途
WO2022162161A1 (en) 2021-01-30 2022-08-04 E-Therapeutics Plc Conjugated oligonucleotide compounds, methods of making and uses thereof
EP4175964A1 (en) 2021-01-30 2023-05-10 E-Therapeutics plc Conjugated oligonucleotide compounds, methods of making and uses thereof
AR126675A1 (es) 2021-08-03 2023-11-01 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA LA TRANSTIRRETINA (TTR) Y SUS MÉTODOS DE USO
EP4763287A2 (en) 2021-09-03 2026-06-24 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
EP4401792A4 (en) 2021-09-16 2025-12-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
EP4499834A2 (en) 2022-03-28 2025-02-05 Empirico Inc. Modified oligonucleotides
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
EP4560020A1 (en) 2023-11-22 2025-05-28 Sylentis S.A.U. Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions
EP4560019A1 (en) 2023-11-22 2025-05-28 Sylentis S.A.U. Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions
WO2025170027A1 (ja) 2024-02-08 2025-08-14 テルモ株式会社 オリゴヌクレオチド

Family Cites Families (85)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB992744A (en) 1962-01-18 1965-05-19 Dennis Harold Ward Plastic containers and lifting means therefore
US4217294A (en) 1979-03-30 1980-08-12 Union Carbide Corporation Method for producing mercaptan-containing organosilicon compounds
WO1991006556A1 (en) 1989-10-24 1991-05-16 Gilead Sciences, Inc. 2' modified oligonucleotides
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5955589A (en) * 1991-12-24 1999-09-21 Isis Pharmaceuticals Inc. Gapped 2' modified oligonucleotides
US5914396A (en) 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US6153737A (en) 1990-01-11 2000-11-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5872232A (en) 1990-01-11 1999-02-16 Isis Pharmaceuticals Inc. 2'-O-modified oligonucleotides
US6399754B1 (en) 1991-12-24 2002-06-04 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides
US5811533A (en) 1990-06-11 1998-09-22 Nexstar Pharmaceuticals, Inc. High-affinity oligonucleotide ligands to vascular endothelial growth factor (VEGF)
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
AU649074B2 (en) † 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
FR2675803B1 (fr) 1991-04-25 1996-09-06 Genset Sa Oligonucleotides fermes, antisens et sens et leurs applications.
FR2678512B1 (fr) 1991-07-03 1995-06-30 Novacell Machine a internaliser.
ATE515510T1 (de) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
WO1994001550A1 (en) 1992-07-02 1994-01-20 Hybridon, Inc. Self-stabilized oligonucleotides as therapeutic agents
US5514546A (en) * 1993-09-01 1996-05-07 Research Corporation Technologies, Inc. Stem-loop oligonucleotides containing parallel and antiparallel binding domains
DE4338704A1 (de) 1993-11-12 1995-05-18 Hoechst Ag Stabilisierte Oligonucleotide und deren Verwendung
EP0743859A4 (en) * 1993-11-16 1998-10-21 Genta Inc CHEMICAL OLIGONUCLEOSIDE COMPOUNDS
US6060456A (en) * 1993-11-16 2000-05-09 Genta Incorporated Chimeric oligonucleoside compounds
CA2207593A1 (en) 1994-12-13 1996-06-20 John Gustofson Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
GB9425118D0 (en) 1994-12-13 1995-02-08 Henkel Kgaa Two component natural hair colorant
US5760012A (en) * 1996-05-01 1998-06-02 Thomas Jefferson University Methods and compounds for curing diseases caused by mutations
DE19618797C2 (de) 1996-05-10 2000-03-23 Bertling Wolf Vehikel zum Transport molekularer Substanz
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
DE19631919C2 (de) 1996-08-07 1998-07-16 Deutsches Krebsforsch Anti-Sinn-RNA mit Sekundärstruktur
US6284267B1 (en) 1996-08-14 2001-09-04 Nutrimed Biotech Amphiphilic materials and liposome formulations thereof
GB9710475D0 (en) 1997-05-21 1997-07-16 Zeneca Ltd Gene silencing
US6133246A (en) * 1997-08-13 2000-10-17 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the modulation of JNK proteins
GB9720148D0 (en) 1997-09-22 1997-11-26 Innes John Centre Innov Ltd Gene silencing materials and methods
US6129912A (en) 1997-09-26 2000-10-10 Uab Research Foundation Polyethylene glycol-protein compositions which reduce the antigenic display of red blood cells
US6617438B1 (en) * 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
CN101818145A (zh) 1998-03-20 2010-09-01 联邦科学和工业研究组织 控制基因表达
DK1068311T3 (da) 1998-04-08 2011-08-08 Commw Scient Ind Res Org Fremgangsmåder og midler til opnåelse af modificerede fænotyper
CA2326823A1 (en) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
JP2002512794A (ja) 1998-04-29 2002-05-08 リボザイム・ファーマシューティカルズ・インコーポレーテッド ヌクレオチド三リン酸およびリボザイム内へのそれらの取り込み
AR020078A1 (es) 1998-05-26 2002-04-10 Syngenta Participations Ag Metodo para alterar la expresion de un gen objetivo en una celula de planta
GB9827152D0 (en) 1998-07-03 1999-02-03 Devgen Nv Characterisation of gene function using double stranded rna inhibition
AU776150B2 (en) 1999-01-28 2004-08-26 Medical College Of Georgia Research Institute, Inc. Composition and method for (in vivo) and (in vitro) attenuation of gene expression using double stranded RNA
US6200924B1 (en) 1999-01-29 2001-03-13 E. I. Du Pont De Nemours And Company Porous highly fluorinated acidic polymer catalyst
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
IL145778A0 (en) 1999-04-21 2002-07-25 American Home Prod Methods and compositions for inhibiting the function of polynucleotide sequences
DE60026313D1 (de) 1999-07-23 2006-04-27 Uutech Ltd Sensibilisierung von roten blutkörperchen gegenüber ultraschall durch einwirkung eines elektrischen feldes
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
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체
CA2456008A1 (en) 2000-08-19 2002-02-28 Axordia Limited Stem cell differentiation
US20030190635A1 (en) 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
CN100523215C (zh) 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
WO2002097114A2 (en) 2001-05-29 2002-12-05 Sirna Therapeutics, Inc. Nucleic acid treatment of diseases or conditions related to levels of ras, her2 and hiv
WO2003070912A2 (en) * 2001-06-06 2003-08-28 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF EPIDERMAL GROWTH FACTOR RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US20060142226A1 (en) 2001-05-18 2006-06-29 Sirna Therapeutics, Inc. RNA interference mediated inhibition of cholesteryl ester transfer protein (CETP) gene expression using short interfering nucleic acid (siNA)
KR100626258B1 (ko) 2001-06-22 2006-09-21 보레알리스 테크놀로지 오와이 4-, 5-, 6- 또는 7-위치에서 실록시 또는 게르밀옥시 기로치환된 인데닐 잔기를 함유하는 메탈로센 촉매
DE10163098B4 (de) 2001-10-12 2005-06-02 Alnylam Europe Ag Verfahren zur Hemmung der Replikation von Viren
US20030135033A1 (en) 2002-01-04 2003-07-17 Anke Klippel-Giese Compounds and methods for the identification and/ or validation of a target
DE10202419A1 (de) 2002-01-22 2003-08-07 Ribopharma Ag Verfahren zur Hemmung der Expression eines durch eine Chromosomen-Aberration entstandenen Zielgens
DK1470144T3 (da) * 2002-02-01 2009-04-06 Univ Mcgill Oligonukleotider omfattende alternerende segmenter og anvendelser deraf
US20030166282A1 (en) 2002-02-01 2003-09-04 David Brown 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
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
EP1857547B2 (en) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Further novel forms of interfering RNA molecules
EP1393742A1 (en) 2002-08-14 2004-03-03 atugen AG Use of protein kinase N beta
MXPA05001766A (es) 2002-08-14 2005-08-19 Atugen Ag Uso de proteina quinasa n beta.
ES2319891T3 (es) 2002-10-18 2009-05-14 Silence Therapeutics Ag Factor implicado en la metastasis y usos del mismo.
US7289445B2 (en) * 2002-11-25 2007-10-30 Intel Corporation Managing a protocol control block cache in a network device
ES2905724T3 (es) 2003-06-13 2022-04-11 Alnylam Europe Ag Acido ribonucleico bicatenario con elevada eficacia en un organismo
WO2005000320A2 (en) 2003-06-27 2005-01-06 Atugen Ag Use of double-stranded ribonucleic acid for inducing cell lysis
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
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
US20050217329A1 (en) * 2004-04-02 2005-10-06 Wen-Kwei Chang Anti-drilling structure of a latch
DK1771206T3 (en) 2004-05-05 2018-05-22 Silence Therapeutics Gmbh LIPIDS, LIPID COMPLEXES AND USE THEREOF
EP1765847A4 (en) 2004-05-27 2010-10-20 Alnylam Pharmaceuticals Inc NUCLEASERESISTENT DOUBLE-STRANDED RIBONUCLEIC ACID
NZ553162A (en) 2004-08-16 2010-03-26 Quark Biotech Inc Double stranded siRNA compounds that inhibit RRTP801
BRPI0519468A2 (pt) 2004-12-27 2009-01-27 Silence Therapeutics Ag complexos de lipÍdeos revestido e seu uso
US20070027097A1 (en) 2005-07-28 2007-02-01 Lewis David L Small interfering RNA with improved activity
AU2006308399A1 (en) 2005-10-28 2007-05-03 Topigen Pharmaceuticals Inc. Small interfering ribonucleic acid duplexes comprising arabinose modified nucleotides
EP1948674A4 (en) 2005-11-02 2009-02-04 Protiva Biotherapeutics Inc MODIFIED SIRNA MOLECULES AND APPLICATIONS THEREOF
UY30097A1 (es) 2006-01-20 2007-08-31 Atugen Ag Usos terapeuticos de inhibidores de rtp801
US7541994B2 (en) 2006-05-17 2009-06-02 Raytheon Company Refractive compact range
CA2701845A1 (en) 2007-10-03 2009-04-09 Quark Pharmaceuticals, Inc. Novel sirna structures

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