JP2021533804A5 - - Google Patents

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Publication number
JP2021533804A5
JP2021533804A5 JP2021509991A JP2021509991A JP2021533804A5 JP 2021533804 A5 JP2021533804 A5 JP 2021533804A5 JP 2021509991 A JP2021509991 A JP 2021509991A JP 2021509991 A JP2021509991 A JP 2021509991A JP 2021533804 A5 JP2021533804 A5 JP 2021533804A5
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nucleotides
oligonucleotide
necessary
positions
antisense strand
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JP2021509991A
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JP7627042B2 (ja
JP2021533804A (ja
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Priority claimed from PCT/US2019/048027 external-priority patent/WO2020041769A1/en
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JP2021509991A 2018-08-23 2019-08-23 O-メチルリッチ完全安定化オリゴヌクレオチド Active JP7627042B2 (ja)

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US201862721993P 2018-08-23 2018-08-23
US62/721,993 2018-08-23
PCT/US2019/048027 WO2020041769A1 (en) 2018-08-23 2019-08-23 O-methyl rich fully stabilized oligonucleotides

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JP2021533804A5 true JP2021533804A5 (enExample) 2022-08-31
JP7627042B2 JP7627042B2 (ja) 2025-02-05

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US (2) US11279930B2 (enExample)
EP (1) EP3840759A4 (enExample)
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WO (1) WO2020041769A1 (enExample)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
US11753638B2 (en) 2016-08-12 2023-09-12 University Of Massachusetts Conjugated oligonucleotides
CA3064590A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts Two-tailed self-delivering sirna and related methods
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ O-メチルリッチ完全安定化オリゴヌクレオチド
WO2020150636A1 (en) 2019-01-18 2020-07-23 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
CA3149835A1 (en) 2019-08-09 2021-02-18 University Of Massachusetts Chemically modified oligonucleotides targeting snps
WO2021041247A1 (en) * 2019-08-23 2021-03-04 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
US12258566B2 (en) 2020-02-28 2025-03-25 University Of Massachusetts Oligonucleotides for PRNP modulation
CA3171246A1 (en) * 2020-03-18 2021-09-23 University Of Massachusetts Oligonucleotides for mapt modulation
US12146136B2 (en) * 2020-03-26 2024-11-19 University Of Massachusetts Synthesis of modified oligonucleotides with increased stability
EP4157289A4 (en) 2020-05-26 2024-06-26 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
AR126207A1 (es) 2021-06-23 2023-09-27 Univ Massachusetts Compuestos de oligonucleotidos anti-flt1 optimizados para el tratamiento de la preeclampsia y otros desordenes angiogénicos
WO2023014654A2 (en) * 2021-08-02 2023-02-09 University Of Massachusetts Oligonucleotides for htt-1a modulation
US20250340883A1 (en) * 2021-11-18 2025-11-06 Resonance Health Analysis Services Pty Ltd Method For Treating Cyclophilin B Associated Diseases
US20250188478A1 (en) * 2022-03-16 2025-06-12 Daiichi Sankyo Company, Limited siRNA SUPPRESSING EXPRESSION OF TRANSFERRIN RECEPTOR 2
WO2025063238A1 (ja) * 2023-09-20 2025-03-27 第一三共株式会社 ホスホランバンの発現を抑制するsiRNA

Family Cites Families (231)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US5240848A (en) 1988-11-21 1993-08-31 Monsanto Company Dna sequences encoding human vascular permeability factor having 189 amino acids
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5332671A (en) 1989-05-12 1994-07-26 Genetech, Inc. Production of vascular endothelial cell growth factor and DNA encoding same
US5219739A (en) 1989-07-27 1993-06-15 Scios Nova Inc. DNA sequences encoding bVEGF120 and hVEGF121 and methods for the production of bovine and human vascular endothelial cell growth factors, bVEGF120 and hVEGF121
US5194596A (en) 1989-07-27 1993-03-16 California Biotechnology Inc. Production of vascular endothelial cell growth factor
ATE162219T1 (de) 1989-11-16 1998-01-15 Univ Duke Transformation von tierischen hautzellen mit hilfe von partikeln
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5672697A (en) 1991-02-08 1997-09-30 Gilead Sciences, Inc. Nucleoside 5'-methylene phosphonates
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
JPH0641183A (ja) 1992-07-23 1994-02-15 Mitsubishi Kasei Corp オリゴヌクレオチド単分子膜
US5985558A (en) 1997-04-14 1999-11-16 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the inibition of c-Jun and c-Fos
US6291438B1 (en) 1993-02-24 2001-09-18 Jui H. Wang Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use
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
JPH08508490A (ja) 1993-03-31 1996-09-10 スターリング ウィンスロップ インコーポレイティド 新規5’−置換ヌクレオシド及びそれから得られるオリゴマー
TW360548B (en) 1993-04-08 1999-06-11 Powderject Res Ltd Products for therapeutic use
ATE188479T1 (de) 1994-06-01 2000-01-15 Hybridon Inc Verzweigte oligonukleotide als pathogenhemmende mittel
AU2991295A (en) 1994-07-26 1996-02-22 Kaken Pharmaceutical Co., Ltd. Sustance with antiviral activity
US6383814B1 (en) 1994-12-09 2002-05-07 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
WO1996033761A1 (en) 1995-04-28 1996-10-31 Medtronic, Inc. Intraparenchymal infusion catheter system
US6025335A (en) 1995-09-21 2000-02-15 Lipitek International, Inc. L-Nucleoside Dimer Compounds and therapeutic uses
US5684143A (en) 1996-02-21 1997-11-04 Lynx Therapeutics, Inc. Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates
US5735814A (en) 1996-04-30 1998-04-07 Medtronic, Inc. Techniques of treating neurodegenerative disorders by brain infusion
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
US6042820A (en) 1996-12-20 2000-03-28 Connaught Laboratories Limited Biodegradable copolymer containing α-hydroxy acid and α-amino acid units
AUPO974597A0 (en) 1997-10-10 1997-11-06 Rhone-Poulenc Agro Methods for obtaining plant varieties
US6472375B1 (en) 1998-04-16 2002-10-29 John Wayne Cancer Institute DNA vaccine and methods for its use
US6242589B1 (en) 1998-07-14 2001-06-05 Isis Pharmaceuticals, Inc. Phosphorothioate oligonucleotides having modified internucleoside linkages
EP1016726A1 (en) 1998-12-30 2000-07-05 Introgene B.V. Gene therapy to promote angiogenesis
US7608681B2 (en) 1999-12-24 2009-10-27 Genentech, Inc. Methods and compositions for prolonging elimination half-times of bioactive compounds
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
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)
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
KR100872437B1 (ko) 2000-12-01 2008-12-05 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Rna 간섭을 매개하는 작은 rna 분자
CA2462144C (en) 2001-09-28 2016-09-20 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Micro-rna molecules
WO2003053363A2 (en) 2001-12-19 2003-07-03 Millennium Pharmaceuticals, Inc. Human diacylglycerol acyltransferase 2 (dgat2) family members and uses therefor
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
US20050096284A1 (en) 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases 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)
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)
AU2003223775A1 (en) 2002-04-30 2003-11-17 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
BRPI0312818B8 (pt) 2002-07-19 2021-05-25 Beth Israel Deaconess Medical Ct Inc uso de uma medida do nível do polipeptídeo sflt-1 em uma amostra de uma paciente e uso de uma medida dos níveis de pelo menos dois dos peptídeos sflt1, vegf livre ou polipeptídio pigf livre em uma amostra de um paciente usando um métrico
US20050106731A1 (en) 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
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)
US8097752B2 (en) 2002-10-30 2012-01-17 Makscientific, Llc Antiprotozoal ring-substituted phospholipids
WO2004043979A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
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
US7790867B2 (en) 2002-12-05 2010-09-07 Rosetta Genomics Inc. Vaccinia virus-related nucleic acids and microRNA
US6913890B2 (en) 2002-12-18 2005-07-05 Palo Alto Research Center Incorporated Process for preparing albumin protein conjugated oligonucleotide probes
WO2004061081A2 (en) 2002-12-27 2004-07-22 Ichem Technologies Sirna compounds and methods for the downregulation of gene expression
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
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
ES2712695T3 (es) 2003-06-02 2019-05-14 Univ Massachusetts Métodos y composiciones para controlar la eficacia de la silenciación del ARN
DE10326302A1 (de) 2003-06-11 2004-12-30 Gnothis Holding Sa Bi-Fluorophor-markierte Sonden zum Nachweis von Nukleinsäuren
WO2004113496A2 (en) 2003-06-20 2004-12-29 Isis Pharmaceuticals, Inc. Double stranded compositions comprising a 3’-endo modified strand for use in gene modulation
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
SG146682A1 (en) 2003-09-18 2008-10-30 Isis Pharmaceuticals Inc Modulation of eif4e expression
CN1906308B (zh) 2003-12-19 2011-09-14 诺华疫苗和诊断公司 小干扰rna的细胞转染制剂、相关组合物以及制备和使用方法
US20050176045A1 (en) 2004-02-06 2005-08-11 Dharmacon, Inc. SNP discriminatory siRNA
US20060078542A1 (en) 2004-02-10 2006-04-13 Mah Cathryn S Gel-based delivery of recombinant adeno-associated virus vectors
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
KR101147147B1 (ko) * 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
CA2564616C (en) 2004-04-20 2016-08-30 Nastech Pharmaceutical Company Inc. Methods and compositions for enhancing delivery of double-stranded rna or a double-stranded hybrid nucleic acid to regulate gene expression in mammalian cells
EP1752536A4 (en) 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
JP2008504840A (ja) 2004-06-30 2008-02-21 アルニラム ファーマスーティカルズ インコーポレイテッド 非リン酸骨格結合を含むオリゴヌクレオチド
US7361468B2 (en) 2004-07-02 2008-04-22 Affymetrix, Inc. Methods for genotyping polymorphisms in humans
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
JP2009504782A (ja) 2005-08-18 2009-02-05 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 神経疾患を治療するための方法および組成物
DK1931780T3 (en) * 2005-08-29 2016-01-25 Regulus Therapeutics Inc Antisense-forbindelser med forøget anti-microrna-aktivitet
AU2006305886C1 (en) 2005-10-28 2011-03-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of huntingtin gene
WO2007051303A1 (en) 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. MODIFIED siRNA MOLECULES AND USES THEREOF
WO2007056153A2 (en) 2005-11-04 2007-05-18 Nastech Pharmaceutical Company Inc. Peptide-dicer substrate rna conjugates as delivery vehicles for sirna
US7825099B2 (en) 2006-01-20 2010-11-02 Quark Pharmaceuticals, Inc. Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes
WO2007087451A2 (en) 2006-01-25 2007-08-02 University Of Massachusetts Compositions and methods for enhancing discriminatory rna interference
US9198981B2 (en) 2006-02-01 2015-12-01 The University Of Kentucky Modulation of angiogenesis
WO2007091269A2 (en) 2006-02-08 2007-08-16 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
WO2007094218A1 (ja) 2006-02-16 2007-08-23 Gifu University 修飾オリゴヌクレオチド
US20100184209A1 (en) 2006-02-17 2010-07-22 Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
US20090306178A1 (en) 2006-03-27 2009-12-10 Balkrishen Bhat Conjugated double strand compositions for use in gene modulation
CA2662704A1 (en) 2006-07-07 2008-01-10 University Of Massachusetts Rna silencing compositions and methods for the treatment of huntington's disease
US20080039415A1 (en) 2006-08-11 2008-02-14 Gregory Robert Stewart Retrograde transport of sirna and therapeutic uses to treat neurologic disorders
US8809514B2 (en) 2006-09-22 2014-08-19 Ge Healthcare Dharmacon, Inc. Tripartite oligonucleotide complexes and methods for gene silencing by RNA interference
AU2007310982A1 (en) 2006-10-18 2008-04-24 Marina Biotech, Inc. Nicked or gapped nucleic acid molecules and uses thereof
US8906874B2 (en) 2006-11-09 2014-12-09 Gradalis, Inc. Bi-functional shRNA targeting Stathmin 1 and uses thereof
JP2010510810A (ja) 2006-11-27 2010-04-08 エンゾン ファーマスーティカルズ インコーポレイテッド ポリマー低分子干渉rna複合体
EP2126081A2 (en) 2007-03-02 2009-12-02 MDRNA, Inc. Nucleic acid compounds for inhibiting hif1a gene expression and uses thereof
WO2008154482A2 (en) 2007-06-08 2008-12-18 Sirnaomics, Inc. Sirna compositions and methods of use in treatment of ocular diseases
WO2009002944A1 (en) * 2007-06-22 2008-12-31 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
AU2008290545A1 (en) 2007-08-23 2009-02-26 Novartis Ag Methods for detecting oligonucleotides
EP2193140B1 (en) 2007-08-27 2016-11-02 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
CN101199858A (zh) 2007-10-18 2008-06-18 广州拓谱基因技术有限公司 治疗眼科疾病的多靶点小干扰rna鸡尾酒制剂及制备方法
TW200927177A (en) 2007-10-24 2009-07-01 Nat Inst Of Advanced Ind Scien Lipid-modified double-stranded RNA having potent RNA interference effect
CN102006890A (zh) 2007-12-04 2011-04-06 阿尔尼拉姆医药品有限公司 靶向脂质
US9029524B2 (en) 2007-12-10 2015-05-12 California Institute Of Technology Signal activated RNA interference
WO2009099991A2 (en) 2008-01-31 2009-08-13 The Brigham And Women's Hospital, Inc. Treatment of cancer
EP2247729B1 (en) 2008-02-11 2019-05-01 Phio Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010008582A2 (en) 2008-07-18 2010-01-21 Rxi Pharmaceuticals Corporation Phagocytic cell drug delivery system
EP2318528A1 (en) 2008-07-24 2011-05-11 Rxi Pharmaceuticals Corporation Rnai constructs and uses therof
CA2732229C (en) * 2008-07-25 2023-10-17 Alnylam Pharmaceuticals, Inc. Enhancement of sirna silencing activity using universal bases or mismatches in the sense strand
EP2340309A2 (en) 2008-09-22 2011-07-06 Rxi Pharmaceuticals Corporation Neutral nanotransporters
WO2010043512A1 (en) 2008-10-15 2010-04-22 F. Hoffmann-La Roche Ag Oligonucleotide detection method
CN102994500B (zh) 2008-10-22 2015-04-01 夸克制药公司 治疗眼部疾病的方法
WO2010048585A2 (en) 2008-10-24 2010-04-29 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
US9074211B2 (en) 2008-11-19 2015-07-07 Rxi Pharmaceuticals Corporation Inhibition of MAP4K4 through RNAI
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
WO2010083615A1 (en) 2009-01-26 2010-07-29 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
KR101141544B1 (ko) 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
EP2411518A2 (en) 2009-03-27 2012-02-01 Merck Sharp&Dohme Corp. Rna interference mediated inhibition of the high affinity ige receptor alpha chain (fcerla) gene expression using short interfering nucleic acid (sina)
US8309356B2 (en) 2009-04-01 2012-11-13 Yale University Pseudocomplementary oligonucleotides for targeted gene therapy
WO2010118263A1 (en) 2009-04-08 2010-10-14 University Of Massachusetts Single-nucleotide polymorphism (snp) targeting therapies for the treatment of huntington's disease
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
GB0910723D0 (en) 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
SI2470656T1 (sl) 2009-08-27 2015-07-31 Idera Pharmaceuticals, Inc. Sestavek za inhibicijo ekspresije genov in njegova uporaba
RU2562861C2 (ru) 2009-09-11 2015-09-10 Айсис Фармасьютикалс, Инк. Модуляция экспрессии гентингтина
US20150025122A1 (en) 2009-10-12 2015-01-22 Larry J. Smith Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro
US8877439B2 (en) * 2009-12-17 2014-11-04 Sirna Therapeutics, Inc. Method for rapidly evaluating performance of short interfering RNA with novel chemical modifications
EP3561060A1 (en) 2010-02-08 2019-10-30 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
WO2011109698A1 (en) 2010-03-04 2011-09-09 Rxi Pharmaceuticals Corporation Formulations and methods for targeted delivery to phagocyte cells
EP2550000A4 (en) 2010-03-24 2014-03-26 Advirna Inc RNAI COMPOUNDS OF REDUCED SIZE ADMINISTERING
BR112012024049A2 (pt) 2010-03-24 2017-03-01 Rxi Pharmaceuticals Corp interferência de rna em indicações dérmicas e fibróticas
CN106074591B (zh) 2010-03-24 2020-01-14 菲奥医药公司 眼部症候中的rna干扰
WO2011125943A1 (ja) 2010-04-01 2011-10-13 日本新薬株式会社 修飾オリゴヌクレオチド
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
WO2012005898A2 (en) 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof
JP5941842B2 (ja) 2010-06-18 2016-06-29 Lsipファンド運営合同会社 優性アレル発現抑制剤
US8586301B2 (en) 2010-06-30 2013-11-19 Stratos Genomics, Inc. Multiplexed identification of nucleic acid sequences
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
EP3766975A1 (en) 2010-10-29 2021-01-20 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acid (sina)
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
US10378016B2 (en) 2010-12-06 2019-08-13 Children's Medical Center Corporation Composition and method for oligonucleotide delivery
CN103492382A (zh) 2010-12-16 2014-01-01 沃泰克斯药物股份有限公司 流感病毒复制的抑制剂
KR20130128435A (ko) 2010-12-16 2013-11-26 버텍스 파마슈티칼스 인코포레이티드 인플루엔자 바이러스 복제의 억제제
AU2011343642A1 (en) 2010-12-16 2013-05-02 Vertex Pharmaceuticals Incorporated Inhibitors of influenza viruses replication
BR112013013434A2 (pt) 2010-12-17 2016-08-16 Arrowhead Res Corp metade que tem por alvo modulador fármaco cinético em cacho de galactose para sirna
WO2012118911A1 (en) 2011-03-03 2012-09-07 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
GB201105137D0 (en) 2011-03-28 2011-05-11 Isis Innovation Therapeutic molecules for use in the suppression of Parkinson's disease
WO2012143796A2 (en) 2011-04-21 2012-10-26 Institut Pasteur Korea Anti-inflammation compounds
EP2723865B1 (en) 2011-06-21 2019-03-27 Alnylam Pharmaceuticals, Inc. METHODS FOR DETERMINING ACTIVITY OF RNAi IN A SUBJECT
EP3358013B1 (en) 2012-05-02 2020-06-24 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
DK2853597T3 (en) 2012-05-22 2019-04-08 Olix Pharmaceuticals Inc RNA INTERFERENCE-INducing NUCLEIC ACID MOLECULES WITH CELL PENETENING EQUIPMENT AND USE THEREOF
AU2013288738A1 (en) 2012-07-10 2015-01-29 Baseclick Gmbh Anandamide-modified nucleic acid molecules
CA2884608C (en) 2012-09-14 2025-05-20 Translate Bio Ma, Inc. MULTIMERIC OLIGONUCLEOTIDE COMPOUNDS
SG10201804331TA (en) 2012-11-15 2018-07-30 Roche Innovation Ct Copenhagen As Oligonucleotide conjugates
KR102096014B1 (ko) 2012-12-05 2020-04-03 알닐람 파마슈티칼스 인코포레이티드 PCSK9 iRNA 조성물 및 그 사용 방법
EP2931319B1 (en) 2012-12-13 2019-08-21 ModernaTX, Inc. Modified nucleic acid molecules and uses thereof
US20140200124A1 (en) 2013-01-15 2014-07-17 Karen Michelle JAMES Physical Therapy Device and Methods for Use Thereof
CA2921162A1 (en) 2013-05-01 2014-11-06 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
WO2014201306A1 (en) 2013-06-12 2014-12-18 Oncoimmunin, Inc. Systemic in vivo delivery of oligonucleotides
EP3010514B1 (en) 2013-06-16 2021-01-20 National University Corporation Tokyo Medical and Dental University Double-stranded antisense nucleic acid with exon-skipping effect
US9862350B2 (en) 2013-08-12 2018-01-09 Tk Holdings Inc. Dual chambered passenger airbag
GB201315141D0 (en) 2013-08-23 2013-10-09 Logicor R & D Ltd Improvements to electric heating systems and method of use thereof
WO2015057847A1 (en) 2013-10-16 2015-04-23 Cerulean Pharma Inc. Conjugates, particles, compositions, and related methods
US10752940B2 (en) 2013-11-08 2020-08-25 Ionis Pharmaceuticals, Inc. Compounds and methods for detecting oligonucleotides
EP3099795A4 (en) 2014-01-27 2018-01-17 The Board of Trustees of the Leland Stanford Junior University Oligonucleotides and methods for treatment of cardiomyopathy using rna interference
EP3132044B1 (en) 2014-04-18 2020-04-08 University of Massachusetts Exosomal loading using hydrophobically modified oligonucleotides
MA39941A (fr) 2014-05-01 2017-03-08 Larry J Smith Procédés et modifications permettant de produire des composés arni simple brin à activité, potentiel et durée d'effet améliorés
WO2015171918A2 (en) 2014-05-07 2015-11-12 Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
BR122020023687B1 (pt) 2014-05-22 2023-03-07 Alnylam Pharmaceuticals, Inc Agente de ácido ribonucleico (rnai) de fita dupla, seus usos e composição farmacêutica
US10588980B2 (en) 2014-06-23 2020-03-17 Novartis Ag Fatty acids and their use in conjugation to biomolecules
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
US20170327524A1 (en) 2014-10-10 2017-11-16 Hoffmann-La Roche, Inc. Galnac phosphoramidites, nucleic acid conjugates thereof and their use
SG11201703638XA (en) 2014-11-10 2017-06-29 Alnylam Pharmaceuticals Inc Hepatitis b virus (hbv) irna compositions and methods of use thereof
WO2016083623A1 (en) 2014-11-28 2016-06-02 Silence Therapeutics Gmbh Means for the treatment of pre-eclampsia
EP3237618B1 (en) 2014-12-24 2019-05-22 uniQure IP B.V. Rnai induced huntingtin gene suppression
US10450563B2 (en) 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
CA2979703A1 (en) * 2015-03-17 2016-09-22 Arrowhead Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression of factor xii
WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
EP3277815B1 (en) 2015-04-03 2021-09-22 University of Massachusetts Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
JP7009356B2 (ja) 2015-04-03 2022-01-25 ユニバーシティ・オブ・マサチューセッツ ハンチンチンmRNAをターゲティングするオリゴヌクレオチド化合物
CA2982369A1 (en) 2015-04-10 2016-10-13 Hudsonalpha Institute For Biotechnology Method for blocking mirna
IL316159A (en) 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
US20170009304A1 (en) 2015-07-07 2017-01-12 Splicingcodes.Com Method and kit for detecting fusion transcripts
WO2017015575A1 (en) 2015-07-22 2017-01-26 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
EP3331573A4 (en) 2015-08-06 2019-02-27 City of Hope THERAPEUTIC CELL-INTERNALIZING CONJUGATES
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
EP3359523A4 (en) * 2015-10-09 2019-07-24 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHOD THEREFOR
CN105194689A (zh) 2015-10-10 2015-12-30 湖南中楚博生物科技有限公司 一种基于siRNA的双偶联复合物
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
EP3440222B1 (en) 2016-04-04 2025-02-19 F. Hoffmann-La Roche AG Nucleic acid sample preparation methods
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)
US11753638B2 (en) 2016-08-12 2023-09-12 University Of Massachusetts Conjugated oligonucleotides
CA3035293A1 (en) 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
RS63836B1 (sr) * 2017-04-05 2023-01-31 Silence Therapeutics Gmbh Proizvodi i sastavi
CN111050806A (zh) 2017-06-02 2020-04-21 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
CA3064590A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts Two-tailed self-delivering sirna and related methods
CA3071086A1 (en) 2017-08-18 2019-02-21 The Procter & Gamble Company Cleaning agent
SG11202003310XA (en) 2017-10-12 2020-05-28 Avery Dennison Corp Low outgassing clean adhesive
IL273875B2 (en) 2017-10-13 2025-07-01 Dicerna Pharmaceuticals Inc Methods and compositions for inhibiting expression of ldha
WO2019099949A1 (en) 2017-11-17 2019-05-23 The Regents Of The University Of California Manipulation of the retinoic acid signaling pathway
EP3550021A1 (en) * 2018-04-05 2019-10-09 Silence Therapeutics GmbH Products and compositions for inhibiting expression of a target gene
EP3790970A1 (en) 2018-05-07 2021-03-17 Alnylam Pharmaceuticals Inc. Extrahepatic delivery
CN120247995A (zh) 2018-05-30 2025-07-04 诺华股份有限公司 脂质修饰的核酸化合物和方法
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ O-メチルリッチ完全安定化オリゴヌクレオチド
WO2020150636A1 (en) 2019-01-18 2020-07-23 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
AU2020239987A1 (en) 2019-03-15 2021-11-04 University Of Massachusetts Oligonucleotides for tissue specific ApoE modulation
US11820985B2 (en) 2019-03-26 2023-11-21 University Of Massachusetts Modified oligonucleotides with increased stability
EP3946374A4 (en) 2019-03-29 2023-07-19 University of Massachusetts OLIGONUCLEOTIDE-BASED MODULATION OF C9ORF72
CA3149835A1 (en) 2019-08-09 2021-02-18 University Of Massachusetts Chemically modified oligonucleotides targeting snps
WO2021041247A1 (en) * 2019-08-23 2021-03-04 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
EP4090744A4 (en) 2020-01-17 2024-04-10 Anastasia Khvorova UNIVERSAL DYNAMIC PHARMACOKINETIC MODIFYING ANCHORS
US12258566B2 (en) 2020-02-28 2025-03-25 University Of Massachusetts Oligonucleotides for PRNP modulation
CA3171246A1 (en) 2020-03-18 2021-09-23 University Of Massachusetts Oligonucleotides for mapt modulation
PL4126040T3 (pl) 2020-03-26 2025-04-28 Nutrivert Inc. Lipofilowe enancjomery deacetyloglukozamino–muramylodipeptydu o aktywności przeciwzapalnej i wspomagającej wzrost
US12146136B2 (en) 2020-03-26 2024-11-19 University Of Massachusetts Synthesis of modified oligonucleotides with increased stability
WO2021195510A1 (en) 2020-03-27 2021-09-30 University Of Massachusetts Dual-acting sirna based modulation of c9orf72
JP2023522701A (ja) 2020-04-20 2023-05-31 ユニバーシティー オブ マサチューセッツ Msh3調節のためのオリゴヌクレオチド
EP4157289A4 (en) 2020-05-26 2024-06-26 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
EP4158028A4 (en) 2020-05-28 2024-10-02 University Of Massachusetts OLIGONUCLEOTIDES FOR THE MODULATION OF SARS-COV-2
CA3174068A1 (en) 2020-07-16 2022-01-20 Anastasia Khvorova Conjugated oligonucleotides for tissue specific delivery
WO2022031591A2 (en) 2020-08-03 2022-02-10 University Of Massachusetts Oligonucleotides for htt-1a modulation
AU2021382621A1 (en) 2020-11-23 2023-06-22 University Of Massachusetts Oligonucleotides for dgat2 modulation
AR126207A1 (es) 2021-06-23 2023-09-27 Univ Massachusetts Compuestos de oligonucleotidos anti-flt1 optimizados para el tratamiento de la preeclampsia y otros desordenes angiogénicos
WO2023014654A2 (en) 2021-08-02 2023-02-09 University Of Massachusetts Oligonucleotides for htt-1a modulation
US20230193281A1 (en) 2021-11-08 2023-06-22 University Of Massachusetts Oligonucleotides for sod1 modulation
WO2023114989A2 (en) 2021-12-17 2023-06-22 University Of Massachusetts Oligonucleotides for mlh3 modulation
US20230340475A1 (en) 2021-12-17 2023-10-26 University Of Massachusetts Oligonucleotides for mlh1 modulation
WO2023133550A2 (en) 2022-01-10 2023-07-13 University Of Massachusetts Oligonucleotides for mecp2 modulation
US20230392146A1 (en) 2022-03-11 2023-12-07 University Of Massachusetts Oligonucleotides for app modulation
WO2023201281A2 (en) 2022-04-14 2023-10-19 University Of Massachusetts Oligonucleotides for atn1 modulation
CN120076786A (zh) 2022-09-30 2025-05-30 马萨诸塞大学 寡核苷酸的经眼递送

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