CN113481201A - 用于减少tau表达的化合物和方法 - Google Patents

用于减少tau表达的化合物和方法 Download PDF

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Publication number
CN113481201A
CN113481201A CN202110697520.2A CN202110697520A CN113481201A CN 113481201 A CN113481201 A CN 113481201A CN 202110697520 A CN202110697520 A CN 202110697520A CN 113481201 A CN113481201 A CN 113481201A
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certain embodiments
modified
oligonucleotide
tau
compound
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CN202110697520.2A
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Chinese (zh)
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H·柯达赛维茨
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Biogen MA Inc
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Ionis Pharmaceuticals Inc
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Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of CN113481201A publication Critical patent/CN113481201A/zh
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CN202110697520.2A 2016-09-29 2017-09-29 用于减少tau表达的化合物和方法 Pending CN113481201A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662401723P 2016-09-29 2016-09-29
US62/401,723 2016-09-29
US201762450469P 2017-01-25 2017-01-25
US62/450,469 2017-01-25
CN201780059823.3A CN109790543B (zh) 2016-09-29 2017-09-29 用于减少tau表达的化合物和方法

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EP (2) EP4335923A3 (OSRAM)
JP (4) JP6738004B2 (OSRAM)
KR (2) KR20210097228A (OSRAM)
CN (2) CN113481201A (OSRAM)
AU (3) AU2017336093B2 (OSRAM)
BR (2) BR122020019785B1 (OSRAM)
CA (1) CA3038891C (OSRAM)
CL (2) CL2019000703A1 (OSRAM)
CO (1) CO2019001385A2 (OSRAM)
CR (2) CR20190214A (OSRAM)
DK (1) DK3519572T5 (OSRAM)
ES (1) ES2963428T3 (OSRAM)
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IL (3) IL299615A (OSRAM)
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SM (1) SMT202300441T1 (OSRAM)
WO (1) WO2018064593A1 (OSRAM)
ZA (2) ZA201900920B (OSRAM)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013148260A1 (en) 2012-03-30 2013-10-03 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
EP2839006B1 (en) * 2012-04-20 2018-01-03 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
MX2015012621A (es) 2013-03-14 2016-05-31 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de tau.
TW202246503A (zh) 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
JOP20190065A1 (ar) * 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
AU2019208011B2 (en) 2018-01-12 2025-06-05 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
WO2019140231A1 (en) 2018-01-12 2019-07-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CR20210178A (es) * 2018-07-03 2021-05-11 Hoffmann La Roche OLIGONUCLEÓTIDOS PARA MODULAR LA EXPRESIÓN DE TAU (Divisional 2021-0058)
WO2022125984A1 (en) * 2020-12-11 2022-06-16 Eisai R&D Management Co., Ltd. Tau-targeting oligonucleotide gapmers
US20240167031A1 (en) 2021-04-01 2024-05-23 Biogen Ma Inc. Nucleic acid delivery to the central nervous system
AU2022293556A1 (en) * 2021-06-18 2024-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ifnar1 expression
EP4373938A2 (en) * 2021-07-23 2024-05-29 University Of Kentucky Research Foundation Inhibition of neurofibrillary tangles using oligonucleotides against circular rnas from the microtubule associated protein tau (mapt) locus
KR20240040759A (ko) * 2021-07-23 2024-03-28 바이오젠 엠에이 인코포레이티드 타우 발현을 감소시키는 방법
WO2025043202A1 (en) * 2023-08-23 2025-02-27 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atn1 expression

Family Cites Families (144)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1027347A (en) 1910-11-23 1912-05-21 Columbus K Lassiter Multiple-spindle drill.
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4880635B1 (en) 1984-08-08 1996-07-02 Liposome Company Dehydrated liposomes
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US4921757A (en) 1985-04-26 1990-05-01 Massachusetts Institute Of Technology System for delayed and pulsed release of biologically active substances
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
JPH0825869B2 (ja) 1987-02-09 1996-03-13 株式会社ビタミン研究所 抗腫瘍剤包埋リポソ−ム製剤
US4917951A (en) 1987-07-28 1990-04-17 Micro-Pak, Inc. Lipid vesicles formed of surfactants and steroids
US4911928A (en) 1987-03-13 1990-03-27 Micro-Pak, Inc. Paucilamellar lipid vesicles
CA1340032C (en) 1987-06-24 1998-09-08 Jim Haralambidis Lucleoside derivatives
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5457191A (en) 1990-01-11 1995-10-10 Isis Pharmaceuticals, Inc. 3-deazapurines
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5859221A (en) 1990-01-11 1999-01-12 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US5614617A (en) 1990-07-27 1997-03-25 Isis Pharmaceuticals, Inc. Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
US5948903A (en) 1991-01-11 1999-09-07 Isis Pharmaceuticals, Inc. Synthesis of 3-deazapurines
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
JP3739785B2 (ja) 1991-11-26 2006-01-25 アイシス ファーマシューティカルズ，インコーポレイティド 修飾されたピリミジンを含有するオリゴマーを使用する増強された三重らせんおよび二重らせんの成形
US20040054156A1 (en) 1992-05-14 2004-03-18 Kenneth Draper Method and reagent for inhibiting hepatitis B viral replication
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
KR940021073A (ko) 1993-03-02 1994-10-17 미야베 요시가즈 알쯔하이머병의 예방 또는 치료제, 그 스크리닝 방법 및 인간 유래의 타우 단백질 키나아제 i
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
USRE44779E1 (en) 1997-03-07 2014-02-25 Santaris Pharma A/S Bicyclonucleoside and oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
WO1999014226A2 (en) 1997-09-12 1999-03-25 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues
US20030228597A1 (en) 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
AU4322999A (en) 1998-06-01 1999-12-20 Advanced Research And Technology Institute, Inc. Methods and compositions for diagnosing tauopathies
ATE287897T2 (de) 1999-02-12 2005-02-15 Sankyo Co Analoga von nukleosiden und oligonukleotiden
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
IL145778A0 (en) 1999-04-21 2002-07-25 American Home Prod Methods and compositions for inhibiting the function of polynucleotide sequences
CN102180924A (zh) 1999-05-04 2011-09-14 桑塔里斯制药公司 L-核糖-lna类似物
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
EP1097941A1 (en) 1999-11-05 2001-05-09 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Substance capable of controlling the inclusion of exon 10 of the tau gene, and its therapeutic use against tauopathies
WO2001072765A1 (en) 2000-03-28 2001-10-04 Isis Pharmaceuticals, Inc. ALTERATION OF CELLULAR BEHAVIOR BY ANTISENSE MODULATION OF mRNA PROCESSING
US20040241651A1 (en) 2000-04-07 2004-12-02 Alexander Olek Detection of single nucleotide polymorphisms (snp's) and cytosine-methylations
JP2004532022A (ja) 2001-03-26 2004-10-21 サーナ・セラピューティクス・インコーポレイテッド Ｂ型肝炎ウイルスおよびｃ型肝炎ウイルスの複製のオリゴヌクレオチド媒介性阻害
US20050019915A1 (en) 2001-06-21 2005-01-27 Bennett C. Frank Antisense modulation of superoxide dismutase 1, soluble expression
JP2005504020A (ja) 2001-07-03 2005-02-10 アイシス・ファーマシューティカルス・インコーポレーテッド ヌクレアーゼ耐性キメラオリゴヌクレオチド
US20030158403A1 (en) 2001-07-03 2003-08-21 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
US20030175906A1 (en) 2001-07-03 2003-09-18 Muthiah Manoharan Nuclease resistant chimeric oligonucleotides
WO2003056030A2 (en) 2001-11-08 2003-07-10 The Johns Hopkins University Methods and systems of nucleic acid sequencing
US20070203333A1 (en) 2001-11-30 2007-08-30 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20050153336A1 (en) 2002-03-29 2005-07-14 Bennett C. F. Compositions and their uses directed to nucleic acid binding proteins
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
WO2004011613A2 (en) 2002-07-29 2004-02-05 Epigenesis Pharmaceuticals, Inc. Composition & methods for treatment and screening
US20050106731A1 (en) 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
WO2004017072A1 (en) 2002-08-14 2004-02-26 Universität Zürich A cellular assay system for neurofibrillary tangle formation
US20060035344A1 (en) 2002-10-18 2006-02-16 Pachuk Catherine J Double-stranded rna structures and constructs, and methods for generating and using the same
AU2003291753B2 (en) 2002-11-05 2010-07-08 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2004044136A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2’-modified nucleosides for use in gene modulation
EP1560931B1 (en) 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
US20080318210A1 (en) 2003-08-27 2008-12-25 Rosetta Genomics Bioinformatically detectable group of novel regulatory viral and viral associated oligonucleotides and uses thereof
US6673661B1 (en) 2002-12-20 2004-01-06 Taiwan Semiconductor Manufacturing Co., Ltd. Self-aligned method for forming dual gate thin film transistor (TFT) device
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
AU2003904328A0 (en) 2003-08-13 2003-08-28 Garvan Institute Of Medical Research Diagnosis and treatment of neurodegenerative disorders
ATE555118T1 (de) 2003-08-28 2012-05-15 Takeshi Imanishi Neue synthetische nukleidsäuren vom typ mit quervernetzter n-o-bindung
JP5379347B2 (ja) 2003-09-18 2013-12-25 アイシス ファーマシューティカルズ， インコーポレーテッド ４’−チオヌクレオシドおよびオリゴマー化合物
WO2005030928A2 (en) 2003-09-23 2005-04-07 Chihiro Koike PORCINE INVARIANT CHAIN PROTEIN, FULL LENGTH cDNA, GENOMIC ORGANIZATION, AND REGULATORY REGION
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
CN100410383C (zh) 2004-05-11 2008-08-13 中国农业科学院生物技术研究所 一种昆虫杆状病毒生物反应器的制备方法
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
ES2415665T3 (es) 2004-06-21 2013-07-26 Proteome Sciences Plc Procedimientos de cribado usando c-abl, fyn y sky en combinación con la proteína tau
WO2006047673A2 (en) 2004-10-27 2006-05-04 Vanderbilt University Mammalian genes involved in infection
US20080003570A1 (en) 2004-12-22 2008-01-03 The General Hospital Corporation Translation enhancer elements of genes encoding human Tau protein and human alpha-synuclein protein
US20060216722A1 (en) 2005-03-25 2006-09-28 Christer Betsholtz Glomerular expression profiling
EP2338991B1 (en) 2005-08-29 2017-01-18 Regulus Therapeutics Inc. Methods for use in modulating MIR-122a
US7858747B2 (en) 2005-08-30 2010-12-28 Novo Nordisk A/S Expression of proteins in E.coli
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
JP5342881B2 (ja) 2006-01-27 2013-11-13 アイシス ファーマシューティカルズ， インコーポレーテッド ６−修飾された二環式核酸類似体
US8178503B2 (en) 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
EP2505646A1 (en) 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of CRP
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
US7547684B2 (en) 2006-05-11 2009-06-16 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
WO2008043561A2 (en) 2006-10-11 2008-04-17 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Influenza targets
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
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
EP2578692B1 (en) 2007-04-05 2016-06-08 The J. David Gladstone Institutes Agents that reduce neuronal overexcitation
KR20170056032A (ko) 2007-05-01 2017-05-22 로슈 이노베이션 센터 코펜하겐 에이/에스 Ｔｎｆ 슈퍼패밀리 수용체에 대한 스플라이스 스위칭 올리고머 및 질병 치료에 있어서의 그의 용도
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
WO2008151631A2 (en) 2007-06-15 2008-12-18 Exiqon A/S Use of short oligonucleotides for reagent redundancy experiments in rna functional analysis
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
AU2008286771B2 (en) 2007-08-15 2013-08-15 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
US20090076725A1 (en) 2007-09-14 2009-03-19 Kulvir Singh Bhogal Conveyance mode aware navigation device
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
EP2265627A2 (en) 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
EP2268813A4 (en) 2008-04-07 2012-10-10 Univ Queensland RNA MOLECULES AND USES THEREOF
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
SI3305302T1 (sl) 2009-06-17 2018-12-31 Biogen Ma Inc. Sestave in metode za modulacijo združevanja smn2 pri subjektu
CA2767225A1 (en) 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for enhancing production of a biological product
EP2451476A4 (en) 2009-07-06 2013-07-03 Alnylam Pharmaceuticals Inc BIOTRAITEMENT BASED ON CELLS
US20110041191A1 (en) 2009-07-09 2011-02-17 Bettina Platt Animal model, and products and methods useful for the production thereof
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
DK2534248T3 (en) 2010-02-08 2018-11-19 Ionis Pharmaceuticals Inc SELECTIVE REDUCTION OF ALLELVARIANS
US9102938B2 (en) 2010-04-01 2015-08-11 Alnylam Pharmaceuticals, Inc. 2′ and 5′ modified monomers and oligonucleotides
DK2560687T3 (en) 2010-04-19 2017-09-18 Nlife Therapeutics S L Preparations and Methods for Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types
WO2011133876A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
WO2011139702A2 (en) 2010-04-28 2011-11-10 Isis 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
WO2012018881A2 (en) 2010-08-03 2012-02-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for the regulation of rna
US20140155462A1 (en) 2011-04-22 2014-06-05 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibitions of egfr by double-stranded rna
WO2012177639A2 (en) 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
US9670488B2 (en) 2011-11-04 2017-06-06 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of met by double stranded RNA
WO2013148260A1 (en) 2012-03-30 2013-10-03 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
BR112014028645A2 (pt) 2012-05-16 2017-06-27 Rana Therapeutics Inc composições e métodos para modulação da expressão de apoa1 e abca1.
EP2872147B1 (en) * 2012-07-13 2022-12-21 Wave Life Sciences Ltd. Method for making chiral oligonucleotides
GB201301233D0 (en) 2013-01-24 2013-03-06 Queen Mary & Westfield College Method
MX2015012621A (es) 2013-03-14 2016-05-31 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de tau.
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
TW202246503A (zh) * 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
US10119136B2 (en) 2014-01-09 2018-11-06 Alnylam Pharmaceuticals, Inc. RNAi agents modified at the 4′-C position
KR101614358B1 (ko) * 2014-03-02 2016-04-21 신창섭 지면 밀착식 박판형 확대 지압판
WO2016019063A1 (en) 2014-07-29 2016-02-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
WO2016127002A1 (en) 2015-02-04 2016-08-11 Bristol-Myers Squibb Company Lna oligonucleotides with alternating flanks
PE20171766A1 (es) 2015-02-04 2017-12-21 Hoffmann La Roche Oligomeros antisentido de tau y usos de estos
ES2820713T3 (es) 2015-03-25 2021-04-22 Univ Degli Studi Di Trento Terapia mediada por ARN interferente para enfermedades neurodegenerativas
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
JOP20200228A1 (ar) 2015-12-21 2017-06-16 Novartis Ag تركيبات وطرق لخفض تعبير البروتين tau
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau

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