[go: up one dir, main page]

ATE485394T1 - Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai - Google Patents

Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai

Info

Publication number
ATE485394T1
ATE485394T1 AT04753972T AT04753972T ATE485394T1 AT E485394 T1 ATE485394 T1 AT E485394T1 AT 04753972 T AT04753972 T AT 04753972T AT 04753972 T AT04753972 T AT 04753972T AT E485394 T1 ATE485394 T1 AT E485394T1
Authority
AT
Austria
Prior art keywords
sirna duplex
antisense strand
fnai
specificity
effectiveness
Prior art date
Application number
AT04753972T
Other languages
English (en)
Inventor
Phillip Zamore
Gyoergy Hutvagner
Dianne Schwarz
Martin Simard
Original Assignee
Univ Massachusetts
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=33556382&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ATE485394(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Univ Massachusetts filed Critical Univ Massachusetts
Application granted granted Critical
Publication of ATE485394T1 publication Critical patent/ATE485394T1/de

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/331—Universal or degenerate base
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Electrotherapy Devices (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AT04753972T 2003-06-02 2004-06-02 Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai ATE485394T1 (de)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US47533103P 2003-06-02 2003-06-02
US50792803P 2003-09-30 2003-09-30
US57526804P 2004-05-28 2004-05-28
PCT/US2004/017256 WO2005001043A2 (en) 2003-06-02 2004-06-02 METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF FNAi

Publications (1)

Publication Number Publication Date
ATE485394T1 true ATE485394T1 (de) 2010-11-15

Family

ID=33556382

Family Applications (1)

Application Number Title Priority Date Filing Date
AT04753972T ATE485394T1 (de) 2003-06-02 2004-06-02 Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai

Country Status (8)

Country Link
US (1) US8309704B2 (de)
EP (2) EP2314692B1 (de)
AT (1) ATE485394T1 (de)
AU (2) AU2004252442B2 (de)
CA (1) CA2527958C (de)
DE (1) DE602004029678D1 (de)
ES (2) ES2357116T5 (de)
WO (1) WO2005001043A2 (de)

Families Citing this family (126)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
AU2003265978A1 (en) 2002-05-03 2003-11-17 Duke University A method of regulating gene expression
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
US20090227780A1 (en) * 2002-11-14 2009-09-10 Dharmacon, Inc. siRNA targeting connexin 43
US20080268457A1 (en) * 2002-11-14 2008-10-30 Dharmacon, Inc. siRNA targeting forkhead box P3 (FOXP3)
WO2006006948A2 (en) * 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US7977471B2 (en) * 2002-11-14 2011-07-12 Dharmacon, Inc. siRNA targeting TNFα
US10011836B2 (en) 2002-11-14 2018-07-03 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US7612196B2 (en) 2002-11-14 2009-11-03 Dharmacon, Inc. siRNA targeting cyclin-dependent kinase inhibitor 1B (p27, Kip1) (CDKN1B)
US7592442B2 (en) * 2002-11-14 2009-09-22 Dharmacon, Inc. siRNA targeting ribonucleotide reductase M2 polypeptide (RRM2 or RNR-R2)
US9839649B2 (en) 2002-11-14 2017-12-12 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US8198427B1 (en) 2002-11-14 2012-06-12 Dharmacon, Inc. SiRNA targeting catenin, beta-1 (CTNNB1)
US7635770B2 (en) 2002-11-14 2009-12-22 Dharmacon, Inc. siRNA targeting protein kinase N-3 (PKN-3)
US7781575B2 (en) 2002-11-14 2010-08-24 Dharmacon, Inc. siRNA targeting tumor protein 53 (p53)
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US7691998B2 (en) * 2002-11-14 2010-04-06 Dharmacon, Inc. siRNA targeting nucleoporin 62kDa (Nup62)
US20100113307A1 (en) * 2002-11-14 2010-05-06 Dharmacon, Inc. siRNA targeting vascular endothelial growth factor (VEGF)
US9771586B2 (en) 2002-11-14 2017-09-26 Thermo Fisher Scientific Inc. RNAi targeting ZNF205
US9879266B2 (en) 2002-11-14 2018-01-30 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US9719092B2 (en) 2002-11-14 2017-08-01 Thermo Fisher Scientific Inc. RNAi targeting CNTD2
US7619081B2 (en) * 2002-11-14 2009-11-17 Dharmacon, Inc. siRNA targeting coatomer protein complex, subunit beta 2 (COPB2)
US9719094B2 (en) 2002-11-14 2017-08-01 Thermo Fisher Scientific Inc. RNAi targeting SEC61G
PT2284266E (pt) * 2002-11-14 2013-12-17 Thermo Fisher Scient Biosciences Inc Siarn contra tp53
US7951935B2 (en) 2002-11-14 2011-05-31 Dharmacon, Inc. siRNA targeting v-myc myelocytomatosis viral oncogene homolog (MYC)
US20040191905A1 (en) * 2002-11-22 2004-09-30 University Of Massachusetts Modulation of HIV replication by RNA interference
EP2239329A1 (de) 2003-03-07 2010-10-13 Alnylam Pharmaceuticals, Inc. Therapeutische Zusammensetzungen
DK1633767T3 (en) 2003-06-02 2019-03-25 Univ Massachusetts METHODS AND COMPOSITIONS FOR MANAGING THE EFFECT OF RNA SILENCING
ATE485394T1 (de) 2003-06-02 2010-11-15 Univ Massachusetts Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
US20070117767A1 (en) * 2003-08-18 2007-05-24 Japan Health Sciences Foundation siRNA Molecules and Method of Suppressing Gene Expression With the Use of the Same
EP2821085B1 (de) 2003-09-12 2020-04-29 University of Massachusetts Rna-interferenz zur behandlung von funktionszunahmestörungen
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
WO2005035769A2 (en) 2003-10-09 2005-04-21 E. I. Du Pont De Nemours And Company Gene silencing by using micro-rna molecules
EP1793674B1 (de) 2003-11-26 2018-05-30 University of Massachusetts Sequenzspezifische hemmung der funktion von kleiner rna
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
WO2005079532A2 (en) * 2004-02-17 2005-09-01 University Of Massachusetts Methods and compositions for enhancing risc activity in vitro and in vivo
US20060069050A1 (en) * 2004-02-17 2006-03-30 University Of Massachusetts Methods and compositions for mediating gene silencing
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
US7605250B2 (en) * 2004-05-12 2009-10-20 Dharmacon, Inc. siRNA targeting cAMP-specific phosphodiesterase 4D
EP2290066B1 (de) 2004-05-28 2015-03-18 Asuragen, Inc. Verfahren und Zusammensetzungen mit MicroRNA
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
EP1765415A4 (de) * 2004-06-03 2010-03-24 Isis Pharmaceuticals Inc Oligomere verbindungen zur erleichterung der risc-beladung
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
CA2583690C (en) * 2004-10-12 2016-04-05 The Rockefeller University Microrna constructs for the suppression of the expression of targeted genes or the down regulation of targeted genes and methods therefore
ES2534303T3 (es) 2004-11-12 2015-04-21 Asuragen, Inc. Procedimientos y composiciones que implican miARN y moléculas inhibidoras de miARN
US20100069620A1 (en) * 2004-12-02 2010-03-18 Rxi Pharmaceuticals Corp. Novel compositions of chemically modified small interfering rna
US20070105803A1 (en) 2005-08-18 2007-05-10 Muthiah Manoharan Methods and compositions for treating neurological disease
US20090018097A1 (en) * 2005-09-02 2009-01-15 Mdrna, Inc Modification of double-stranded ribonucleic acid molecules
FR2890859B1 (fr) * 2005-09-21 2012-12-21 Oreal Oligonucleotide d'arn double brin inhibant l'expression de la tyrosinase
US7951784B2 (en) * 2006-01-26 2011-05-31 University Of Massachusetts RNA interference agents for therapeutic use
WO2008104978A2 (en) * 2007-02-28 2008-09-04 Quark Pharmaceuticals, Inc. Novel sirna structures
WO2008143774A2 (en) * 2007-05-01 2008-11-27 University Of Massachusetts Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy
WO2008143786A1 (en) * 2007-05-14 2008-11-27 The Rockefeller University Production of artificial micrornas using synthetic microrna precursors
US8361714B2 (en) 2007-09-14 2013-01-29 Asuragen, Inc. Micrornas differentially expressed in cervical cancer and uses thereof
WO2009039300A2 (en) * 2007-09-18 2009-03-26 Intradigm Corporation Compositions comprising hif-1 alpha sirna and methods of use thereof
JP2010538678A (ja) * 2007-09-18 2010-12-16 イントラドイグム コーポレーション Ｋ−ｒａｓｓｉＲＮＡ含有組成物及びそれらの使用法
WO2009039173A2 (en) 2007-09-19 2009-03-26 Applied Biosystems Inc. SiRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAi, AND STABILIZED FORMS THEREOF
US8614309B2 (en) * 2007-10-03 2013-12-24 Quark Pharmaceuticals, Inc. Double-stranded RNA directed to CASP2 and methods of use thereof
WO2009070805A2 (en) 2007-12-01 2009-06-04 Asuragen, Inc. Mir-124 regulated genes and pathways as targets for therapeutic intervention
WO2009074990A2 (en) * 2007-12-12 2009-06-18 Quark Pharmaceuticals, Inc. Rtp801l 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
EP2242854A4 (de) * 2008-01-15 2012-08-15 Quark Pharmaceuticals Inc Sirna-verbindungen und verfahren zur verwendung davon
EP2247729B1 (de) 2008-02-11 2019-05-01 Phio Pharmaceuticals Corp. Modifizierte rnai-polynukleotide und verwendungen davon
MX2010010303A (es) * 2008-03-20 2010-10-20 Quark Pharmaceuticals Inc Nuevos compuestos de acido ribonucleico de pequeña interferencia para inhibir rtp801.
US8278287B2 (en) * 2008-04-15 2012-10-02 Quark Pharmaceuticals Inc. siRNA compounds for inhibiting NRF2
EP2285960B1 (de) 2008-05-08 2015-07-08 Asuragen, INC. Zusammensetzungen und verfahren in zusammenhang mit der mirna-184modulation von neovaskularisation oder angiogenese
JP5524189B2 (ja) * 2008-06-06 2014-06-18 クォーク ファーマシューティカルズ インコーポレーティッド 耳障害治療のための組成物および方法
WO2010008582A2 (en) 2008-07-18 2010-01-21 Rxi Pharmaceuticals Corporation Phagocytic cell drug delivery system
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
CA2743981C (en) 2008-09-22 2019-01-29 Rxi Pharmaceuticals Corporation Reduced size self-delivering rnai compounds
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
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
US9799416B2 (en) * 2009-11-06 2017-10-24 Terrapower, Llc Methods and systems for migrating fuel assemblies in a nuclear fission reactor
WO2011057171A1 (en) 2009-11-08 2011-05-12 Quark Pharmaceuticals, Inc. METHODS FOR DELIVERY OF siRNA TO THE SPINAL CORD AND THERAPIES ARISING THEREFROM
WO2011066475A1 (en) 2009-11-26 2011-06-03 Quark Pharmaceuticals, Inc. Sirna compounds comprising terminal substitutions
TWI611021B (zh) 2009-12-09 2018-01-11 日東電工股份有限公司 Ｈｓｐ４７表現之調節
WO2011084193A1 (en) 2010-01-07 2011-07-14 Quark Pharmaceuticals, Inc. Oligonucleotide compounds comprising non-nucleotide overhangs
US20130018086A1 (en) * 2010-03-16 2013-01-17 Massachusette Institute Of Technology Sirnas targeting exon 10 of pyruvate kinase m2
EP2550000A4 (de) 2010-03-24 2014-03-26 Advirna Inc Selbstfreisetzende rnai-verbindungen von reduzierter grösse
US9340786B2 (en) 2010-03-24 2016-05-17 Rxi Pharmaceuticals Corporation RNA interference in dermal and fibrotic indications
EP2550001B1 (de) 2010-03-24 2019-05-22 Phio Pharmaceuticals Corp. Rna-interferenz bei augenerkrankungen
KR101553753B1 (ko) * 2010-06-24 2015-09-16 쿠아크 파마수티칼스 인코퍼레이티드 Ｒｈｏａ에 대한 이중 가닥 ｒｎａ 및 그의 용도
SG190412A1 (en) 2010-12-06 2013-06-28 Quark Pharmaceuticals Inc Double stranded oligonucleotide compounds comprising threose modifications
WO2013040251A2 (en) 2011-09-13 2013-03-21 Asurgen, Inc. Methods and compositions involving mir-135b for distinguishing pancreatic cancer from benign pancreatic disease
US9777277B2 (en) 2013-05-31 2017-10-03 Brandeis University Organic small hairpin RNAs
WO2015015496A1 (en) 2013-07-31 2015-02-05 Qbi Enterprises Ltd. Sphingolipid-polyalkylamine-oligonucleotide compounds
WO2015015498A1 (en) 2013-07-31 2015-02-05 Qbi Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
RU2744194C2 (ru) 2013-12-02 2021-03-03 Фио Фармасьютикалс Корп Иммунотерапия рака
EP3137119B1 (de) 2014-04-28 2020-07-01 Phio Pharmaceuticals Corp. Verfahren zur behandlung von krebs mithilfe von einer nukleinsaüre gegen mdm2
KR101670254B1 (ko) * 2014-07-09 2016-10-28 서울대학교산학협력단 목적 유전자의 발현 억제 특이성이 증가된 siRNA 분자
CN107073294A (zh) 2014-09-05 2017-08-18 阿克赛医药公司 使用靶向tyr或mmp1的核酸治疗老化和皮肤病症的方法
JP2017535266A (ja) 2014-11-14 2017-11-30 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法
EP3277815B1 (de) 2015-04-03 2021-09-22 University of Massachusetts Oligonukleotidverbindungen zur behandlung von präeklampsie und anderen angiogenen erkrankungen
CA2980337A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mrna
WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
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
KR20180026739A (ko) 2015-07-06 2018-03-13 알엑스아이 파마슈티칼스 코포레이션 슈퍼옥시드 디스뮤타제 1 (sod1)을 표적화하는 핵산 분자
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP2018531037A (ja) 2015-10-19 2018-10-25 アールエックスアイ ファーマシューティカルズ コーポレーション 長い非コードｒｎａを標的とする減少したサイズの自己送達型核酸化合物
AU2017210726B2 (en) 2016-01-31 2023-08-03 University Of Massachusetts Branched oligonucleotides
MA45469A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de bêta-caténine et leurs utilisations
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45470A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques kras et leurs utilisations
MA45349A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques egfr et leurs utilisations
EP3458589A4 (de) * 2016-05-18 2020-01-01 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von morbus huntington
WO2018031933A2 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
US10443055B2 (en) 2016-12-22 2019-10-15 Beth Israel Deaconess Medical Center Compounds that target MYC microRNA responsive elements for the treatment of MYC-associated cancer
EP3618839A4 (de) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von amyotropher lateralsklerose (als)
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
JP7502991B2 (ja) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症（ａｌｓ）の治療
CN118638788A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
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
US11492619B2 (en) 2019-01-18 2022-11-08 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
EP3966328A4 (de) 2019-05-06 2023-10-18 University Of Massachusetts Anti-c9orf72-oligonukleotide und zugehörige verfahren
AU2020329155A1 (en) 2019-08-09 2022-03-10 University Of Massachusetts Chemically modified oligonucleotides targeting SNPs
JP2023505737A (ja) * 2019-12-11 2023-02-10 アベリノ ラボ ユーエスエー インコーポレイテッド Ｒ１２４ｈ突然変異を備えたトランスフォーミング増殖因子ベータ誘導遺伝子の短鎖干渉ｒｎａを使用したアレル特異的サイレンシング
EP4081302A4 (de) 2019-12-23 2025-01-29 Univ Massachusetts Oligonukleotide zur gewebespezifischen genexpressionsmodulation
MX2022011499A (es) 2020-03-19 2022-10-07 Avidity Biosciences Inc Composiciones y metodos para tratar la distrofia muscular facioescapulohumeral.
AU2021244600A1 (en) 2020-03-26 2022-11-03 University Of Massachusetts Synthesis of modified oligonucleotides with increased stability
CN115698291A (zh) 2020-05-28 2023-02-03 马萨诸塞大学 用于SARS-CoV-2调节的寡核苷酸
WO2022271786A1 (en) 2021-06-23 2022-12-29 University Of Massachusetts Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders

Family Cites Families (71)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3023787A1 (de) 1980-06-25 1982-01-21 Studiengesellschaft Kohle mbH, 4330 Mülheim Verfahren zur erhoehung der inkorporation und der expression von genetischem material in die kerne von intakten zellen mit hilfe von liposomen
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
US4980286A (en) 1985-07-05 1990-12-25 Whitehead Institute For Biomedical Research In vivo introduction and expression of foreign genetic material in epithelial cells
AU6131086A (en) 1985-07-05 1987-01-30 Whitehead Institute For Biomedical Research Epithelial cells expressing foreign genetic material
JP2627899B2 (ja) 1987-08-19 1997-07-09 株式会社 ビタミン研究所 遺伝子封入リポソームの製法
US5175385A (en) 1987-09-03 1992-12-29 Ohio University/Edison Animal Biotechnolgy Center Virus-resistant transgenic mice
US5252726A (en) 1987-09-04 1993-10-12 Novo Nordisk A/S Promoters for use in aspergillus
JP3015383B2 (ja) 1987-09-11 2000-03-06 ホワイトヘツド・インスチチユート・フオー・バイオメデイカル・リサーチ 形質導入した線維芽およびそれらの使用
EP0391960B1 (de) 1987-12-11 1994-08-17 Whitehead Institute For Biomedical Research Genetische modifizierung von endothelialen zellen
DE68927996T2 (de) 1988-02-05 1997-12-04 Hughes Howard Med Inst Modifizierte hepatozyten und deren anwendung
US5070020A (en) 1988-05-09 1991-12-03 Eli Lilly And Company Recombinant dna expression vectors and dna compounds that encode deacetoxycephalosporin c synthetase
US5223409A (en) 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
US5175384A (en) 1988-12-05 1992-12-29 Genpharm International Transgenic mice depleted in mature t-cells and methods for making transgenic mice
US5175383A (en) 1989-02-17 1992-12-29 President And Fellows Of Harvard College Animal model for benign prostatic disease
JP3056782B2 (ja) 1989-11-03 2000-06-26 ヴァンダービルト ユニバーシティ 標的器官内での遺伝子の発現用医薬組成物
US6946587B1 (en) 1990-01-22 2005-09-20 Dekalb Genetics Corporation Method for preparing fertile transgenic corn plants
US5204253A (en) 1990-05-29 1993-04-20 E. I. Du Pont De Nemours And Company Method and apparatus for introducing biological substances into living cells
AU656544B2 (en) 1990-10-31 1995-02-09 Brigham And Women's Hospital Genetic modification of endothelial cells
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
GB9517779D0 (en) 1995-08-31 1995-11-01 Roslin Inst Edinburgh Biological manipulation
GB9517780D0 (en) 1995-08-31 1995-11-01 Roslin Inst Edinburgh Biological manipulation
US5684143A (en) 1996-02-21 1997-11-04 Lynx Therapeutics, Inc. Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US20040002153A1 (en) 1999-07-21 2004-01-01 Monia Brett P. Modulation of PTEN expression via oligomeric compounds
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
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
WO2002081628A2 (en) 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
EP2028278B1 (de) 2000-03-30 2014-03-19 Whitehead Institute For Biomedical Research RNA-sequenzspezifische Mediatoren von RNA-Interferenz
US20020173478A1 (en) 2000-11-14 2002-11-21 The Trustees Of The University Of Pennsylvania Post-transcriptional gene silencing by RNAi in mammalian cells
ATE373724T2 (de) 2000-12-01 2007-10-15 Max Planck Gesellschaft Kleine rns moleküle, die rns-interferenz vermitteln
WO2003035869A1 (de) 2001-10-26 2003-05-01 Ribopharma Ag Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
WO2003006477A1 (en) 2001-07-12 2003-01-23 University Of Massachusetts IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING
US20030198627A1 (en) 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
EP2224003B1 (de) 2001-09-28 2018-04-18 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MikroRNA-Moleküle
EP1572902B1 (de) 2002-02-01 2014-06-11 Life Technologies Corporation HOCHWIRKSAME siRNAS ZUR REDUZIERUNG DER EXPRESSION VON ZIELGENEN
ES2312753T5 (es) 2002-02-14 2012-12-13 City Of Hope Procedimientos para producir moléculas de ARN de interferencia en células de mamífero y usos terapéuticos para tales moléculas
AU2003265978A1 (en) 2002-05-03 2003-11-17 Duke University A method of regulating gene expression
WO2004007718A2 (en) 2002-07-10 2004-01-22 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Rna-interference by single-stranded rna molecules
DK3222724T3 (en) 2002-08-05 2018-12-03 Silence Therapeutics Gmbh ADDITIONALLY UNKNOWN FORMS OF INTERFERRING RNA MOLECULES
ES2389024T3 (es) 2002-08-05 2012-10-22 Silence Therapeutics Aktiengesellschaft Moléculas de RNA interferentes de extremos romos
AU2003261449A1 (en) 2002-08-07 2004-02-25 Compositions for rna interference and methods of use thereof
AU2003273336A1 (en) 2002-09-18 2004-04-08 Isis Pharmaceuticals, Inc. Efficient reduction of target rna's by single- and double-stranded oligomeric compounds
ATE513843T1 (de) 2002-09-25 2011-07-15 Univ Massachusetts Abstellen von genen in vivo durch chemischmodifizierte und stabile sirna
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
CA2504915A1 (en) 2002-11-04 2004-05-21 University Of Massachusetts Allele-specific rna interference
PT2284266E (pt) 2002-11-14 2013-12-17 Thermo Fisher Scient Biosciences Inc Siarn contra tp53
US20040214198A1 (en) 2002-11-15 2004-10-28 University Of Massachusetts Allele-targeted RNA interference
US20040248299A1 (en) 2002-12-27 2004-12-09 Sumedha Jayasena RNA interference
US20040224328A1 (en) 2003-01-15 2004-11-11 Hans Prydz siRNA screening method
WO2004063375A1 (en) 2003-01-15 2004-07-29 Hans Prydz OPTIMIZING siRNA BY RNAi ANTISENSE
WO2004064737A2 (en) 2003-01-17 2004-08-05 Alnylam Pharmaceuticals Therapeutics compositions
WO2004065613A2 (en) 2003-01-17 2004-08-05 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Inducible small interfering rna (sirna) expression constructs for targeted gene silencing
US20070104688A1 (en) 2003-02-13 2007-05-10 City Of Hope Small interfering RNA mediated transcriptional gene silencing in mammalian cells
EP2239329A1 (de) 2003-03-07 2010-10-13 Alnylam Pharmaceuticals, Inc. Therapeutische Zusammensetzungen
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
ATE485394T1 (de) 2003-06-02 2010-11-15 Univ Massachusetts Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai
DK1633767T3 (en) 2003-06-02 2019-03-25 Univ Massachusetts METHODS AND COMPOSITIONS FOR MANAGING THE EFFECT OF RNA SILENCING
EP1793674B1 (de) 2003-11-26 2018-05-30 University of Massachusetts Sequenzspezifische hemmung der funktion von kleiner rna
WO2005062937A2 (en) 2003-12-22 2005-07-14 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna
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
WO2005069987A2 (en) 2004-01-23 2005-08-04 City Of Hope Amplifying interfering rna (rnai) expression and effects
WO2005078096A2 (en) 2004-02-09 2005-08-25 University Of Massachusetts Dual functional oligonucleotides for use in repressing mutant gene expression
US20050182005A1 (en) 2004-02-13 2005-08-18 Tuschl Thomas H. Anti-microRNA oligonucleotide molecules
WO2005079532A2 (en) 2004-02-17 2005-09-01 University Of Massachusetts Methods and compositions for enhancing risc activity in vitro and in vivo
US20060069050A1 (en) 2004-02-17 2006-03-30 University Of Massachusetts Methods and compositions for mediating gene silencing
JP5243789B2 (ja) 2004-03-15 2013-07-24 シティ・オブ・ホープ 二本鎖ｒｎａによる遺伝子発現の特異的阻害のための方法及び組成物
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
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Ｒｎａ 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
US20060178334A1 (en) 2005-02-04 2006-08-10 City Of Hope Double-stranded and single-stranded RNA molecules with 5 ' triphosphates and their use for inducing interferon

Also Published As

Publication number Publication date
EP1633890B1 (de) 2010-10-20
DE602004029678D1 (de) 2010-12-02
AU2010202861A1 (en) 2010-08-05
WO2005001043A2 (en) 2005-01-06
EP2314692B1 (de) 2021-02-24
ES2357116T3 (es) 2011-04-18
AU2010202861B2 (en) 2013-05-23
CA2527958C (en) 2014-04-08
EP2314692A2 (de) 2011-04-27
AU2004252442A1 (en) 2005-01-06
CA2527958A1 (en) 2005-01-06
AU2004252442B2 (en) 2010-04-08
ES2357116T5 (es) 2021-09-03
US8309704B2 (en) 2012-11-13
EP1633890A4 (de) 2006-11-29
EP1633890B2 (de) 2020-11-18
EP2314692A3 (de) 2011-08-03
WO2005001043A3 (en) 2005-12-29
EP1633890A2 (de) 2006-03-15
US20050181382A1 (en) 2005-08-18
ES2864206T3 (es) 2021-10-13

Similar Documents

Publication Publication Date Title
ATE485394T1 (de) Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai
EP1325019A4 (de) Antisense-modulation der clusterinexpression
ATE550025T1 (de) Behandlung neurodegenerativer erkrankungen mittels intrakranialer freisetzung von small- interfering-rna (sirna)
EP1392867A4 (de) Antisense-modulation der ptp18-expression
DE60114680D1 (de) Oberflächenbehandlungsmittel ohne Chromat, Verfahren zur Oberflächenbehandlung und behandeltes Stahl
EP1248791A4 (de) Antisens-modulierung der caspase 3 expression
EP1569695A4 (de) Antisense-modulationder apolipoprotein-b-expression
ATE508188T1 (de) Oligonukleotid-zusammensetzungen mit verbesserter wirksamkeit
ATE513843T1 (de) Abstellen von genen in vivo durch chemischmodifizierte und stabile sirna
EP1419168A4 (de) Antisense-modulation der expression von apolipoprotein b
EP1250455A4 (de) Antisense inhibierung der ptp1b expression.
HRP20130750T1 (hr) Postupak identifikacije molekula za vezanje koje mogu neutralizirati virus bjesnoä†e
EP1417351A4 (de) Antisense-modulation der expression von apolipoprotein(a)
DE69533697D1 (de) ANTISENSE OLIGONUKLEOTIDMODULATION DER raf GENEXPRESSION
EP1250157A4 (de) Antisens-modulation der expression der induzierbaren stickstoffmonoxid-synthase
WO2005049832A3 (en) FcϜRIIA-SPECIFIC NUCLEIC ACID INTERFERENCE
WO2003046132A3 (en) Antisense modulation of myd88 expression
WO2003057843A8 (en) Methods and materials for modulating trpc4
EP1648910A4 (de) Antisense-modulation der expression von stearoyl coa desaturase
EP1436308A4 (de) Antisense-modulierung der il-1-rezeptor-assoziierten kinase-4-expression
WO2003012031A3 (en) Antisense modulation of stearoyl-coa desaturase expression
ATE361755T1 (de) Verwendung von thrombomodulinanaloga zur regenerierung von rückenmarkverletzungen
DE60235867D1 (de) Verwendung von speziellen Aminosilikonen zur Vor- oder Nachbehandlung in der Entfärbung von Keratinfasern
WO2003057847A8 (en) METHODS AND MATERIALS FOR MODULATING ENaC-BETA
DE50109972D1 (de) Verfahren zur herstellung von hydrolysestabilen ammoniumnitrilen

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties