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=***_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

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/111Antisense spanning the whole gene, or a large part of it
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/331Universal or degenerate base
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity

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  • 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) ES2864206T3 (de)
WO (1) WO2005001043A2 (de)

Families Citing this family (122)

* 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
CA2524569C (en) 2002-05-03 2013-10-22 Duke University A method of regulating gene expression
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
EP1578765A4 (de) 2002-11-05 2008-04-23 Isis Pharmaceuticals Inc Oligomere verbindungen,die zuckerersatzstoffe enthalten, und zusammensetzung zur verwendung in dergenmodulation
US20080268457A1 (en) * 2002-11-14 2008-10-30 Dharmacon, Inc. siRNA targeting forkhead box P3 (FOXP3)
US9879266B2 (en) 2002-11-14 2018-01-30 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US7781575B2 (en) 2002-11-14 2010-08-24 Dharmacon, Inc. siRNA targeting tumor protein 53 (p53)
US9839649B2 (en) 2002-11-14 2017-12-12 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)
US9719094B2 (en) 2002-11-14 2017-08-01 Thermo Fisher Scientific Inc. RNAi targeting SEC61G
US7977471B2 (en) * 2002-11-14 2011-07-12 Dharmacon, Inc. siRNA targeting TNFα
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US8090542B2 (en) * 2002-11-14 2012-01-03 Dharmacon Inc. Functional and hyperfunctional siRNA
US9771586B2 (en) 2002-11-14 2017-09-26 Thermo Fisher Scientific Inc. RNAi targeting ZNF205
US20100113307A1 (en) * 2002-11-14 2010-05-06 Dharmacon, Inc. siRNA targeting vascular endothelial growth factor (VEGF)
WO2006006948A2 (en) * 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US7619081B2 (en) * 2002-11-14 2009-11-17 Dharmacon, Inc. siRNA targeting coatomer protein complex, subunit beta 2 (COPB2)
US8198427B1 (en) 2002-11-14 2012-06-12 Dharmacon, Inc. SiRNA targeting catenin, beta-1 (CTNNB1)
US7592442B2 (en) 2002-11-14 2009-09-22 Dharmacon, Inc. siRNA targeting ribonucleotide reductase M2 polypeptide (RRM2 or RNR-R2)
US7691998B2 (en) 2002-11-14 2010-04-06 Dharmacon, Inc. siRNA targeting nucleoporin 62kDa (Nup62)
US7951935B2 (en) 2002-11-14 2011-05-31 Dharmacon, Inc. siRNA targeting v-myc myelocytomatosis viral oncogene homolog (MYC)
US7635770B2 (en) * 2002-11-14 2009-12-22 Dharmacon, Inc. siRNA targeting protein kinase N-3 (PKN-3)
US20090227780A1 (en) * 2002-11-14 2009-09-10 Dharmacon, Inc. siRNA targeting connexin 43
US9719092B2 (en) 2002-11-14 2017-08-01 Thermo Fisher Scientific Inc. RNAi targeting CNTD2
US10011836B2 (en) 2002-11-14 2018-07-03 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
AU2003298718A1 (en) * 2002-11-22 2004-06-18 University Of Massachusetts Modulation of hiv replication by rna interference
US8110674B2 (en) 2003-03-07 2012-02-07 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
DE602004029678D1 (de) 2003-06-02 2010-12-02 Univ Massachusetts Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai
AU2004248136B2 (en) * 2003-06-02 2011-09-15 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
EP1666593A4 (de) * 2003-08-18 2009-07-15 Japan Health Science Found Verbessertes sirna-molekül und verfahren zur hemmung der genexpression unter verwendung davon
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
ES2485848T3 (es) 2003-09-12 2014-08-14 University Of Massachusetts ARN de interferencia para el tratamiento de trastornos relacionados con la ganancia de función
WO2005052170A2 (en) 2003-10-09 2005-06-09 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
WO2005079533A2 (en) * 2004-02-17 2005-09-01 University Of Massachusetts Methods and compositions for mediating gene silencing
US20050273868A1 (en) * 2004-02-17 2005-12-08 University Of Massachusetts Methods and compositions for enhancing RISC activity in vitro and in vivo
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
EP2290070B1 (de) 2004-05-28 2015-03-25 Asuragen, Inc. Verfahren und Zusammensetzungen mit MicroRNA
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
WO2005121372A2 (en) * 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
BRPI0516874A (pt) * 2004-10-12 2008-09-23 Univ Rockefeller micrornas
EP2322616A1 (de) * 2004-11-12 2011-05-18 Asuragen, Inc. Verfahren und Zusammensetzungen, die miRNAs und miRNA-inhibitorischen Molekülen verbunden sind
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
EP1986699A4 (de) * 2006-01-26 2010-12-15 Univ Massachusetts Rna-interferenz-wirkstoffe zur therapeutischen verwendung
US20100292301A1 (en) * 2007-02-28 2010-11-18 Elena Feinstein 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
US20100286241A1 (en) * 2007-09-18 2010-11-11 Intradigm Corporation Compositions comprising k-ras sirna and methods of use
US20100280097A1 (en) * 2007-09-18 2010-11-04 Intradigm Corporation Compositions comprising hif-1 alpha sirna and methods of use thereof
DK2548962T3 (en) 2007-09-19 2016-04-11 Applied Biosystems Llc Sirna sequence-independent modification formats to reduce off-target phenotype effects in RNAI and stabilized forms thereof
WO2009044392A2 (en) * 2007-10-03 2009-04-09 Quark Pharmaceuticals, Inc. Novel sirna structures
WO2009070805A2 (en) 2007-12-01 2009-06-04 Asuragen, Inc. Mir-124 regulated genes and pathways as targets for therapeutic intervention
US8614311B2 (en) 2007-12-12 2013-12-24 Quark Pharmaceuticals, Inc. RTP801L siRNA compounds and methods of use thereof
US20110105584A1 (en) * 2007-12-12 2011-05-05 Elena Feinstein Rtp80il sirna compounds and methods of use thereof
WO2009090639A2 (en) * 2008-01-15 2009-07-23 Quark Pharmaceuticals, Inc. Sirna compounds and methods of use thereof
CN104975020B (zh) 2008-02-11 2020-01-17 菲奥医药公司 经修饰的RNAi多核苷酸及其用途
AU2009227549A1 (en) * 2008-03-20 2009-09-24 Quark Pharmaceuticals, Inc. Novel siRNA compounds for inhibiting RTP801
EP2285385A4 (de) * 2008-04-15 2013-01-16 Quark Pharmaceuticals Inc siRNA-VERBINDUNGEN ZUR NRF2-HEMMUNG
EP2990487A1 (de) 2008-05-08 2016-03-02 Asuragen, INC. Zusammensetzungen und verfahren in zusammenhang mit der mirna-modulation von neovaskularisation oder angiogenese
WO2009147684A2 (en) 2008-06-06 2009-12-10 Quark Pharmaceuticals, Inc. Compositions and methods for treatment of ear disorders
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
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
CN108165548B (zh) 2008-09-22 2022-10-14 菲奥医药公司 减小大小的自递送RNAi化合物
US9074211B2 (en) 2008-11-19 2015-07-07 Rxi Pharmaceuticals Corporation Inhibition of MAP4K4 through RNAI
WO2010078536A1 (en) 2009-01-05 2010-07-08 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
US9799416B2 (en) * 2009-11-06 2017-10-24 Terrapower, Llc Methods and systems for migrating fuel assemblies in a nuclear fission reactor
US8901097B2 (en) 2009-11-08 2014-12-02 Quark Pharmaceuticals, Inc. Methods for delivery of siRNA to the spinal cord and therapies arising therefrom
ES2759003T3 (es) 2009-11-26 2020-05-07 Quark Pharmaceuticals Inc Compuestos de ARNip que comprenden sustituciones terminales
CN102741410B (zh) 2009-12-09 2016-11-16 日东电工株式会社 Hsp47表达的调节
WO2011084193A1 (en) 2010-01-07 2011-07-14 Quark Pharmaceuticals, Inc. Oligonucleotide compounds comprising non-nucleotide overhangs
WO2011115810A2 (en) * 2010-03-16 2011-09-22 Massachusetts Institute Of Technology Isoform-specific rnai based on codon redundancy/degeneracy
CN110042099A (zh) 2010-03-24 2019-07-23 菲奥医药公司 皮肤与纤维化症候中的rna干扰
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
WO2011119871A1 (en) 2010-03-24 2011-09-29 Rxi Phrmaceuticals Corporation Rna interference in ocular indications
KR101553753B1 (ko) * 2010-06-24 2015-09-16 쿠아크 파마수티칼스 인코퍼레이티드 Rhoa에 대한 이중 가닥 rna 및 그의 용도
CN103298939A (zh) 2010-12-06 2013-09-11 夸克医药公司 包含位置修饰的双链寡核苷酸化合物
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
WO2014193489A1 (en) * 2013-05-31 2014-12-04 Brandeis University Organic small hairpin rnas
EP3027222A1 (de) 2013-07-31 2016-06-08 QBI Enterprises Ltd. Sphingolipid-polyalkylamin-oligonukleotidverbindungen
WO2015015498A1 (en) 2013-07-31 2015-02-05 Qbi Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
EP3079707A4 (de) 2013-12-02 2017-10-18 RXi Pharmaceuticals Corporation Immuntherapie für krebs
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
EP3188799B1 (de) 2014-09-05 2022-07-06 Phio Pharmaceuticals Corp. Verfahren zur behandlung von alterung und hauterkrankungen mit auf tyr oder mmp1 gerichteten nukleinsäuren
SG11201703281RA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
US9809817B2 (en) 2015-04-03 2017-11-07 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mRNA
US9862952B2 (en) 2015-04-03 2018-01-09 University Of Massachusetts Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
CA2991598A1 (en) 2015-07-06 2017-01-12 Rxi Pharmaceuticals Corporation Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
WO2017007825A1 (en) 2015-07-06 2017-01-12 Rxi Pharmaceuticals Corporation Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
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
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
MA45469A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de bêta-caténine et leurs utilisations
MA45349A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques egfr et leurs utilisations
MA45470A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques kras et leurs utilisations
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
CA3024449A1 (en) * 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
CA3033368A1 (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
CN110913866A (zh) 2017-05-05 2020-03-24 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
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
CN111479924B (zh) 2017-10-16 2024-06-14 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
KR102443358B1 (ko) 2017-12-06 2022-09-14 어비디티 바이오사이언시스 인크. 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법
EP3833763A4 (de) 2018-08-10 2023-07-19 University of Massachusetts Modifizierte oligonukleotide zum targeting von snps
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
EP3966328A4 (de) 2019-05-06 2023-10-18 University Of Massachusetts Anti-c9orf72-oligonukleotide und zugehörige verfahren
CN114502730A (zh) 2019-08-09 2022-05-13 马萨诸塞大学 经化学修饰的靶向snp的寡核苷酸
US12037585B2 (en) 2019-12-23 2024-07-16 University Of Massachusetts Oligonucleotides for tissue specific gene expression modulation
US11555190B2 (en) 2020-03-19 2023-01-17 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
MX2023015523A (es) 2021-06-23 2024-03-11 Univ Massachusetts Compuestos de oligonucleotidos anti-flt1 optimizados para el tratamiento de la preeclampsia y otros desordenes angiogenicos.

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
EP0228458B2 (de) 1985-07-05 1997-10-22 Whitehead Institute For Biomedical Research Expression von fremdem genetischem material in epithelzellen
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
EP0633318A1 (de) 1987-09-11 1995-01-11 Whitehead Institute For Biomedical Research Transduktionsveränderte Fibroblasten und ihre Anwendung
ATE110108T1 (de) 1987-12-11 1994-09-15 Whitehead Biomedical Inst Genetische modifizierung von endothelialen zellen.
WO1989007136A2 (en) 1988-02-05 1989-08-10 Whitehead Institute For Biomedical Research Modified hepatocytes and uses therefor
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
AU625013B2 (en) 1989-11-03 1992-06-25 Vanderbilt University Method of in vivo delivery of functioning foreign genes
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
JP3418982B2 (ja) 1990-10-31 2003-06-23 ソマティクス セラピー コーポレイション 内皮細胞の遺伝的変性
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
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
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
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
WO2001075164A2 (en) 2000-03-30 2001-10-11 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
US20020173478A1 (en) 2000-11-14 2002-11-21 The Trustees Of The University Of Pennsylvania Post-transcriptional gene silencing by RNAi in mammalian cells
DK2813582T3 (en) 2000-12-01 2017-07-31 Max-Planck-Gesellschaft Zur Förderung Der Wss E V Small RNA molecules that mediate RNA interference
WO2003035869A1 (de) 2001-10-26 2003-05-01 Ribopharma Ag Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
JP4210737B2 (ja) 2001-07-12 2009-01-21 ユニバーシティー オブ マサチューセッツ 遺伝子サイレンシングを仲介する低分子干渉リボ核酸のインビボにおける製造方法
US20030198627A1 (en) 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
CA2462144C (en) 2001-09-28 2016-09-20 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Micro-rna molecules
EP1572902B1 (de) 2002-02-01 2014-06-11 Life Technologies Corporation HOCHWIRKSAME siRNAS ZUR REDUZIERUNG DER EXPRESSION VON ZIELGENEN
CA2476530A1 (en) 2002-02-14 2003-08-21 City Of Hope Methods for producing interfering rna molecules in mammalian cells and therapeutic uses for such molecules
CA2524569C (en) 2002-05-03 2013-10-22 Duke University A method of regulating gene expression
AU2003254334A1 (en) 2002-07-10 2004-02-02 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Rna-interference by single-stranded rna molecules
DK1389637T3 (da) 2002-08-05 2012-09-03 Silence Therapeutics Ag Interfererende RNA-molekyler med stumpe ender
DK1527176T4 (en) 2002-08-05 2017-07-03 Silence Therapeutics Gmbh ADDITIONAL NEW FORMS OF INTERFERRING RNA MOLECULES
US8729036B2 (en) 2002-08-07 2014-05-20 University Of Massachusetts Compositions for RNA interference and methods of use thereof
EP1546344A4 (de) 2002-09-18 2007-10-03 Isis Pharmaceuticals Inc Effiziente produktion von ziel-rnas mittels einzel- und doppelsträngiger oligomerverbindungen
EP1556402B1 (de) 2002-09-25 2011-06-22 University of 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
DE60329416D1 (de) 2002-11-04 2009-11-05 Univ Massachusetts Allelspezifische rna-interferenz
US8090542B2 (en) 2002-11-14 2012-01-03 Dharmacon Inc. Functional and hyperfunctional siRNA
AU2003295539A1 (en) 2002-11-15 2004-06-15 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
EP2333063A1 (de) 2003-01-17 2011-06-15 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Induzierbare Sirna Expressionskonstrukte zur gezielten Genabschaltung
US20070104688A1 (en) 2003-02-13 2007-05-10 City Of Hope Small interfering RNA mediated transcriptional gene silencing in mammalian cells
US8110674B2 (en) * 2003-03-07 2012-02-07 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
AU2004248136B2 (en) 2003-06-02 2011-09-15 University Of Massachusetts Methods and compositions for controlling efficacy of RNA silencing
DE602004029678D1 (de) 2003-06-02 2010-12-02 Univ Massachusetts Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai
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
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
US20050227940A1 (en) 2004-01-23 2005-10-13 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
US20050273868A1 (en) 2004-02-17 2005-12-08 University Of Massachusetts Methods and compositions for enhancing RISC activity in vitro and in vivo
WO2005079533A2 (en) 2004-02-17 2005-09-01 University Of Massachusetts Methods and compositions for mediating gene silencing
US20050277610A1 (en) 2004-03-15 2005-12-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
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

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EP1633890B2 (de) 2020-11-18
AU2010202861A1 (en) 2010-08-05
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AU2004252442B2 (en) 2010-04-08
EP1633890B1 (de) 2010-10-20
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US8309704B2 (en) 2012-11-13
EP2314692A3 (de) 2011-08-03
EP1633890A2 (de) 2006-03-15
EP1633890A4 (de) 2006-11-29
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EP2314692A2 (de) 2011-04-27

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