ATE490321T1 - Induzierbare sirna expressionskonstrukte zur gezielten genabschaltung - Google Patents

Induzierbare sirna expressionskonstrukte zur gezielten genabschaltung

Info

Publication number
ATE490321T1
ATE490321T1 AT04702315T AT04702315T ATE490321T1 AT E490321 T1 ATE490321 T1 AT E490321T1 AT 04702315 T AT04702315 T AT 04702315T AT 04702315 T AT04702315 T AT 04702315T AT E490321 T1 ATE490321 T1 AT E490321T1
Authority
AT
Austria
Prior art keywords
expression constructs
targeted gene
sirna expression
inducible sirna
turning
Prior art date
Application number
AT04702315T
Other languages
English (en)
Inventor
Thomas Tuschl
Tilmann Achsel
Reinhard Luehrmann
Jutta Dammann
Original Assignee
Max Planck Gesellschaft
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
Application filed by Max Planck Gesellschaft filed Critical Max Planck Gesellschaft
Application granted granted Critical
Publication of ATE490321T1 publication Critical patent/ATE490321T1/de

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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 nucleic acids [NA]
    • 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
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Pain & Pain Management (AREA)
  • Immunology (AREA)
  • Rheumatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Circuits Of Receivers In General (AREA)
  • Compression Or Coding Systems Of Tv Signals (AREA)
  • Lock And Its Accessories (AREA)
AT04702315T 2003-01-17 2004-01-15 Induzierbare sirna expressionskonstrukte zur gezielten genabschaltung ATE490321T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP03001058 2003-01-17
PCT/EP2004/000265 WO2004065613A2 (en) 2003-01-17 2004-01-15 Inducible small interfering rna (sirna) expression constructs for targeted gene silencing

Publications (1)

Publication Number Publication Date
ATE490321T1 true ATE490321T1 (de) 2010-12-15

Family

ID=32748770

Family Applications (1)

Application Number Title Priority Date Filing Date
AT04702315T ATE490321T1 (de) 2003-01-17 2004-01-15 Induzierbare sirna expressionskonstrukte zur gezielten genabschaltung

Country Status (6)

Country Link
US (2) US8198077B2 (de)
EP (3) EP1583832B1 (de)
AT (1) ATE490321T1 (de)
DE (1) DE602004030315D1 (de)
ES (1) ES2356910T3 (de)
WO (1) WO2004065613A2 (de)

Families Citing this family (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE450621T2 (de) 2000-03-30 2009-12-15 Whitehead Biomedical Inst Mediatoren von rns-interferenz, die rns- sequenzspezifisch sind
KR100872437B1 (ko) 2000-12-01 2008-12-05 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Ｒｎａ 간섭을 매개하는 작은 ｒｎａ 분자
EP1504126B1 (de) 2002-05-03 2014-02-26 Duke University Verfahren zur regulation der genexpression
WO2004014933A1 (en) 2002-08-07 2004-02-19 University Of Massachusetts Compositions for rna interference and methods of use thereof
US7892793B2 (en) * 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
AU2003298718A1 (en) * 2002-11-22 2004-06-18 University Of Massachusetts Modulation of hiv replication by rna interference
AU2004252442B2 (en) * 2003-06-02 2010-04-08 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of FNAi
ES2712695T3 (es) * 2003-06-02 2019-05-14 Univ Massachusetts Métodos y composiciones para controlar la eficacia de la silenciación del ARN
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
WO2005027980A1 (en) * 2003-09-12 2005-03-31 University Of Massachusetts Rna interference for the treatment of gain-of-function disorders
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
GB0327585D0 (en) * 2003-11-27 2003-12-31 Genomica Sau Inducible nucleic acid expression system
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
US20050273868A1 (en) * 2004-02-17 2005-12-08 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
US20060031945A1 (en) * 2004-08-06 2006-02-09 Yu Shen Inducible expression systems for modulating the expression of target genes in eukaryotic cells and non-human animals
WO2006112239A1 (ja) * 2005-04-15 2006-10-26 National University Corporation Tottori University ｈＴＥＲＴ発現調節遺伝子
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
EP2386564B1 (de) * 2005-10-01 2014-07-16 Charles Stout Regulierbare Fusionspromoter
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
WO2008143774A2 (en) * 2007-05-01 2008-11-27 University Of Massachusetts Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy
WO2009026576A1 (en) * 2007-08-23 2009-02-26 Keren Pharmaceuticals Targeting rna with external guide sequences
CN101849008A (zh) 2007-09-19 2010-09-29 应用生物系统有限公司 用于减少RNAi中的脱靶表型效应的siRNA的不依赖于序列的修饰形式和其稳定形式
US8901288B2 (en) * 2007-10-26 2014-12-02 Cold Spring Harbor Laboratory High throughput methods for functionally determining RNA interference efficiency
US8217161B2 (en) * 2008-04-22 2012-07-10 Clemson University Research Foundation Methods of inhibiting multiple cytochrome P450 genes with siRNA
US9089610B2 (en) * 2008-08-19 2015-07-28 Nektar Therapeutics Complexes of small-interfering nucleic acids
EP2440666B1 (de) 2009-06-10 2017-03-01 Temasek Life Sciences Laboratory Limited Virus-induzierte genausschaltung (vigs) zur funktionsanalyse von genen in baumwolle
US8916693B2 (en) 2009-09-17 2014-12-23 Nektar Therapeutics Monoconjugated chitosans as delivery agents for small interfering nucleic acids
WO2012101118A1 (en) 2011-01-24 2012-08-02 Bayer Cropscience N.V. Use of the rd29 promoter or fragments thereof for stress-inducible expression of transgenes in cotton
EP2723865B1 (de) 2011-06-21 2019-03-27 Alnylam Pharmaceuticals, Inc. VERFAHREN ZUR BESTIMMUNG DER AKTIVITÄT VON RNAi IN EINEM PATIENTEN
US20140215655A1 (en) 2011-08-12 2014-07-31 Bayer Cropscience Nv Guard cell-specific expression of transgenes in cotton
AU2013201323A1 (en) 2011-10-12 2013-05-02 Bayer Cropscience Ag Plants with decreased activity of a starch dephosphorylating enzyme
US20140251317A1 (en) 2011-10-12 2014-09-11 Bayer Cropscience Ag Plants with decreased activity of a starch dephosphorylating enzyme
EP2773777B1 (de) 2011-10-31 2020-05-13 University of Utah Research Foundation Genetische veränderungen bei glioblastomen
WO2014123967A2 (en) * 2013-02-05 2014-08-14 University Of Georgia Research Foundation, Inc. Cell lines for virus production and methods of use

Family Cites Families (9)

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US5891718A (en) * 1996-03-27 1999-04-06 Vical Incorporated Tetracycline inducible/repressible systems
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US20050148530A1 (en) * 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
AU2001286171B2 (en) * 2001-05-25 2008-01-10 Serono Genetics Institute S.A. Human CDNAs and proteins and uses thereof
US7195916B2 (en) * 2001-09-13 2007-03-27 California Institute Of Technology Method for expression of small antiviral RNA molecules within a cell
JP2005512598A (ja) * 2001-12-21 2005-05-12 オックスフォード バイオメディカ （ユーケー） リミテッド Ｅｉａｖなどのレンチウイルス発現ベクターを使用するトランスジェニック生物の作製方法
US20050071893A1 (en) * 2002-10-17 2005-03-31 Jost Seibler SiRNA mediated gene silencing in transgenic animals
US20060218654A1 (en) * 2002-10-17 2006-09-28 Artemis Pharmaceuticals Gmbh Sirna mediated gene silencing in transgenic animals
AU2003299732A1 (en) * 2002-12-18 2004-07-14 Genpath Pharmaceuticals, Incorporated Vectors for inducible rna interference

Also Published As

Publication number Publication date
WO2004065613A3 (en) 2004-10-07
US8198077B2 (en) 2012-06-12
EP2333063A1 (de) 2011-06-15
EP2314687B1 (de) 2017-12-27
US20060212950A1 (en) 2006-09-21
DE602004030315D1 (de) 2011-01-13
US8470797B2 (en) 2013-06-25
ES2356910T3 (es) 2011-04-14
EP2314687A1 (de) 2011-04-27
EP1583832A2 (de) 2005-10-12
EP1583832B1 (de) 2010-12-01
US20120255045A1 (en) 2012-10-04
WO2004065613A2 (en) 2004-08-05

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