DE602004030315D1 - Induzierbare sirna expressionskonstrukte zur gezielten genabschaltung - Google Patents

Induzierbare sirna expressionskonstrukte zur gezielten genabschaltung

Info

Publication number
DE602004030315D1
DE602004030315D1 DE602004030315T DE602004030315T DE602004030315D1 DE 602004030315 D1 DE602004030315 D1 DE 602004030315D1 DE 602004030315 T DE602004030315 T DE 602004030315T DE 602004030315 T DE602004030315 T DE 602004030315T DE 602004030315 D1 DE602004030315 D1 DE 602004030315D1
Authority
DE
Germany
Prior art keywords
expression constructs
sirna expression
general switching
target general
inducible sirna
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Lifetime
Application number
DE602004030315T
Other languages
English (en)
Inventor
Thomas Tuschl
Tilmann Achsel
Reinhard Luehrmann
Jutta Dammann
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Max Planck Gesellschaft zur Foerderung der Wissenschaften eV
Original Assignee
Max Planck Gesellschaft zur Foerderung der Wissenschaften eV
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 zur Foerderung der Wissenschaften eV filed Critical Max Planck Gesellschaft zur Foerderung der Wissenschaften eV
Publication of DE602004030315D1 publication Critical patent/DE602004030315D1/de
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

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
    • 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 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
    • C12N2310/50—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)
  • Compression Or Coding Systems Of Tv Signals (AREA)
  • Lock And Its Accessories (AREA)
  • Circuits Of Receivers In General (AREA)
DE602004030315T 2003-01-17 2004-01-15 Induzierbare sirna expressionskonstrukte zur gezielten genabschaltung Expired - Lifetime DE602004030315D1 (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
DE602004030315D1 true DE602004030315D1 (de) 2011-01-13

Family

ID=32748770

Family Applications (1)

Application Number Title Priority Date Filing Date
DE602004030315T Expired - Lifetime DE602004030315D1 (de) 2003-01-17 2004-01-15 Induzierbare sirna expressionskonstrukte zur gezielten genabschaltung

Country Status (6)

Country Link
US (2) US8198077B2 (de)
EP (3) EP2314687B1 (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
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
EP1873259B1 (de) 2000-12-01 2012-01-25 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Durch kleine 21nt- und 22nt-RNAs vermittelte RNA-Interferenz
PT1504126E (pt) 2002-05-03 2014-06-02 Univ Duke Um método para regular a expressão génica
AU2003261449A1 (en) 2002-08-07 2004-02-25 Compositions for rna interference and methods of use thereof
US7892793B2 (en) * 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
US20040191905A1 (en) * 2002-11-22 2004-09-30 University Of Massachusetts Modulation of HIV replication by RNA interference
DK1633767T3 (en) * 2003-06-02 2019-03-25 Univ Massachusetts METHODS AND COMPOSITIONS FOR MANAGING THE EFFECT OF RNA SILENCING
ES2357116T5 (es) * 2003-06-02 2021-09-03 Univ Massachusetts Métodos y composiciones para mejorar la eficacia y especificad de FNAi
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
ES2808561T3 (es) * 2003-09-12 2021-03-01 Univ Massachusetts Interferencia por ARN para el tratamiento de trastornos de ganancia de función
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
US20060069050A1 (en) * 2004-02-17 2006-03-30 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
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
EP2098593B1 (de) * 2005-04-15 2011-12-28 National University Corporation Tottori University Gen zur Regulierung der hTERT-Expression
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
EP1943265B1 (de) * 2005-10-01 2012-09-12 Charles Stout Regulierbare fusionspromotoren
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
EP2192925A4 (de) * 2007-08-23 2013-04-03 Keren Pharmaceuticals Zielen auf rna mit externen führungssequenzen
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
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
US9433684B2 (en) 2008-08-19 2016-09-06 Nektar Therapeutics Conjugates of small-interfering nucleic acids
CN102803496A (zh) 2009-06-10 2012-11-28 淡马锡生命科学研究院有限公司 用于棉花中基因功能性分析的病毒诱导的基因沉默(vigs)
WO2011035065A1 (en) 2009-09-17 2011-03-24 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
JP2014526882A (ja) 2011-06-21 2014-10-09 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 対象中の治療剤の活性を判定するアッセイおよび方法
WO2013023992A1 (en) 2011-08-12 2013-02-21 Bayer Cropscience Nv Guard cell-specific expression of transgenes in cotton
AU2013201355B2 (en) 2011-10-12 2015-07-09 Bayer Cropscience Ag Plants with decreased activity of a starch dephosphorylating enzyme
AU2012323042B2 (en) 2011-10-12 2015-01-15 Bayer Cropscience Ag Plants with decreased activity of a starch dephosphorylating enzyme
WO2013067050A1 (en) 2011-10-31 2013-05-10 University Of Utah Research Foundation Genetic alterations in glioblastoma
AU2014215025B2 (en) * 2013-02-05 2018-08-30 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Cell lines for virus production and methods of use

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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)
US7074901B2 (en) * 2001-05-25 2006-07-11 Serono Genetics Institute S.A. Isolated human vCOL16A1 polypeptide and fragments 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
CA2502664A1 (en) * 2002-10-17 2004-04-29 Artemis Pharmaceuticals Gmbh Sirna mediated gene silencing in transgenic animals
US20050071893A1 (en) * 2002-10-17 2005-03-31 Jost Seibler 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
EP2314687A1 (de) 2011-04-27
EP2314687B1 (de) 2017-12-27
EP1583832B1 (de) 2010-12-01
ATE490321T1 (de) 2010-12-15
ES2356910T3 (es) 2011-04-14
WO2004065613A3 (en) 2004-10-07
WO2004065613A2 (en) 2004-08-05
US8198077B2 (en) 2012-06-12
US20120255045A1 (en) 2012-10-04
EP2333063A1 (de) 2011-06-15
EP1583832A2 (de) 2005-10-12
US8470797B2 (en) 2013-06-25
US20060212950A1 (en) 2006-09-21

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