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WO2001023541A3 - Compositions and methods for altering gene expression - Google Patents

Compositions and methods for altering gene expression Download PDF

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Publication number
WO2001023541A3
WO2001023541A3 PCT/US2000/027065 US0027065W WO0123541A3 WO 2001023541 A3 WO2001023541 A3 WO 2001023541A3 US 0027065 W US0027065 W US 0027065W WO 0123541 A3 WO0123541 A3 WO 0123541A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
compositions
gene expression
altering gene
target gene
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.)
Ceased
Application number
PCT/US2000/027065
Other languages
French (fr)
Other versions
WO2001023541A2 (en
Inventor
William L Fodor
Jagdeece J Ramsoondar
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.)
Alexion Pharmaceuticals Inc
Original Assignee
Alexion Pharmaceuticals Inc
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 Alexion Pharmaceuticals Inc filed Critical Alexion Pharmaceuticals Inc
Priority to AU77448/00A priority Critical patent/AU7744800A/en
Priority to MXPA02003232A priority patent/MXPA02003232A/en
Priority to JP2001526924A priority patent/JP2003510072A/en
Priority to CA002385162A priority patent/CA2385162A1/en
Priority to EP00967217A priority patent/EP1220928A2/en
Publication of WO2001023541A2 publication Critical patent/WO2001023541A2/en
Publication of WO2001023541A3 publication Critical patent/WO2001023541A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/60—Vectors containing traps for, e.g. exons, promoters
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Environmental Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Husbandry (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Materials For Medical Uses (AREA)

Abstract

Disclosed are novel compositions and methods useful for modulating expression of a target gene in a cell by insertion of exogenous DNA sequence into the target gene. The compositions and methods of the invention are useful for generation of knockout animals including mammals.
PCT/US2000/027065 1999-09-30 2000-10-02 Compositions and methods for altering gene expression Ceased WO2001023541A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
AU77448/00A AU7744800A (en) 1999-09-30 2000-10-02 Compositions and methods for altering gene expression
MXPA02003232A MXPA02003232A (en) 1999-09-30 2000-10-02 Compositions and methods for altering gene expression.
JP2001526924A JP2003510072A (en) 1999-09-30 2000-10-02 Compositions and methods for altering gene expression
CA002385162A CA2385162A1 (en) 1999-09-30 2000-10-02 Compositions and methods for altering gene expression
EP00967217A EP1220928A2 (en) 1999-09-30 2000-10-02 Compositions and methods for altering gene expression

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US15695399P 1999-09-30 1999-09-30
US60/156,953 1999-09-30

Publications (2)

Publication Number Publication Date
WO2001023541A2 WO2001023541A2 (en) 2001-04-05
WO2001023541A3 true WO2001023541A3 (en) 2002-01-17

Family

ID=22561777

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2000/027065 Ceased WO2001023541A2 (en) 1999-09-30 2000-10-02 Compositions and methods for altering gene expression

Country Status (6)

Country Link
EP (1) EP1220928A2 (en)
JP (1) JP2003510072A (en)
AU (1) AU7744800A (en)
CA (1) CA2385162A1 (en)
MX (1) MXPA02003232A (en)
WO (1) WO2001023541A2 (en)

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7074983B2 (en) 1999-11-19 2006-07-11 Kirin Beer Kabushiki Kaisha Transgenic bovine comprising human immunoglobulin loci and producing human immunoglobulin
US7253334B2 (en) 2000-12-22 2007-08-07 Aurox, Llc Methods for cloning non-human mammals using reprogrammed donor chromatin or donor cells
US7414170B2 (en) 1999-11-19 2008-08-19 Kirin Beer Kabushiki Kaisha Transgenic bovines capable of human antibody production
US7420099B2 (en) 2004-04-22 2008-09-02 Kirin Holdings Kabushiki Kaisha Transgenic animals and uses thereof
US7429690B2 (en) 2002-11-08 2008-09-30 Kirin Holdings Kabushiki Kaisha Transgenic bovines having reduced prion protein production
US7491534B2 (en) 2000-12-22 2009-02-17 Kirin Holdings Kabushiki Kaisha Methods for altering cell fate to generate T-cells specific for an antigen of interest

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6902890B1 (en) 1999-11-04 2005-06-07 Diadexus, Inc. Method of diagnosing monitoring, staging, imaging and treating cancer
US7879540B1 (en) 2000-08-24 2011-02-01 Promega Corporation Synthetic nucleic acid molecule compositions and methods of preparation
AU2012227227B2 (en) * 2001-12-21 2015-05-07 Immerge Biotherapeutics, Inc. Knockout swine and methods for making the same
AU2002364596A1 (en) 2001-12-21 2003-07-15 Immerge Biotherapeutics, Inc. Knockout swine and methods for making the same
EP1534819B1 (en) 2002-08-21 2009-12-09 Revivicor, Inc. Porcine animals lacking any expression of functional alpha 1,3 galactosyltransferase
AU2004290006A1 (en) 2003-11-05 2005-05-26 University Of Pittsburgh Porcine isogloboside 3 synthase protein, cDNA, genomic organization, and regulatory region
CA2857051A1 (en) 2004-03-17 2005-09-29 David Ayares Tissue products derived from animals lacking any expression of functional alpha 1,3 galactosyltransferase
AU2005243169A1 (en) 2004-05-07 2005-11-24 University Of Pittsburgh Of The Commonwealth System Of Higher Education Porcine Forssman synthetase protein, cDNA, genomic organization, and regulatory region
US7728118B2 (en) 2004-09-17 2010-06-01 Promega Corporation Synthetic nucleic acid molecule compositions and methods of preparation
EP1829961A4 (en) * 2004-12-22 2008-06-04 Chugai Pharmaceutical Co Ltd Method of preparing antibody by use of cell having its fucose transporter function inhibited
GB0909660D0 (en) * 2009-06-04 2009-07-22 Gene Bridges Gmbh Method of altering nucleic acids
WO2013163346A1 (en) * 2012-04-25 2013-10-31 The Regents Of The University Of California Synthetic promoter for modulating gene expression
KR102210310B1 (en) 2014-03-03 2021-02-01 트린세오 유럽 게엠베하 Styrenic composition containing long fibers
WO2016140353A1 (en) * 2015-03-04 2016-09-09 株式会社 医療実験用大動物供給事業準備会社 Disease model pig exhibiting stable phenotype, and production method thereof

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995028412A1 (en) * 1994-04-13 1995-10-26 Biotransplant, Inc. α(1,3) GALACTOSYLTRANSFERASE NEGATIVE SWINE
US5464764A (en) * 1989-08-22 1995-11-07 University Of Utah Research Foundation Positive-negative selection methods and vectors
US5627058A (en) * 1990-05-14 1997-05-06 Massachusetts Institute Of Technology Retrovirus promoter-trap vectors
US5916810A (en) * 1993-01-05 1999-06-29 Jarvik; Jonathan W. Method for producing tagged genes transcripts and proteins
WO1999061604A2 (en) * 1998-05-22 1999-12-02 Japan Science And Technology Corporation A vector for gene trap, and a method for gene trapping by using the vector
WO2001030992A2 (en) * 1999-10-22 2001-05-03 University Of Pittsburgh Of The Commonwealth System Of Higher Education α1-3 GALACTOSYLTRANSFERASE GENE AND PROMOTER

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5464764A (en) * 1989-08-22 1995-11-07 University Of Utah Research Foundation Positive-negative selection methods and vectors
US5627058A (en) * 1990-05-14 1997-05-06 Massachusetts Institute Of Technology Retrovirus promoter-trap vectors
US5916810A (en) * 1993-01-05 1999-06-29 Jarvik; Jonathan W. Method for producing tagged genes transcripts and proteins
WO1995028412A1 (en) * 1994-04-13 1995-10-26 Biotransplant, Inc. α(1,3) GALACTOSYLTRANSFERASE NEGATIVE SWINE
WO1999061604A2 (en) * 1998-05-22 1999-12-02 Japan Science And Technology Corporation A vector for gene trap, and a method for gene trapping by using the vector
WO2001030992A2 (en) * 1999-10-22 2001-05-03 University Of Pittsburgh Of The Commonwealth System Of Higher Education α1-3 GALACTOSYLTRANSFERASE GENE AND PROMOTER

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
HOOPES C.W. & PLATT J.L.: "A molecular epidemiological probe for pig microchimerism", TRANSPLANTATION (BALTIMORE), vol. 64, no. 2, 1997, pages 347 - 350, XP000982842 *
KARAPLIS ET AL: "Gene targeting", PRINCIPLES OF BONE BIOLOGY,XX,XX, 1996, pages 1189 - 1201, XP002090123 *

Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7074983B2 (en) 1999-11-19 2006-07-11 Kirin Beer Kabushiki Kaisha Transgenic bovine comprising human immunoglobulin loci and producing human immunoglobulin
US7414170B2 (en) 1999-11-19 2008-08-19 Kirin Beer Kabushiki Kaisha Transgenic bovines capable of human antibody production
US7491867B2 (en) 1999-11-19 2009-02-17 Kyowa Hakko Kirin Co., Ltd. Expression of xenogenous (human) immunoglobulins in cloned, transgenic ungulates
US7253334B2 (en) 2000-12-22 2007-08-07 Aurox, Llc Methods for cloning non-human mammals using reprogrammed donor chromatin or donor cells
US7491534B2 (en) 2000-12-22 2009-02-17 Kirin Holdings Kabushiki Kaisha Methods for altering cell fate to generate T-cells specific for an antigen of interest
US7429690B2 (en) 2002-11-08 2008-09-30 Kirin Holdings Kabushiki Kaisha Transgenic bovines having reduced prion protein production
US7420099B2 (en) 2004-04-22 2008-09-02 Kirin Holdings Kabushiki Kaisha Transgenic animals and uses thereof

Also Published As

Publication number Publication date
EP1220928A2 (en) 2002-07-10
MXPA02003232A (en) 2003-09-22
CA2385162A1 (en) 2001-04-05
JP2003510072A (en) 2003-03-18
AU7744800A (en) 2001-04-30
WO2001023541A2 (en) 2001-04-05

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