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

Compositions and methods for altering gene expression Download PDF

Info

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
Application number
PCT/US2000/027065
Other languages
French (fr)
Other versions
WO2001023541A2 (en
Inventor
William L Fodor
Jagdeece J Ramsoondar
Original Assignee
Alexion Pharma Inc
William L Fodor
Jagdeece J Ramsoondar
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 Pharma Inc, William L Fodor, Jagdeece J Ramsoondar filed Critical Alexion Pharma Inc
Priority to CA002385162A priority Critical patent/CA2385162A1/en
Priority to JP2001526924A priority patent/JP2003510072A/en
Priority to AU77448/00A priority patent/AU7744800A/en
Priority to EP00967217A priority patent/EP1220928A2/en
Priority to MXPA02003232A priority patent/MXPA02003232A/en
Publication of WO2001023541A2 publication Critical patent/WO2001023541A2/en
Publication of WO2001023541A3 publication Critical patent/WO2001023541A3/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
    • A01K67/027—New 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; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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

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 WO2001023541A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
CA002385162A CA2385162A1 (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
AU77448/00A AU7744800A (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
MXPA02003232A MXPA02003232A (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 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)

Families Citing this family (16)

* 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
CA2471035C (en) * 2001-12-21 2014-05-13 The Curators Of The University Of Missouri Knockout swine and methods for making the same
AU2012227227B2 (en) * 2001-12-21 2015-05-07 Immerge Biotherapeutics, Inc. Knockout swine and methods for making the same
JP2005536228A (en) 2002-08-21 2005-12-02 レビビコア， インコーポレイテッド Pig animals lacking any expression of functional alpha-1,3-galactosyltransferase
MXPA05004870A (en) 2002-11-08 2005-11-04 Hematech Llc Transgenic ungulates having reduced prion protein activity and uses thereof.
CA2548080A1 (en) 2003-11-05 2005-05-26 University Of Pittsburgh Porcine isogloboside 3 synthase protein, cdna, genomic organization, and regulatory region
AU2005223617A1 (en) 2004-03-17 2005-09-29 Revivicor, Inc. Tissue products from animals lacking functional alpha 1,3 galactosyl transferase
US7420099B2 (en) 2004-04-22 2008-09-02 Kirin Holdings Kabushiki Kaisha Transgenic animals and uses thereof
EP1745128A2 (en) 2004-05-07 2007-01-24 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
EP2208783A1 (en) * 2004-12-22 2010-07-21 Chugai Seiyaku Kabushiki Kaisha Method of producing an antibody using a cell in which the function of fucose transporter is 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
CN106061994A (en) 2014-03-03 2016-10-26 盛禧奥欧洲有限责任公司 Styrenic composition containing long fibers
US11246299B2 (en) 2015-03-04 2022-02-15 Pormedtec Co., Ltd. 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 *

Also Published As

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

Similar Documents

Publication Publication Date Title
WO2001023541A3 (en) Compositions and methods for altering gene expression
AU2001294520A1 (en) Methods and compositions for increasing protein yield from a cell culture
AU5095699A (en) Liver stem cell
CA2323086A1 (en) Regulation of endogenous gene expression in cells using zinc finger proteins
WO2003093469A3 (en) Methods for delivering nucleic acid molecules into cells and assessment thereof
DE69739688D1 (en) Hybrid polyketide synthase I gene
WO2002008409A3 (en) Methods for the replacement, translocation and stacking of dna in eukaryotic genomes
CA2263725A1 (en) Immortalized cell lines for virus growth
WO2001090345A3 (en) Methods and compositions for enhancing the delivery of a nucleic acid to a cell
WO2002006457A3 (en) Novel lipase genes
AU4784996A (en) Homologous recombination in mismatch repair inactivated eukaryotic cells
AU5539898A (en) Nucleic acid construct for the cell cycle regulated expression of structural genes
AU2001278734A1 (en) Method of extensive culture of antigen-specific cytotoxic t cells
AU3644299A (en) A method for the targeting of proteins produced by (yersinia) into the cytosol of eukaryotic cells
WO1999021976A3 (en) Rapid generation of stable mammalian cell lines producing high levels of recombinant proteins
WO2002068635A3 (en) Methods of inhibiting expression of a target gene in mammalian cells
WO2004105697A3 (en) Novel lipids for transfection of nucleic acids
AU6229498A (en) Composition for gene introduction into cell
WO2000009755A3 (en) Domain specific gene evolution
WO2001049721A3 (en) Bacterial genes and proteins that are essential for cell viability and their uses
WO2000029551A3 (en) Cells, culture methods, and their use in autologous transplantation therapy
AU2002337769A1 (en) Methods and compositions for gene targeting by homologous recombination
AU1140101A (en) Improved mutation analysis of the nf1 gene
AU2001278700A1 (en) Method of culturing human chondrocytes
AU2001256232A1 (en) Genes isolated from dendritic cells, gene products and methods employing the same

Legal Events

Date Code Title Description
AK Designated states

Kind code of ref document: A2

Designated state(s): AE AL AM AT AU AZ BA BB BG BR BY CA CH CN CR CU CZ DE DK DM EE ES FI GB GD GE GH GM HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MA MD MG MK MN MW MX NO NZ PL PT RO RU SD SE SG SI SK SL TJ TM TR TT TZ UA UG US UZ VN YU ZA ZW

AL Designated countries for regional patents

Kind code of ref document: A2

Designated state(s): GH GM KE LS MW MZ SD SL SZ TZ UG ZW AM AZ BY KG KZ MD RU TJ TM AT BE CH CY DE DK ES FI FR GB GR IE IT LU MC NL PT SE BF BJ CF CG CI CM GA GN GW ML MR NE SN TD TG

121 Ep: the epo has been informed by wipo that ep was designated in this application
DFPE Request for preliminary examination filed prior to expiration of 19th month from priority date (pct application filed before 20040101)
AK Designated states

Kind code of ref document: A3

Designated state(s): AE AL AM AT AU AZ BA BB BG BR BY CA CH CN CR CU CZ DE DK DM EE ES FI GB GD GE GH GM HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MA MD MG MK MN MW MX NO NZ PL PT RO RU SD SE SG SI SK SL TJ TM TR TT TZ UA UG US UZ VN YU ZA ZW

AL Designated countries for regional patents

Kind code of ref document: A3

Designated state(s): GH GM KE LS MW MZ SD SL SZ TZ UG ZW AM AZ BY KG KZ MD RU TJ TM AT BE CH CY DE DK ES FI FR GB GR IE IT LU MC NL PT SE BF BJ CF CG CI CM GA GN GW ML MR NE SN TD TG

WWE Wipo information: entry into national phase

Ref document number: PA/a/2002/003232

Country of ref document: MX

WWE Wipo information: entry into national phase

Ref document number: 2385162

Country of ref document: CA

ENP Entry into the national phase

Ref country code: JP

Ref document number: 2001 526924

Kind code of ref document: A

Format of ref document f/p: F

WWE Wipo information: entry into national phase

Ref document number: 77448/00

Country of ref document: AU

WWE Wipo information: entry into national phase

Ref document number: 2000967217

Country of ref document: EP

WWP Wipo information: published in national office

Ref document number: 2000967217

Country of ref document: EP

REG Reference to national code

Ref country code: DE

Ref legal event code: 8642

WWW Wipo information: withdrawn in national office

Ref document number: 2000967217

Country of ref document: EP

DPE2 Request for preliminary examination filed before expiration of 19th month from priority date (pct application filed from 20040101)