WO2009118524A3 - Efficient insertion of dna into embryonic stem cells - Google Patents

Efficient insertion of dna into embryonic stem cells Download PDF

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Publication number
WO2009118524A3
WO2009118524A3 PCT/GB2009/000790 GB2009000790W WO2009118524A3 WO 2009118524 A3 WO2009118524 A3 WO 2009118524A3 GB 2009000790 W GB2009000790 W GB 2009000790W WO 2009118524 A3 WO2009118524 A3 WO 2009118524A3
Authority
WO
WIPO (PCT)
Prior art keywords
embryonic stem
dna
stem cells
host
sequence
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/GB2009/000790
Other languages
French (fr)
Other versions
WO2009118524A2 (en
Inventor
Jost Seibler
Nico Scheer
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.)
ITI Scotland Ltd
Original Assignee
ITI Scotland Ltd
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
Priority claimed from GB0805487A external-priority patent/GB0805487D0/en
Priority claimed from GB0903120A external-priority patent/GB0903120D0/en
Application filed by ITI Scotland Ltd filed Critical ITI Scotland Ltd
Priority to CA2718517A priority Critical patent/CA2718517A1/en
Priority to US12/934,337 priority patent/US20110107445A1/en
Priority to EP09723802A priority patent/EP2271207A2/en
Priority to JP2011501288A priority patent/JP2011515098A/en
Publication of WO2009118524A2 publication Critical patent/WO2009118524A2/en
Publication of WO2009118524A3 publication Critical patent/WO2009118524A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • 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
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • 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
    • C12N15/86—Viral vectors
    • 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
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C12N9/0077—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14) with a reduced iron-sulfur protein as one donor (1.14.15)
    • 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
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • 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
    • 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
    • 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
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • 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
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Environmental Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Rheumatology (AREA)
  • Urology & Nephrology (AREA)
  • Toxicology (AREA)
  • Public Health (AREA)
  • Endocrinology (AREA)
  • Diabetes (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

The present invention relates, in general, to a method for introducing a heterologous replacement gene sequence into a host embryonic stem cell to replace an endogenous host gene target sequence. In particular, the invention relates to a method for inserting large pieces of DNA into embryonic stem cells with improved efficiency, by first deleting the endogenous host gene target sequence, and subsequently utilising two proximally positioned site-specific recombinase target (RT) sites to insert a heterologous replacement gene sequence into the host chromosome.
PCT/GB2009/000790 2008-03-26 2009-03-25 Efficient insertion of dna into embryonic stem cells Ceased WO2009118524A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
CA2718517A CA2718517A1 (en) 2008-03-26 2009-03-25 Efficient insertion of dna into embryonic stem cells
US12/934,337 US20110107445A1 (en) 2008-03-26 2009-03-25 Efficient Insertion of DNA Into Embryonic Stem Cells
EP09723802A EP2271207A2 (en) 2008-03-26 2009-03-25 Efficient insertion of dna into embryonic stem cells
JP2011501288A JP2011515098A (en) 2008-03-26 2009-03-25 Efficient insertion of DNA into embryonic stem cells

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
GB0805487A GB0805487D0 (en) 2008-03-26 2008-03-26 Efficient insertion of DNA into ES cells
GB0805487.6 2008-03-26
GB0903120A GB0903120D0 (en) 2009-02-24 2009-02-24 Efficient insertion of DNA into embryonic stem cells
GB0903120.4 2009-02-24

Publications (2)

Publication Number Publication Date
WO2009118524A2 WO2009118524A2 (en) 2009-10-01
WO2009118524A3 true WO2009118524A3 (en) 2009-11-26

Family

ID=40935738

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2009/000790 Ceased WO2009118524A2 (en) 2008-03-26 2009-03-25 Efficient insertion of dna into embryonic stem cells

Country Status (5)

Country Link
US (1) US20110107445A1 (en)
EP (1) EP2271207A2 (en)
JP (1) JP2011515098A (en)
CA (1) CA2718517A1 (en)
WO (1) WO2009118524A2 (en)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9445581B2 (en) 2012-03-28 2016-09-20 Kymab Limited Animal models and therapeutic molecules
DK3622815T3 (en) 2009-07-08 2023-06-26 Kymab Ltd RODENT MODELS AND THERAPEUTIC MOLECULES
CN103228130B (en) * 2010-06-17 2016-03-16 科马布有限公司 Animal Models and Therapeutic Molecules
BR112014006394A2 (en) 2011-09-19 2017-03-28 Kymab Ltd manipulation of immunoglobulin genetic diversity and therapeutic multi-antibodies
WO2013045916A1 (en) 2011-09-26 2013-04-04 Kymab Limited Chimaeric surrogate light chains (slc) comprising human vpreb
US9253965B2 (en) 2012-03-28 2016-02-09 Kymab Limited Animal models and therapeutic molecules
US10251377B2 (en) 2012-03-28 2019-04-09 Kymab Limited Transgenic non-human vertebrate for the expression of class-switched, fully human, antibodies
GB2502127A (en) 2012-05-17 2013-11-20 Kymab Ltd Multivalent antibodies and in vivo methods for their production
US9788534B2 (en) 2013-03-18 2017-10-17 Kymab Limited Animal models and therapeutic molecules
US9783618B2 (en) 2013-05-01 2017-10-10 Kymab Limited Manipulation of immunoglobulin gene diversity and multi-antibody therapeutics
US11707056B2 (en) 2013-05-02 2023-07-25 Kymab Limited Animals, repertoires and methods
US9783593B2 (en) 2013-05-02 2017-10-10 Kymab Limited Antibodies, variable domains and chains tailored for human use
AU2014330922A1 (en) 2013-10-01 2016-03-03 Kymab Limited Animal models and therapeutic molecules

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2256972T3 (en) * 1997-11-18 2006-07-16 Pioneer Hi-Bred International, Inc. NEW METHOD FOR THE INTEGRATION OF A FORANEOUS DNA WITHIN EUCARIOTE GENOME.
AU2001257078A1 (en) * 2000-04-18 2001-10-30 Iconix Pharmaceuticals, Inc. Vector and method for targeted replacement and disruption of an integrated dna sequence

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
SORRELL ET AL: "Targeted modification of mammalian genomes", BIOTECHNOLOGY ADVANCES, ELSEVIER PUBLISHING, BARKING, GB, vol. 23, no. 7-8, 1 November 2005 (2005-11-01), pages 431 - 469, XP005076498, ISSN: 0734-9750 *
WALLACE HELEN A C ET AL: "Manipulating the mouse genome to engineer precise functional syntenic replacements with human sequence", CELL, CELL PRESS, CAMBRIDGE, NA, US, vol. 128, no. 1, 1 January 2007 (2007-01-01), pages 197 - 209, XP002481713, ISSN: 0092-8674 *
WIRTH ET AL: "Road to precision: recombinase-based targeting technologies for genome engineering", CURRENT OPINION IN BIOTECHNOLOGY, LONDON, GB, vol. 18, no. 5, 1 October 2007 (2007-10-01), pages 411 - 419, XP022350911, ISSN: 0958-1669 *

Also Published As

Publication number Publication date
WO2009118524A2 (en) 2009-10-01
CA2718517A1 (en) 2009-10-01
US20110107445A1 (en) 2011-05-05
EP2271207A2 (en) 2011-01-12
JP2011515098A (en) 2011-05-19

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