AU2014262867B2 - Delivery methods and compositions for nuclease-mediated genome engineering - Google Patents

Delivery methods and compositions for nuclease-mediated genome engineering Download PDF

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Publication number
AU2014262867B2
AU2014262867B2 AU2014262867A AU2014262867A AU2014262867B2 AU 2014262867 B2 AU2014262867 B2 AU 2014262867B2 AU 2014262867 A AU2014262867 A AU 2014262867A AU 2014262867 A AU2014262867 A AU 2014262867A AU 2014262867 B2 AU2014262867 B2 AU 2014262867B2
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cell
dna
sequence
nuclease
gene
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AU2014262867A1 (en
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Paula M. Cannon
Colin Michael EXLINE
Michael C. Holmes
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Sangamo Therapeutics Inc
University of Southern California
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University of Southern California
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    • 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
    • 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
    • A01K2207/12—Animals modified by administration of exogenous cells
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0381—Animal model for diseases of the hematopoietic system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
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  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
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  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Developmental Biology & Embryology (AREA)
  • Environmental Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Epidemiology (AREA)
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  • Enzymes And Modification Thereof (AREA)
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AU2014262867A 2013-05-10 2014-05-06 Delivery methods and compositions for nuclease-mediated genome engineering Ceased AU2014262867B2 (en)

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US201361821872P 2013-05-10 2013-05-10
US61/821,872 2013-05-10
PCT/US2014/036972 WO2014182700A1 (en) 2013-05-10 2014-05-06 Delivery methods and compositions for nuclease-mediated genome engineering

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AU2014262867A1 AU2014262867A1 (en) 2015-11-12
AU2014262867B2 true AU2014262867B2 (en) 2019-12-05

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EP (1) EP2994531B1 (enExample)
JP (1) JP2016518142A (enExample)
AU (1) AU2014262867B2 (enExample)
CA (1) CA2910427C (enExample)
WO (1) WO2014182700A1 (enExample)

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