AU2013359212B2 - Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation - Google Patents

Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation Download PDF

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Publication number
AU2013359212B2
AU2013359212B2 AU2013359212A AU2013359212A AU2013359212B2 AU 2013359212 B2 AU2013359212 B2 AU 2013359212B2 AU 2013359212 A AU2013359212 A AU 2013359212A AU 2013359212 A AU2013359212 A AU 2013359212A AU 2013359212 B2 AU2013359212 B2 AU 2013359212B2
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crispr
sequence
cas system
expression
dna
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AU2013359212A1 (en
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Fei RAN
Ophir SHALEM
Feng Zhang
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Massachusetts Institute of Technology
Broad Institute
Harvard University
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Massachusetts Institute of Technology
Broad Institute
Harvard University
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Priority to AU2017202542A priority Critical patent/AU2017202542B2/en
Priority to AU2019236591A priority patent/AU2019236591B2/en
Priority to AU2022202048A priority patent/AU2022202048B2/en
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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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C12N15/82—Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
    • C12N15/8201—Methods for introducing genetic material into plant cells, e.g. DNA, RNA, stable or transient incorporation, tissue culture methods adapted for transformation
    • C12N15/8213—Targeted insertion of genes into the plant genome by homologous recombination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/10—Vectors comprising a non-peptidic targeting moiety
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Immobilizing And Processing Of Enzymes And Microorganisms (AREA)
AU2013359212A 2012-12-12 2013-12-12 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation Active AU2013359212B2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
AU2017202542A AU2017202542B2 (en) 2012-12-12 2017-04-19 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
AU2019236591A AU2019236591B2 (en) 2012-12-12 2019-09-23 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
AU2022202048A AU2022202048B2 (en) 2012-12-12 2022-03-24 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation

Applications Claiming Priority (25)

Application Number Priority Date Filing Date Title
US201261736527P 2012-12-12 2012-12-12
US61/736,527 2012-12-12
US201361748427P 2013-01-02 2013-01-02
US61/748,427 2013-01-02
US201361758468P 2013-01-30 2013-01-30
US61/758,468 2013-01-30
US201361769046P 2013-02-25 2013-02-25
US61/769,046 2013-02-25
US201361791409P 2013-03-15 2013-03-15
US201361802174P 2013-03-15 2013-03-15
US61/802,174 2013-03-15
US61/791,409 2013-03-15
US201361806375P 2013-03-28 2013-03-28
US61/806,375 2013-03-28
US201361814263P 2013-04-20 2013-04-20
US61/814,263 2013-04-20
US201361819803P 2013-05-06 2013-05-06
US61/819,803 2013-05-06
US201361828130P 2013-05-28 2013-05-28
US61/828,130 2013-05-28
US201361836101P 2013-06-17 2013-06-17
US201361835931P 2013-06-17 2013-06-17
US61/836,101 2013-06-17
US61/835,931 2013-06-17
PCT/US2013/074691 WO2014093635A1 (en) 2012-12-12 2013-12-12 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2017202542A Division AU2017202542B2 (en) 2012-12-12 2017-04-19 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation

Publications (2)

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AU2013359212A1 AU2013359212A1 (en) 2015-07-30
AU2013359212B2 true AU2013359212B2 (en) 2017-01-19

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AU2013359212A Active AU2013359212B2 (en) 2012-12-12 2013-12-12 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
AU2017202542A Active AU2017202542B2 (en) 2012-12-12 2017-04-19 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
AU2019236591A Active AU2019236591B2 (en) 2012-12-12 2019-09-23 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
AU2022202048A Active AU2022202048B2 (en) 2012-12-12 2022-03-24 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation

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AU2017202542A Active AU2017202542B2 (en) 2012-12-12 2017-04-19 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
AU2019236591A Active AU2019236591B2 (en) 2012-12-12 2019-09-23 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
AU2022202048A Active AU2022202048B2 (en) 2012-12-12 2022-03-24 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation

Country Status (15)

Country Link
US (6) US20140186919A1 (enExample)
EP (1) EP2898075B1 (enExample)
JP (6) JP6552965B2 (enExample)
KR (1) KR20150105634A (enExample)
CN (2) CN105209621B (enExample)
AU (4) AU2013359212B2 (enExample)
CA (1) CA2894684A1 (enExample)
DK (2) DK2898075T3 (enExample)
ES (2) ES2786193T3 (enExample)
IL (2) IL300461B1 (enExample)
PL (1) PL2898075T3 (enExample)
PT (1) PT2898075E (enExample)
SG (3) SG11201504519TA (enExample)
WO (1) WO2014093635A1 (enExample)
ZA (1) ZA201504081B (enExample)

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