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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Prior art keywords
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 Inc
Harvard University
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Massachusetts Institute of Technology
Broad Institute Inc
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
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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
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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
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    • 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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    • 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
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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)
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  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (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)
  • Immobilizing And Processing Of Enzymes And Microorganisms (AREA)
  • Peptides Or Proteins (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
US201361802174P 2013-03-15 2013-03-15
US201361791409P 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

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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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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 (cg-RX-API-DMAC7.html)
EP (1) EP2898075B1 (cg-RX-API-DMAC7.html)
JP (6) JP6552965B2 (cg-RX-API-DMAC7.html)
KR (1) KR20150105634A (cg-RX-API-DMAC7.html)
CN (2) CN105209621B (cg-RX-API-DMAC7.html)
AU (4) AU2013359212B2 (cg-RX-API-DMAC7.html)
CA (1) CA2894684A1 (cg-RX-API-DMAC7.html)
DK (2) DK2898075T3 (cg-RX-API-DMAC7.html)
ES (2) ES2786193T3 (cg-RX-API-DMAC7.html)
IL (2) IL300461A (cg-RX-API-DMAC7.html)
PL (1) PL2898075T3 (cg-RX-API-DMAC7.html)
PT (1) PT2898075E (cg-RX-API-DMAC7.html)
SG (3) SG11201504519TA (cg-RX-API-DMAC7.html)
WO (1) WO2014093635A1 (cg-RX-API-DMAC7.html)
ZA (1) ZA201504081B (cg-RX-API-DMAC7.html)

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