DK3079725T3 - Administration, brug og terapeutiske anvendelser af crispr-cas-systemerne og sammensætninger til genomredigering - Google Patents

Administration, brug og terapeutiske anvendelser af crispr-cas-systemerne og sammensætninger til genomredigering Download PDF

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DK3079725T3
DK3079725T3 DK14821438.0T DK14821438T DK3079725T3 DK 3079725 T3 DK3079725 T3 DK 3079725T3 DK 14821438 T DK14821438 T DK 14821438T DK 3079725 T3 DK3079725 T3 DK 3079725T3
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administration
returning
compositions
cas systems
crispr cas
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DK14821438.0T
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Le Cong
Matthias Heidenreich
Randall Jeffrey Platt
Lukasz Swiech
Feng Zhang
David Benjamin Turitz Cox
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Broad Inst Inc
Massachusetts Inst Technology
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61K9/0048—Eye, e.g. artificial tears
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    • 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
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    • 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
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    • 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; CARE OF BIRDS, FISHES, INSECTS; 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
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • 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
DK14821438.0T 2013-12-12 2014-12-12 Administration, brug og terapeutiske anvendelser af crispr-cas-systemerne og sammensætninger til genomredigering DK3079725T3 (da)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201361915176P 2013-12-12 2013-12-12
US201361915145P 2013-12-12 2013-12-12
US201361915148P 2013-12-12 2013-12-12
US201361915215P 2013-12-12 2013-12-12
US201361915153P 2013-12-12 2013-12-12
US201361915107P 2013-12-12 2013-12-12
US201361915192P 2013-12-12 2013-12-12
PCT/US2014/070127 WO2015089462A1 (en) 2013-12-12 2014-12-12 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing

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DK3079725T3 true DK3079725T3 (da) 2020-01-20

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US (2) US11407985B2 (da)
EP (2) EP3653229A1 (da)
JP (3) JP7103750B2 (da)
KR (1) KR20160089527A (da)
CN (2) CN111206032A (da)
AU (3) AU2014361781B2 (da)
BR (1) BR112016013201B1 (da)
CA (1) CA2932478A1 (da)
DK (1) DK3079725T3 (da)
ES (1) ES2765481T3 (da)
IL (2) IL246115B (da)
MX (2) MX2016007328A (da)
SG (1) SG10201804976YA (da)
WO (1) WO2015089462A1 (da)

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