WO2008102274A3 - Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof - Google Patents

Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof Download PDF

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Publication number
WO2008102274A3
WO2008102274A3 PCT/IB2008/001334 IB2008001334W WO2008102274A3 WO 2008102274 A3 WO2008102274 A3 WO 2008102274A3 IB 2008001334 W IB2008001334 W IB 2008001334W WO 2008102274 A3 WO2008102274 A3 WO 2008102274A3
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Prior art keywords
beta
target sequence
dna target
variant
microglobulin gene
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PCT/IB2008/001334
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French (fr)
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WO2008102274A2 (en
Inventor
Sylvain Arnould
Andre Choulika
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Cellectis
Sylvain Arnould
Andre Choulika
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Application filed by Cellectis, Sylvain Arnould, Andre Choulika filed Critical Cellectis
Priority to CN200880005471A priority Critical patent/CN101678126A/en
Priority to CA002678709A priority patent/CA2678709A1/en
Priority to JP2009550342A priority patent/JP2010518832A/en
Priority to EP08751044A priority patent/EP2121036A2/en
Priority to AU2008218605A priority patent/AU2008218605A1/en
Publication of WO2008102274A2 publication Critical patent/WO2008102274A2/en
Publication of WO2008102274A3 publication Critical patent/WO2008102274A3/en

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    • C—CHEMISTRY; METALLURGY
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    • 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 RNAses, DNAses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
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    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • 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
    • 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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  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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Abstract

An I-CreI variant which has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated from positions 26 to 40 and 44 to 77 of I-Crel, said variant being able to cleave a DNA target sequence from the beta-2 microglobulin gene. Use of said variant and derived products for the prevention and the treatment of xenograft rejection and pathological conditions associated with a fibrillar conformation of the beta-2 microglobulin,, as well as for the engineering of transgenic animals and recombinant cell lines expressing an heterologous protein of interest.
PCT/IB2008/001334 2007-02-20 2008-02-20 Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof WO2008102274A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
CN200880005471A CN101678126A (en) 2007-02-20 2008-02-20 Cutting is from meganuclease variant of the DNA target sequence of beta-2-microglobulin gene and uses thereof
CA002678709A CA2678709A1 (en) 2007-02-20 2008-02-20 Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof
JP2009550342A JP2010518832A (en) 2007-02-20 2008-02-20 Meganuclease variants that cleave DNA target sequences from beta-2-microglobulin genes and uses thereof
EP08751044A EP2121036A2 (en) 2007-02-20 2008-02-20 Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof
AU2008218605A AU2008218605A1 (en) 2007-02-20 2008-02-20 Meganuclease variants cleaving a DNA target sequence from the beta-2-microglobulin gene and uses thereof

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
PCT/IB2007/001532 WO2008102199A1 (en) 2007-02-20 2007-02-20 Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof
IBPCT/IB2007/001532 2007-02-20

Publications (2)

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WO2008102274A2 WO2008102274A2 (en) 2008-08-28
WO2008102274A3 true WO2008102274A3 (en) 2009-02-12

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
PCT/IB2007/001532 WO2008102199A1 (en) 2007-02-20 2007-02-20 Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof
PCT/IB2008/001334 WO2008102274A2 (en) 2007-02-20 2008-02-20 Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PCT/IB2007/001532 WO2008102199A1 (en) 2007-02-20 2007-02-20 Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof

Country Status (6)

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EP (1) EP2121036A2 (en)
JP (1) JP2010518832A (en)
CN (1) CN101678126A (en)
AU (1) AU2008218605A1 (en)
CA (1) CA2678709A1 (en)
WO (2) WO2008102199A1 (en)

Families Citing this family (39)

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Publication number Priority date Publication date Assignee Title
EP2660317B1 (en) 2007-10-31 2016-04-06 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
WO2009114321A2 (en) 2008-03-11 2009-09-17 Precision Biosciencs, Inc. Rationally-designed meganucleases for maize genome engineering
DK2313498T3 (en) 2008-07-14 2017-05-22 Prec Biosciences Inc RECOGNITION SEQUENCES FOR I-CREI-DERIVED MECHANUCLEASES AND APPLICATIONS THEREOF
EP2352821B1 (en) * 2008-09-08 2016-11-23 Cellectis Meganuclease variants cleaving a dna target sequence from a glutamine synthetase gene and uses thereof
WO2010122367A2 (en) * 2009-04-21 2010-10-28 Cellectis Meganuclease variants cleaving the genomic insertion of a virus and uses thereof
EP2435560A2 (en) 2009-05-26 2012-04-04 Cellectis Meganuclease variants cleaving the genome of a pathogenic non-integrating virus and uses thereof
WO2010136841A2 (en) * 2009-05-26 2010-12-02 Cellectis Meganuclease variants cleaving the genome of a non-genomically integrating virus and uses thereof
WO2011007193A1 (en) 2009-07-17 2011-01-20 Cellectis Viral vectors encoding a dna repair matrix and containing a virion-associated site specific meganuclease for gene targeting
WO2011021062A1 (en) 2009-08-21 2011-02-24 Cellectis Meganuclease variants cleaving a dna target sequence from the human lysosomal acid alpha-glucosidase gene and uses thereof
WO2011141825A1 (en) 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the rhodopsin gene and uses thereof
US20130145487A1 (en) 2010-05-12 2013-06-06 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
AU2011264074B2 (en) * 2010-06-09 2015-01-22 Bayer Cropscience Nv Methods and means to modify a plant genome at a nucleotide sequence commonly used in plant genome engineering
BR112012031323A2 (en) * 2010-06-09 2015-09-08 Bayer Cropscience Nv methods and means for modifying plant genomes in a nucleotide sequence commonly used in plant genetic engineering
US20130196320A1 (en) 2010-06-15 2013-08-01 Cellectis Method for improving cleavage of dna by endonuclease sensitive to methylation
JP2013534423A (en) 2010-07-07 2013-09-05 セレクティス Meganuclease variants that cleave DNA target sequences in the NANOG gene and uses thereof
WO2012010976A2 (en) 2010-07-15 2012-01-26 Cellectis Meganuclease variants cleaving a dna target sequence in the tert gene and uses thereof
WO2012007848A2 (en) 2010-07-16 2012-01-19 Cellectis Meganuclease variants cleaving a dna target sequence in the was gene and uses thereof
SG189482A1 (en) 2010-10-27 2013-05-31 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenesis
WO2012138927A2 (en) 2011-04-05 2012-10-11 Philippe Duchateau Method for the generation of compact tale-nucleases and uses thereof
WO2012138901A1 (en) 2011-04-05 2012-10-11 Cellectis Sa Method for enhancing rare-cutting endonuclease efficiency and uses thereof
WO2012158778A1 (en) 2011-05-16 2012-11-22 Cellectis S.A. Doubly secure transgenic algae or cyanobacteria strains to prevent their establishment and spread in natural ecosystems
WO2013009525A1 (en) 2011-07-08 2013-01-17 Cellectis S.A. Method for increasing the efficiency of double-strand break-induced mutagenssis
CN104046593A (en) * 2013-03-14 2014-09-17 浙江大学 Human cell with low immunogenicity and preparation method thereof
EP2981617B1 (en) * 2013-04-04 2023-07-05 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
WO2015136001A1 (en) 2014-03-11 2015-09-17 Cellectis Method for generating t-cells compatible for allogenic transplantation
CA2986314C (en) * 2015-06-30 2024-04-23 Cellectis Methods for improving functionality in nk cell by gene inactivation using specific endonuclease
EP3756682B8 (en) 2015-10-05 2022-04-27 Precision Biosciences, Inc. Genetically-modified cells comprising a modified human t cell receptor alpha constant region gene
AU2016333886B2 (en) 2015-10-05 2020-10-08 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human T cell receptor alpha constant region gene
IL297018A (en) 2015-12-18 2022-12-01 Sangamo Therapeutics Inc Targeted disruption of the mhc cell receptor
JP6846429B2 (en) 2015-12-23 2021-03-24 プレシジョン バイオサイエンシズ，インク． An engineered meganuclease with a recognition sequence found in the human β-2 microglobulin gene
US11690873B2 (en) 2017-03-31 2023-07-04 Cellectis Sa Universal chimeric antigen receptor T cells specific for CD22
US11053484B2 (en) 2017-06-30 2021-07-06 Precision Biosciences, Inc. Genetically-modified T cells comprising a modified intron in the T cell receptor alpha gene
TW201923073A (en) * 2017-11-20 2019-06-16 新加坡商泰莎治療私人有限公司 Modified K562 cell
KR102617818B1 (en) 2018-04-12 2023-12-27 프리시젼 바이오사이언시스 인코포레이티드 Optimized engineered nuclease with specificity for human T cell receptor alpha constant region gene
CA3117709A1 (en) * 2018-10-25 2020-04-30 United States Government As Represented By The Department Of Veterans Affairs Compositions and methods for selecting biallelic gene editing
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EP3966322A1 (en) * 2019-05-07 2022-03-16 Precision BioSciences, Inc. Optimization of engineered meganucleases for recognition sequences
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Title
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Publication number Publication date
CA2678709A1 (en) 2008-08-28
AU2008218605A1 (en) 2008-08-28
EP2121036A2 (en) 2009-11-25
JP2010518832A (en) 2010-06-03
CN101678126A (en) 2010-03-24
WO2008102274A2 (en) 2008-08-28
WO2008102199A1 (en) 2008-08-28

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