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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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WIPO (PCT)
Prior art keywords
beta
target sequence
dna target
variant
microglobulin gene
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/IB2008/001334
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French (fr)
Other versions
WO2008102274A2 (en
Inventor
Sylvain Arnould
Andre Choulika
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Cellectis SA
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Cellectis SA
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Publication date
Application filed by Cellectis SA filed Critical Cellectis SA
Priority to AU2008218605A priority Critical patent/AU2008218605A1/en
Priority to CA002678709A priority patent/CA2678709A1/en
Priority to JP2009550342A priority patent/JP2010518832A/en
Priority to EP08751044A priority patent/EP2121036A2/en
Priority to CN200880005471A priority patent/CN101678126A/en
Publication of WO2008102274A2 publication Critical patent/WO2008102274A2/en
Publication of WO2008102274A3 publication Critical patent/WO2008102274A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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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
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • 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
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Urology & Nephrology (AREA)
  • Physics & Mathematics (AREA)
  • Transplantation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)

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 Ceased WO2008102274A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
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
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
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

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)

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

Family

ID=39167465

Family Applications (2)

Application Number Title Priority Date Filing Date
PCT/IB2007/001532 Ceased 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 Ceased 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 Ceased 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)

Country Link
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 (41)

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DK2215223T3 (en) 2007-10-31 2013-07-22 Prec Biosciences Inc Rationally constructed single chain mechanucleases with non-palindrome recognition sequences
WO2009114321A2 (en) 2008-03-11 2009-09-17 Precision Biosciencs, Inc. Rationally-designed meganucleases for maize genome engineering
EP4001408A3 (en) 2008-07-14 2022-06-08 Precision Biosciences, Inc. Recognition sequences for i-crei-derived meganucleases and uses thereof
US9273296B2 (en) * 2008-09-08 2016-03-01 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
US20120171191A1 (en) 2009-05-26 2012-07-05 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
EP2569424A1 (en) 2010-05-12 2013-03-20 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
CA2801834A1 (en) * 2010-06-09 2011-12-15 Kathleen D'halluin Methods and means to modify a plant genome at a nucleotide sequence commonly used in plant genome engineering
SG185668A1 (en) * 2010-06-09 2012-12-28 Bayer Cropscience Nv Methods and means to modify a plant genome at a nucleotide sequence commonly used in plant genome engineering
US20130196320A1 (en) 2010-06-15 2013-08-01 Cellectis Method for improving cleavage of dna by endonuclease sensitive to methylation
AU2011275460A1 (en) 2010-07-07 2013-01-24 Cellectis Meganucleases variants cleaving a DNA target sequence 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
AU2011319725A1 (en) 2010-10-27 2013-05-30 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenesis
WO2012138901A1 (en) 2011-04-05 2012-10-11 Cellectis Sa Method for enhancing rare-cutting endonuclease efficiency and uses thereof
CA3111953C (en) 2011-04-05 2023-10-24 Cellectis Method for the generation of compact tale-nucleases 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
US9540623B2 (en) 2011-07-08 2017-01-10 Cellectis Method for increasing the efficiency of double-strand-break induced mutagenesis
CN104046593A (en) * 2013-03-14 2014-09-17 浙江大学 Human cell with low immunogenicity and preparation method thereof
EP4286517A3 (en) * 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
RU2752933C2 (en) * 2014-03-11 2021-08-11 Селлектис Method for creation of t-cells suitable for allogeneic transplantation
AU2016261600B2 (en) 2015-05-08 2021-09-23 President And Fellows Of Harvard College Universal donor stem cells and related methods
AU2016287440B2 (en) * 2015-06-30 2022-02-10 Cellectis Methods for improving functionality in NK cell by gene inactivation using specific endonuclease
IL296340A (en) 2015-10-05 2022-11-01 Prec Biosciences Inc Genetically-modified cells comprising a modified human t cell receptor alpha constant region gene
DK3680329T3 (en) 2015-10-05 2021-08-23 Prec Biosciences Inc MODIFIED MEGANUCLEASES WITH RECOGNITION SEQUENCES FOUND IN THE HUMAN T-CELL RECEPTOR ALPHA CONSTANT REGION
CN108699132B (en) * 2015-12-18 2023-08-11 桑格摩生物治疗股份有限公司 Targeted disruption of MHC cell receptors
CA3009637A1 (en) * 2015-12-23 2017-06-29 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human beta-2 microglobulin gene
PT3592379T (en) 2017-03-31 2024-05-31 Cellectis Sa Universal anti-cd22 chimeric antigen receptor engineered immune cells
CA3068465A1 (en) 2017-06-30 2019-01-03 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
WO2019097083A1 (en) * 2017-11-20 2019-05-23 Tessa Therapeutics Pte. Ltd. Modified k562 cell
JP7304888B2 (en) 2018-04-12 2023-07-07 プレシジョン バイオサイエンシズ，インク． Optimized engineered nucleases with specificity for the human T-cell receptor alpha constant region gene
WO2020087010A1 (en) * 2018-10-25 2020-04-30 Ling Ming Compositions and methods for selecting biallelic gene editing
CN116200342A (en) 2019-04-03 2023-06-02 精密生物科学公司 Genetically modified immune cells containing microRNA-adapted shRNAs (shRNAmiRs)
MX2021013502A (en) * 2019-05-07 2022-02-03 Prec Biosciences Inc OPTIMIZATION OF MEGANUCLASES DESIGNED FOR RECOGNITION SEQUENCES.
US11661459B2 (en) 2020-12-03 2023-05-30 Century Therapeutics, Inc. Artificial cell death polypeptide for chimeric antigen receptor and uses thereof
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Title
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Also Published As

Publication number Publication date
CN101678126A (en) 2010-03-24
WO2008102199A1 (en) 2008-08-28
WO2008102274A2 (en) 2008-08-28
AU2008218605A1 (en) 2008-08-28
JP2010518832A (en) 2010-06-03
EP2121036A2 (en) 2009-11-25
CA2678709A1 (en) 2008-08-28

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