CA3219160A1 - Genomic safe harbors - Google Patents

Genomic safe harbors Download PDF

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Publication number
CA3219160A1
CA3219160A1 CA3219160A CA3219160A CA3219160A1 CA 3219160 A1 CA3219160 A1 CA 3219160A1 CA 3219160 A CA3219160 A CA 3219160A CA 3219160 A CA3219160 A CA 3219160A CA 3219160 A1 CA3219160 A1 CA 3219160A1
Authority
CA
Canada
Prior art keywords
cell
nucleic acid
gsh
protein
promoter
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.)
Pending
Application number
CA3219160A
Other languages
English (en)
French (fr)
Inventor
Robert Kotin
Charlotte Mcguinness
Sebastian AGUIRRE
Shannon LONCAR
Robert Gifford
Matthew A. Campbell
Marco Antonio Quezada Ramirez
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Synteny Therapeutics Inc
University of Massachusetts Amherst
Original Assignee
Individual
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Individual filed Critical Individual
Publication of CA3219160A1 publication Critical patent/CA3219160A1/en
Pending legal-status Critical Current

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Classifications

    • 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
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B25/00—ICT specially adapted for hybridisation; ICT specially adapted for gene or protein expression
    • G16B25/10—Gene or protein expression profiling; Expression-ratio estimation or normalisation
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • 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
    • 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
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6897—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters
    • 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
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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
    • 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

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Environmental Sciences (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Analytical Chemistry (AREA)
  • Evolutionary Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medical Informatics (AREA)
  • Spectroscopy & Molecular Physics (AREA)
  • Theoretical Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Animal Husbandry (AREA)
  • Virology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3219160A 2021-05-20 2022-05-19 Genomic safe harbors Pending CA3219160A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163190996P 2021-05-20 2021-05-20
US63/190,996 2021-05-20
PCT/US2022/030024 WO2022246063A1 (en) 2021-05-20 2022-05-19 Genomic safe harbors

Publications (1)

Publication Number Publication Date
CA3219160A1 true CA3219160A1 (en) 2022-11-24

Family

ID=84141733

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3219160A Pending CA3219160A1 (en) 2021-05-20 2022-05-19 Genomic safe harbors

Country Status (7)

Country Link
US (1) US20250087304A1 (https=)
EP (1) EP4352519A4 (https=)
JP (1) JP2024521679A (https=)
KR (1) KR20240023030A (https=)
AU (1) AU2022277688A1 (https=)
CA (1) CA3219160A1 (https=)
WO (1) WO2022246063A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025049807A1 (en) * 2023-08-31 2025-03-06 Ryne Biotechnology Inc. Methods and compositions for engineered da neuronal cells
CN118256559B (zh) * 2024-01-24 2024-09-17 中国医学科学院阜外医院 制备射血分数保留的心衰的动物模型的方法
WO2026064739A1 (en) * 2024-09-23 2026-03-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011104382A1 (en) * 2010-02-26 2011-09-01 Cellectis Use of endonucleases for inserting transgenes into safe harbor loci
CN110891420B (zh) * 2017-07-31 2022-06-03 瑞泽恩制药公司 Cas转基因小鼠胚胎干细胞和小鼠及其应用
CA3084185A1 (en) * 2017-12-06 2019-06-13 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
MA52116A (fr) * 2018-03-02 2021-01-06 Generation Bio Co Vecteurs d'adn à extrémité fermée (cedna) pour l'insertion de transgènes au niveau de havres génomiques sécuritaires (gsh) dans des génomes humains et murins
EP3759226A4 (en) * 2018-03-02 2022-06-15 Generation Bio Co. IDENTIFICATION AND CHARACTERIZATION OF GENOMIC SAFE HARBOR (GSH) IN HUMAN AND MURINE GENOMES AND VIRAL AND NON-VIRAL VECTOR COMPOSITIONS FOR TARGETED INTEGRATION AT IDENTIFIED GSH LOCI
US20200370067A1 (en) * 2019-05-21 2020-11-26 University Of Washington Method to identify and validate genomic safe harbor sites for targeted genome engineering
WO2021055592A1 (en) * 2019-09-17 2021-03-25 Memorial Sloan-Kettering Cancer Center Methods for identifying genomic safe harbors
EP4032092A4 (en) * 2019-09-17 2023-12-06 Memorial Sloan Kettering Cancer Center Genomic safe harbors for transgene integration
EP4034640A4 (en) * 2019-09-23 2023-10-25 Regents Of The University Of Minnesota GENETICALLY EDITED IMMUNE CELLS AND THERAPY METHODS
EP4045539A4 (en) * 2019-10-17 2024-03-13 Fate Therapeutics, Inc. Enhanced chimeric antigen receptor for immune effector cell engineering and use thereof

Also Published As

Publication number Publication date
JP2024521679A (ja) 2024-06-04
WO2022246063A1 (en) 2022-11-24
EP4352519A4 (en) 2025-05-14
EP4352519A1 (en) 2024-04-17
AU2022277688A1 (en) 2023-12-21
US20250087304A1 (en) 2025-03-13
KR20240023030A (ko) 2024-02-20

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