JP2024521679A - ゲノムセーフハーバー - Google Patents

ゲノムセーフハーバー Download PDF

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Publication number
JP2024521679A
JP2024521679A JP2023571569A JP2023571569A JP2024521679A JP 2024521679 A JP2024521679 A JP 2024521679A JP 2023571569 A JP2023571569 A JP 2023571569A JP 2023571569 A JP2023571569 A JP 2023571569A JP 2024521679 A JP2024521679 A JP 2024521679A
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cell
nucleic acid
gsh
cells
protein
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JP2023571569A
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Japanese (ja)
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JP2024521679A5 (https=
Inventor
ロバート コティン，
シャーロット マクギネス，
セバスチャン アギーレ，
シャノン ロンカー，
ロバート ギフォード，
マシュー エー． キャンベル，
ラミレス， マルコ アントニオ ケサダ
Original Assignee
シンテニー セラピューティクス， インコーポレイテッド
ユニバーシティ オブ マサチューセッツ
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Publication of JP2024521679A publication Critical patent/JP2024521679A/ja
Publication of JP2024521679A5 publication Critical patent/JP2024521679A5/ja
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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
    • 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

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  • 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)
JP2023571569A 2021-05-20 2022-05-19 ゲノムセーフハーバー Pending JP2024521679A (ja)

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 (2)

Publication Number Publication Date
JP2024521679A true JP2024521679A (ja) 2024-06-04
JP2024521679A5 JP2024521679A5 (https=) 2025-05-26

Family

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Family Applications (1)

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JP2023571569A Pending JP2024521679A (ja) 2021-05-20 2022-05-19 ゲノムセーフハーバー

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

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20200390072A1 (en) * 2018-03-02 2020-12-17 Generation Bio Co. Identifying and characterizing genomic safe harbors (gsh) in humans and murine genomes, and viral and non-viral vector compositions for targeted integration at an identified gsh loci
JP2021505159A (ja) * 2017-12-06 2021-02-18 ジェネレーション バイオ カンパニー 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集
WO2021061832A1 (en) * 2019-09-23 2021-04-01 Regents Of The University Of Minnesota Genetically-edited immune cells and methods of therapy
WO2021077117A1 (en) * 2019-10-17 2021-04-22 Fate Therapeutics, Inc. Enhanced chimeric antigen receptor for immune effector cell engineering and use thereof

Family Cites Families (6)

* 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
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
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

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021505159A (ja) * 2017-12-06 2021-02-18 ジェネレーション バイオ カンパニー 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集
US20200390072A1 (en) * 2018-03-02 2020-12-17 Generation Bio Co. Identifying and characterizing genomic safe harbors (gsh) in humans and murine genomes, and viral and non-viral vector compositions for targeted integration at an identified gsh loci
WO2021061832A1 (en) * 2019-09-23 2021-04-01 Regents Of The University Of Minnesota Genetically-edited immune cells and methods of therapy
WO2021077117A1 (en) * 2019-10-17 2021-04-22 Fate Therapeutics, Inc. Enhanced chimeric antigen receptor for immune effector cell engineering and use thereof

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
FINDLAY M. GREGORY ET AL.: "Saturation editing of genomic regions by multiplex homology-directed repair", NATURE, vol. 513, JPN6026009226, 4 September 2014 (2014-09-04), pages 120 - 123, ISSN: 0005811313 *
PAPAPETROU P. EIRINI ET AL.: "Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy", HUMAN GENE THERAPY, vol. 24, no. 4, JPN6026009224, 8 March 2016 (2016-03-08), pages 678 - 684, ISSN: 0005811311 *
RAVN DE SUK SEE ET AL.: "Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulo", NATURE BIOTECHNOLOGY, vol. 34, no. 4, JPN6026009225, 7 March 2016 (2016-03-07), pages 424 - 431, ISSN: 0005811312 *
SADELAIN MICHEL ET AL.: "Safe harbours for the integration of new DNA in the human genome", NATURE REVIEWS CANCER, vol. 12, JPN6026009227, 1 December 2011 (2011-12-01), pages 51 - 58, ISSN: 0005811314 *

Also Published As

Publication number Publication date
CA3219160A1 (en) 2022-11-24
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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