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 (OSRAM
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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (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 (OSRAM) 2025-05-26

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
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 (OSRAM)
EP (1) EP4352519A4 (OSRAM)
JP (1) JP2024521679A (OSRAM)
KR (1) KR20240023030A (OSRAM)
AU (1) AU2022277688A1 (OSRAM)
CA (1) CA3219160A1 (OSRAM)
WO (1) WO2022246063A1 (OSRAM)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024334482C1 (en) * 2023-08-31 2026-04-23 Kenai Therapeutics 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
EP2539445B1 (en) * 2010-02-26 2018-03-21 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
WO2019169233A1 (en) * 2018-03-02 2019-09-06 Generation Bio Co. Closed-ended dna (cedna) vectors for insertion of transgenes at genomic safe harbors (gsh) in humans and murine genomes
US20200370067A1 (en) * 2019-05-21 2020-11-26 University Of Washington Method to identify and validate genomic safe harbor sites for targeted genome engineering
AU2020348776A1 (en) * 2019-09-17 2022-05-05 Memorial Sloan-Kettering Cancer Center Genomic safe harbors for transgene integration
WO2021055592A1 (en) * 2019-09-17 2021-03-25 Memorial Sloan-Kettering Cancer Center Methods for identifying genomic safe harbors

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
US20250087304A1 (en) 2025-03-13
WO2022246063A1 (en) 2022-11-24
EP4352519A1 (en) 2024-04-17
EP4352519A4 (en) 2025-05-14
AU2022277688A1 (en) 2023-12-21
CA3219160A1 (en) 2022-11-24
KR20240023030A (ko) 2024-02-20

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