AU2016389495B2 - Systems and methods for in vivo dual recombinase-mediated cassette exchange (dRMCE) and disease models thereof - Google Patents

Systems and methods for in vivo dual recombinase-mediated cassette exchange (dRMCE) and disease models thereof Download PDF

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AU2016389495B2
AU2016389495B2 AU2016389495A AU2016389495A AU2016389495B2 AU 2016389495 B2 AU2016389495 B2 AU 2016389495B2 AU 2016389495 A AU2016389495 A AU 2016389495A AU 2016389495 A AU2016389495 A AU 2016389495A AU 2016389495 B2 AU2016389495 B2 AU 2016389495B2
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rna
cells
transgene
promoter
recombinase
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AU2016389495A1 (en
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Joshua BREUNIG
Moise Danielpour
Gi Bum Kim
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Cedars-Sinai Medical Center
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    • 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
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    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
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    • 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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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
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    • A—HUMAN NECESSITIES
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    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0331—Animal model for proliferative diseases
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    • 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
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    • 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
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    • 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
    • C12N2015/8527—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases
    • C12N2015/8572—Animal models for proliferative diseases, e.g. comprising an oncogene
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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AU2016389495A 2016-01-26 2016-12-30 Systems and methods for in vivo dual recombinase-mediated cassette exchange (dRMCE) and disease models thereof Active AU2016389495B2 (en)

Applications Claiming Priority (3)

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US201662287197P 2016-01-26 2016-01-26
US62/287,197 2016-01-26
PCT/US2016/069442 WO2017131926A1 (en) 2016-01-26 2016-12-30 SYSTEMS AND METHODS FOR IN VIVO DUAL RECOMBINASE-MEDIATED CASSETTE EXCHANGE (dRMCE) AND DISEASE MODELS THEREOF

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AU2016389495B2 true AU2016389495B2 (en) 2023-03-30

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US (2) US11466290B2 (enExample)
EP (1) EP3408396A4 (enExample)
JP (3) JP2019511243A (enExample)
KR (1) KR102811061B1 (enExample)
CN (1) CN108884472A (enExample)
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CA (2) CA3303511A1 (enExample)
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CA3303511A1 (en) 2016-01-26 2026-05-13 Cedars-Sinai Medical Center SYSTEMS AND METHODS FOR IN VIVO DUAL RECOMBINASE-MEDIATED CASSETTE EXCHANGE (dRMCE) AND DISEASE MODELS THEREOF
AU2019236288B2 (en) 2018-03-16 2025-06-05 Cedars-Sinai Medical Center Methods and compositions for inducible expression of neurotrophic factors
CN109321599B (zh) * 2018-09-27 2023-08-22 同济大学 人多能干细胞中的谱系示踪系统的构建与应用
CN109943564B (zh) * 2019-03-28 2022-03-15 上海市中医老年医学研究所 Amh基因定点敲入2A-Cre的杂合子小鼠模型构建方法及其应用
EP3983000A4 (en) * 2019-06-17 2023-10-11 Cedars-Sinai Medical Center SYSTEMS AND METHODS OF DOUBLE RECOMBINASE-MEDIATED CASSETTE EXCHANGE (DRMCE) IN VIVO AND ASSOCIATED DISEASE MODELS
CA3140297A1 (en) * 2019-06-19 2020-12-24 Simon Auslaender Method for the generation of a protein expressing cell by targeted integration using cre mrna
CN119301241A (zh) 2022-06-06 2025-01-10 协和发酵生化株式会社 提高了3'-唾液酸乳糖的生产率的微生物和3'-唾液酸乳糖的制造方法
WO2024218715A1 (en) 2023-04-19 2024-10-24 Btl Healthcare Technologies A.S. Culture media based on protein hydrolysate and a process for preparing thereof
US12668774B2 (en) 2023-10-12 2026-06-30 Btl Healthcare Technologies A.S. Product, system and method of cell cultivation
WO2026050378A1 (en) * 2024-08-28 2026-03-05 Cedars-Sinai Medical Center Humanized genetically-engineered autochthonous brain tumors compatible with use in immunocompetent rodents

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KR20180101535A (ko) 2018-09-12
JP2019511243A (ja) 2019-04-25
CA3303511A1 (en) 2026-05-13
US11466290B2 (en) 2022-10-11
EP3408396A4 (en) 2019-10-09
CN108884472A (zh) 2018-11-23
US20190390222A1 (en) 2019-12-26
AU2016389495A1 (en) 2018-08-16
WO2017131926A1 (en) 2017-08-03
US20230084201A1 (en) 2023-03-16
KR102811061B1 (ko) 2025-05-21
EP3408396A1 (en) 2018-12-05
CA3012042A1 (en) 2017-08-03
JP2022091801A (ja) 2022-06-21
JP2024063065A (ja) 2024-05-10

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