CA3143981A1 - 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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Publication number
CA3143981A1
CA3143981A1 CA3143981A CA3143981A CA3143981A1 CA 3143981 A1 CA3143981 A1 CA 3143981A1 CA 3143981 A CA3143981 A CA 3143981A CA 3143981 A CA3143981 A CA 3143981A CA 3143981 A1 CA3143981 A1 CA 3143981A1
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Prior art keywords
cell
promoter
transgene
recombinase
cells
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CA3143981A
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English (en)
French (fr)
Inventor
Alberto AYALA-SARMIENTO
Amy Yang
Joshua BREUNIG
Moise Danielpour
Gi Bum Kim
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Cedars Sinai Medical Center
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Cedars Sinai Medical Center
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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • 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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
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  • Health & Medical Sciences (AREA)
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CA3143981A 2019-06-17 2020-06-16 Systems and methods for in vivo dual recombinase-mediated cassette exchange (drmce) and disease models thereof Pending CA3143981A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962862576P 2019-06-17 2019-06-17
US62/862,576 2019-06-17
PCT/US2020/037946 WO2020257205A1 (en) 2019-06-17 2020-06-16 SYSTEMS AND METHODS FOR IN VIVO DUAL RECOMBINASE-MEDIATED CASSETTE EXCHANGE (dRMCE) AND DISEASE MODELS THEREOF

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Publication Number Publication Date
CA3143981A1 true CA3143981A1 (en) 2020-12-24

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CA3143981A Pending CA3143981A1 (en) 2019-06-17 2020-06-16 Systems and methods for in vivo dual recombinase-mediated cassette exchange (drmce) and disease models thereof

Country Status (8)

Country Link
US (1) US20220304286A1 (enExample)
EP (1) EP3983000A4 (enExample)
JP (1) JP2022536934A (enExample)
KR (1) KR20220024527A (enExample)
CN (1) CN114761035A (enExample)
AU (1) AU2020297427A1 (enExample)
CA (1) CA3143981A1 (enExample)
WO (1) WO2020257205A1 (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017131926A1 (en) 2016-01-26 2017-08-03 Cedars-Sinai Medical Center SYSTEMS AND METHODS FOR IN VIVO DUAL RECOMBINASE-MEDIATED CASSETTE EXCHANGE (dRMCE) AND DISEASE MODELS THEREOF
WO2019178550A1 (en) 2018-03-16 2019-09-19 Cedars-Sinai Medical Center Methods and compositions for inducible expression of neurotrophic factors
CN114480457A (zh) * 2021-12-21 2022-05-13 浙江大学 一种基于pCAG-flox-neo载体的高效稳定的定点整合基因敲入方法
CN114438039A (zh) * 2022-04-07 2022-05-06 中国科学院天津工业生物技术研究所 一种哺乳动物细胞疾病模型的高通量构建方法
EP4596686A1 (en) * 2022-09-29 2025-08-06 Keio University Searching method for functional molecule for causing response in cell
CN118460688A (zh) * 2023-02-09 2024-08-09 中国人民解放军军事科学院军事医学研究院 多聚甲醛固定细胞的单细胞转录组及多重靶向蛋白质同时检测方法的建立
CN116463380A (zh) * 2023-03-30 2023-07-21 中国科学院深圳先进技术研究院 一种重组腺相关病毒载体组合物、外源基因表达系统及方法
WO2024243040A2 (en) * 2023-05-19 2024-11-28 The Board Of Trustees Of The Leland Stanford Junior University High-throughput multiomic readout of rna and genomic dna within single cells
IT202300019377A1 (it) * 2023-09-20 2025-03-20 Univ Degli Studi Di Pavia Linee di cellule staminali contenenti proteine endogene marcate con espressione differenziale, metodi di produzione e loro utilizzo per l’identificazione del ciclo cellulare
CN118086394B (zh) * 2024-04-24 2024-07-12 中国农业科学院北京畜牧兽医研究所 猪脂肪组织发育示踪系统及应用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1614755A1 (en) * 2004-07-07 2006-01-11 ARTEMIS Pharmaceuticals GmbH Target transgenesis of short hairpin RNA expression cassette using recombinase mediated cassette exchange
WO2011001247A2 (en) * 2009-06-29 2011-01-06 University Of Basel Methods and kits for high efficiency engineering of conditional mouse alleles
WO2017131926A1 (en) * 2016-01-26 2017-08-03 Cedars-Sinai Medical Center SYSTEMS AND METHODS FOR IN VIVO DUAL RECOMBINASE-MEDIATED CASSETTE EXCHANGE (dRMCE) AND DISEASE MODELS THEREOF
JP6856919B2 (ja) * 2016-05-10 2021-04-14 大学共同利用機関法人情報・システム研究機構 ベクターシステム、遺伝子発現方法、標的遺伝子ノックアウト方法、標的遺伝子ノックダウン方法、標的遺伝子編集方法、及び遺伝子発現キット

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Publication number Publication date
KR20220024527A (ko) 2022-03-03
WO2020257205A1 (en) 2020-12-24
CN114761035A (zh) 2022-07-15
EP3983000A4 (en) 2023-10-11
AU2020297427A1 (en) 2022-02-03
US20220304286A1 (en) 2022-09-29
EP3983000A1 (en) 2022-04-20
JP2022536934A (ja) 2022-08-22

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