JP5908405B2 - 多機能性対立遺伝子 - Google Patents

多機能性対立遺伝子 Download PDF

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Publication number
JP5908405B2
JP5908405B2 JP2012537099A JP2012537099A JP5908405B2 JP 5908405 B2 JP5908405 B2 JP 5908405B2 JP 2012537099 A JP2012537099 A JP 2012537099A JP 2012537099 A JP2012537099 A JP 2012537099A JP 5908405 B2 JP5908405 B2 JP 5908405B2
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recombinase
nsi
coin
sense
site
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JP2013509192A (ja
Inventor
アリス エヌ. エコノミデス,
アリス エヌ. エコノミデス,
アンドリュー ジェイ. マーフィー,
アンドリュー ジェイ. マーフィー,
ピーター マシュー レンゲル,
ピーター マシュー レンゲル,
ピーター エイチ. エー. ヤング,
ピーター エイチ. エー. ヤング,
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • 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
    • A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • 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
    • A01K2217/20—Animal model comprising regulated expression system
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/60—Vectors containing traps for, e.g. exons, promoters
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2012537099A 2009-10-29 2010-10-29 多機能性対立遺伝子 Active JP5908405B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US25607809P 2009-10-29 2009-10-29
US61/256,078 2009-10-29
PCT/US2010/054654 WO2011059799A1 (en) 2009-10-29 2010-10-29 Multifunctional alleles

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2016017198A Division JP6085696B2 (ja) 2009-10-29 2016-02-01 多機能性対立遺伝子

Publications (2)

Publication Number Publication Date
JP2013509192A JP2013509192A (ja) 2013-03-14
JP5908405B2 true JP5908405B2 (ja) 2016-04-26

Family

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Family Applications (3)

Application Number Title Priority Date Filing Date
JP2012537099A Active JP5908405B2 (ja) 2009-10-29 2010-10-29 多機能性対立遺伝子
JP2016017198A Active JP6085696B2 (ja) 2009-10-29 2016-02-01 多機能性対立遺伝子
JP2016148272A Active JP6333316B2 (ja) 2009-10-29 2016-07-28 多機能性対立遺伝子

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JP2016017198A Active JP6085696B2 (ja) 2009-10-29 2016-02-01 多機能性対立遺伝子
JP2016148272A Active JP6333316B2 (ja) 2009-10-29 2016-07-28 多機能性対立遺伝子

Country Status (13)

Country Link
US (5) US20110104799A1 (cg-RX-API-DMAC10.html)
EP (2) EP2494047B1 (cg-RX-API-DMAC10.html)
JP (3) JP5908405B2 (cg-RX-API-DMAC10.html)
CN (2) CN102666854B (cg-RX-API-DMAC10.html)
AU (1) AU2010319894B2 (cg-RX-API-DMAC10.html)
CA (1) CA2779858C (cg-RX-API-DMAC10.html)
CY (2) CY1118659T1 (cg-RX-API-DMAC10.html)
DK (2) DK2494047T3 (cg-RX-API-DMAC10.html)
ES (2) ES2613662T3 (cg-RX-API-DMAC10.html)
PL (2) PL3147362T3 (cg-RX-API-DMAC10.html)
PT (2) PT3147362T (cg-RX-API-DMAC10.html)
TR (1) TR201903376T4 (cg-RX-API-DMAC10.html)
WO (1) WO2011059799A1 (cg-RX-API-DMAC10.html)

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CN102666854B (zh) 2009-10-29 2016-08-24 瑞泽恩制药公司 多功能等位基因
ES2683071T3 (es) 2012-04-25 2018-09-24 Regeneron Pharmaceuticals, Inc. Direccionamiento mediado por nucleasas con grandes vectores de direccionamiento
US9510569B2 (en) * 2013-03-13 2016-12-06 Regeneron Pharmaceuticals, Inc. Genetically modified mouse with an inducible Acvr1 gene with a mutant R206H exon 5 that has ectopic bone formation
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
HK1220990A1 (zh) * 2013-08-07 2017-05-19 瑞泽恩制药公司 缺乏lincrna的非人类动物
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
EP3460063B1 (en) 2013-12-11 2024-03-13 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
DK3152312T3 (da) 2014-06-06 2020-04-06 Regeneron Pharma Fremgangsmåder og sammensætninger til modifikation af et mållocus
US9738897B2 (en) 2014-06-23 2017-08-22 Regeneron Pharmaceuticals, Inc. Nuclease-mediated DNA assembly
DK3161128T3 (en) 2014-06-26 2018-11-05 Regeneron Pharma METHODS AND COMPOSITIONS FOR TARGETED GENTICAL MODIFICATIONS AND PROCEDURES FOR USE THEREOF
BR112017007770A2 (pt) 2014-10-15 2018-01-16 Regeneron Pharma cultura in vitro, população de hipscs, método para modificar um locus-alvo genômico, e, hipsc.
EP3221457B1 (en) 2014-11-21 2019-03-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
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US10285387B2 (en) 2015-03-16 2019-05-14 Regeneron Pharmaceuticals, Inc. Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception
US11505792B2 (en) * 2016-04-11 2022-11-22 Applied Stemcell, Inc. Site-specific integration of transgenes
SG10202102885UA (en) 2016-05-20 2021-05-28 Regeneron Pharma Methods for breaking immunological tolerance using multiple guide rnas
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CN107779462B (zh) * 2016-08-29 2021-06-04 中国科学院分子细胞科学卓越创新中心 双同源重组谱系示踪技术
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WO2019067875A1 (en) 2017-09-29 2019-04-04 Regeneron Pharmaceuticals, Inc. NON-HUMAN ANIMALS COMPRISING A HUMANIZED TTR LOCUS AND METHODS OF USE
ES2911249T3 (es) 2017-11-10 2022-05-18 Regeneron Pharma Animales no humanos que comprenden una mutación de slc30a8 y métodos de uso
JP7361031B2 (ja) 2017-11-30 2023-10-13 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ヒト化trkb遺伝子座を含む非ヒト動物
SG11202010891SA (en) 2018-06-13 2020-11-27 Regeneron Pharma A rodent model of fibrodysplasia ossificans progressiva
WO2020131632A1 (en) 2018-12-20 2020-06-25 Regeneron Pharmaceuticals, Inc. Nuclease-mediated repeat expansion
EP3945800A1 (en) 2019-04-03 2022-02-09 Regeneron Pharmaceuticals, Inc. Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
JP7506686B2 (ja) 2019-04-04 2024-06-26 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ヒト化凝固因子12遺伝子座を含む非ヒト動物
WO2020247452A1 (en) 2019-06-04 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use
CN113939595A (zh) 2019-06-07 2022-01-14 瑞泽恩制药公司 包括人源化白蛋白基因座的非人动物
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
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
WO2021154791A1 (en) 2020-01-28 2021-08-05 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized pnpla3 locus and methods of use
EP4099821A1 (en) 2020-02-07 2022-12-14 Regeneron Pharmaceuticals, Inc. <smallcaps/>? ? ?klkb1? ? ? ? ?non-human animals comprising a humanizedlocus and methods of use
US20230102342A1 (en) 2020-03-23 2023-03-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
EP4171215A2 (en) 2020-06-26 2023-05-03 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ace2 locus
EP4259279A1 (en) 2020-12-14 2023-10-18 Regeneron Pharmaceuticals, Inc. Methods of treating metabolic disorders and cardiovascular disease with inhibin subunit beta e (inhbe) inhibitors
WO2023076944A1 (en) 2021-10-26 2023-05-04 Regeneron Pharmaceuticals, Inc. Overexpression of lemd2, lemd3, or chmp7 as a therapeutic modality for tauopathy
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Also Published As

Publication number Publication date
CA2779858C (en) 2019-10-29
US12473574B2 (en) 2025-11-18
PT2494047T (pt) 2017-02-08
CN106191126B (zh) 2020-04-14
US20190390230A1 (en) 2019-12-26
TR201903376T4 (tr) 2019-04-22
CY1121457T1 (el) 2020-05-29
PT3147362T (pt) 2019-04-02
US10392633B2 (en) 2019-08-27
DK2494047T3 (en) 2017-04-10
CN106191126A (zh) 2016-12-07
JP6333316B2 (ja) 2018-05-30
CY1118659T1 (el) 2017-07-12
AU2010319894B2 (en) 2015-03-05
US20130302899A1 (en) 2013-11-14
EP2494047A1 (en) 2012-09-05
US11319557B2 (en) 2022-05-03
EP3147362B1 (en) 2019-01-02
PL2494047T3 (pl) 2017-05-31
US20260234672A1 (en) 2026-08-13
ES2716981T3 (es) 2019-06-18
EP2494047B1 (en) 2017-01-04
US20110104799A1 (en) 2011-05-05
US20220220510A1 (en) 2022-07-14
CN102666854A (zh) 2012-09-12
JP2013509192A (ja) 2013-03-14
PL3147362T3 (pl) 2019-06-28
WO2011059799A1 (en) 2011-05-19
EP3147362A1 (en) 2017-03-29
CA2779858A1 (en) 2011-05-19
CN102666854B (zh) 2016-08-24
JP6085696B2 (ja) 2017-02-22
AU2010319894A1 (en) 2012-05-31
JP2016063839A (ja) 2016-04-28
JP2016185166A (ja) 2016-10-27
ES2613662T3 (es) 2017-05-25
DK3147362T3 (en) 2019-04-08

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