PL2494047T3 - Allele wielofunkcyjne - Google Patents

Allele wielofunkcyjne

Info

Publication number
PL2494047T3
PL2494047T3 PL10774391T PL10774391T PL2494047T3 PL 2494047 T3 PL2494047 T3 PL 2494047T3 PL 10774391 T PL10774391 T PL 10774391T PL 10774391 T PL10774391 T PL 10774391T PL 2494047 T3 PL2494047 T3 PL 2494047T3
Authority
PL
Poland
Prior art keywords
alleles
multifunctional
multifunctional alleles
Prior art date
Application number
PL10774391T
Other languages
English (en)
Inventor
Aris N. Economides
Andrew J. Murphy
Peter Matthew Lengyel
Peter H. A. Yang
Original Assignee
Regeneron Pharmaceuticals, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Regeneron Pharmaceuticals, Inc. filed Critical Regeneron Pharmaceuticals, Inc.
Publication of PL2494047T3 publication Critical patent/PL2494047T3/pl

Links

Classifications

    • 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

Landscapes

  • 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)
PL10774391T 2009-10-29 2010-10-29 Allele wielofunkcyjne PL2494047T3 (pl)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US25607809P 2009-10-29 2009-10-29

Publications (1)

Publication Number Publication Date
PL2494047T3 true PL2494047T3 (pl) 2017-05-31

Family

ID=43244777

Family Applications (2)

Application Number Title Priority Date Filing Date
PL16196614T PL3147362T3 (pl) 2009-10-29 2010-10-29 Allele wielofunkcyjne
PL10774391T PL2494047T3 (pl) 2009-10-29 2010-10-29 Allele wielofunkcyjne

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PL16196614T PL3147362T3 (pl) 2009-10-29 2010-10-29 Allele wielofunkcyjne

Country Status (13)

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

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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
MX388784B (es) 2014-12-19 2025-03-20 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida a través de direccionamiento múltiple de una sola etapa.
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
CN109803530A (zh) 2016-07-29 2019-05-24 瑞泽恩制药公司 包含引起c-截短的原纤维蛋白-1表达的突变的小鼠
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
WO2018096356A1 (en) * 2016-11-28 2018-05-31 Horizon Discovery Limited Methods for conditional gene knock-out
CA3049980A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
WO2019006034A1 (en) 2017-06-27 2019-01-03 Regeneron Pharmaceuticals, Inc. NON-HUMAN ANIMALS COMPRISING A HUMANIZED ASGR1 LOCUS
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
EP4423271A2 (en) 2021-10-28 2024-09-04 Regeneron Pharmaceuticals, Inc. Crispr/cas-related methods and compositions for knocking out c5
AU2022408167A1 (en) 2021-12-08 2024-06-06 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
EP4451863A1 (en) 2021-12-20 2024-10-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising humanized ace2 and tmprss loci
WO2023150798A1 (en) 2022-02-07 2023-08-10 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
AU2023269134A1 (en) 2022-05-09 2024-12-12 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
WO2023220697A1 (en) * 2022-05-12 2023-11-16 Memorial Sloan-Kettering Cancer Center Inducible recombinase systems
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
JP7635182B2 (ja) * 2022-08-04 2025-02-25 株式会社豊田中央研究所 相同組換え方法
EP4565603A1 (en) 2022-08-05 2025-06-11 Regeneron Pharmaceuticals, Inc. Aggregation-resistant variants of tdp-43
KR20250144399A (ko) 2023-02-01 2025-10-10 리제너론 파마슈티칼스 인코포레이티드 변형된 klhdc7b 유전자좌를 포함하는 동물
IL324966A (en) 2023-06-08 2026-01-01 Regeneron Pharma An animal model with rapid onset of Alzheimer's beta-amyloid plaque pathology
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing
WO2026102284A1 (en) 2024-11-08 2026-05-15 Regeneron Pharmaceuticals, Inc. Humanized stathmin2 allele to track human tdp-43-dependent splicing changes caused by tdp-43 pathology

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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
JP5908405B2 (ja) 2016-04-26
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
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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