PL3147362T3 - Allele wielofunkcyjne - Google Patents

Allele wielofunkcyjne

Info

Publication number
PL3147362T3
PL3147362T3 PL16196614T PL16196614T PL3147362T3 PL 3147362 T3 PL3147362 T3 PL 3147362T3 PL 16196614 T PL16196614 T PL 16196614T PL 16196614 T PL16196614 T PL 16196614T PL 3147362 T3 PL3147362 T3 PL 3147362T3
Authority
PL
Poland
Prior art keywords
allels
multifunctional
multifunctional allels
Prior art date
Application number
PL16196614T
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 PL3147362T3 publication Critical patent/PL3147362T3/pl

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/15Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/20Animal model comprising regulated expression system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/30Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/60Vectors containing traps for, e.g. exons, promoters
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/44Vectors 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)
PL16196614T 2009-10-29 2010-10-29 Allele wielofunkcyjne PL3147362T3 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US25607809P 2009-10-29 2009-10-29
PCT/US2010/054654 WO2011059799A1 (en) 2009-10-29 2010-10-29 Multifunctional alleles
EP16196614.8A EP3147362B1 (en) 2009-10-29 2010-10-29 Multifunctional alleles
EP10774391.6A EP2494047B1 (en) 2009-10-29 2010-10-29 Multifunctional alleles

Publications (1)

Publication Number Publication Date
PL3147362T3 true PL3147362T3 (pl) 2019-06-28

Family

ID=43244777

Family Applications (2)

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

Family Applications Before (1)

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

Country Status (13)

Country Link
US (4) US20110104799A1 (pl)
EP (2) EP3147362B1 (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) PL2494047T3 (pl)
PT (2) PT2494047T (pl)
TR (1) TR201903376T4 (pl)
WO (1) WO2011059799A1 (pl)

Families Citing this family (51)

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PT2494047T (pt) 2009-10-29 2017-02-08 Regeneron Pharma Alelos multifuncionais
RU2645475C2 (ru) 2012-04-25 2018-02-21 Регенерон Фармасьютикалз, Инк. Опосредованное нуклеазой нацеливание с большими нацеливающими векторами
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
DK2986729T3 (en) 2013-04-16 2018-10-29 Regeneron Pharma TARGETED MODIFICATION OF ROOT THROUGH
EP3178928B1 (en) * 2013-08-07 2020-06-10 Regeneron Pharmaceuticals, Inc. Pint knockout mouse exhibiting a premature aging-associated phenotype
WO2015088643A1 (en) 2013-12-11 2015-06-18 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
PT3080279T (pt) 2013-12-11 2018-12-17 Regeneron Pharma Métodos e composições para a modificação direcionada de um genoma
RU2704283C9 (ru) 2014-06-06 2020-02-07 Регенерон Фармасьютикалз, Инк. Способы и композиции для модификации целевого локуса
RS60366B1 (sr) 2014-06-23 2020-07-31 Regeneron Pharma Sastavljanje dnk posredovano nukleazom
PT3161128T (pt) 2014-06-26 2018-11-21 Regeneron Pharma Métodos e composições para modificações genéticas alvejadas e métodos de utilização
SG11201702309RA (en) 2014-10-15 2017-04-27 Regeneron Pharma Methods and compositions for generating or maintaining pluripotent cells
CA2968440A1 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
BR112017013104A2 (en) 2014-12-19 2018-05-15 Regeneron Pharmaceuticals, Inc. methods for modifying a target genomic locus in a cell, for enhancing homologous recombination at a target genomic locus in a cell, and for producing an f0 generation from a nonhuman animal.
AU2016233250C1 (en) 2015-03-16 2022-08-18 Regeneron Pharmaceuticals, Inc. Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception
WO2017180669A1 (en) * 2016-04-11 2017-10-19 Applied Stemcell, Inc. Site-specific integration of transgenes
US20170332610A1 (en) 2016-05-20 2017-11-23 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
KR20190041476A (ko) 2016-07-29 2019-04-22 리제너론 파마슈티칼스 인코포레이티드 C-절단된 피브릴린-1의 발현을 유도하는 돌연변이를 포함하는 마우스
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
SG11201906735RA (en) 2017-01-23 2019-08-27 Regeneron Pharma Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
EP4484562A3 (en) 2017-06-27 2025-03-26 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized asgr1 locus
JP7139419B2 (ja) 2017-09-29 2022-09-20 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ttr遺伝子座を含む非ヒト動物および使用方法
ES2911249T3 (es) 2017-11-10 2022-05-18 Regeneron Pharma Animales no humanos que comprenden una mutación de slc30a8 y métodos de uso
IL274740B2 (en) 2017-11-30 2024-06-01 Regeneron Pharma Non-human animals comprising a humanized trkb locus
US11419319B2 (en) 2018-06-13 2022-08-23 Regeneran Pharmaceuticals, Inc. Genetically modified rodent with an inducible ACVR1 mutation in exon 7 that causes ectopic bone formation
US11690362B2 (en) 2018-12-20 2023-07-04 Regeneran Pharmaceuticals, Inc. Nuclease-mediated repeat expansion
JP7524214B2 (ja) 2019-04-03 2024-07-29 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 抗体コード配列をセーフハーバー遺伝子座に挿入するための方法および組成物
ES2966625T3 (es) 2019-04-04 2024-04-23 Regeneron Pharma Roedores que comprenden un locus del factor de coagulación 12 humanizado
US11891618B2 (en) 2019-06-04 2024-02-06 Regeneron Pharmaceuticals, Inc. Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use
CA3137764A1 (en) 2019-06-07 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
BR112021025425A2 (pt) * 2019-06-19 2022-02-01 Hoffmann La Roche Método para produzir uma célula de mamífero recombinante e uso de mrna de recombinase cre
JP7756639B2 (ja) 2019-11-08 2025-10-20 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 X連鎖性若年網膜分離療法のためのcrisprおよびaav戦略
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
CN115175559A (zh) 2020-01-28 2022-10-11 瑞泽恩制药公司 包含人源化pnpla3基因座的非人动物及其使用方法
WO2021158883A1 (en) 2020-02-07 2021-08-12 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized klkb1 locus and methods of use
EP4125348A1 (en) 2020-03-23 2023-02-08 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
US20230232796A1 (en) 2020-06-26 2023-07-27 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ace2 locus
MX2023007012A (es) 2020-12-14 2023-06-27 Regeneron Pharma Metodos para tratar los trastornos metabolicos y las enfermedades cardiovasculares con inhibidores de la subunidad beta e de la inhibina (inhbe).
EP4422661A1 (en) 2021-10-26 2024-09-04 Regeneron Pharmaceuticals, Inc. Overexpression of lemd2, lemd3, or chmp7 as a therapeutic modality for tauopathy
JP2024540086A (ja) 2021-10-28 2024-10-31 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 C5をノックアウトするためのcrispr/cas関連方法及び組成物
US12582104B2 (en) 2021-12-08 2026-03-24 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
EP4475671A1 (en) 2022-02-07 2024-12-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
EP4522726A1 (en) 2022-05-09 2025-03-19 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
KR20250044256A (ko) 2022-08-05 2025-03-31 리제너론 파마슈티칼스 인코포레이티드 Tdp-43의 응집 저항성 변이체
WO2024163650A1 (en) 2023-02-01 2024-08-08 Regeneron Pharmaceuticals, Inc. Animals comprising a modified klhdc7b locus
AU2024285988A1 (en) 2023-06-08 2025-11-20 Regeneron Pharmaceuticals, Inc. Animal model with rapid onset of alzheimer's amyloid beta plaque pathology
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing

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US7582741B2 (en) * 2004-07-26 2009-09-01 University Of Massachusetts Conditional disruption of dicer1 in cell lines and non-human mammals
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PT2494047T (pt) 2009-10-29 2017-02-08 Regeneron Pharma Alelos multifuncionais

Also Published As

Publication number Publication date
US20130302899A1 (en) 2013-11-14
CY1118659T1 (el) 2017-07-12
JP2013509192A (ja) 2013-03-14
US10392633B2 (en) 2019-08-27
EP3147362A1 (en) 2017-03-29
PL2494047T3 (pl) 2017-05-31
CN102666854A (zh) 2012-09-12
EP2494047B1 (en) 2017-01-04
CA2779858C (en) 2019-10-29
US20220220510A1 (en) 2022-07-14
US20190390230A1 (en) 2019-12-26
US12473574B2 (en) 2025-11-18
JP6085696B2 (ja) 2017-02-22
WO2011059799A1 (en) 2011-05-19
DK2494047T3 (en) 2017-04-10
JP5908405B2 (ja) 2016-04-26
CA2779858A1 (en) 2011-05-19
US11319557B2 (en) 2022-05-03
PT2494047T (pt) 2017-02-08
CN106191126A (zh) 2016-12-07
EP2494047A1 (en) 2012-09-05
JP2016185166A (ja) 2016-10-27
CN106191126B (zh) 2020-04-14
DK3147362T3 (en) 2019-04-08
JP6333316B2 (ja) 2018-05-30
EP3147362B1 (en) 2019-01-02
ES2716981T3 (es) 2019-06-18
TR201903376T4 (tr) 2019-04-22
ES2613662T3 (es) 2017-05-25
CN102666854B (zh) 2016-08-24
AU2010319894A1 (en) 2012-05-31
CY1121457T1 (el) 2020-05-29
PT3147362T (pt) 2019-04-02
JP2016063839A (ja) 2016-04-28
AU2010319894B2 (en) 2015-03-05
US20110104799A1 (en) 2011-05-05

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