PT3147362T - Alelos multifuncionais - Google Patents

Alelos multifuncionais

Info

Publication number
PT3147362T
PT3147362T PT16196614T PT16196614T PT3147362T PT 3147362 T PT3147362 T PT 3147362T PT 16196614 T PT16196614 T PT 16196614T PT 16196614 T PT16196614 T PT 16196614T PT 3147362 T PT3147362 T PT 3147362T
Authority
PT
Portugal
Prior art keywords
alleles
multifunctional
multifunctional alleles
Prior art date
Application number
PT16196614T
Other languages
English (en)
Inventor
J Murphy Andrew
N Economides Aris
Matthew Lengyel Peter
H A Yang Peter
Original Assignee
Regeneron Pharma
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 Pharma filed Critical Regeneron Pharma
Publication of PT3147362T publication Critical patent/PT3147362T/pt

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)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (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)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PT16196614T 2009-10-29 2010-10-29 Alelos multifuncionais PT3147362T (pt)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
PT3147362T true PT3147362T (pt) 2019-04-02

Family

ID=43244777

Family Applications (2)

Application Number Title Priority Date Filing Date
PT16196614T PT3147362T (pt) 2009-10-29 2010-10-29 Alelos multifuncionais
PT107743916T PT2494047T (pt) 2009-10-29 2010-10-29 Alelos multifuncionais

Family Applications After (1)

Application Number Title Priority Date Filing Date
PT107743916T PT2494047T (pt) 2009-10-29 2010-10-29 Alelos multifuncionais

Country Status (13)

Country Link
US (4) US20110104799A1 (pt)
EP (2) EP2494047B1 (pt)
JP (3) JP5908405B2 (pt)
CN (2) CN102666854B (pt)
AU (1) AU2010319894B2 (pt)
CA (1) CA2779858C (pt)
CY (2) CY1118659T1 (pt)
DK (2) DK2494047T3 (pt)
ES (2) ES2716981T3 (pt)
PL (2) PL2494047T3 (pt)
PT (2) PT3147362T (pt)
TR (1) TR201903376T4 (pt)
WO (1) WO2011059799A1 (pt)

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TR201903376T4 (tr) 2009-10-29 2019-04-22 Regeneron Pharma Çok fonksiyonlu alleller.
SG11201406547YA (en) 2012-04-25 2014-11-27 Regeneron Pharma Nuclease-mediated targeting with large targeting vectors
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
DK3178928T3 (da) * 2013-08-07 2020-08-24 Regeneron Pharma Pint-knockout-mus med en præmatur, aldersassocieret fænotype
RU2685914C1 (ru) 2013-12-11 2019-04-23 Регенерон Фармасьютикалс, Инк. Способы и композиции для направленной модификации генома
EP3460063B1 (en) 2013-12-11 2024-03-13 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
KR102374379B1 (ko) 2014-06-06 2022-03-17 리제너론 파마슈티칼스 인코포레이티드 표적화된 좌를 변형시키는 방법 및 조성물
EP3155099B1 (en) 2014-06-23 2018-03-21 Regeneron Pharmaceuticals, Inc. Nuclease-mediated dna assembly
ES2901074T3 (es) 2014-06-26 2022-03-21 Regeneron Pharma Métodos y composiciones para modificaciones genéticas objetivo y métodos de uso
MX2017004890A (es) 2014-10-15 2018-02-12 Regeneron Pharma Metodos y composiciones para la generación o el mantenimiento de células pluripotentes.
NZ731962A (en) 2014-11-21 2022-07-01 Regeneron Pharma Methods and compositions for targeted genetic modification using paired guide rnas
MX2017008190A (es) 2014-12-19 2018-03-23 Regeneron Pharma Metodos y composiciones para la modificacion genetica dirigida a traves de direccionamiento multiple de una sola etapa.
EP3270688B1 (en) 2015-03-16 2020-08-12 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
MX2018014172A (es) 2016-05-20 2019-08-22 Regeneron Pharma Métodos para romper la tolerancia inmunológica usando múltiples arn guías.
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
RU2019126483A (ru) 2017-01-23 2021-02-24 Ридженерон Фармасьютикалз, Инк. Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение
SG11201911886PA (en) 2017-06-27 2020-01-30 Regeneron Pharma Non-human animals comprising a humanized asgr1 locus
US20190098879A1 (en) 2017-09-29 2019-04-04 Regeneron Pharmaceuticals, Inc. Non-Human Animals Comprising A Humanized TTR Locus And Methods Of Use
KR102444458B1 (ko) 2017-11-10 2022-09-19 리제너론 파마슈티칼스 인코포레이티드 Slc30a8 돌연변이를 포함하는 비인간 동물 및 사용 방법
EP4299732A3 (en) 2017-11-30 2024-03-27 Regeneron Pharmaceuticals, Inc. Rats comprising a humanized trkb locus
MX2020013545A (es) 2018-06-13 2021-02-26 Regeneron Pharma Modelo en roedores de fibrodisplasia osificante progresiva.
EP3732291A1 (en) 2018-12-20 2020-11-04 Regeneron Pharmaceuticals, Inc. Nuclease-mediated repeat expansion
AU2020256225A1 (en) 2019-04-03 2021-09-02 Regeneron Pharmaceuticals, Inc. Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
CA3133360A1 (en) 2019-04-04 2020-10-08 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized coagulation factor 12 locus
CN113874510A (zh) 2019-06-04 2021-12-31 瑞泽恩制药公司 包括具有β滑移突变的人源化TTR基因座的非人动物和使用方法
KR20220017939A (ko) 2019-06-07 2022-02-14 리제너론 파마슈티칼스 인코포레이티드 인간화 알부민 좌위를 포함하는 비-인간 동물
AU2020294880B2 (en) * 2019-06-19 2024-05-02 F. Hoffmann-La Roche Ag Method for the generation of a protein expressing cell by targeted integration using Cre mRNA
CN114746125A (zh) 2019-11-08 2022-07-12 瑞泽恩制药公司 用于x连锁青少年型视网膜劈裂症疗法的crispr和aav策略
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
US20230081547A1 (en) 2020-02-07 2023-03-16 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized klkb1 locus and methods of use
WO2021195079A1 (en) 2020-03-23 2021-09-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
WO2021263146A2 (en) 2020-06-26 2021-12-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ace2 locus
KR20230119130A (ko) 2020-12-14 2023-08-16 리제너론 파마슈티칼스 인코포레이티드 인히빈 서브유닛 베타 e(inhbe) 억제제로 대사 장애 및 심혈관 질환을 치료하는 방법
US20230293727A1 (en) 2021-10-26 2023-09-21 Regeneron Pharmaceuticals, Inc. Overexpression of lemd2, lemd3, or chmp7 as a therapeutic modality for tauopathy
WO2023077053A2 (en) 2021-10-28 2023-05-04 Regeneron Pharmaceuticals, Inc. Crispr/cas-related methods and compositions for knocking out c5
WO2023108047A1 (en) 2021-12-08 2023-06-15 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
WO2023122506A1 (en) 2021-12-20 2023-06-29 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
WO2023220603A1 (en) 2022-05-09 2023-11-16 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
JP2024021835A (ja) * 2022-08-04 2024-02-16 株式会社豊田中央研究所 相同組換え方法
WO2024031053A1 (en) 2022-08-05 2024-02-08 Regeneron Pharmaceuticals, Inc. Aggregation-resistant variants of tdp-43

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Also Published As

Publication number Publication date
US20110104799A1 (en) 2011-05-05
EP3147362B1 (en) 2019-01-02
EP3147362A1 (en) 2017-03-29
AU2010319894B2 (en) 2015-03-05
JP6333316B2 (ja) 2018-05-30
DK3147362T3 (en) 2019-04-08
CN106191126A (zh) 2016-12-07
EP2494047A1 (en) 2012-09-05
ES2716981T3 (es) 2019-06-18
CA2779858C (en) 2019-10-29
DK2494047T3 (en) 2017-04-10
PT2494047T (pt) 2017-02-08
US20220220510A1 (en) 2022-07-14
EP2494047B1 (en) 2017-01-04
US11319557B2 (en) 2022-05-03
AU2010319894A1 (en) 2012-05-31
JP2016063839A (ja) 2016-04-28
CN106191126B (zh) 2020-04-14
TR201903376T4 (tr) 2019-04-22
CY1118659T1 (el) 2017-07-12
ES2613662T3 (es) 2017-05-25
CN102666854A (zh) 2012-09-12
US20130302899A1 (en) 2013-11-14
CN102666854B (zh) 2016-08-24
JP2016185166A (ja) 2016-10-27
JP5908405B2 (ja) 2016-04-26
US10392633B2 (en) 2019-08-27
JP6085696B2 (ja) 2017-02-22
CA2779858A1 (en) 2011-05-19
PL2494047T3 (pl) 2017-05-31
CY1121457T1 (el) 2020-05-29
JP2013509192A (ja) 2013-03-14
WO2011059799A1 (en) 2011-05-19
PL3147362T3 (pl) 2019-06-28
US20190390230A1 (en) 2019-12-26

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