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)
  • 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)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (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
PL16196614T PL3147362T3 (pl) 2009-10-29 2010-10-29 Allele wielofunkcyjne
PL10774391T PL2494047T3 (pl) 2009-10-29 2010-10-29 Allele wielofunkcyjne

Family Applications After (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) EP2494047B1 (pl)
JP (3) JP5908405B2 (pl)
CN (2) CN106191126B (pl)
AU (1) AU2010319894B2 (pl)
CA (1) CA2779858C (pl)
CY (2) CY1118659T1 (pl)
DK (2) DK2494047T3 (pl)
ES (2) ES2716981T3 (pl)
PL (2) PL3147362T3 (pl)
PT (2) PT3147362T (pl)
TR (1) TR201903376T4 (pl)
WO (1) WO2011059799A1 (pl)

Families Citing this family (51)

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CA2779858C (en) 2009-10-29 2019-10-29 Regeneron Pharmaceuticals, Inc. Multifunctional alleles
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
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
MY198884A (en) * 2013-08-07 2023-10-02 Regeneron Pharma Lincrna-deficient non-human animals
KR102170502B1 (ko) 2013-12-11 2020-10-28 리제너론 파마슈티칼스 인코포레이티드 게놈의 표적화된 변형을 위한 방법 및 조성물
EP3080279B1 (en) 2013-12-11 2018-09-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
HRP20200529T1 (hr) 2014-06-06 2020-09-04 Regeneron Pharmaceuticals, Inc. Postupci i pripravci za modificiranje ciljnog lokusa
ES2781323T3 (es) 2014-06-23 2020-09-01 Regeneron Pharma Montaje de ADN mediado por nucleasas
SI3161128T1 (sl) 2014-06-26 2019-02-28 Regeneron Pharmaceuticals, Inc. Postopki in sestavki za ciljano genetsko spremembo in postopki uporabe
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.
KR102531016B1 (ko) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물
ES2947714T3 (es) 2014-12-19 2023-08-17 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida a través de múltiples direccionamientos en un solo paso
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
CN108884475A (zh) * 2016-04-11 2018-11-23 应用干细胞有限公司 转基因的位点特异性整合
CN113831407B (zh) 2016-05-20 2024-06-11 瑞泽恩制药公司 用于使用多个引导rna来破坏免疫耐受性的方法
JP2019523009A (ja) 2016-07-29 2019-08-22 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Regeneron Pharmaceuticals, Inc. C末端切断型フィブリリン−1の発現をもたらす変異を有するマウス
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
ES2996886T3 (en) 2017-06-27 2025-02-13 Regeneron Pharma Rodents 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
JP7097440B2 (ja) 2017-11-10 2022-07-07 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 Slc30a8変異を含む非ヒト動物および使用方法
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
CA3099441A1 (en) 2018-06-13 2019-12-19 Regeneron Pharmaceuticals, Inc. A rodent model of fibrodysplasia ossificans progressiva
KR102925054B1 (ko) 2018-12-20 2026-02-10 리제너론 파마슈티칼스 인코포레이티드 뉴클레아제-매개 반복부 팽창
AU2020256225B9 (en) 2019-04-03 2025-04-10 Regeneron Pharmaceuticals, Inc. Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
KR102661779B1 (ko) 2019-04-04 2024-04-30 리제너론 파마슈티칼스 인코포레이티드 인간화 응고 인자 12 좌위를 포함하는 비-인간 동물
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
MX2021015122A (es) 2019-06-07 2022-04-06 Regeneron Pharma Animales no humanos que comprenden un locus de albumina humanizado.
CN118547004A (zh) * 2019-06-19 2024-08-27 豪夫迈·罗氏有限公司 通过使用Cre mRNA进行的靶向整合来产生蛋白质表达细胞的方法
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
WO2021263146A2 (en) 2020-06-26 2021-12-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ace2 locus
CA3201466A1 (en) 2020-12-14 2022-06-23 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
US20240415980A1 (en) 2021-10-28 2024-12-19 Regeneron Pharmaceuticals, Inc. Crispr/cas-related methods and compositions for knocking out c5
KR20240117571A (ko) 2021-12-08 2024-08-01 리제너론 파마슈티칼스 인코포레이티드 돌연변이 마이오실린 질환 모델 및 이의 용도
US20250049006A1 (en) 2021-12-20 2025-02-13 C/O 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
US20250302998A1 (en) 2022-05-09 2025-10-02 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
JP2025525745A (ja) 2022-08-05 2025-08-07 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 Tdp-43の凝集耐性バリアント
AU2024214456A1 (en) 2023-02-01 2025-07-03 Regeneron Pharmaceuticals, Inc. Animals comprising a modified klhdc7b locus
WO2024254376A1 (en) 2023-06-08 2024-12-12 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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Also Published As

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

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