JP7469806B2 - ゲノム編集方法、組成物、細胞、細胞製剤、及び細胞製剤の製造方法 - Google Patents

ゲノム編集方法、組成物、細胞、細胞製剤、及び細胞製剤の製造方法 Download PDF

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JP7469806B2
JP7469806B2 JP2020509319A JP2020509319A JP7469806B2 JP 7469806 B2 JP7469806 B2 JP 7469806B2 JP 2020509319 A JP2020509319 A JP 2020509319A JP 2020509319 A JP2020509319 A JP 2020509319A JP 7469806 B2 JP7469806 B2 JP 7469806B2
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homologous
cells
length
cell
dna
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JPWO2019189573A1 (ja
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豊 花園
弘真 原
英毅 魚崎
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Jichi Medical University
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
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    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/715—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
    • C07K14/7155—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons for interleukins [IL]
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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/108—Swine
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
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  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
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  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Environmental Sciences (AREA)
  • Developmental Biology & Embryology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Epidemiology (AREA)
  • Animal Husbandry (AREA)
JP2020509319A 2018-03-29 2019-03-28 ゲノム編集方法、組成物、細胞、細胞製剤、及び細胞製剤の製造方法 Active JP7469806B2 (ja)

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JP2018066174 2018-03-29
JP2018066174 2018-03-29
PCT/JP2019/013602 WO2019189573A1 (ja) 2018-03-29 2019-03-28 ゲノム編集方法、組成物、細胞、細胞製剤、及び細胞製剤の製造方法

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JPWO2019189573A1 JPWO2019189573A1 (ja) 2021-04-15
JPWO2019189573A5 JPWO2019189573A5 (https=) 2022-04-04
JP7469806B2 true JP7469806B2 (ja) 2024-04-17

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US (1) US20210071202A1 (https=)
EP (1) EP3778888A4 (https=)
JP (1) JP7469806B2 (https=)
CN (1) CN112534051B (https=)
WO (1) WO2019189573A1 (https=)

Families Citing this family (1)

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Publication number Priority date Publication date Assignee Title
CN116162621B (zh) * 2021-11-25 2025-09-16 中国科学院大连化学物理研究所 Dna双链序列及应用和肺腺癌细胞增殖抑制剂或在肺腺癌药物

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016183345A1 (en) 2015-05-13 2016-11-17 Seattle Children' S Hospital (Dba Seattle Children 's Research Institute) Enhancing endonuclease based gene editing in primary cells
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2731437T3 (es) 2014-11-21 2019-11-15 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida mediante el uso de pares de ARN guías
EP3280803B1 (en) * 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
JP6937740B2 (ja) * 2015-07-28 2021-09-22 ダニスコ・ユーエス・インク ゲノム編集システムおよび使用方法
WO2017096041A1 (en) * 2015-12-02 2017-06-08 The Regents Of The University Of California Compositions and methods for modifying a target nucleic acid
JP6776090B2 (ja) 2016-10-19 2020-10-28 アルプスアルパイン株式会社 位置判定装置、位置判定方法及びプログラム並びにキーレスエントリーシステム

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016183345A1 (en) 2015-05-13 2016-11-17 Seattle Children' S Hospital (Dba Seattle Children 's Research Institute) Enhancing endonuclease based gene editing in primary cells
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
HENDEL, Ayal et al.，Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing，Cell Rep.，2014年，Vol. 7，pp. 293-305
MORGAN, Richard A. et al.，Hematopoietic Stem Cell Gene Therapy : Progress and Lessons Learned，Cell Stem Cell，2017年，Vol. 21，pp. 574-590
SUZUKI, Keiichiro et al.，In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration，Nature，2016年，Vol. 540，pp. 144-149
ZHANG, Jian-Ping et al.，Efficient precise knockin with a double cut HDR donor after CRISPR/Cas9-mediated double-stranded DNA cleavage，Genome Biology，2017年，Vol.18: 35，pp. 1-18

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Publication number Publication date
EP3778888A1 (en) 2021-02-17
WO2019189573A1 (ja) 2019-10-03
CN112534051A (zh) 2021-03-19
US20210071202A1 (en) 2021-03-11
CN112534051B (zh) 2025-07-29
JPWO2019189573A1 (ja) 2021-04-15
EP3778888A4 (en) 2022-04-13

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