CN112534051B - 基因组编辑方法、组合物、细胞、细胞制剂、以及细胞制剂的制造方法 - Google Patents

基因组编辑方法、组合物、细胞、细胞制剂、以及细胞制剂的制造方法

Info

Publication number
CN112534051B
CN112534051B CN201980034209.0A CN201980034209A CN112534051B CN 112534051 B CN112534051 B CN 112534051B CN 201980034209 A CN201980034209 A CN 201980034209A CN 112534051 B CN112534051 B CN 112534051B
Authority
CN
China
Prior art keywords
cells
homologous recombination
dna
cell
genome
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CN201980034209.0A
Other languages
English (en)
Chinese (zh)
Other versions
CN112534051A (zh
Inventor
花园丰
原弘真
鱼崎英毅
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Jichi Medical University
Original Assignee
Jichi Medical University
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 Jichi Medical University filed Critical Jichi Medical University
Publication of CN112534051A publication Critical patent/CN112534051A/zh
Application granted granted Critical
Publication of CN112534051B publication Critical patent/CN112534051B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/715Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
    • C07K14/7155Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons for interleukins [IL]
    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0647Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10Cells modified by introduction of foreign genetic material
    • 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
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/072Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/108Swine
    • 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
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • 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)
CN201980034209.0A 2018-03-29 2019-03-28 基因组编辑方法、组合物、细胞、细胞制剂、以及细胞制剂的制造方法 Active CN112534051B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2018066174 2018-03-29
JP2018-066174 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

Publications (2)

Publication Number Publication Date
CN112534051A CN112534051A (zh) 2021-03-19
CN112534051B true CN112534051B (zh) 2025-07-29

Family

ID=68060174

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980034209.0A Active CN112534051B (zh) 2018-03-29 2019-03-28 基因组编辑方法、组合物、细胞、细胞制剂、以及细胞制剂的制造方法

Country Status (5)

Country Link
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)

* Cited by examiner, † Cited by third party
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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107787367A (zh) * 2015-04-06 2018-03-09 里兰斯坦福初级大学理事会 用于crispr/cas介导的基因调控的化学修饰的引导rna

Family Cites Families (6)

* 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
US20180119174A1 (en) * 2015-05-13 2018-05-03 Seattle Children's Hospita (dba Seattle Children's Research Institute Enhancing endonuclease based gene editing in primary cells
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
US11866726B2 (en) * 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107787367A (zh) * 2015-04-06 2018-03-09 里兰斯坦福初级大学理事会 用于crispr/cas介导的基因调控的化学修饰的引导rna

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Efficient precise knockin with a double cut HDR donor after CRISPR/Cas9-mediated double-stranded DNA cleavage;Jian-Ping Zhang等;Genome Biology;20170220;第18卷(第1期);第4页左栏第2段、图2 *
Hematopoietic stem cell gne therapy:progress and lesions learned;Richard A Morgan等;cell stem cell;20171102;第21卷(第5期);摘要、第9页最后1段、图3 *

Also Published As

Publication number Publication date
EP3778888A1 (en) 2021-02-17
JP7469806B2 (ja) 2024-04-17
WO2019189573A1 (ja) 2019-10-03
CN112534051A (zh) 2021-03-19
US20210071202A1 (en) 2021-03-11
JPWO2019189573A1 (ja) 2021-04-15
EP3778888A4 (en) 2022-04-13

Similar Documents

Publication Publication Date Title
US12529039B2 (en) Method of insterting a tranposon into nucleic acids
JP5175814B2 (ja) ヒト人工染色体(hac)ベクター
ES2980050T3 (es) Método para fabricar células eucariotas editadas con ADN
JP7197363B2 (ja) ヌクレアーゼを使用するヒト神経幹細胞のゲノム編集
JP6772067B2 (ja) 哺乳動物の標的ゲノム領域にdnaをノックインする方法及び細胞
EP3673732A2 (en) Engineering of humanized car t-cells and platelets by genetic complementation
JP2021505187A (ja) 遺伝子編集のためのcpf1関連方法及び組成物
EP4051792A1 (en) Pam-reduced and pam-abolished cas derivatives compositions and uses thereof in genetic modulation
WO2008013067A1 (fr) Vecteur de chromosome artificiel humain (cah), et substance pharmaceutique à base de cellules humaines contenant un vecteur de chromosome artificiel humain (cah)
Zhao et al. Generation of transgenic cloned buffalo embryos harboring the EGFP gene in the Y chromosome using CRISPR/Cas9-mediated targeted integration
CN112534051B (zh) 基因组编辑方法、组合物、细胞、细胞制剂、以及细胞制剂的制造方法
Liu et al. Electroporation-based Easi-CRISPR yields biallelic insertions of EGFP-HiBiT cassette in immortalized chicken oviduct epithelial cells
US20240301349A1 (en) Compositions and methods for improving embryo development
HK40041694A (en) Genome editing method, composition, cell, cell preparation, and method for producing cell preparation
HK40041694B (zh) 基因组编辑方法、组合物、细胞、细胞制剂、以及细胞制剂的制造方法
US20240374758A1 (en) Homology dna repair enhancement of cas derivatives compositions and uses thereof in genetic modulation
JP2022531930A (ja) 栄養要求性調節可能な細胞を使用する方法および組成物
KR102472344B1 (ko) 프라임 편집을 이용한 개의 고관절 이형성증 치료용 조성물
CN117363649A (zh) 基因整合方法及应用
CN120290568A (zh) 一种Pcdh15基因增强子及其应用

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40041694

Country of ref document: HK

GR01 Patent grant
GR01 Patent grant