JP2025510574A - 真核生物遺伝子編集のための組成物、システムおよび方法 - Google Patents

真核生物遺伝子編集のための組成物、システムおよび方法 Download PDF

Info

Publication number
JP2025510574A
JP2025510574A JP2024553395A JP2024553395A JP2025510574A JP 2025510574 A JP2025510574 A JP 2025510574A JP 2024553395 A JP2024553395 A JP 2024553395A JP 2024553395 A JP2024553395 A JP 2024553395A JP 2025510574 A JP2025510574 A JP 2025510574A
Authority
JP
Japan
Prior art keywords
cells
protein
sequence
coding sequence
dna
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.)
Pending
Application number
JP2024553395A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025510574A5 (https=
Inventor
バイソン ルー,
アンソニー アタラ,
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.)
Wake Forest University Health Sciences
Original Assignee
Wake Forest University Health Sciences
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 Wake Forest University Health Sciences filed Critical Wake Forest University Health Sciences
Publication of JP2025510574A publication Critical patent/JP2025510574A/ja
Publication of JP2025510574A5 publication Critical patent/JP2025510574A5/ja
Pending legal-status Critical Current

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
    • 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]
    • C12N9/222Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226Class 2 CAS enzyme complex, e.g. single CAS protein
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • 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
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241Nucleotidyltransferases (2.7.7)
    • C12N9/1252DNA-directed DNA polymerase (2.7.7.7), i.e. DNA replicase
    • 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]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07Nucleotidyltransferases (2.7.7)
    • C12Y207/07007DNA-directed DNA polymerase (2.7.7.7), i.e. DNA replicase
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • 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
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • 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]
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2795/00Bacteriophages
    • C12N2795/00011Details
    • C12N2795/10011Details dsDNA Bacteriophages
    • C12N2795/10311Siphoviridae
    • C12N2795/10322New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2795/00Bacteriophages
    • C12N2795/00011Details
    • C12N2795/18011Details ssRNA Bacteriophages positive-sense
    • C12N2795/18111Leviviridae
    • C12N2795/18122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2024553395A 2022-03-08 2023-03-08 真核生物遺伝子編集のための組成物、システムおよび方法 Pending JP2025510574A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263317720P 2022-03-08 2022-03-08
US63/317,720 2022-03-08
PCT/US2023/063954 WO2023172966A1 (en) 2022-03-08 2023-03-08 Compositions, systems and methods for eukaryotic gene editing

Publications (2)

Publication Number Publication Date
JP2025510574A true JP2025510574A (ja) 2025-04-15
JP2025510574A5 JP2025510574A5 (https=) 2026-03-16

Family

ID=87935949

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024553395A Pending JP2025510574A (ja) 2022-03-08 2023-03-08 真核生物遺伝子編集のための組成物、システムおよび方法

Country Status (6)

Country Link
EP (1) EP4489772A4 (https=)
JP (1) JP2025510574A (https=)
KR (1) KR20240155953A (https=)
AU (1) AU2023232138A1 (https=)
CA (1) CA3245468A1 (https=)
WO (1) WO2023172966A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025072446A1 (en) * 2023-09-27 2025-04-03 Keith Brown Cell specific activation

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2004225520A1 (en) * 2003-03-25 2004-10-14 Stratagene DNA polymerase fusions and uses thereof
US20120202276A1 (en) * 2010-02-26 2012-08-09 Life Technologies Corporation Modified Proteins and Methods of Making and Using Same
EP3788155A1 (en) * 2018-05-01 2021-03-10 Wake Forest University Health Sciences Lentiviral-based vectors and related systems and methods for eukaryotic gene editing
US10947517B2 (en) * 2019-02-15 2021-03-16 Sigma-Aldrich Co. Llc CRISPR/Cas fusion proteins and systems
WO2020191233A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences

Also Published As

Publication number Publication date
KR20240155953A (ko) 2024-10-29
WO2023172966A1 (en) 2023-09-14
AU2023232138A1 (en) 2024-09-26
EP4489772A1 (en) 2025-01-15
EP4489772A4 (en) 2026-03-25
CA3245468A1 (en) 2023-09-14

Similar Documents

Publication Publication Date Title
JP7832700B2 (ja) 真核生物の遺伝子編集のためのレンチウイルスベースのベクターならびに関連システムおよび方法
JP7472121B2 (ja) アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法
CN114230675B (zh) Rna指导的基因编辑和基因调节
AU2022202558A1 (en) Compositions and methods for the treatment of hemoglobinopathies
JP2019530467A (ja) 安全性向上のための自己制限Cas9回路網(SLiCES)プラスミドおよびそのレンチウイルス系
CN113272428A (zh) 核酸构建体和使用方法
JP2021522783A5 (https=)
JP2023515710A (ja) CRISPR媒介式エクソン欠失用の最適なgRNA対を発見するためのハイスループットスクリーニング法
WO2019173248A1 (en) Engineered nucleic acid-targeting nucleic acids
JP2025510574A (ja) 真核生物遺伝子編集のための組成物、システムおよび方法
US20230405116A1 (en) Vectors, systems and methods for eukaryotic gene editing
CN111518812B (zh) 一种编辑绵羊FGF5基因实现选择性剪接的sgRNA、成套核酸分子和应用
KR20260032655A (ko) Hbv 유전자 발현의 후성유전학적 조절을 위한 조성물 및 방법
KR20250084928A (ko) 유전자 전달 효율이 향상된 트랜스포사제 돌연변이
WO2025096936A2 (en) Use of prime editing in correcting mutations in cdkl5
KR20250005273A (ko) CAS-PLUS 변이체를 사용하는 DNA 폴리머라제의 변이체에 의한 CRISPR-Cas 유도된 유전자 편집의 안전성과 정밀성의 향상
WO2026085201A2 (en) Base editing to correct inborn errors of immunity
WO2025076306A1 (en) Prime editors having improved prime editing efficiency
HK40071050B (zh) Rna指导的基因编辑和基因调节
WO2025049792A1 (en) Compositions and methods for epigenetic regulation of tgfbr2 expression
WO2025049877A1 (en) Chemo-sensitive dominant clone for adaptive therapy
CN120897761A (zh) 用于使用基因组工程产生调节性t细胞(treg)的方法
HK40126455A (zh) Rna指导的基因编辑和基因调节
HK40061317A (en) Compositions and methods for transgene expression from an albumin locus
HK40051560A (en) Nucleic acid constructs and methods of use

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260306

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20260306