KR20240135617A - 부위-특이적 유전자 편집을 위한 세린 재조합효소 시스템 - Google Patents

부위-특이적 유전자 편집을 위한 세린 재조합효소 시스템 Download PDF

Info

Publication number
KR20240135617A
KR20240135617A KR1020247025476A KR20247025476A KR20240135617A KR 20240135617 A KR20240135617 A KR 20240135617A KR 1020247025476 A KR1020247025476 A KR 1020247025476A KR 20247025476 A KR20247025476 A KR 20247025476A KR 20240135617 A KR20240135617 A KR 20240135617A
Authority
KR
South Korea
Prior art keywords
recombinase
cell
bxb1
variant
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
KR1020247025476A
Other languages
English (en)
Korean (ko)
Inventor
세바스티안 아란군디
프리드리히 에이. 포저
제프리 씨. 밀러
니콜라스 스칼롯
Original Assignee
상가모 테라퓨틱스, 인코포레이티드
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 상가모 테라퓨틱스, 인코포레이티드 filed Critical 상가모 테라퓨틱스, 인코포레이티드
Publication of KR20240135617A publication Critical patent/KR20240135617A/ko
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]
    • 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
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/22Endodeoxyribonucleases producing 3'-phosphomonoesters (3.1.22)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020247025476A 2022-01-28 2023-01-27 부위-특이적 유전자 편집을 위한 세린 재조합효소 시스템 Pending KR20240135617A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263304565P 2022-01-28 2022-01-28
US63/304,565 2022-01-28
PCT/US2023/061500 WO2023147507A1 (en) 2022-01-28 2023-01-27 Serine recombinase systems for site-specific gene editing

Publications (1)

Publication Number Publication Date
KR20240135617A true KR20240135617A (ko) 2024-09-11

Family

ID=85328719

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247025476A Pending KR20240135617A (ko) 2022-01-28 2023-01-27 부위-특이적 유전자 편집을 위한 세린 재조합효소 시스템

Country Status (9)

Country Link
US (1) US20250243512A1 (https=)
EP (1) EP4469569A1 (https=)
JP (1) JP2025504019A (https=)
KR (1) KR20240135617A (https=)
CN (1) CN118591625A (https=)
AU (1) AU2023214005A1 (https=)
CA (1) CA3243345A1 (https=)
IL (1) IL314478A (https=)
WO (1) WO2023147507A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121693564A (zh) 2023-06-26 2026-03-17 夏威夷大学 进化的整合酶和其用于基因组编辑的方法
WO2025160202A1 (en) * 2024-01-22 2025-07-31 Arc Researrch Institute Engineered large serine recombinases
WO2025212976A2 (en) * 2024-04-03 2025-10-09 Sangamo Therapeutics, Inc. Engineered modular recombinase compositions, methods, and systems for site-specific dna recombination

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB202006462D0 (en) * 2020-05-04 2020-06-17 Mote Res Limited Modifying genomes with integrase

Also Published As

Publication number Publication date
IL314478A (en) 2024-09-01
CA3243345A1 (en) 2023-08-03
JP2025504019A (ja) 2025-02-06
EP4469569A1 (en) 2024-12-04
CN118591625A (zh) 2024-09-03
WO2023147507A1 (en) 2023-08-03
AU2023214005A1 (en) 2024-08-15
US20250243512A1 (en) 2025-07-31

Similar Documents

Publication Publication Date Title
KR20240135617A (ko) 부위-특이적 유전자 편집을 위한 세린 재조합효소 시스템
JP6840796B2 (ja) Dna結合ドメインと切断ドメインとを連結するための組成物
JP2023510352A (ja) Pcsk9の標的化のための組成物および方法
CN109069568B (zh) 用于连接dna结合结构域和切割结构域的组合物
JP2021522273A (ja) Cns変性のための遺伝子治療法
AU2021391783A1 (en) Compositions and methods for the targeting of bcl11a
CA3173889A1 (en) Transposition-based therapies
JP2023551874A (ja) 筋強直性ジストロフィー1型を処置するためのrna標的化組成物および方法
CA3229345A1 (en) Closed-end dna production with inverted terminal repeat sequences
EP4240426A1 (en) Enhancement of predictable and template-free gene editing by the association of cas with dna polymerase
JP2025535368A (ja) 逆転写酵素を含む遺伝子編集システム
KR20240117153A (ko) 핵염기 편집을 위한 신규한 아연 핑거 융합 단백질
US20240335561A1 (en) A method for in vivo gene therapy to cure scd without myeloablative toxicity
WO2023220654A2 (en) Effector protein compositions and methods of use thereof
WO2025024285A1 (en) Compositions for the modification of the human c9orf72 gene
WO2024263707A1 (en) Compositions for the treatment of amyotrophic lateral sclerosis
WO2025072763A2 (en) Compositions and methods for precision editing with cas dimers
HK40076804A (en) Compositions and methods for the targeting of pcsk9
HK1262757B (en) Compositions for linking dna-binding domains and cleavage domains
HK1262757A1 (en) Compositions for linking dna-binding domains and cleavage domains

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000