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WO2025010350A3 - Compositions et procédés d'édition précise du génome à l'aide de rétrons - Google Patents

Compositions et procédés d'édition précise du génome à l'aide de rétrons Download PDF

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Publication number
WO2025010350A3
WO2025010350A3 PCT/US2024/036763 US2024036763W WO2025010350A3 WO 2025010350 A3 WO2025010350 A3 WO 2025010350A3 US 2024036763 W US2024036763 W US 2024036763W WO 2025010350 A3 WO2025010350 A3 WO 2025010350A3
Authority
WO
WIPO (PCT)
Prior art keywords
retron
retrons
compositions
methods
genome editing
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
PCT/US2024/036763
Other languages
English (en)
Other versions
WO2025010350A2 (fr
Inventor
Ilya J. FINKELSTEIN
Hung-Che KUO
Kuang Hu
Jesse BUFFINGTON
Kamyab JAVANMANDI
You-Chiun CHANG
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.)
University of Texas System
University of Texas at Austin
Original Assignee
University of Texas System
University of Texas at Austin
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 University of Texas System, University of Texas at Austin filed Critical University of Texas System
Publication of WO2025010350A2 publication Critical patent/WO2025010350A2/fr
Publication of WO2025010350A3 publication Critical patent/WO2025010350A3/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • 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]
    • 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]

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

Les éditeurs de rétrons permettent l'insertion d'un segment d'ADN défini par l'utilisateur dans un génome de cellule hôte. Pour maximiser l'activité d'éditeurs de rétrons, ils ont été optimisés par des lieurs améliorés, des séquences de localisation nucléaire et des séquences d'ARN. L'éditeur de rétrons résultant peut insérer, supprimer ou modifier de manière programmable le génome d'une cellule ou d'un organisme hôte, y compris des génomes humains.
PCT/US2024/036763 2023-07-03 2024-07-03 Compositions et procédés d'édition précise du génome à l'aide de rétrons Pending WO2025010350A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363511699P 2023-07-03 2023-07-03
US63/511,699 2023-07-03

Publications (2)

Publication Number Publication Date
WO2025010350A2 WO2025010350A2 (fr) 2025-01-09
WO2025010350A3 true WO2025010350A3 (fr) 2025-05-30

Family

ID=94172134

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/036763 Pending WO2025010350A2 (fr) 2023-07-03 2024-07-03 Compositions et procédés d'édition précise du génome à l'aide de rétrons

Country Status (1)

Country Link
WO (1) WO2025010350A2 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120518784A (zh) * 2025-07-24 2025-08-22 崖州湾国家实验室 一种核苷酸序列编辑方法及具有编辑作用的融合蛋白

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023019164A2 (fr) * 2021-08-11 2023-02-16 The Board Of Trustees Of The Leland Stanford Junior University Édition de génome de précision à haut rendement dans des cellules humaines

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023019164A2 (fr) * 2021-08-11 2023-02-16 The Board Of Trustees Of The Leland Stanford Junior University Édition de génome de précision à haut rendement dans des cellules humaines

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
HALL: "Integrons and gene cassettes: hotspots of diversity in bacterial genomes", ANN. N.Y. ACAD. SCI., vol. 1267, no. 1, 6 September 2012 (2012-09-06), pages 71 - 78, XP071408508, DOI: 10.1111/j.1749-6632.2012.06588.x *
TING-WEI WILL CHIANG, CARLOS LE SAGE, DELPHINE LARRIEU, MUKERREM DEMIR, STEPHEN P. JACKSON: "CRISPR-Cas9D10A nickase-based genotypic and phenotypic screening to enhance genome editing", SCIENTIFIC REPORTS, vol. 6, no. 1, 1 July 2016 (2016-07-01), XP055463907, DOI: 10.1038/srep24356 *

Also Published As

Publication number Publication date
WO2025010350A2 (fr) 2025-01-09

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