WO2023141602A3 - Engineered retrons and methods of use - Google Patents

Engineered retrons and methods of use Download PDF

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Publication number
WO2023141602A3
WO2023141602A3 PCT/US2023/061038 US2023061038W WO2023141602A3 WO 2023141602 A3 WO2023141602 A3 WO 2023141602A3 US 2023061038 W US2023061038 W US 2023061038W WO 2023141602 A3 WO2023141602 A3 WO 2023141602A3
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WIPO (PCT)
Prior art keywords
engineered
retrons
methods
mammalian
host
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PCT/US2023/061038
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French (fr)
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WO2023141602A2 (en
Inventor
Alim Ladha
Vladimir PRESNYAK
Inna SHCHERBAKOVA
Brian Goodman
Mario Rodriguez MESTRE
Kyong-Rim KIEFFER-KWON
Socheata LY
Devin Scott QUINLAN
Muthusamy Jayaraman
Stephen Scully
Jacob LAYER
Abril Fleitas BEITANS
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Renagade Therapeutics Management Inc.
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Application filed by Renagade Therapeutics Management Inc. filed Critical Renagade Therapeutics Management Inc.
Publication of WO2023141602A2 publication Critical patent/WO2023141602A2/en
Priority to PCT/US2023/072872 priority Critical patent/WO2024044723A1/en
Publication of WO2023141602A3 publication Critical patent/WO2023141602A3/en

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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/87—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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • 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 RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • C—CHEMISTRY; METALLURGY
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    • 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 [CRISPRs]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes

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

Abstract

Disclosed are engineered retrons and methods of use such as to modify the genome of a host (e.g., mammalian) cell by delivering the engineered retron or the encoded ncRNA in vitro or in vivo to the host (e.g., mammalian) cell.
PCT/US2023/061038 2022-01-21 2023-01-20 Engineered retrons and methods of use WO2023141602A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
PCT/US2023/072872 WO2024044723A1 (en) 2022-08-25 2023-08-24 Engineered retrons and methods of use

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US202263301936P 2022-01-21 2022-01-21
US63/301,936 2022-01-21
US202263370880P 2022-08-09 2022-08-09
US63/370,880 2022-08-09
US202263373545P 2022-08-25 2022-08-25
US63/373,545 2022-08-25
US202263476900P 2022-12-22 2022-12-22
US63/476,900 2022-12-22

Publications (2)

Publication Number Publication Date
WO2023141602A2 WO2023141602A2 (en) 2023-07-27
WO2023141602A3 true WO2023141602A3 (en) 2023-11-02

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Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024044673A1 (en) * 2022-08-24 2024-02-29 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Dual cut retron editors for genomic insertions and deletions
WO2024044723A1 (en) * 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
CN118127022A (en) * 2024-04-30 2024-06-04 四川大学 Streptococcus mutans circular RNA circcsbD and application thereof, over-expression strain, construction method and application thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021178709A1 (en) * 2020-03-04 2021-09-10 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
WO2021178720A2 (en) * 2020-03-04 2021-09-10 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
WO2023081756A1 (en) * 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons

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