CA3203876A1 - Prime editor variants, constructs, and methods for enhancing prime editing efficiency and precision - Google Patents

Prime editor variants, constructs, and methods for enhancing prime editing efficiency and precision Download PDF

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Publication number
CA3203876A1
CA3203876A1 CA3203876A CA3203876A CA3203876A1 CA 3203876 A1 CA3203876 A1 CA 3203876A1 CA 3203876 A CA3203876 A CA 3203876A CA 3203876 A CA3203876 A CA 3203876A CA 3203876 A1 CA3203876 A1 CA 3203876A1
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CA
Canada
Prior art keywords
fold
seq
dna
amino acid
basepair
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
CA3203876A
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English (en)
French (fr)
Inventor
David R. Liu
Peter J. Chen
Brittany ADAMSON
Jeffrey HUSSMANN
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.)
Harvard College
University of California
Princeton University
Broad Institute Inc
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Harvard College
University of California
Princeton University
Broad Institute Inc
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 Harvard College, University of California, Princeton University, Broad Institute Inc filed Critical Harvard College
Publication of CA3203876A1 publication Critical patent/CA3203876A1/en
Pending legal-status Critical Current

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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N9/10—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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • 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 [CRISPRs]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Steroid Compounds (AREA)
  • Document Processing Apparatus (AREA)
  • Steering-Linkage Mechanisms And Four-Wheel Steering (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA3203876A 2021-01-11 2022-01-11 Prime editor variants, constructs, and methods for enhancing prime editing efficiency and precision Pending CA3203876A1 (en)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US202163136194P 2021-01-11 2021-01-11
US63/136,194 2021-01-11
US202163176202P 2021-04-16 2021-04-16
US202163176180P 2021-04-16 2021-04-16
US63/176,202 2021-04-16
US63/176,180 2021-04-16
US202163194913P 2021-05-28 2021-05-28
US202163194865P 2021-05-28 2021-05-28
US63/194,913 2021-05-28
US63/194,865 2021-05-28
US202163231230P 2021-08-09 2021-08-09
US63/231,230 2021-08-09
US202163255897P 2021-10-14 2021-10-14
US63/255,897 2021-10-14
PCT/US2022/012054 WO2022150790A2 (en) 2021-01-11 2022-01-11 Prime editor variants, constructs, and methods for enhancing prime editing efficiency and precision

Publications (1)

Publication Number Publication Date
CA3203876A1 true CA3203876A1 (en) 2022-07-14

Family

ID=80119064

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3203876A Pending CA3203876A1 (en) 2021-01-11 2022-01-11 Prime editor variants, constructs, and methods for enhancing prime editing efficiency and precision

Country Status (5)

Country Link
EP (1) EP4274894A2 (de)
JP (1) JP2024503437A (de)
AU (1) AU2022206476A1 (de)
CA (1) CA3203876A1 (de)
WO (1) WO2022150790A2 (de)

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EP4114941A4 (de) 2020-03-04 2024-10-16 Flagship Pioneering Innovations Vi Llc Verbesserte verfahren und zusammensetzungen zur modulierung eines genoms
MX2024002927A (es) 2021-09-08 2024-05-29 Flagship Pioneering Innovations Vi Llc Metodos y composiciones para modular un genoma.
EP4437106A2 (de) * 2021-11-24 2024-10-02 Prime Medicine, Inc. Verfahren und zusammensetzungen zur verhinderung der reparatur von fehlpaarungen
WO2024026344A1 (en) * 2022-07-27 2024-02-01 Inscripta, Inc. Modulating cellular repair mechanisms for genomic editing
WO2024077267A1 (en) 2022-10-07 2024-04-11 The Broad Institute, Inc. Prime editing methods and compositions for treating triplet repeat disorders
WO2024178144A1 (en) * 2023-02-22 2024-08-29 Prime Medicine, Inc. Methods and compositions for editing nucleotide sequences
WO2024187173A1 (en) * 2023-03-09 2024-09-12 The Regents Of The University Of California High throughput prime editing screens identify functional dna variants in the human genome
WO2024192141A1 (en) 2023-03-13 2024-09-19 Dana-Farber Cancer Institute, Inc. Treatment of cancers having a drug-resistant mesenchymal cell state
WO2024197185A1 (en) 2023-03-21 2024-09-26 The Broad Institute, Inc. Methods and compositions for dissecting organelle physiology
WO2024206125A1 (en) * 2023-03-24 2024-10-03 The Broad Institute, Inc. Use of prime editing for treating sickle cell disease

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Also Published As

Publication number Publication date
WO2022150790A2 (en) 2022-07-14
WO2022150790A3 (en) 2022-08-11
JP2024503437A (ja) 2024-01-25
EP4274894A2 (de) 2023-11-15
AU2022206476A1 (en) 2023-07-20

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