AU2021350835A1 - Prime editing guide rnas, compositions thereof, and methods of using the same - Google Patents

Prime editing guide rnas, compositions thereof, and methods of using the same Download PDF

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Publication number
AU2021350835A1
AU2021350835A1 AU2021350835A AU2021350835A AU2021350835A1 AU 2021350835 A1 AU2021350835 A1 AU 2021350835A1 AU 2021350835 A AU2021350835 A AU 2021350835A AU 2021350835 A AU2021350835 A AU 2021350835A AU 2021350835 A1 AU2021350835 A1 AU 2021350835A1
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AU
Australia
Prior art keywords
pegrna
nucleotides
napdnabp
dna
nucleic acid
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AU2021350835A
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AU2021350835A2 (en
Inventor
Andrew Vito ANZALONE
Peter J. Chen
Kelcee EVERETTE
David R. Liu
James William NELSON
Peyton Barksdale RANDOLPH
Simon Shen
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Broad Institute Inc
Harvard University
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Broad Institute Inc
Harvard University
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Publication of AU2021350835A1 publication Critical patent/AU2021350835A1/en
Publication of AU2021350835A2 publication Critical patent/AU2021350835A2/en
Pending legal-status Critical Current

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    • 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
    • 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
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • 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
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2021350835A 2020-09-24 2021-09-24 Prime editing guide rnas, compositions thereof, and methods of using the same Pending AU2021350835A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202063083067P 2020-09-24 2020-09-24
US63/083,067 2020-09-24
US202063091272P 2020-10-13 2020-10-13
US63/091,272 2020-10-13
US202163182633P 2021-04-30 2021-04-30
US63/182,633 2021-04-30
US202163231231P 2021-08-09 2021-08-09
US63/231,231 2021-08-09
PCT/US2021/052097 WO2022067130A2 (en) 2020-09-24 2021-09-24 Prime editing guide rnas, compositions thereof, and methods of using the same

Publications (2)

Publication Number Publication Date
AU2021350835A1 true AU2021350835A1 (en) 2023-04-27
AU2021350835A2 AU2021350835A2 (en) 2023-04-27

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Family Applications (1)

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AU2021350835A Pending AU2021350835A1 (en) 2020-09-24 2021-09-24 Prime editing guide rnas, compositions thereof, and methods of using the same

Country Status (6)

Country Link
US (1) US20230357766A1 (https=)
EP (1) EP4217490A2 (https=)
JP (1) JP2023543803A (https=)
AU (1) AU2021350835A1 (https=)
CA (1) CA3193099A1 (https=)
WO (1) WO2022067130A2 (https=)

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