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AR125191A1 - Enzimas modificadoras del adn y sus fragmentos activos y variantes y métodos de uso - Google Patents

Enzimas modificadoras del adn y sus fragmentos activos y variantes y métodos de uso

Info

Publication number
AR125191A1
AR125191A1 ARP220100668A ARP220100668A AR125191A1 AR 125191 A1 AR125191 A1 AR 125191A1 AR P220100668 A ARP220100668 A AR P220100668A AR P220100668 A ARP220100668 A AR P220100668A AR 125191 A1 AR125191 A1 AR 125191A1
Authority
AR
Argentina
Prior art keywords
amino acid
seq
acid sequence
deaminase
fusion protein
Prior art date
Application number
ARP220100668A
Other languages
English (en)
Original Assignee
Lifeedit Therapeutics 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 Lifeedit Therapeutics Inc filed Critical Lifeedit Therapeutics Inc
Publication of AR125191A1 publication Critical patent/AR125191A1/es

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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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • 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
    • 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
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C12N9/80—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04001—Cytosine deaminase (3.5.4.1)

Landscapes

  • 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)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Se proporcionan composiciones y métodos que comprenden polipéptidos de desaminasa para la edición dirigida de ácidos nucleicos. Las composiciones comprenden polipéptidos de desaminasa. También se proporcionan proteínas de fusión que comprenden un polipéptido de unión a ADN y una desaminasa de la invención. Las proteínas de fusión incluyen nucleasas guiadas por ARN fusionadas con desaminasas, opcionalmente en complejo con ARN guía. Las composiciones también incluyen moléculas de ácido nucleico que codifican las desaminasas o las proteínas de fusión. También se proporcionan vectores y células huésped que comprenden las moléculas de ácido nucleico que codifican las desaminasas o las proteínas de fusión. Reivindicación 1: Un polipéptido que comprende una secuencia de aminoácidos seleccionada del grupo que consiste en: a) una secuencia de aminoácidos que tiene al menos un 90% de identidad de secuencia con cualquiera de las SEQ ID Nº 2 y 7 - 12; y b) una secuencia de aminoácidos que tiene al menos un 95% de identidad de secuencia con SEQ ID Nº 4 ó 6; en donde dicho polipéptido tiene actividad de desaminasa. Reivindicación 5: La molécula de ácido nucleico de acuerdo con la reivindicación 3, en donde la desaminasa está codificada por una secuencia de nucleótidos que tiene una identidad de secuencia del 100% con cualquiera de las SEQ ID Nº 109, 111 y 113 - 119. Reivindicación 13: El método de acuerdo con la reivindicación 11 ó 12, que comprende además purificar dicha desaminasa. Reivindicación 14: Una proteína de fusión que comprende un polipéptido de unión a ADN y una desaminasa que tiene una secuencia de aminoácidos seleccionada del grupo que consiste en: a) una secuencia de aminoácidos que tiene al menos un 90% de identidad de secuencia con cualquiera de las SEQ ID Nº 2 y 7 - 12; y b) una secuencia de aminoácidos que tiene al menos un 95% de identidad de secuencia con SEQ ID Nº 4 ó 6. Reivindicación 27: La proteína de fusión de acuerdo con la reivindicación 19, en donde la RGN tiene una secuencia de aminoácidos de cualquiera de las SEQ ID Nº 74, 82, 87, 106 y 107. Reivindicación 30: La proteína de fusión de acuerdo con cualquiera de las reivindicaciones 14 - 29, en donde la proteína de fusión comprende además al menos una señal de localización nuclear (NLS). Reivindicación 33: La proteína de fusión de acuerdo con la reivindicación 14, en donde dicha proteína de fusión tiene una secuencia de aminoácidos de cualquiera de las SEQ ID Nº 67, 68, 146 y 147.
ARP220100668A 2021-03-22 2022-03-22 Enzimas modificadoras del adn y sus fragmentos activos y variantes y métodos de uso AR125191A1 (es)

Applications Claiming Priority (1)

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US202163164273P 2021-03-22 2021-03-22

Publications (1)

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AR125191A1 true AR125191A1 (es) 2023-06-21

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Country Status (9)

Country Link
US (1) US20240301385A1 (es)
EP (1) EP4314266A1 (es)
JP (1) JP2024511131A (es)
CN (1) CN117295817A (es)
AR (1) AR125191A1 (es)
AU (1) AU2022242754A1 (es)
CA (1) CA3173950A1 (es)
TW (1) TW202300649A (es)
WO (1) WO2022204093A1 (es)

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Also Published As

Publication number Publication date
WO2022204093A1 (en) 2022-09-29
CN117295817A (zh) 2023-12-26
AU2022242754A9 (en) 2023-11-16
US20240301385A1 (en) 2024-09-12
TW202300649A (zh) 2023-01-01
CA3173950A1 (en) 2022-09-22
JP2024511131A (ja) 2024-03-12
AU2022242754A1 (en) 2023-11-02
EP4314266A1 (en) 2024-02-07

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