ES2970169T3 - Polipéptidos útiles para edición génica y métodos de uso - Google Patents

Polipéptidos útiles para edición génica y métodos de uso Download PDF

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Publication number
ES2970169T3
ES2970169T3 ES19839624T ES19839624T ES2970169T3 ES 2970169 T3 ES2970169 T3 ES 2970169T3 ES 19839624 T ES19839624 T ES 19839624T ES 19839624 T ES19839624 T ES 19839624T ES 2970169 T3 ES2970169 T3 ES 2970169T3
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interest
target sequence
crispr
binding
compositions
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ES19839624T
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English (en)
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Tyson D Bowen
Alexandra Briner Crawley
Tedd D Elich
Mark Moore
Rodolphe Barrangou
Michael Lassner
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Lifeedit Therapeutics Inc
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Lifeedit Therapeutics Inc
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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
    • C12N15/113—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
    • 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
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • 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
    • 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)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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]

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

Abstract

Se proporcionan composiciones y métodos para unirse a una secuencia diana de interés. Las composiciones incluyen proteínas de fusión entre proteínas o dominios proteicos de unión a ADN y proteínas o dominios proteicos modificadores de ácidos nucleicos. Las composiciones encuentran uso para escindir o modificar una secuencia diana de interés, visualizar una secuencia diana de interés y modificar la expresión de una secuencia de interés. Las composiciones comprenden polipéptidos de nucleasa guiados por ARN, ARN CRISPR, ARN CRISPR transactivadores, ARN guía, desaminasas y moléculas de ácido nucleico que codifican los mismos. También se proporcionan vectores y células huésped que comprenden las moléculas de ácido nucleico. Además se proporcionan sistemas CRISPR para unir una secuencia diana de interés, en los que el sistema CRISPR comprende un polipéptido de nucleasa guiado por ARN y uno o más ARN guía. También se proporcionan desaminasas que pueden fusionarse a un polipéptido de unión al ADN y pueden ser útiles para la edición de genes. (Traducción automática con Google Translate, sin valor legal)
ES19839624T 2018-12-27 2019-12-20 Polipéptidos útiles para edición génica y métodos de uso Active ES2970169T3 (es)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201862785391P 2018-12-27 2018-12-27
US201962790262P 2019-01-09 2019-01-09
US201962790261P 2019-01-09 2019-01-09
US201962790266P 2019-01-09 2019-01-09
US201962790256P 2019-01-09 2019-01-09
US201962790258P 2019-01-09 2019-01-09
US201962932169P 2019-11-07 2019-11-07
PCT/US2019/068079 WO2020139783A2 (en) 2018-12-27 2019-12-20 Polypeptides useful for gene editing and methods of use

Publications (1)

Publication Number Publication Date
ES2970169T3 true ES2970169T3 (es) 2024-05-27

Family

ID=69182752

Family Applications (1)

Application Number Title Priority Date Filing Date
ES19839624T Active ES2970169T3 (es) 2018-12-27 2019-12-20 Polipéptidos útiles para edición génica y métodos de uso

Country Status (14)

Country Link
US (2) US20220145296A1 (es)
EP (2) EP4339286A3 (es)
JP (2) JP2024050676A (es)
KR (1) KR20210149686A (es)
CN (1) CN113631704A (es)
AU (1) AU2019416108A1 (es)
BR (1) BR112021012665A2 (es)
ES (1) ES2970169T3 (es)
IL (2) IL312708A (es)
MX (1) MX2021007835A (es)
SG (1) SG11202106977PA (es)
TW (1) TW202039847A (es)
WO (1) WO2020139783A2 (es)
ZA (1) ZA202105281B (es)

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WO2022015969A1 (en) 2020-07-15 2022-01-20 LifeEDIT Therapeutics, Inc. Uracil stabilizing proteins and active fragments and variants thereof and methods of use
CA3173886A1 (en) 2020-09-11 2022-03-17 Tyson D. BOWEN Dna modifying enzymes and active fragments and variants thereof and methods of use
AR125191A1 (es) 2021-03-22 2023-06-21 Lifeedit Therapeutics Inc Enzimas modificadoras del adn y sus fragmentos activos y variantes y métodos de uso
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TW202424186A (zh) 2022-08-25 2024-06-16 美商生命編輯治療學公司 Rna引導核酸酶中介基因編輯用之具鎖核酸引導 rna之化學修飾
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SG11202106977PA (en) 2021-07-29
MX2021007835A (es) 2021-08-11
JP2024137967A (ja) 2024-10-07
BR112021012665A2 (pt) 2021-11-03
EP3902911B1 (en) 2023-12-13
EP3902911C0 (en) 2023-12-13
KR20210149686A (ko) 2021-12-09
TW202039847A (zh) 2020-11-01
CN113631704A (zh) 2021-11-09
JP2024050676A (ja) 2024-04-10
US20220145296A1 (en) 2022-05-12
WO2020139783A2 (en) 2020-07-02
IL284400A (en) 2021-08-31
AU2019416108A1 (en) 2021-08-12
US20230272398A1 (en) 2023-08-31
IL312708A (en) 2024-07-01
ZA202105281B (en) 2024-05-30
WO2020139783A3 (en) 2020-07-23
EP4339286A3 (en) 2024-08-28
JP2022516130A (ja) 2022-02-24
EP4339286A2 (en) 2024-03-20
EP3902911A2 (en) 2021-11-03

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