BR112022002695A2 - Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso - Google Patents

Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso

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Publication number
BR112022002695A2
BR112022002695A2 BR112022002695A BR112022002695A BR112022002695A2 BR 112022002695 A2 BR112022002695 A2 BR 112022002695A2 BR 112022002695 A BR112022002695 A BR 112022002695A BR 112022002695 A BR112022002695 A BR 112022002695A BR 112022002695 A2 BR112022002695 A2 BR 112022002695A2
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interest
rna
methods
target sequence
variants
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BR112022002695A
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English (en)
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D Bowen Tyson
Briner Crawley Alexandra
D Elich Tedd
Coyle Michael
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Lifeedit Therapeutics Inc
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Publication of BR112022002695A2 publication Critical patent/BR112022002695A2/pt

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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
    • C12N15/10—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
    • C12N15/113—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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813—Hybridisation assays
    • C12Q1/6816—Hybridisation assays characterised by the detection means
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813—Hybridisation assays
    • C12Q1/6816—Hybridisation assays characterised by the detection means
    • C12Q1/6818—Hybridisation assays characterised by the detection means involving interaction of two or more labels, e.g. resonant energy transfer
    • 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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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Immunology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

NUCLEASES GUIADAS POR RNA, FRAGMENTOS ATIVOS E VARIANTES DAS MESMAS E MÉTODOS DE USO. São fornecidas composições e métodos de ligação a uma sequência alvo de interesse. As composições são úteis para clivar ou modificar uma sequência alvo de interesse, visualizar uma sequência alvo de interesse e modificar a expressão de uma sequência de interesse. As composições compreendem polipeptídeos de nuclease guiada por RNA, RNAs CRISPR, RNAs CRISPR transativadores, RNAs-guias, bem como moléculas de ácido nucleico que codificam os mesmos. Também são fornecidos vetores e células hospedeiras que compreendem as moléculas de ácido nucleico. São fornecidos, ainda, sistemas de CRISPR para ligar uma sequência alvo de interesse, em que o sistema de CRISPR compreende um polipeptídeo de nuclease guiada por RNA e um ou mais RNAs-guias. Métodos e kits para detectar uma sequência de DNA alvo também são fornecidos.
BR112022002695A 2019-08-12 2020-08-11 Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso BR112022002695A2 (pt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962885483P 2019-08-12 2019-08-12
US201962901875P 2019-09-18 2019-09-18
US202063030088P 2020-05-26 2020-05-26
PCT/US2020/045759 WO2021030344A1 (en) 2019-08-12 2020-08-11 Rna-guided nucleases and active fragments and variants thereof and methods of use

Publications (1)

Publication Number Publication Date
BR112022002695A2 true BR112022002695A2 (pt) 2022-08-23

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BR112022002695A BR112022002695A2 (pt) 2019-08-12 2020-08-11 Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso

Country Status (12)

Country Link
US (1) US20220364074A1 (pt)
EP (1) EP4013864A1 (pt)
JP (1) JP2022545385A (pt)
KR (1) KR20220062289A (pt)
CN (1) CN114641568A (pt)
AU (1) AU2020329912A1 (pt)
BR (1) BR112022002695A2 (pt)
CA (1) CA3147783A1 (pt)
IL (1) IL290562A (pt)
MX (1) MX2022001849A (pt)
TW (1) TW202120688A (pt)
WO (1) WO2021030344A1 (pt)

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JP2023541400A (ja) 2020-09-11 2023-10-02 ライフエディット セラピューティクス，インコーポレイティド Ｄｎａ修飾酵素並びにその活性断片及びバリアント並びにその使用方法
TW202300649A (zh) 2021-03-22 2023-01-01 美商生命編輯治療學公司 Ｄｎａ修飾酶及活性片段及其變體及使用方法
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IL290562A (en) 2022-04-01
CA3147783A1 (en) 2021-02-18
JP2022545385A (ja) 2022-10-27
EP4013864A1 (en) 2022-06-22
KR20220062289A (ko) 2022-05-16
CN114641568A (zh) 2022-06-17
WO2021030344A1 (en) 2021-02-18
US20220364074A1 (en) 2022-11-17
AU2020329912A1 (en) 2022-03-24
TW202120688A (zh) 2021-06-01
MX2022001849A (es) 2022-03-11

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