BR112022021459A2 - 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
BR112022021459A2
BR112022021459A2 BR112022021459A BR112022021459A BR112022021459A2 BR 112022021459 A2 BR112022021459 A2 BR 112022021459A2 BR 112022021459 A BR112022021459 A BR 112022021459A BR 112022021459 A BR112022021459 A BR 112022021459A BR 112022021459 A2 BR112022021459 A2 BR 112022021459A2
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BR
Brazil
Prior art keywords
rna
interest
methods
target sequence
variants
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BR112022021459A
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English (en)
Inventor
D Bowen Tyson
Coyle Michael
Briner Crawley Alexandra
D Elich Tedd
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Lifeedit Therapeutics Inc
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Publication date
Application filed by Lifeedit Therapeutics Inc filed Critical Lifeedit Therapeutics Inc
Publication of BR112022021459A2 publication Critical patent/BR112022021459A2/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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • 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
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
    • 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
    • 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
    • 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
    • C12Q2521/00—Reaction characterised by the enzymatic activity
    • C12Q2521/30—Phosphoric diester hydrolysing, i.e. nuclease
    • C12Q2521/301—Endonuclease

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 (RGN), 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. Também são fornecidos sistemas RGN para ligar uma sequência alvo de interesse, em que o sistema RGN compreende um polipeptídeo de nuclease guiado por RNA e um ou mais RNAs-guias.
BR112022021459A 2020-04-24 2021-04-23 Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso BR112022021459A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063014970P 2020-04-24 2020-04-24
US202063077211P 2020-09-11 2020-09-11
PCT/US2021/028843 WO2021217002A1 (en) 2020-04-24 2021-04-23 Rna-guided nucleases and active fragments and variants thereof and methods of use

Publications (1)

Publication Number Publication Date
BR112022021459A2 true BR112022021459A2 (pt) 2023-01-17

Family

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BR112022021459A BR112022021459A2 (pt) 2020-04-24 2021-04-23 Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso

Country Status (12)

Country Link
US (3) US20230175018A1 (pt)
EP (2) EP4242306A3 (pt)
JP (1) JP2023516226A (pt)
KR (1) KR20230014700A (pt)
CN (1) CN116096879A (pt)
AU (1) AU2021258273A1 (pt)
BR (1) BR112022021459A2 (pt)
CA (1) CA3174124A1 (pt)
IL (1) IL297500A (pt)
MX (1) MX2022013187A (pt)
TW (1) TW202208626A (pt)
WO (1) WO2021217002A1 (pt)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3173950A1 (en) 2021-03-22 2022-09-22 Tyson D. BOWEN Dna modifyng enzymes and active fragments and variants thereof and methods of use
CA3173953A1 (en) 2021-06-11 2023-12-10 Tyson D. BOWEN Rna polymerase iii promoters and methods of use
WO2024033901A1 (en) 2022-08-12 2024-02-15 LifeEDIT Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2024042489A1 (en) 2022-08-25 2024-02-29 LifeEDIT Therapeutics, Inc. Chemical modification of guide rnas with locked nucleic acid for rna guided nuclease-mediated gene editing

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WO2021217002A1 (en) 2021-10-28
EP4118198A1 (en) 2023-01-18
CA3174124A1 (en) 2021-10-28
AU2021258273A1 (en) 2022-12-01
US20230235360A1 (en) 2023-07-27
CN116096879A (zh) 2023-05-09
US11859181B2 (en) 2024-01-02
US20230175018A1 (en) 2023-06-08
US20230257742A1 (en) 2023-08-17
EP4242306A2 (en) 2023-09-13
TW202208626A (zh) 2022-03-01
JP2023516226A (ja) 2023-04-18
EP4242306A3 (en) 2023-10-18
IL297500A (en) 2022-12-01
KR20230014700A (ko) 2023-01-30
MX2022013187A (es) 2022-11-14
US20240026384A1 (en) 2024-01-25

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