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WO2023244934A3 - Protéines acr modifiées pour moduler l'activité de crispr - Google Patents

Protéines acr modifiées pour moduler l'activité de crispr Download PDF

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Publication number
WO2023244934A3
WO2023244934A3 PCT/US2023/068149 US2023068149W WO2023244934A3 WO 2023244934 A3 WO2023244934 A3 WO 2023244934A3 US 2023068149 W US2023068149 W US 2023068149W WO 2023244934 A3 WO2023244934 A3 WO 2023244934A3
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
acr
subject
target
engineered
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/US2023/068149
Other languages
English (en)
Other versions
WO2023244934A2 (fr
Inventor
David Rabuka
Allison SHARRAR
Luisa Mayumi Arake DE TACCA
Michael SHELLE
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Acrigen Biosciences Inc
Original Assignee
Acrigen Biosciences 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 Acrigen Biosciences Inc filed Critical Acrigen Biosciences Inc
Priority to JP2024573948A priority Critical patent/JP2025520547A/ja
Priority to EP23824718.3A priority patent/EP4540376A2/fr
Priority to CN202380058718.3A priority patent/CN120051567A/zh
Publication of WO2023244934A2 publication Critical patent/WO2023244934A2/fr
Publication of WO2023244934A3 publication Critical patent/WO2023244934A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

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
    • 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
    • C12N15/63—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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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]

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  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

L'invention concerne des compositions et des procédés qui comprennent une protéine anti-CRISPR (Acr) modifiée qui peut atteindre un équilibre dans lequel les protéines Cas conservent leur activité pour effectuer des fonctions de ciblage d'acide nucléique sur cible (par exemple, le clivage de l'ADN pour des applications d'édition de gène), mais sont inhibées par une protéine Acr sujet à un degré qui diminue l'activité hors cible, ce qui permet d'obtenir un rapport accru entre les événements de ciblage d'acide nucléique sur cible et hors cible. Dans certains cas, une composition d'objet (par exemple, un système d'objet) comprend une nucléase CRISPR ou un acide nucléique codant pour celle-ci. L'invention concerne également des procédés d'utilisation d'un polypeptide Acr d'objet conjointement avec une nucléase CRISPR pour obtenir une modification d'acide nucléique cible.
PCT/US2023/068149 2022-06-15 2023-06-08 Protéines acr modifiées pour moduler l'activité de crispr Ceased WO2023244934A2 (fr)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2024573948A JP2025520547A (ja) 2022-06-15 2023-06-08 ＣＲＩＳＰＲ活性を調節するための操作Ａｃｒタンパク質
EP23824718.3A EP4540376A2 (fr) 2022-06-15 2023-06-08 Protéines acr modifiées pour moduler l'activité de crispr
CN202380058718.3A CN120051567A (zh) 2022-06-15 2023-06-08 用于调节CRISPR活性的工程化Acr蛋白

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263352494P 2022-06-15 2022-06-15
US63/352,494 2022-06-15

Publications (2)

Publication Number Publication Date
WO2023244934A2 WO2023244934A2 (fr) 2023-12-21
WO2023244934A3 true WO2023244934A3 (fr) 2024-02-08

Family

ID=89191962

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/068149 Ceased WO2023244934A2 (fr) 2022-06-15 2023-06-08 Protéines acr modifiées pour moduler l'activité de crispr

Country Status (4)

Country Link
EP (1) EP4540376A2 (fr)
JP (1) JP2025520547A (fr)
CN (1) CN120051567A (fr)
WO (1) WO2023244934A2 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240309349A1 (en) * 2021-07-09 2024-09-19 Acrigen Biosciences Compositions and methods for nucleic acid modifications

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020068196A2 (fr) * 2018-06-18 2020-04-02 The Regents Of The University Of California Protéines qui inhibent cas12a (cpf1) ou la nucléase crispr-cas
WO2022072673A1 (fr) * 2020-10-02 2022-04-07 Acrigen Biosciences, Inc. Compositions et procédés permettant d'améliorer la spécificité de ciblage des acides nucléiques

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020068196A2 (fr) * 2018-06-18 2020-04-02 The Regents Of The University Of California Protéines qui inhibent cas12a (cpf1) ou la nucléase crispr-cas
WO2022072673A1 (fr) * 2020-10-02 2022-04-07 Acrigen Biosciences, Inc. Compositions et procédés permettant d'améliorer la spécificité de ciblage des acides nucléiques

Also Published As

Publication number Publication date
WO2023244934A2 (fr) 2023-12-21
CN120051567A (zh) 2025-05-27
EP4540376A2 (fr) 2025-04-23
JP2025520547A (ja) 2025-07-03

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