SG11202012228QA - MODIFIED Cas9 PROTEIN AND USE THEREOF - Google Patents

MODIFIED Cas9 PROTEIN AND USE THEREOF

Info

Publication number
SG11202012228QA
SG11202012228QA SG11202012228QA SG11202012228QA SG11202012228QA SG 11202012228Q A SG11202012228Q A SG 11202012228QA SG 11202012228Q A SG11202012228Q A SG 11202012228QA SG 11202012228Q A SG11202012228Q A SG 11202012228QA SG 11202012228Q A SG11202012228Q A SG 11202012228QA
Authority
SG
Singapore
Prior art keywords
cas9 protein
modified cas9
modified
protein
cas9
Prior art date
Application number
SG11202012228QA
Other languages
English (en)
Inventor
Tetsuya Yamagata
Yuanbo Qin
Original Assignee
Modalis Therapeutics Corp
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 Modalis Therapeutics Corp filed Critical Modalis Therapeutics Corp
Publication of SG11202012228QA publication Critical patent/SG11202012228QA/en

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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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • 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
    • 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
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • C12N15/11—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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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)
    • 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
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
SG11202012228QA 2018-06-08 2019-06-07 MODIFIED Cas9 PROTEIN AND USE THEREOF SG11202012228QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862682244P 2018-06-08 2018-06-08
PCT/JP2019/022795 WO2019235627A1 (fr) 2018-06-08 2019-06-07 PROTÉINE Cas9 MODIFIÉE ET UTILISATION DE CELLE-CI

Publications (1)

Publication Number Publication Date
SG11202012228QA true SG11202012228QA (en) 2021-01-28

Family

ID=68770337

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202012228QA SG11202012228QA (en) 2018-06-08 2019-06-07 MODIFIED Cas9 PROTEIN AND USE THEREOF

Country Status (13)

Country Link
US (1) US20220017881A1 (fr)
EP (1) EP3805386A4 (fr)
JP (1) JP7412001B2 (fr)
KR (1) KR20210025046A (fr)
CN (1) CN112513266A (fr)
AU (1) AU2019281158A1 (fr)
BR (1) BR112020024992A2 (fr)
CA (1) CA3103088A1 (fr)
IL (1) IL279178A (fr)
MX (1) MX2020013158A (fr)
SG (1) SG11202012228QA (fr)
WO (1) WO2019235627A1 (fr)
ZA (1) ZA202100092B (fr)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2021014478A (es) 2019-05-28 2022-01-06 Astellas Pharma Inc Metodo para tratar distrofia muscular por direccionamiento del gen dmpk.
WO2021230385A1 (fr) 2020-05-15 2021-11-18 Astellas Pharma Inc. Procédé de traitement de la dystrophie musculaire par ciblage d'un gène utrophine
WO2022045366A1 (fr) * 2020-08-31 2022-03-03 Modalis Therapeutics Corporation Procédé de traitement de la dystrophie musculaire faceoscapulohumerale (dfmh) par le ciblage du gène dux4
AR124143A1 (es) 2020-11-25 2023-02-15 Astellas Pharma Inc Método para tratar la distrofia muscular mediante el direccionamiento al gen dmpk

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8343539B2 (en) 2006-07-14 2013-01-01 Regents Of The University Of Minnesota Compounds that bind α5β1 integrin and methods of use
CN101563358A (zh) 2006-10-13 2009-10-21 诺沃-诺迪斯克保健股份有限公司 融合于碱性蛋白标签的加工酶
US20150110825A1 (en) 2013-09-24 2015-04-23 Massachusetts Institute Of Technology Self-assembled nanoparticle vaccines
CA2959220A1 (fr) 2014-09-01 2016-03-10 Vib Vzw Pores csgg mutants
KR102670705B1 (ko) 2015-01-02 2024-05-31 다케다 파머수티컬 컴패니 리미티드 혈장 칼리크레인 및 인자 xii에 대한 이중특이성 항체
CA2978314A1 (fr) 2015-03-03 2016-09-09 The General Hospital Corporation Nucleases crispr-cas9 genetiquement modifiees presentant une specificite pam modifiee
EP3303634B1 (fr) * 2015-06-03 2023-08-30 The Regents of The University of California Variants de cas9 et procédés d'utilisation associés
WO2016205759A1 (fr) * 2015-06-18 2016-12-22 The Broad Institute Inc. Modification et optimisation de systèmes, de méthodes, d'enzymes et d'échafaudages guides d'orthologues de cas9 et variant pour la manipulation de séquences
IL293323B2 (en) * 2015-06-18 2024-01-01 Massachusetts Inst Technology CRISPR enzyme mutations that reduce unintended effects
US20180201912A1 (en) 2015-07-14 2018-07-19 The University Of Tokyo Modified fncas9 protein and use thereof
EP3473728B1 (fr) 2016-06-15 2021-10-20 Toolgen Incorporated Procédé de criblage de ciseaux génétiques ciblés à l'aide d'un système à cibles multiples d'activité sur cible et hors cible et son utilisation
WO2018074979A1 (fr) 2016-10-17 2018-04-26 Nanyang Technological University Protéines crispr-cas tronquées pour ciblage d'adn
EP3625338A4 (fr) 2017-05-19 2021-01-20 Tsinghua University Ingénierie d'un système crispr/cas minimal sacas9 pour l'édition de gènes et la régulation transcriptionnelle optimisée par un arn guide amélioré
WO2019089910A1 (fr) * 2017-11-01 2019-05-09 Ohio State Innovation Foundation Régulateurs transcriptionnels à base de cas9 très compacts pour la régulation de gènes in vivo
TW202033224A (zh) * 2018-11-16 2020-09-16 日商安斯泰來製藥股份有限公司 藉靶向肌營養相關蛋白基因治療肌肉萎縮症之方法

Also Published As

Publication number Publication date
JPWO2019235627A1 (ja) 2021-06-24
JP7412001B2 (ja) 2024-01-12
EP3805386A4 (fr) 2022-03-23
ZA202100092B (en) 2021-10-27
US20220017881A1 (en) 2022-01-20
BR112020024992A2 (pt) 2021-03-23
KR20210025046A (ko) 2021-03-08
EP3805386A1 (fr) 2021-04-14
CN112513266A (zh) 2021-03-16
CA3103088A1 (fr) 2019-12-12
MX2020013158A (es) 2021-04-29
IL279178A (en) 2021-01-31
AU2019281158A1 (en) 2021-01-14
WO2019235627A1 (fr) 2019-12-12

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