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MX2020013158A - Proteina cas9 modificada y uso de la misma. - Google Patents

Proteina cas9 modificada y uso de la misma.

Info

Publication number
MX2020013158A
MX2020013158A MX2020013158A MX2020013158A MX2020013158A MX 2020013158 A MX2020013158 A MX 2020013158A MX 2020013158 A MX2020013158 A MX 2020013158A MX 2020013158 A MX2020013158 A MX 2020013158A MX 2020013158 A MX2020013158 A MX 2020013158A
Authority
MX
Mexico
Prior art keywords
deletion region
amino acid
positions
domain
protein
Prior art date
Application number
MX2020013158A
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 MX2020013158A publication Critical patent/MX2020013158A/es

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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
    • 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]
    • 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 [CRISPR]
    • 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

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  • 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)

Abstract

Se describe una proteína que tiene una capacidad de unión al ARN guía y que consiste de una secuencia que contiene una secuencia de aminoácidos en donde una región de eliminación continua está presente entre la posición 481 y la posición 649 en la secuencia de aminoácidos mostrada en la SEQ ID NO: 2, la región de eliminación que contiene (i) todo o una parte del dominio L1 (posiciones 481 hasta 519), y (ii) el dominio HNH completo (posiciones 520 hasta 628), y de manera opcional además que contiene (iii) todo o una parte del dominio L2 (posiciones 629 hasta 649), en donde los aminoácidos adyacentes a cada una de la región de eliminación se ligan por un ligador que consiste de 3 hasta 10 residuos de aminoácidos que funciona como una proteína dSaCas9 miniaturizada mientras mantiene afinidad de unión al ADN. El uso de la proteína dSaCas9 miniaturizada hace posible montar muchos genes en vectores.
MX2020013158A 2018-06-08 2019-06-07 Proteina cas9 modificada y uso de la misma. MX2020013158A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862682244P 2018-06-08 2018-06-08
PCT/JP2019/022795 WO2019235627A1 (ja) 2018-06-08 2019-06-07 改変されたＣａｓ９タンパク質及びその用途

Publications (1)

Publication Number Publication Date
MX2020013158A true MX2020013158A (es) 2021-04-29

Family

ID=68770337

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020013158A MX2020013158A (es) 2018-06-08 2019-06-07 Proteina cas9 modificada y uso de la misma.

Country Status (13)

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

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 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
JP7736329B2 (ja) * 2020-08-31 2025-09-09 株式会社モダリス Ｄｕｘ４遺伝子を標的とした顔面肩甲上腕型筋ジストロフィーの治療方法
WO2022114243A1 (en) 2020-11-25 2022-06-02 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene

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
ES2531372T3 (es) 2006-10-13 2015-03-13 Novo Nordisk Healthcare Ag Enzimas de procesamiento fusionadas a marcadores de proteínas básicas
US20150110825A1 (en) 2013-09-24 2015-04-23 Massachusetts Institute Of Technology Self-assembled nanoparticle vaccines
WO2016034591A2 (en) 2014-09-01 2016-03-10 Vib Vzw Mutant pores
JP6744313B2 (ja) 2015-01-02 2020-08-26 ダイアックス コーポレーション 血漿カリクレインおよび第ｘｉｉ因子に対する二重特異性抗体
WO2016141224A1 (en) 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
EP3303634B1 (en) 2015-06-03 2023-08-30 The Regents of The University of California Cas9 variants and methods of use thereof
CA2989830A1 (en) * 2015-06-18 2016-12-22 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
US20180201912A1 (en) 2015-07-14 2018-07-19 The University Of Tokyo Modified fncas9 protein and use thereof
WO2017217768A1 (ko) 2016-06-15 2017-12-21 주식회사 툴젠 온타겟 및 오프타겟의 다중 타겟 시스템을 이용하는, 표적 특이적 유전자 가위 스크리닝 방법 및 이의 용도
WO2018074979A1 (en) 2016-10-17 2018-04-26 Nanyang Technological University Truncated crispr-cas proteins for dna targeting
EP3625338A4 (en) 2017-05-19 2021-01-20 Tsinghua University CONSTRUCTION OF A MINIMAL SACAS9-CRISPR / CAS SYSTEM FOR GENEDITATION AND TRANSCRIPTION REGULATION, OPTIMIZED BY EXTENDED GUIDE-RNA
WO2019089910A1 (en) * 2017-11-01 2019-05-09 Ohio State Innovation Foundation Highly compact cas9-based transcriptional regulators for in vivo gene regulation
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
IL279178A (en) 2021-01-31
US20220017881A1 (en) 2022-01-20
WO2019235627A1 (ja) 2019-12-12
EP3805386A4 (en) 2022-03-23
JPWO2019235627A1 (ja) 2021-06-24
BR112020024992A2 (pt) 2021-03-23
CA3103088A1 (en) 2019-12-12
CN112513266A (zh) 2021-03-16
KR20210025046A (ko) 2021-03-08
SG11202012228QA (en) 2021-01-28
AU2019281158A1 (en) 2021-01-14
JP7412001B2 (ja) 2024-01-12
EP3805386A1 (en) 2021-04-14
ZA202100092B (en) 2021-10-27

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