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

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
    • 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)

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
AU2020283372A1 (en) 2019-05-28 2021-11-18 Modalis Therapeutics Corporation Method for treating muscular dystrophy by targeting DMPK gene
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
WO2022045366A1 (en) * 2020-08-31 2022-03-03 Modalis Therapeutics Corporation Method for treating facioscapulohumeral muscular dystrophy (fshd) by targeting dux4 gene
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
WO2008008523A1 (en) * 2006-07-14 2008-01-17 Regents Of The University Of Minnesota COMPOUNDS THAT BIND α5β1 INTEGRIN AND METHODS OF USE
JP2010505437A (ja) * 2006-10-13 2010-02-25 ノボ ノルディスク ヘルス ケア アーゲー 塩基性タンパク質タグに融合したプロセシング酵素
US20150110825A1 (en) * 2013-09-24 2015-04-23 Massachusetts Institute Of Technology Self-assembled nanoparticle vaccines
EP3224274B1 (en) * 2014-09-01 2024-02-14 Vib Vzw Mutant csgg pores
KR102670705B1 (ko) * 2015-01-02 2024-05-31 다케다 파머수티컬 컴패니 리미티드 혈장 칼리크레인 및 인자 xii에 대한 이중특이성 항체
EP3265559B1 (en) 2015-03-03 2021-01-06 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
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
JP7107683B2 (ja) * 2015-06-18 2022-07-27 ザ・ブロード・インスティテュート・インコーポレイテッド オフターゲット効果を低下させるｃｒｉｓｐｒ酵素突然変異
JPWO2017010543A1 (ja) 2015-07-14 2018-06-14 国立大学法人 東京大学 改変されたＦｎＣａｓ９タンパク質及びその使用
CN109312386B (zh) * 2016-06-15 2022-10-25 株式会社图尔金 使用中靶靶标和脱靶靶标的多重靶标系统筛选靶特异性核酸酶的方法及其用途
SG10201913505WA (en) 2016-10-17 2020-02-27 Univ Nanyang Tech Truncated crispr-cas proteins for dna targeting
CN110662835B (zh) 2017-05-19 2023-04-28 清华大学 工程化改造用于由增强的指导RNA优化的基因编辑和转录调节的最小化SaCas9 CRISPR/Cas系统
WO2019089910A1 (en) * 2017-11-01 2019-05-09 Ohio State Innovation Foundation Highly compact cas9-based transcriptional regulators for in vivo gene regulation
BR112021009481A2 (pt) * 2018-11-16 2021-08-17 Astellas Pharma Inc. método para tratamento da distrofia muscular direcionando gene de utrofina.

Also Published As

Publication number Publication date
IL279178A (en) 2021-01-31
JP7412001B2 (ja) 2024-01-12
CN112513266A (zh) 2021-03-16
CA3103088A1 (en) 2019-12-12
KR20210025046A (ko) 2021-03-08
WO2019235627A1 (ja) 2019-12-12
US20220017881A1 (en) 2022-01-20
BR112020024992A2 (pt) 2021-03-23
EP3805386A4 (en) 2022-03-23
JPWO2019235627A1 (ja) 2021-06-24
EP3805386A1 (en) 2021-04-14
AU2019281158A1 (en) 2021-01-14
ZA202100092B (en) 2021-10-27
SG11202012228QA (en) 2021-01-28

Similar Documents

Publication Publication Date Title
MX2020013158A (es) Proteina cas9 modificada y uso de la misma.
MX2020004578A (es) Composiciones de casz y metodos de uso.
MX2018010824A (es) Proteinas de union inducibles y metodos de uso.
AU2017248121A1 (en) T cell receptors
AU2017248120A1 (en) T cell receptors
MX2022001134A (es) Proteinas quimericas de factor viii y usos de estas.
NZ606584A (en) Binding proteins for hepcidin
NZ602634A (en) Vista regulatory t cell mediator protein, vista binding agents and use thereof
MX360208B (es) Composicion farmaceutica para el tratamiento y/o prevencion del cancer.
MX363136B (es) Composicion farmaceutica para el tratamiento y/o prevencion del cancer.
MX2021004714A (es) Proteina cas9 modificada, y uso de la misma.
MX2016004579A (es) Proteinas de union del miembro 1 de la familia de transmembrana 4 l-seis (tm4sf1) y metodos de uso de las mismas.
MX2018005399A (es) Nuevo polipeptido con afinidad por pd-l1.
EP4269562A3 (en) Antigen binding molecules and methods of use thereof
PH12017502213A1 (en) Compositions and methods for treating celiac sprue disease
MX2021001578A (es) Proteina asociada a crispr novedosa y uso de la misma.
WO2016130628A8 (en) Griffithsin mutants
MX2019006515A (es) Composiciones que comprenden peptido wkdeagkplvk.
MX2011007963A (es) Peptidos neil3 y vacunas que incluyen los mismos.
WO2019094938A3 (en) Methods and compositions for the treatment of wounds
MX2019015143A (es) Rna guias modificados, complejos crispr-ribonucleoproteina y metodos de uso.
MX2021010837A (es) Receptores en linfocitos t de la proteina 1 de tumor de wilms (wt1) de alta avidez.
MX2018003162A (es) Variante en el numero de copias que genera resistencia a virus.
MX2015009867A (es) Mutantes del factor x.
PH12018500468A1 (en) Fusion protein