BR112022026885A2 - Método para tratar doença de alzheimer por direcionamento de gene mapt - Google Patents

Método para tratar doença de alzheimer por direcionamento de gene mapt

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Publication number
BR112022026885A2
BR112022026885A2 BR112022026885A BR112022026885A BR112022026885A2 BR 112022026885 A2 BR112022026885 A2 BR 112022026885A2 BR 112022026885 A BR112022026885 A BR 112022026885A BR 112022026885 A BR112022026885 A BR 112022026885A BR 112022026885 A2 BR112022026885 A2 BR 112022026885A2
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Brazil
Prior art keywords
targetment
gene
mapt
alzheimer disease
treat alzheimer
Prior art date
Application number
BR112022026885A
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English (en)
Inventor
Robert Thompson Iain
Yamagata Tetsuya
Akbulut Talha
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Modalis Therapeutics Corp
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Publication date
Application filed by Modalis Therapeutics Corp filed Critical Modalis Therapeutics Corp
Publication of BR112022026885A2 publication Critical patent/BR112022026885A2/pt

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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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]
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
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  • Veterinary Medicine (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

MÉTODO PARA TRATAR DOENÇA DE ALZHEIMER POR DIRECIONAMENTO DE GENE MAPT. A presente invenção refere-se a um polinucleotídeo, compreendendo as seguintes sequências de base: (a) uma sequência de base que codifica uma proteína de fusão de uma proteína efetora CRISPR deficiente em nuclease e um repressor de transcrição, e (b) uma sequência de base que codifica um RNA guia direcionado a uma região contínua de 18 a 24 nucleotídeos de comprimento em uma região apresentada na SEQ ID NO: 54, 55, 56, 57, 68, 153 ou 97 na região reguladora de expressão do gene MAPT humano. Espera-se que sejam úteis para tratar ou prevenir tauopatia, incluindo doença de Alzheimer.
BR112022026885A 2020-07-09 2021-07-09 Método para tratar doença de alzheimer por direcionamento de gene mapt BR112022026885A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063049736P 2020-07-09 2020-07-09
US202163212429P 2021-06-18 2021-06-18
PCT/JP2021/025974 WO2022009987A1 (en) 2020-07-09 2021-07-09 Method for treating alzheimer's disease by targeting mapt gene

Publications (1)

Publication Number Publication Date
BR112022026885A2 true BR112022026885A2 (pt) 2023-01-24

Family

ID=79552554

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022026885A BR112022026885A2 (pt) 2020-07-09 2021-07-09 Método para tratar doença de alzheimer por direcionamento de gene mapt

Country Status (11)

Country Link
US (1) US20230248810A1 (pt)
EP (1) EP4179082A1 (pt)
JP (1) JP2023533988A (pt)
KR (1) KR20230037586A (pt)
CN (1) CN115867652A (pt)
AU (1) AU2021304639A1 (pt)
BR (1) BR112022026885A2 (pt)
CA (1) CA3182390A1 (pt)
IL (1) IL298856A (pt)
MX (1) MX2023000223A (pt)
WO (1) WO2022009987A1 (pt)

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE202013012242U1 (de) 2012-05-25 2016-02-02 Emmanuelle Charpentier Zusammensetzungen für die durch RNA gesteuerte Modifikation einer Ziel-DNA und für die durch RNA gesteuerte Modulation der Transkription
WO2015188056A1 (en) * 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design
WO2017180915A2 (en) * 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
KR20190085529A (ko) * 2016-12-01 2019-07-18 상가모 테라퓨틱스, 인코포레이티드 Tau 조절제 및 그것의 전달을 위한 방법 및 조성물
MX2019011772A (es) * 2017-04-03 2020-01-09 Encoded Therapeutics Inc Expresion transgenica selectiva de tejidos.
EP3860618A4 (en) * 2018-10-02 2022-06-08 Sangamo Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING TAU PROTEINS
WO2020072684A1 (en) * 2018-10-02 2020-04-09 Sangamo Therapeutics, Inc. Engineered genetic modulators

Also Published As

Publication number Publication date
KR20230037586A (ko) 2023-03-16
WO2022009987A1 (en) 2022-01-13
US20230248810A1 (en) 2023-08-10
MX2023000223A (es) 2023-02-09
AU2021304639A1 (en) 2023-01-19
CN115867652A (zh) 2023-03-28
IL298856A (en) 2023-02-01
JP2023533988A (ja) 2023-08-07
EP4179082A1 (en) 2023-05-17
CA3182390A1 (en) 2022-01-13

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