MX2023000223A - Metodo para tratar la enfermedad de alzheimer mediante el direccionamiento al gen mapt. - Google Patents

Metodo para tratar la enfermedad de alzheimer mediante el direccionamiento al gen mapt.

Info

Publication number
MX2023000223A
MX2023000223A MX2023000223A MX2023000223A MX2023000223A MX 2023000223 A MX2023000223 A MX 2023000223A MX 2023000223 A MX2023000223 A MX 2023000223A MX 2023000223 A MX2023000223 A MX 2023000223A MX 2023000223 A MX2023000223 A MX 2023000223A
Authority
MX
Mexico
Prior art keywords
disease
mapt gene
treating alzheimer
sequence encoding
base sequence
Prior art date
Application number
MX2023000223A
Other languages
English (en)
Inventor
Tetsuya Yamagata
Iain Robert Thompson
Talha Akbulut
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 MX2023000223A publication Critical patent/MX2023000223A/es

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    • 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
    • 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)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • 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
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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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    • C12N9/14—Hydrolases (3)
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    • C12N9/22—Ribonucleases RNAses, DNAses
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    • 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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    • 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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    • C12N2750/14011—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
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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)
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  • Animal Behavior & Ethology (AREA)
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  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • 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

Un polinucleótido, que comprende las siguientes secuencias de bases: (a) una secuencia de bases que codifica una proteína de fusión de una proteína efectora CRISPR deficiente en nucleasa y un represor de la transcripción, y (b) una secuencia de bases que codifica un ARN guía dirigido a una región continua de 18 a 24 nucleótidos de longitud en una región establecida en la SEQ ID NO: 54, 55, 56, 57, 68, 153 o 97 en la región reguladora de la expresión del gen MAPT humano. Se espera que sean útiles para tratar o prevenir la taupatía, incluida la enfermedad de Alzheimer.
MX2023000223A 2020-07-09 2021-07-09 Metodo para tratar la enfermedad de alzheimer mediante el direccionamiento al gen mapt. MX2023000223A (es)

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
MX2023000223A true MX2023000223A (es) 2023-02-09

Family

ID=79552554

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023000223A MX2023000223A (es) 2020-07-09 2021-07-09 Metodo para tratar la enfermedad de alzheimer mediante el direccionamiento al gen mapt.

Country Status (11)

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

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ728024A (en) 2012-05-25 2019-05-31 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2015188065A1 (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
BR112019010014A2 (pt) * 2016-12-01 2019-08-20 Sangamo Therapeutics Inc moduladores de tau e métodos e composições para entrega dos mesmos
BR112019020777A2 (pt) * 2017-04-03 2020-04-28 Encoded Therapeutics Inc expressão de transgene seletiva para tecido
EP3860618A4 (en) * 2018-10-02 2022-06-08 Sangamo Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING TAU PROTEINS
JP2022504075A (ja) * 2018-10-02 2022-01-13 サンガモ セラピューティクス， インコーポレイテッド 操作された遺伝子モジュレーター

Also Published As

Publication number Publication date
CN115867652A (zh) 2023-03-28
EP4179082A1 (en) 2023-05-17
AU2021304639A1 (en) 2023-01-19
IL298856A (en) 2023-02-01
CA3182390A1 (en) 2022-01-13
JP2023533988A (ja) 2023-08-07
WO2022009987A1 (en) 2022-01-13
KR20230037586A (ko) 2023-03-16
BR112022026885A2 (pt) 2023-01-24
US20230248810A1 (en) 2023-08-10

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