MX378789B - Métodos y composiciones para tratar la enfermedad de huntington. - Google Patents

Métodos y composiciones para tratar la enfermedad de huntington.

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Publication number
MX378789B
MX378789B MX2016014565A MX2016014565A MX378789B MX 378789 B MX378789 B MX 378789B MX 2016014565 A MX2016014565 A MX 2016014565A MX 2016014565 A MX2016014565 A MX 2016014565A MX 378789 B MX378789 B MX 378789B
Authority
MX
Mexico
Prior art keywords
methods
compositions
disease
subject
treating huntington
Prior art date
Application number
MX2016014565A
Other languages
English (en)
Other versions
MX2016014565A (es
Inventor
H Steve Zhang
Ignacio Munoz-Sanjuan
Seung Kwak
Steven Froelich
Original Assignee
Sangamo Biosciences Inc
Chdi Foundation Inc
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 Sangamo Biosciences Inc, Chdi Foundation Inc filed Critical Sangamo Biosciences Inc
Publication of MX2016014565A publication Critical patent/MX2016014565A/es
Publication of MX378789B publication Critical patent/MX378789B/es

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    • 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
    • A—HUMAN NECESSITIES
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    • A61K38/00—Medicinal preparations containing peptides
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    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A—HUMAN NECESSITIES
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/54—Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
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    • A61P25/00—Drugs for disorders of the nervous system
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    • A61P25/00—Drugs for disorders of the nervous system
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
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    • A61K39/001152—Transcription factors, e.g. SOX or c-MYC
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
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    • C07K2319/00—Fusion polypeptide
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invención se refiere a métodos y composiciones para tratar o prevenir la enfermedad de Huntington (EH). De forma específica, se proporcionan métodos y composiciones para reducir y/o eliminar acumulaciones de Huntingtina (Htt), mejorar los déficits motores, aumentar la actividad celular (p. ej., actividad de ATP) y/o reducir la apoptosis en un sujeto con EH, en el que los métodos comprenden administrarle al sujeto un represor de un alelo de Htt mutante (mHtt). Además se describen métodos de diagnóstico de EH y/o control del avance de la enfermedad que comprenden detectar mHtt en un sujeto.
MX2016014565A 2014-05-08 2015-05-07 Métodos y composiciones para tratar la enfermedad de huntington. MX378789B (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461990521P 2014-05-08 2014-05-08
US201462051724P 2014-09-17 2014-09-17
PCT/US2015/029748 WO2015171932A1 (en) 2014-05-08 2015-05-07 Methods and compositions for treating huntington's disease

Publications (2)

Publication Number Publication Date
MX2016014565A MX2016014565A (es) 2017-05-23
MX378789B true MX378789B (es) 2025-03-11

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MX2016014565A MX378789B (es) 2014-05-08 2015-05-07 Métodos y composiciones para tratar la enfermedad de huntington.

Country Status (11)

Country Link
US (1) US11110154B2 (es)
EP (1) EP3139966B1 (es)
JP (1) JP2017515819A (es)
KR (1) KR102380324B1 (es)
CN (1) CN106413760B (es)
AU (1) AU2015255877B2 (es)
BR (1) BR112016025849A2 (es)
CA (1) CA2947035A1 (es)
MX (1) MX378789B (es)
RU (1) RU2691102C2 (es)
WO (1) WO2015171932A1 (es)

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