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MX2015014464A - Vectores para terapia genica para tratar cardiomiopatía. - Google Patents

Vectores para terapia genica para tratar cardiomiopatía.

Info

Publication number
MX2015014464A
MX2015014464A MX2015014464A MX2015014464A MX2015014464A MX 2015014464 A MX2015014464 A MX 2015014464A MX 2015014464 A MX2015014464 A MX 2015014464A MX 2015014464 A MX2015014464 A MX 2015014464A MX 2015014464 A MX2015014464 A MX 2015014464A
Authority
MX
Mexico
Prior art keywords
gene therapy
therapy vector
subject
gene
relates
Prior art date
Application number
MX2015014464A
Other languages
English (en)
Inventor
Carrier Lucie
Eschenhagen Thomas
Voit Thomas
MEARINI Giulia
Mueller Oliver
Stimpel Doreen
Mourot-Filiatre Julia
Original Assignee
Universitätsklinikum Hamburg-Eppendorf
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 Universitätsklinikum Hamburg-Eppendorf filed Critical Universitätsklinikum Hamburg-Eppendorf
Publication of MX2015014464A publication Critical patent/MX2015014464A/es

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/34—Muscles; Smooth muscle cells; Heart; Cardiac stem cells; Myoblasts; Myocytes; Cardiomyocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657—Cardiomyocytes; Heart cells
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
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    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
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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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    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/007—Vector systems having a special element relevant for transcription cell cycle specific enhancer/promoter combination
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cardiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
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  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Rheumatology (AREA)
  • Transplantation (AREA)
  • Vascular Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)

Abstract

La presente invención se refiere a un vector de terapia génica que es útil en el tratamiento o prevención de cardiomiopatía hipertrófica en un sujeto con tal necesidad. El vector de terapia génica de la invención comprende una secuencia de ácido nucleico que codifica una proteína sarcomérica y un promotor especifico del cardiomiocito que está operablemente enlazado a tal secuencia de ácido nucleico. La invención además se refiere a una célula que comprende el vector de terapia génica. También se proporcionan composiciones farmacéuticas que comprenden el vector de terapia génica y/o una célula que comprende tal vector. En otro aspecto, la invención se refiere a un método para tratar o prevenir cardiomiopatía hipertrófica en un sujeto mediante la introducción del vector de terapia génica de la invención en un sujeto en necesidad de tratamiento.
MX2015014464A 2013-04-17 2014-04-17 Vectores para terapia genica para tratar cardiomiopatía. MX2015014464A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP13164212 2013-04-17
EP13198201.9A EP2792742A1 (en) 2013-04-17 2013-12-18 Gene-therapy vectors for treating cardiomyopathy
PCT/EP2014/057984 WO2014170470A1 (en) 2013-04-17 2014-04-17 Gene-therapy vectors for treating cardiomyopathy

Publications (1)

Publication Number Publication Date
MX2015014464A true MX2015014464A (es) 2016-08-23

Family

ID=48128179

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2015014464A MX2015014464A (es) 2013-04-17 2014-04-17 Vectores para terapia genica para tratar cardiomiopatía.

Country Status (11)

Country Link
US (4) US10501756B2 (es)
EP (2) EP2792742A1 (es)
JP (1) JP2016516423A (es)
KR (1) KR20160023649A (es)
CN (1) CN105378089A (es)
AU (1) AU2014255655A1 (es)
BR (1) BR112015026145A2 (es)
CA (1) CA2944186A1 (es)
MX (1) MX2015014464A (es)
RU (1) RU2015148929A (es)
WO (1) WO2014170470A1 (es)

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WO2014170470A1 (en) 2014-10-23
CA2944186A1 (en) 2014-10-23
EP2986712B1 (en) 2019-02-13
RU2015148929A (ru) 2017-05-22
US20160108430A1 (en) 2016-04-21
KR20160023649A (ko) 2016-03-03
BR112015026145A2 (pt) 2017-12-19
US20240018548A1 (en) 2024-01-18
JP2016516423A (ja) 2016-06-09
US10501756B2 (en) 2019-12-10
EP2792742A1 (en) 2014-10-22
US11773408B2 (en) 2023-10-03
RU2015148929A3 (es) 2018-03-01
EP2986712A1 (en) 2016-02-24
CN105378089A (zh) 2016-03-02
AU2014255655A1 (en) 2015-12-03
US20230220421A1 (en) 2023-07-13
US20200095609A1 (en) 2020-03-26

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