ATE428777T1 - Muskelspezifische expressionsvektoren - Google Patents

Muskelspezifische expressionsvektoren

Info

Publication number
ATE428777T1
ATE428777T1 AT02739449T AT02739449T ATE428777T1 AT E428777 T1 ATE428777 T1 AT E428777T1 AT 02739449 T AT02739449 T AT 02739449T AT 02739449 T AT02739449 T AT 02739449T AT E428777 T1 ATE428777 T1 AT E428777T1
Authority
AT
Austria
Prior art keywords
muscle
promoter
elements
expression vectors
specific expression
Prior art date
Application number
AT02739449T
Other languages
German (de)
English (en)
Inventor
David Souza
Donna Armentano
Original Assignee
Genzyme 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 Genzyme Corp filed Critical Genzyme Corp
Application granted granted Critical
Publication of ATE428777T1 publication Critical patent/ATE428777T1/de

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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Diabetes (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Hematology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Obesity (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AT02739449T 2001-05-24 2002-05-22 Muskelspezifische expressionsvektoren ATE428777T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US29330401P 2001-05-24 2001-05-24
PCT/US2002/016748 WO2002095006A2 (en) 2001-05-24 2002-05-22 Muscle-specific expression vectors

Publications (1)

Publication Number Publication Date
ATE428777T1 true ATE428777T1 (de) 2009-05-15

Family

ID=23128549

Family Applications (1)

Application Number Title Priority Date Filing Date
AT02739449T ATE428777T1 (de) 2001-05-24 2002-05-22 Muskelspezifische expressionsvektoren

Country Status (7)

Country Link
US (2) US20030100526A1 (enExample)
EP (2) EP1390490B1 (enExample)
JP (1) JP2004535801A (enExample)
AT (1) ATE428777T1 (enExample)
CA (1) CA2448120A1 (enExample)
DE (1) DE60231975D1 (enExample)
WO (1) WO2002095006A2 (enExample)

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US9217148B2 (en) 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
WO2015035364A1 (en) * 2013-09-09 2015-03-12 University Of Washington Through Its Center For Commercialization Proteins for targeting neuronal nitric oxide synthase to muscle sarcolemma and related methods of use
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US10472408B2 (en) 2015-03-05 2019-11-12 Peter Und Traudl Engelhorn-Stiftung Zur Förderung Der Lebenswissenschaften Fusion proteins comprising partial tetraspanin sequences and a system thereof for presenting peptides on the cell surface
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WO2017223128A1 (en) 2016-06-21 2017-12-28 The Curators Of The University Of Missouri Modified dystrophin proteins
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WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
US20210254103A1 (en) 2018-07-02 2021-08-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
AU2019354793A1 (en) 2018-10-05 2021-05-13 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding AAV production proteins
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MX2021005720A (es) 2018-11-16 2021-07-21 Astellas Pharma Inc Metodo para tratar la distrofia muscular mediante el direccionamiento del gen de utrofina.
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WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
WO2022056291A1 (en) * 2020-09-11 2022-03-17 Nature Technology Corporation Muscle-specific hybrid promoter
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WO2022114243A1 (en) 2020-11-25 2022-06-02 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene
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WO2023012313A1 (en) * 2021-08-04 2023-02-09 Genethon Hybrid promoters for gene expression in muscles and in the cns
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US20250243510A1 (en) * 2022-03-16 2025-07-31 Rutgers, The State University Of New Jersey Controlled muscle-specific gene delivery
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Also Published As

Publication number Publication date
EP1390490B1 (en) 2009-04-15
WO2002095006A2 (en) 2002-11-28
US20030100526A1 (en) 2003-05-29
DE60231975D1 (de) 2009-05-28
CA2448120A1 (en) 2002-11-28
EP1390490A2 (en) 2004-02-25
WO2002095006A3 (en) 2003-03-13
EP1390490A4 (en) 2005-05-11
JP2004535801A (ja) 2004-12-02
EP2017338A1 (en) 2009-01-21
US20110212529A1 (en) 2011-09-01

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