DK1009808T3 - Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer - Google Patents

Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer

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Publication number
DK1009808T3
DK1009808T3 DK98945922.7T DK98945922T DK1009808T3 DK 1009808 T3 DK1009808 T3 DK 1009808T3 DK 98945922 T DK98945922 T DK 98945922T DK 1009808 T3 DK1009808 T3 DK 1009808T3
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help
generation
methods
aav vectors
recombinant aav
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DK98945922.7T
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English (en)
Inventor
Edward Morrow Atkinson
Victor P Fung
Perry C Wilkins
Ryan K Takeya
Thomas C Reynolds
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Genzyme Corp
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
DK98945922.7T 1997-09-05 1998-09-04 Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer DK1009808T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US92581597A 1997-09-05 1997-09-05
US7173398P 1998-01-16 1998-01-16
PCT/US1998/018600 WO1999011764A2 (en) 1997-09-05 1998-09-04 Methods for generating high titer helper-free preparations of recombinant aav vectors

Publications (1)

Publication Number Publication Date
DK1009808T3 true DK1009808T3 (da) 2013-01-21

Family

ID=26752596

Family Applications (2)

Application Number Title Priority Date Filing Date
DK08004542.0T DK1944362T3 (da) 1997-09-05 1998-09-04 Fremgangsmåder til fremstilling af hjælpevirusfri præparater med høj titer af rekombinante AAV-vektorer
DK98945922.7T DK1009808T3 (da) 1997-09-05 1998-09-04 Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DK08004542.0T DK1944362T3 (da) 1997-09-05 1998-09-04 Fremgangsmåder til fremstilling af hjælpevirusfri præparater med høj titer af rekombinante AAV-vektorer

Country Status (9)

Country Link
EP (3) EP2325299A3 (da)
JP (4) JP2001514845A (da)
AU (1) AU758708B2 (da)
CA (4) CA2302992C (da)
CY (2) CY1113731T1 (da)
DK (2) DK1944362T3 (da)
ES (2) ES2399232T3 (da)
PT (2) PT1009808E (da)
WO (1) WO1999011764A2 (da)

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