WO2004111248A3 - Improved aav vector for gene therapy - Google Patents

Improved aav vector for gene therapy Download PDF

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Publication number
WO2004111248A3
WO2004111248A3 PCT/EP2004/006222 EP2004006222W WO2004111248A3 WO 2004111248 A3 WO2004111248 A3 WO 2004111248A3 EP 2004006222 W EP2004006222 W EP 2004006222W WO 2004111248 A3 WO2004111248 A3 WO 2004111248A3
Authority
WO
WIPO (PCT)
Prior art keywords
gene therapy
aav vector
improved aav
eliminated
aav vectors
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/EP2004/006222
Other languages
French (fr)
Other versions
WO2004111248A2 (en
Inventor
Juergen Kleinschmidt
Oliver Mueller
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
German Cancer Research Center
Original Assignee
German Cancer Research Center
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 German Cancer Research Center filed Critical German Cancer Research Center
Priority to US10/560,268 priority Critical patent/US7629322B2/en
Priority to EP04762994A priority patent/EP1664314B1/en
Priority to DE602004016871T priority patent/DE602004016871D1/en
Publication of WO2004111248A2 publication Critical patent/WO2004111248A2/en
Publication of WO2004111248A3 publication Critical patent/WO2004111248A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • 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
    • C12N15/86—Viral vectors
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C12N2750/14145—Special targeting system for viral vectors
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (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)

Abstract

Described are recombinant AAV vectors characterized in that they carry capsid protein modification(s) resulting in a reduced or eliminated heparin binding function. The AAV vectors of the present invention are particularly suitable for gene therapy by systemic application, since (a) transduction of the liver is eliminated or at least drastically reduced and (b) the transduction efficiency of non-hepatic tissues is increased.
PCT/EP2004/006222 2003-06-11 2004-06-09 Improved aav vector for gene therapy Ceased WO2004111248A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
US10/560,268 US7629322B2 (en) 2003-06-11 2004-06-09 AAV vector for gene therapy
EP04762994A EP1664314B1 (en) 2003-06-11 2004-06-09 Improved adeno-associated virus (aav) vector for gene therapy
DE602004016871T DE602004016871D1 (en) 2003-06-11 2004-06-09 FOR GENE THERAPY

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP03013169A EP1486567A1 (en) 2003-06-11 2003-06-11 Improved adeno-associated virus (AAV) vector for gene therapy
EP03013169.2 2003-06-11

Publications (2)

Publication Number Publication Date
WO2004111248A2 WO2004111248A2 (en) 2004-12-23
WO2004111248A3 true WO2004111248A3 (en) 2005-07-07

Family

ID=33185871

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2004/006222 Ceased WO2004111248A2 (en) 2003-06-11 2004-06-09 Improved aav vector for gene therapy

Country Status (5)

Country Link
US (1) US7629322B2 (en)
EP (2) EP1486567A1 (en)
AT (1) ATE409750T1 (en)
DE (1) DE602004016871D1 (en)
WO (1) WO2004111248A2 (en)

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* Cited by examiner, † Cited by third party
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