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WO2002029073A3 - Gene delivery vectors for stem cells - Google Patents

Gene delivery vectors for stem cells Download PDF

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Publication number
WO2002029073A3
WO2002029073A3 PCT/NL2001/000731 NL0100731W WO0229073A3 WO 2002029073 A3 WO2002029073 A3 WO 2002029073A3 NL 0100731 W NL0100731 W NL 0100731W WO 0229073 A3 WO0229073 A3 WO 0229073A3
Authority
WO
WIPO (PCT)
Prior art keywords
disease
stem cells
gene delivery
tropism
adenoviruses
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/NL2001/000731
Other languages
French (fr)
Other versions
WO2002029073A2 (en
Inventor
Menzo Jans Emco Havenga
Abraham Bout
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.)
Janssen Vaccines and Prevention BV
Original Assignee
Crucell Holand BV
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
Priority claimed from EP00203471A external-priority patent/EP1195440A1/en
Application filed by Crucell Holand BV filed Critical Crucell Holand BV
Priority to AU2002211087A priority Critical patent/AU2002211087A1/en
Publication of WO2002029073A2 publication Critical patent/WO2002029073A2/en
Publication of WO2002029073A3 publication Critical patent/WO2002029073A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10322—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10344—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10345—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The invention relates to the field of molecular genetics and medicine. In particular to the field of gene therapy, more in particular to gene therapy using adenoviruses. The invention provides the use of a gene delivery vehicle, more specifically an adenoviruses having tropism for stem cells, more specifically hemopoietic stem cells, wherein said tropism is provided by at least part of an adenoviral fiber protein derived from an adenovirus B serotype or functional equivalent and/or homologue thereof as a vehicle for delivering a nucleic acid to stem cells, for the treatment of Hurlers disease, Hunters disease, Sanfilippos disease, Morquois disease, Gaucher disease, Farbers disease, Niemann-pick disease, Krabbe disease, Metachromatic leucodistrophy. I-Cell disease, Fucosidose deficiency, Thallasemia and Erythropoietic porphyria, AIDS, cancer or other autoimmune diseases.
PCT/NL2001/000731 2000-10-06 2001-10-04 Gene delivery vectors for stem cells Ceased WO2002029073A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2002211087A AU2002211087A1 (en) 2000-10-06 2001-10-04 Gene delivery vectors for stem cells

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US23883000P 2000-10-06 2000-10-06
US60/238,830 2000-10-06
EP00203471.8 2000-10-06
EP00203471A EP1195440A1 (en) 2000-10-06 2000-10-06 Gene delivery vectors for stem cells

Publications (2)

Publication Number Publication Date
WO2002029073A2 WO2002029073A2 (en) 2002-04-11
WO2002029073A3 true WO2002029073A3 (en) 2002-10-03

Family

ID=26072760

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/NL2001/000731 Ceased WO2002029073A2 (en) 2000-10-06 2001-10-04 Gene delivery vectors for stem cells

Country Status (2)

Country Link
AU (1) AU2002211087A1 (en)
WO (1) WO2002029073A2 (en)

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5911983A (en) * 1992-06-26 1999-06-15 University Of Pittsburgh Gene therapy for Gaucher disease using retroviral vectors
WO1999032647A1 (en) * 1997-12-23 1999-07-01 Introgene B.V. Adeno-associated virus and adenovirus chimeric recombinant viruses useful for the integration of foreign genetic information into the chromosomal dna of target cells
WO2000003029A2 (en) * 1998-07-08 2000-01-20 Introgene B.V. Chimaeric adenoviruses
WO2000052186A1 (en) * 1999-03-04 2000-09-08 Introgene B.V. Means and methods for fibroblast-like or macrophage-like cell transduction
WO2000070071A1 (en) * 1999-05-17 2000-11-23 Crucell Holland B.V. Adenovirus derived gene delivery vehicles comprising at least one element of adenovirus type 35
WO2000073478A2 (en) * 1999-06-01 2000-12-07 University Of Washington Recombinant adenoviral vectors expressing chimeric fiber proteins for cell specific infection and genome integration
WO2002024933A2 (en) * 2000-09-25 2002-03-28 Galapagos Genomics N.V. Viral vectors having tissue tropism for t-lymphocytes, b- and mast cells
WO2002027006A1 (en) * 2000-09-26 2002-04-04 Crucell Holland B.V. Adenoviral vectors for gene delivery in skeletal muscle cells or myoblasts

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5911983A (en) * 1992-06-26 1999-06-15 University Of Pittsburgh Gene therapy for Gaucher disease using retroviral vectors
WO1999032647A1 (en) * 1997-12-23 1999-07-01 Introgene B.V. Adeno-associated virus and adenovirus chimeric recombinant viruses useful for the integration of foreign genetic information into the chromosomal dna of target cells
WO2000003029A2 (en) * 1998-07-08 2000-01-20 Introgene B.V. Chimaeric adenoviruses
WO2000052186A1 (en) * 1999-03-04 2000-09-08 Introgene B.V. Means and methods for fibroblast-like or macrophage-like cell transduction
WO2000070071A1 (en) * 1999-05-17 2000-11-23 Crucell Holland B.V. Adenovirus derived gene delivery vehicles comprising at least one element of adenovirus type 35
WO2000073478A2 (en) * 1999-06-01 2000-12-07 University Of Washington Recombinant adenoviral vectors expressing chimeric fiber proteins for cell specific infection and genome integration
WO2002024933A2 (en) * 2000-09-25 2002-03-28 Galapagos Genomics N.V. Viral vectors having tissue tropism for t-lymphocytes, b- and mast cells
WO2002027006A1 (en) * 2000-09-26 2002-04-04 Crucell Holland B.V. Adenoviral vectors for gene delivery in skeletal muscle cells or myoblasts

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
DATABASE BIOSIS [online] BIOSCIENCES INFORMATION SERVICE, PHILADELPHIA, PA, US; 1994, BAHNSON ALFRED B ET AL: "Transduction of CD34+ enriched cord blood and Gaucher bone marrow cells by a retroviral vector carrying the glucocerebrosidase gene.", XP002163089, Database accession no. PREV199497497999 *
GENE THERAPY, vol. 1, no. 3, 1994, pages 178 - 184 *
LIEBER ANDRE ET AL: "Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes.", JOURNAL OF VIROLOGY, vol. 73, no. 11, November 1999 (1999-11-01), pages 9314 - 9324, XP002163087, ISSN: 0022-538X *
SHAYAKHMETOV D M ET AL: "EFFICIENT GENE TRANSFER INTO HUMAN CD34+ CELLS BY A RETARGETED ADENOVIRUS VECTOR", JOURNAL OF VIROLOGY,THE AMERICAN SOCIETY FOR MICROBIOLOGY,US, vol. 74, no. 6, March 2000 (2000-03-01), pages 2567 - 2583, XP000938716, ISSN: 0022-538X *
VAN BEUSECHEM V W ET AL: "LONG-TERM EXPRESSION OF HUMAN ADENOSINE DEAMINASE IN RHESUS MONKEYS TRANSPLANTED WITH RETROVIRUS-INFECTED BONE-MARROW CELLS", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES, vol. 89, no. 16, 1992, 1992, pages 7640 - 7644, XP002163088, ISSN: 0027-8424 *

Also Published As

Publication number Publication date
AU2002211087A1 (en) 2002-04-15
WO2002029073A2 (en) 2002-04-11

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