WO2000063369A3 - Gene therapy - Google Patents

Gene therapy Download PDF

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Publication number
WO2000063369A3
WO2000063369A3 PCT/EP2000/003532 EP0003532W WO0063369A3 WO 2000063369 A3 WO2000063369 A3 WO 2000063369A3 EP 0003532 W EP0003532 W EP 0003532W WO 0063369 A3 WO0063369 A3 WO 0063369A3
Authority
WO
WIPO (PCT)
Prior art keywords
cells
gene therapy
fasr
fgfr
progenitors
Prior art date
Application number
PCT/EP2000/003532
Other languages
French (fr)
Other versions
WO2000063369A2 (en
Inventor
Barbara Jutta Ballermann
Pascal Goldschmidt
Rao N Movva
Alfred Sanfilippo
Original Assignee
Novartis Ag
Novartis Erfind Verwalt Gmbh
Univ Johns Hopkins
Barbara Jutta Ballermann
Pascal Goldschmidt
Rao N Movva
Alfred Sanfilippo
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 Novartis Ag, Novartis Erfind Verwalt Gmbh, Univ Johns Hopkins, Barbara Jutta Ballermann, Pascal Goldschmidt, Rao N Movva, Alfred Sanfilippo filed Critical Novartis Ag
Priority to AU45527/00A priority Critical patent/AU4552700A/en
Publication of WO2000063369A2 publication Critical patent/WO2000063369A2/en
Publication of WO2000063369A3 publication Critical patent/WO2000063369A3/en

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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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343Use 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
    • C12N2750/14141Use 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/30Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6009Vectors comprising as targeting moiety peptide derived from defined protein from viruses dsDNA viruses
    • C12N2810/6018Adenoviridae
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
    • C12N2810/855Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian from receptors; from cell surface antigens; from cell surface determinants

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

Abstract

The invention relates to genetic modification of target cells, e.g. vascular cells, to inhibit excessive proliferation thereof, comprising transferring to the cells or progenitors thereof, a DNA sequence encoding a soluble form of FasR or FGFR-1 or a biologically active fragment or derivative thereof.
PCT/EP2000/003532 1999-04-19 2000-04-18 Gene therapy WO2000063369A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU45527/00A AU4552700A (en) 1999-04-19 2000-04-18 Gene therapy

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US29417699A 1999-04-19 1999-04-19
US09/294,176 1999-04-19
US30769099A 1999-05-10 1999-05-10
US09/307,690 1999-05-10

Publications (2)

Publication Number Publication Date
WO2000063369A2 WO2000063369A2 (en) 2000-10-26
WO2000063369A3 true WO2000063369A3 (en) 2001-04-26

Family

ID=26968381

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2000/003532 WO2000063369A2 (en) 1999-04-19 2000-04-18 Gene therapy

Country Status (2)

Country Link
AU (1) AU4552700A (en)
WO (1) WO2000063369A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020014242A1 (en) 2000-07-31 2002-02-07 Abraham Scaria Use of rapamycin to inhibit immune response and induce tolerance to gene therapy vector and encoded transgene products
JP2023509742A (en) * 2020-01-06 2023-03-09 メモリアル スローン ケタリング キャンサー センター Novel dominant-negative FAS polypeptides, cells containing them, and uses thereof

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0510691A1 (en) * 1991-04-26 1992-10-28 Osaka Bioscience Institute DNA coding for human cell surface antigen
US5229501A (en) * 1991-01-11 1993-07-20 Chiron Corporation Expression and use of human fibroblast growth factor receptor
WO1996020206A1 (en) * 1994-12-23 1996-07-04 Uab Research Foundation Secreted human fas antigen
WO1999055861A2 (en) * 1998-04-28 1999-11-04 Eisai Co., Ltd. Fibroblast growth factor mutein compositions and methods of use therefor
WO2000039311A1 (en) * 1998-12-31 2000-07-06 Advanced Research And Technology Institute Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2
WO2000046380A2 (en) * 1999-02-08 2000-08-10 Chiron Corporation Fibroblast growth factor receptor-immunoglobulin fusion

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5229501A (en) * 1991-01-11 1993-07-20 Chiron Corporation Expression and use of human fibroblast growth factor receptor
EP0510691A1 (en) * 1991-04-26 1992-10-28 Osaka Bioscience Institute DNA coding for human cell surface antigen
WO1996020206A1 (en) * 1994-12-23 1996-07-04 Uab Research Foundation Secreted human fas antigen
WO1999055861A2 (en) * 1998-04-28 1999-11-04 Eisai Co., Ltd. Fibroblast growth factor mutein compositions and methods of use therefor
WO2000039311A1 (en) * 1998-12-31 2000-07-06 Advanced Research And Technology Institute Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2
WO2000046380A2 (en) * 1999-02-08 2000-08-10 Chiron Corporation Fibroblast growth factor receptor-immunoglobulin fusion

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BELLOSTA P ET AL: "CLEAVAGE OF K-FGF PRODUCES A TRUNCATED MOLECULE WITH INCREASED BIOLOGICAL ACTIVITY AND RECEPTOR BINDING AFFINITY", JOURNAL OF CELL BIOLOGY,ROCKEFELLER UNIVERSITY PRESS, NEW YORK, US,US, vol. 121, no. 3, May 1993 (1993-05-01), pages 705 - 713, XP000942993, ISSN: 0021-9525 *
LUO W ET AL: "Gene transfer of soluble FGF receptor 1 (sFGFR1) reduces accelerated graft arteriosclerosis (AGA).", JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, vol. 10, no. PROGRAM AND ABSTR. ISSUE, September 1999 (1999-09-01), 32nd Annual Meeting of the American Society of Nephrology;Miami Beach, Florida, USA; November 1-8, 1999, pages 447A, XP000972013, ISSN: 1046-6673 *
MAHIOU JEROME ET AL: "Soluble FasR ligand-binding domain: High-yield production of active fusion and non-fusion recombinant proteins using the baculovirus/insect cell system.", BIOCHEMICAL JOURNAL, vol. 330, no. 2, 1 March 1998 (1998-03-01), pages 1051 - 1058, XP002154103, ISSN: 0264-6021 *

Also Published As

Publication number Publication date
WO2000063369A2 (en) 2000-10-26
AU4552700A (en) 2000-11-02

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