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WO1998053063A3 - Transduction de cellules de mammiferes utilisee en therapie genique - Google Patents

Transduction de cellules de mammiferes utilisee en therapie genique Download PDF

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Publication number
WO1998053063A3
WO1998053063A3 PCT/EP1998/003013 EP9803013W WO9853063A3 WO 1998053063 A3 WO1998053063 A3 WO 1998053063A3 EP 9803013 W EP9803013 W EP 9803013W WO 9853063 A3 WO9853063 A3 WO 9853063A3
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WO
WIPO (PCT)
Prior art keywords
cells
vector
transduction
bone marrow
factor viii
Prior art date
Application number
PCT/EP1998/003013
Other languages
English (en)
Other versions
WO1998053063A2 (fr
Inventor
Driessche Thierry Vanden
Marinee Khim Lay Chuah
Original Assignee
Leuven Res & Dev Vzw
Driessche Thierry Vanden
Marinee Khim Lay Chuah
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 EP98200382A external-priority patent/EP0938904A1/fr
Application filed by Leuven Res & Dev Vzw, Driessche Thierry Vanden, Marinee Khim Lay Chuah filed Critical Leuven Res & Dev Vzw
Priority to EP98930718A priority Critical patent/EP0980264A2/fr
Priority to AU81057/98A priority patent/AU8105798A/en
Priority to CA002289918A priority patent/CA2289918A1/fr
Publication of WO1998053063A2 publication Critical patent/WO1998053063A2/fr
Publication of WO1998053063A3 publication Critical patent/WO1998053063A3/fr

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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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0669—Bone marrow stromal cells; Whole bone marrow
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043—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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

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

Abstract

La présente invention concerne un procédé pour la transduction ex vivo de cellules de mammifères, en particulier, pour la transduction de cellules du stroma de la moelle osseuse. Ces cellules peuvent être transduites avec un gène interessant, en particulier le gène du facteur VIII de l'homme supprimé du domaine B. Dans ce dernier cas, les cellules transduites peuvent permettre de traiter l'hémophilie A. Le procédé pour la transduction ex vivo des cellules du stroma de la moelle osseuse avec le gène du facteur VIII de l'homme consiste à prévoir un vecteur rétroviral à base d'intron, comprenant un ADNc du facteur VIII de l'homme supprimé du domaine B (désigné par MFG-FVIIIΔB); à pseudotyper ce vector avec l'enveloppe du virus (GALV) de la leucémie du singe Gibbon; à transduire les cellules du stroma de la moelle osseuse avec ce vecteur pseudotypé en pré-incubant les cellules pendant un laps de temps approprié dans un milieu de culture cellulaire sans phosphate. Ce procédé consiste à ajouter ensuite un surnageant contenant un vecteur, éventuellement complété par des additifs de transduction des cellules avant de centrifuger le mélange ainsi obtenu, puis à répéter éventuellement les deux étapes précédentes.
PCT/EP1998/003013 1997-05-16 1998-05-18 Transduction de cellules de mammiferes utilisee en therapie genique WO1998053063A2 (fr)

Priority Applications (3)

Application Number Priority Date Filing Date Title
EP98930718A EP0980264A2 (fr) 1997-05-16 1998-05-18 Transduction de cellules de mammiferes utilisee en therapie genique
AU81057/98A AU8105798A (en) 1997-05-16 1998-05-18 Mammalian cell transduction for use in gene therapy
CA002289918A CA2289918A1 (fr) 1997-05-16 1998-05-18 Transduction de cellules de mammiferes utilisee en therapie genique

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP97201480.7 1997-05-16
EP97201480 1997-05-16
EP98200382.4 1998-02-09
EP98200382A EP0938904A1 (fr) 1998-02-09 1998-02-09 Transduction des cellules mammifères pour utilisation dans la thérapie génique

Publications (2)

Publication Number Publication Date
WO1998053063A2 WO1998053063A2 (fr) 1998-11-26
WO1998053063A3 true WO1998053063A3 (fr) 1999-03-18

Family

ID=26146488

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP1998/003013 WO1998053063A2 (fr) 1997-05-16 1998-05-18 Transduction de cellules de mammiferes utilisee en therapie genique

Country Status (4)

Country Link
EP (1) EP0980264A2 (fr)
AU (1) AU8105798A (fr)
CA (1) CA2289918A1 (fr)
WO (1) WO1998053063A2 (fr)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000017375A2 (fr) * 1998-09-23 2000-03-30 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw Procede de traitement de l'hemophilie par therapie genique in vivo a l'aide de vecteurs retroviraux
AU1685201A (en) * 1999-12-01 2001-06-12 Centre For Translational Research In Cancer Drug inducible system and use thereof
GB0325379D0 (en) * 2003-10-30 2003-12-03 Oxford Biomedica Ltd Vectors

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993003143A1 (fr) * 1991-08-07 1993-02-18 Anderson W French Vecteurs retroviraux contenant des sites internes d'entree de ribosome
WO1996009400A1 (fr) * 1994-09-19 1996-03-28 Systemix, Inc. Procedes permettant de modifier genetiquement des cellules souches hematopoietiques
WO1996021035A2 (fr) * 1994-12-30 1996-07-11 Chiron Viagene, Inc. Production retrovirale du facteur viii entier
WO1996030533A1 (fr) * 1995-03-29 1996-10-03 Bavarian Nordic Research Institute A/S Particules retrovirales pseudotypees
WO1997012968A1 (fr) * 1995-10-05 1997-04-10 Chiron Corporation Vecteurs retroviraux pseudotypes a sequence de glycoproteines d'enveloppes srv-3
WO1997030169A1 (fr) * 1996-02-13 1997-08-21 Fred Hutchinson Cancer Research Center Cellules d'encapsidation de vecteurs retroviraux 10a1 et utilisations de ce type de cellules

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993003143A1 (fr) * 1991-08-07 1993-02-18 Anderson W French Vecteurs retroviraux contenant des sites internes d'entree de ribosome
WO1996009400A1 (fr) * 1994-09-19 1996-03-28 Systemix, Inc. Procedes permettant de modifier genetiquement des cellules souches hematopoietiques
WO1996021035A2 (fr) * 1994-12-30 1996-07-11 Chiron Viagene, Inc. Production retrovirale du facteur viii entier
WO1996030533A1 (fr) * 1995-03-29 1996-10-03 Bavarian Nordic Research Institute A/S Particules retrovirales pseudotypees
WO1997012968A1 (fr) * 1995-10-05 1997-04-10 Chiron Corporation Vecteurs retroviraux pseudotypes a sequence de glycoproteines d'enveloppes srv-3
WO1997030169A1 (fr) * 1996-02-13 1997-08-21 Fred Hutchinson Cancer Research Center Cellules d'encapsidation de vecteurs retroviraux 10a1 et utilisations de ce type de cellules

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
BUNNELL ET AL: "HIGH-EFFICIENCY RETROVIRAL MEDIATED GENE TRANSFER INTO HUMAN AND NONHUMAN PRIMATE PERIPHERAL BLOOD LYMPHOCYTES", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES,USA, vol. 92, 1995, pages 7739 - 7743, XP002081773 *
CHUAH ET AL: "BONE MARROW STROMAL CELLS AS TARGETS FOR GENE THERAPY OF HEMOPHILIA A", HUMAN GENE THERAPY, vol. 9, no. 3, 10 February 1998 (1998-02-10), pages 353 - 365, XP002090778 *
DWARKI V J ET AL: "GENE THERAPY FOR HEMOPHILIA A: PRODUCTION OF THERAPEUTIC LEVELS OF HUMAN FACTOR VIII IN VIVO IN MICE", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, vol. 92, no. 4, 14 February 1995 (1995-02-14), pages 1023 - 1027, XP000569659 *
GLIMM ET AL: "EFFICIENT GENE TRANSFER IN PRIMITIVE CD34+/CD38LO HUMAN BONE MARROW CELLS RESELECTED AFTER LONG-TERM EXPOSURE TO GALV-PSEUDOTYPED RETROVIRAL VECTOR", HUMAN GENE THERAPY, vol. 8, 20 November 1997 (1997-11-20), pages 2079 - 2086, XP002081772 *
KAGAMI ET AL: "GENETICALLY MODIFIED HUMAN BONE MARROW STROMAL CELLS USING A RETROVIRAL VECTOR CARRYING THE HUMAN GM-CSF GENE", ADVANCES IN BONE MARROW PURGING AND PROCESSING, 1992, pages 309 - 314, XP002081774 *
KIEM ET AL: "GENE TRANSFER INTO MARROW REPOPULATING CELLS: COMPARISON BETWEEN AMPHOTROPIC AND GIBBON APE LEUKEMIA VIRUS PSEUDOTYPED RETROVIRAL VECTORS IN A COMPETITIVE REPOPULATION ASSAY IN BABOONS", BLOOD, vol. 90, no. 11, 1 December 1997 (1997-12-01), pages 4638 - 4645, XP002081771 *
LI ET AL: "RETROVIRAL-MEDIATED GENE TRANSFER INTO HUMAN BONE MARROW STROMAL CELLS: STUDIES OF EFFICIENCY AND IN VIVO SURVIVAL IN SCID MICE", EUROPEAN JOURNAL OF HAEMATOLOGY, vol. 55, 1995, pages 302 - 306, XP002081775 *

Also Published As

Publication number Publication date
EP0980264A2 (fr) 2000-02-23
WO1998053063A2 (fr) 1998-11-26
CA2289918A1 (fr) 1998-11-26
AU8105798A (en) 1998-12-11

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