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1. (WO1998053063) MAMMALIAN CELL TRANSDUCTION FOR USE IN GENE THERAPY
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/1998/053063 International Application No.: PCT/EP1998/003013
Publication Date: 26.11.1998 International Filing Date: 18.05.1998
Chapter 2 Demand Filed: 16.12.1998
IPC:
A61K 38/37 (2006.01) ,C12N 15/12 (2006.01) ,C12N 15/867 (2006.01) ,C12N 5/077 (2010.01) ,A61K 48/00 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38
Medicinal preparations containing peptides
16
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17
from animals; from humans
36
Blood coagulation or fibrinolysis factors
37
Factors VIII
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
12
Genes encoding animal proteins
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
86
Viral vectors
867
Retroviral vectors
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5
Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
07
Animal cells or tissues
071
Vertebrate cells or tissues, e.g. human cells or tissues
077
Mesenchymal cells, e.g. bone cells, cartilage cells, marrow stromal cells, fat cells or muscle cells
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Applicants:
LEUVEN RESEARCH & DEVELOPMENT VZW [BE/BE]; Groot Begijnhof Benedenstraat 60 B-3000 Leuven, BE (AllExceptUS)
VANDEN DRIESSCHE, Thierry [BE/BE]; BE (UsOnly)
CHUAH, Marinee, Khim, Lay [MY/BE]; BE (UsOnly)
Inventors:
VANDEN DRIESSCHE, Thierry; BE
CHUAH, Marinee, Khim, Lay; BE
Agent:
VAN SOMEREN, Petronella ; Arnold & Siedsma Sweelinckplein 1 NL-2517 GK The Hague, NL
Priority Data:
97201480.716.05.1997EP
98200382.409.02.1998EP
Title (EN) MAMMALIAN CELL TRANSDUCTION FOR USE IN GENE THERAPY
(FR) TRANSDUCTION DE CELLULES DE MAMMIFERES UTILISEE EN THERAPIE GENIQUE
Abstract:
(EN) The present invention relates to a method for the $i(ex vivo) transduction of mammalian cells, in particular to the transduction of bone marrow stromal cells. These cells can be transduced with a gene of interest, in particular a B-domain deleted human factor VIII gene. In the latter case, the transduced cells can be used to treat hemophilia A. The method for the $i(ex vivo) transduction of bone marrow stromal cells with the human factor VIII gene comprises provision of an intron-based retroviral vector comprising a B-domain deleted human factor VIII cDNA (designated as MFG-FVIII$g(D)B); pseudotyping the said vector with the Gibbon ape leukemia virus (GALV) envelope; transducing bone marrow stromal cells with the said pseudotyped vector by pre-incubating the cells for a suitable period of time in cell culture medium without phosphate and subsequently adding a vector-containing supernatant, optionally supplemented with transduction additives to the cells, followed by centrifuging the mixture thus obtained; and optionally repeating the two previous steps.
(FR) La présente invention concerne un procédé pour la transduction $i(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 $i(ex vivo) des cellules du stroma de la moelle osseuse avec le gène du facteur VIII de l'homme consiste à prévoir un vecteur rétroviral à base d'intron, comprenant un ADNc du facteur VIII de l'homme supprimé du domaine B (désigné par MFG-FVIII$g(D)B); 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.
Designated States: AL, AM, AT, AU, AZ, BA, BB, BG, BR, BY, CA, CH, CN, CU, CZ, DE, DK, EE, ES, FI, GB, GE, GH, GM, GW, HU, ID, IL, IS, JP, KE, KG, KP, KR, KZ, LC, LK, LR, LS, LT, LU, LV, MD, MG, MK, MN, MW, MX, NO, NZ, PL, PT, RO, RU, SD, SE, SG, SI, SK, SL, TJ, TM, TR, TT, UA, UG, US, UZ, VN, YU, ZW
African Regional Intellectual Property Organization (ARIPO) (GH, GM, KE, LS, MW, SD, SZ, UG, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, MD, RU, TJ, TM)
European Patent Office (AT, BE, CH, CY, DE, DK, ES, FI, FR, GB, GR, IE, IT, LU, MC, NL, PT, SE)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)
Also published as:
EP0980264CA2289918AU1998081057