CA2289918A1 - Transduction de cellules de mammiferes utilisee en therapie genique - Google Patents
Transduction de cellules de mammiferes utilisee en therapie genique Download PDFInfo
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- CA2289918A1 CA2289918A1 CA002289918A CA2289918A CA2289918A1 CA 2289918 A1 CA2289918 A1 CA 2289918A1 CA 002289918 A CA002289918 A CA 002289918A CA 2289918 A CA2289918 A CA 2289918A CA 2289918 A1 CA2289918 A1 CA 2289918A1
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- cells
- vector
- transduction
- bone marrow
- fviii
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Classifications
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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
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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
- 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
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- 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
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- 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)
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- Genetics & Genomics (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
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- General Health & Medical Sciences (AREA)
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- Medicinal Chemistry (AREA)
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- Pharmacology & Pharmacy (AREA)
- Developmental Biology & Embryology (AREA)
- Rheumatology (AREA)
- Cell Biology (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-FVIII.DELTA.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.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP97201480 | 1997-05-16 | ||
| EP97201480.7 | 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 |
| PCT/EP1998/003013 WO1998053063A2 (fr) | 1997-05-16 | 1998-05-18 | Transduction de cellules de mammiferes utilisee en therapie genique |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2289918A1 true CA2289918A1 (fr) | 1998-11-26 |
Family
ID=26146488
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA002289918A Abandoned CA2289918A1 (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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU6468199A (en) * | 1998-09-23 | 2000-04-10 | Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw | Method to treat haemophilia by (in vivo) gene therapy with retroviral vectors |
| EP1234047A1 (fr) * | 1999-12-01 | 2002-08-28 | Centre for Translational Research in Cancer | Systeme inductible par medicament inductible et utilisation associee |
| GB0325379D0 (en) * | 2003-10-30 | 2003-12-03 | Oxford Biomedica Ltd | Vectors |
Family Cites Families (6)
| 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 |
| AU3635695A (en) * | 1994-09-19 | 1996-04-09 | Board Of Trustees Of The Leland Stanford Junior University | Methods for genetically modifying hematopoietic stem cells |
| US5681746A (en) * | 1994-12-30 | 1997-10-28 | Chiron Viagene, Inc. | Retroviral delivery of full length factor VIII |
| AU5274696A (en) * | 1995-03-29 | 1996-10-16 | Bavarian Nordic Research Institute A/S | Pseudotyped retroviral particles |
| WO1997012968A1 (fr) * | 1995-10-05 | 1997-04-10 | Chiron Corporation | Vecteurs retroviraux pseudotypes a sequence de glycoproteines d'enveloppes srv-3 |
| AU721326B2 (en) * | 1996-02-13 | 2000-06-29 | Fred Hutchinson Cancer Research Center | 10A1 retroviral packaging cells and uses thereof |
-
1998
- 1998-05-18 EP EP98930718A patent/EP0980264A2/fr not_active Withdrawn
- 1998-05-18 WO PCT/EP1998/003013 patent/WO1998053063A2/fr not_active Application Discontinuation
- 1998-05-18 CA CA002289918A patent/CA2289918A1/fr not_active Abandoned
- 1998-05-18 AU AU81057/98A patent/AU8105798A/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP0980264A2 (fr) | 2000-02-23 |
| AU8105798A (en) | 1998-12-11 |
| WO1998053063A2 (fr) | 1998-11-26 |
| WO1998053063A3 (fr) | 1999-03-18 |
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