Expression of human coagulation factor factor VIII in adipocytes transduced with the simian immunodeficiency virus agmTYO1 -based vector for hemophilia a gene therapy

Gene Therapy

Volumn 11, Issue 3, 2004, Pages 253-259

  Ogata, K ^a,c   Mimuro, J ^a   Kikuchi, J ^a   Tabata, T ^a   Ueda, Y ^b   Naito, M ^a   Madoiwa, S ^a   Takano, K ^a   Hasegawa, M ^b   Ozawa, K ^a   Sakata, Y ^a  

^a JICHI MEDICAL UNIVERSITY   (Japan)

^b DNAVEC Research Inc   (Japan)

^c TOKYO MEDICAL UNIVERSITY   (Japan)

Author keywords

Adipocyte; Hemophilia; Simian immunodeficiency virus vector

Indexed keywords

BETA GALACTOSIDASE; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR; VON WILLEBRAND FACTOR;

ADIPOCYTE; ADIPOSE TISSUE; ANIMAL; ANIMAL CELL; ANIMAL TISSUE; ANTIGEN DETECTION; ARTICLE; CELL CULTURE; CONTROLLED STUDY; DOSE RESPONSE; DRUG BLOOD LEVEL; GENE EXPRESSION; GENE THERAPY; GENE VECTOR; GENETIC TRANSCRIPTION; GENETIC TRANSDUCTION; GENETICS; HEMOPHILIA A; HUMAN; HUMAN CELL; IMMUNOFLUORESCENCE; IN VITRO STUDY; IN VIVO STUDY; METABOLISM; METHODOLOGY; MOUSE; MOUSE STRAIN; NONHUMAN; PRIORITY JOURNAL; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SIMIAN IMMUNODEFICIENCY VIRUS; TRANSGENE;

ADIPOCYTES; ANIMALS; BETA-GALACTOSIDASE; CELLS, CULTURED; FACTOR VIII; GENE THERAPY; GENETIC VECTORS; HEMOPHILIA A; HUMANS; MICE; MICE, INBRED C57BL; SIMIAN IMMUNODEFICIENCY VIRUS; TRANSDUCTION, GENETIC;

ANIMALIA; LENTIVIRUS; RNA VIRUSES; SIMIAE; SIMIAN IMMUNODEFICIENCY VIRUS;

EID: 10744226283     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3302174     Document Type: Article

References (22)

1. Hoyer LW. Hemophilia A. N Engl J Med 1994; 330: 38-47.
2. Kay MA, High K. Gene therapy for the hemophilias. Proc Natl Acad Sci USA 1999; 96: 9973-9975.
3. Mohamed-Ali V, Pinkney JH, Coppack SW. Adipose tissue as an endocrine and paracrine organ. Int J Obes Relat Metab Disord 1998; 22: 1145-1158.
4. Kaufman RJ, Wasley LC, Dorner AJ. Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells. J Biol Chem 1988: 6352-6362.
5. Levine JA, Eberhardt NL, Jensen MD, O'Brien T. Adenoviral-mediated gene transfer to human adipocytes in vitro, and human adipose tissue ex vivo and rabbit femoral adipose tissue in vivo. J Nutr Sci Vitaminol 1998; 44: 569-572.
6. y mice. FEBS Lett 2001; 509: 106-110.
7. Miyoshi H et al. Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 1999; 283: 682-686.
8. Chen W et al. Lentiviral vector transduction of hematopoietic stem cells that mediate long-term reconstitution of lethally irradiated mice. Stem Cells 2000; 18: 352-359.
9. Woods NB et al. Lentiviral gene transfer into primary and secondary NOD/SCID repopulating cells. Blood 2000; 96: 3725-3733.
10. Naldini L et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272: 263-267.
11. Honjo S et al. Experimental infection of African green monkeys and cynomolgus monkeys with a SIVAGM strain isolated from a healthy African green monkey. J Med Primatol 1990; 19: 9-20.
12. Roth DA, The factor FIII transkaryotic therapy study group et al. Implantation of non-viral ex vivo genetically modified autologous dermal fibroblaststhat express B-domain deleted human factor VIII in 12 severe hemophilia A study subjects. Blood 2002; 100 (Suppl): 116a (abstract #430).
13. Dwarki VJ et al. Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. Proc Natl Acad Sci USA 1995; 14: 1023-1027.
14. Halvorsen YC et al. Thiazolinediones and glucocortidoids synergistically induce differentiation of human adipose tissue stromal cells: biochemical, cellular, and molecular analysis. Metabolism 2001; 50: 407-413.
15. Harp JB, Franklin D, Vanderpuije AA, Gimble JM. Differential expression of signal transducers and activators of transcription during human adipogenesis. Biochem Biophys Res Commun 2001; 281: 907-912.
16. Weiss GH, Rosen OM, Rubin CS. Regulation of fatty acid synthetase concentration and activity during adipocyte differentiation. J Biol Chem 1980; 255: 4751-4757.
17. Mertens K et al. Biological activity of recombinant factor VIII variants lacking the central B-domain and the heavy-chain sequence Lys713-Arg740: discordant in vitro and in vivo activity. Br J Haematol 1993; 85: 133-142.
18. Lind P et al. Novel forms of B-domain-deleted recombinant factor VIII molecules. Construction and biochemical characterization. Eur J Biochem 1995; 232: 19-27.
19. Nakajima T et al. Development of novel simian immunodeficiency virus vectors carrying a dual gene expression system. Hum Gene Ther 2000; 11: 1863-1874.
20. Naito M et al. Defective sorting to secretory vesicles in the trans Golgi network is partly responsible for protein C deficiency: molecular mechanisms of impaired secretion of abnormal protein C R169W, R352W, and G376D. Circ Res 2003; 92: 865-872.
21. Chua Jr S-C et al. Phenotypes of mouse diabetes and rat fatty due to mutations in the OB (leptin) receptor. Science 1996; 271: 994-996.
22. Yonemura H et al. Efficient production of recombinant human factor VIII by co-expression of the heavy and light chains. Protein Eng 1993; 6: 669-674.