Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver

Blood

Volumn 96, Issue 3, 2000, Pages 1173-1176

  Park, Frank ^a   Ohashi, Kazuo ^a   Kay, Mark A ^a  

^a STANFORD UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; VIRUS VECTOR;

ANIMAL EXPERIMENT; ARTICLE; CELL PROLIFERATION; CONTROLLED STUDY; DOSE RESPONSE; GENE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; HEMOPHILIA; HUMAN IMMUNODEFICIENCY VIRUS 1; LENTIVIRINAE; LIVER CELL; LIVER INJURY; MOUSE; NONHUMAN; PRIORITY JOURNAL;

EID: 0034254320     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v96.3.1173     Document Type: Article

References (24)

1. In vivo gene therapy of hemophilia B: Sustained partial correction in factor IX-deficient dogs
2. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII- deficient mice
3. Retroviral-mediated in vivo gene transfer into muscle cells and synthesis of human factor IX in mice
4. Construction of human factor IX expression vectors in retroviral frames optimized for muscle cells
5. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector
6. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
7. Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector
8. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors
9. Efficient lentiviral transduction of liver requires cell cycling in vivo
10. A third-generation lentivirus vector with a conditional packaging system
11. Transduction of human progenitor hematopoietic stem cells by human immunodeficiency virus type 1-based vectors is cell cycle dependent
12. Successful expression of human factor IX following repeat administration of an adenoviral vector in mice
13. Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice
14. Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy
15. Lentiviral delivery of HIV-1 Vpr protein induces apoptosis in transformed cells
16. Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells
17. Gene transfer by retro-virus vectors occurs only in cells that are actively replicating at the time of infection
18. Sustained correction of bleeding disorder in hemophilia B mice by gene therapy
19. AAV-mediated gene transfer of human blood coagulation factor IX into mouse liver
20. Persistence and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
21. Human immunodeficiency virus type 1 integrase mutant retain in vitro integrase activity yet fail to integrate viral DNA efficiently during infection
22. Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy
23. Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors
24. Effects of keritinocyte and hepatocyte growth factor in vivo: Implications for retrovirus-mediated gene transfer to liver