Non-viral gene transfer results in therapeutic factor IX levels in haemophilia B mice;Nicht-viraler gentransfer führt zu therapeutischen faktor-IX-spiegeln im hämophilie-B-mausmodell

Hamostaseologie

Volumn 28, Issue 4 A/SUPPL. 1, 2008, Pages

  Schüttrumpf, Jörg ^a   Milanov, P ^a   Roth, S ^a   Seifried, E ^a   Tonn, T ^a  

^a UNIVERSITY HOSPITAL FRANKFURT   (Germany)

Author keywords

Factor IX; Gene therapy; Haemophilia B

Indexed keywords

BLOOD CLOTTING FACTOR 9; PLASMID VECTOR; PROTEIN ANTIBODY;

ANIMAL EXPERIMENT; ANIMAL MODEL; CONFERENCE PAPER; DOSE RESPONSE; GENE EXPRESSION; GENE TRANSFER; HEMOPHILIA B; HYDRODYNAMICS BASED TRANSFECTION; KNOCKOUT MOUSE; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NONVIRAL GENE THERAPY; PROTEIN BLOOD LEVEL;

EID: 54549112149     PISSN: 07209355     EISSN: None     Source Type: Journal    
DOI: 10.1055/s-0037-1621423     Document Type: Conference Paper

References (12)

1. Manno CS, Chew AJ, Hutchison S et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 2003; 101: 2963-2972.
2. Jiang H, Pierce GF, Ozelo MC et al. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Mol Ther 2006; 14: 452-455.
3. Manno CS, Pierce GF, Arruda VR et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006; 12: 342-347.
4. Fewell JG, MacLaughlin F, Mehta V et al. Gene therapy for the treatment of hemophilia B using PINC-formulated plasmid delivered to muscle with electroporation. Mol Ther 2001; 3: 574-583.
5. Ye X, Loeb KR, Stafford DW et al. Complete and sustained phenotypic correction of hemophilia B in mice following hepatic gene transfer of a high-expressing human factor IX plasmid. J Thromb Haemost 2003; 1: 103-111.
6. Miao CH, Thompson AR, Loeb K et al. Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo. Mol Ther 2001; 3: 947-957.
7. Schuettrumpf J, Herzog RW, Schlachterman A et al. Factor IX variants improve gene therapy efficacy for hemophilia B. Blood 2005; 105: 2316-2323.
8. Fabre JW, Grehan A, Whitehorne M et al. Hydrodynamic gene delivery to the pig liver via an isolated segment of the inferior vena cava. Gene Ther 2008; 15: 452-462.
9. Khorsandi SE, Bachellier P, Weber JC et al. Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human. Cancer Gene Ther 2008; 15: 225-230.
10. Alino SF, Herrero MJ, Noguera I et al. Pig liver gene therapy by noninvasive interventionist catheterism. Gene Ther 2007; 14: 334-343.
11. Yoshino H, Hashizume K, Kobayashi E. Naked plasmid DNA transfer to the porcine liver using rapid injection with large volume. Gene Ther 2006; 13: 1696-1702.
12. Vigen KK, Hegge JO, Zhang G et al. Magnetic resonance imaging-monitored plasmid DNA delivery in primate limb muscle. Hum Gene Ther 2007; 18: 257-68.