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Perspective

Will gene therapy trump factor treatment in hemophilia?

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Pages 43-48 | Published online: 10 Jan 2014

References

  • Key NS, Negrier C. Coagulation factor concentrates: past, present, and future. Lancet 370(9585), 439–448 (2007).
  • Colvin BT, Astermark J, Fischer K et al.; Inter Disciplinary Working Group. European principles of haemophilia care. Haemophilia 14(2), 361–374 (2008).
  • Collins PW, Björkman S, Fischer K et al. Factor VIII requirement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A: influences of variance in pharmacokinetics and treatment regimens. J. Thromb. Haemost. 8(2), 269–275 (2010).
  • Berntorp E. Joint outcomes in patients with haemophilia: the importance of adherence to preventive regimens. Haemophilia 15(6), 1219–1227 (2009).
  • Powell JS, Nugent DJ, Harrison JA et al. Safety and pharmacokinetics of a recombinant factor VIII with pegylated liposomes in severe hemophilia A. J. Thromb. Haemost. 6(2), 277–283 (2008).
  • Spira J, Plyushch OP, Andreeva TA, Andreev Y. Prolonged bleeding-free period following prophylactic infusion of recombinant factor VIII reconstituted with pegylated liposomes. Blood 108(12), 3668–3673 (2006).
  • Mei B, Pan C, Jiang H et al. Rational design of a fully active, long-acting pegylated factor VIII for hemophilia A treatment. Blood 116(2), 270–279 (2010).
  • Peters RT, Low SC, Kamphaus GD et al. Prolonged activity of factor IX as a monomeric Fc fusion protein. Blood 115(10), 2057–2064 (2010).
  • Pipe SW, Kaufman RJ. Characterization of a genetically engineered inactivation-resistant coagulation factor VIIIa. Proc. Natl Acad. Sci. USA 94(22), 11851–11856 (1997).
  • Shapiro AD, Ragni MV, Valentino LA et al. Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a Phase 1/2a study in hemophilia B patients. Blood 119(3), 666–672 (2012).
  • High KA. Gene therapy for haemophilia: a long and winding road. J. Thromb. Haemost. 9(Suppl. 1), 2–11 (2011).
  • High KA. The gene therapy journey for hemophilia: are we there yet? Hematology Am. Soc. Hematol. Educ. Program 2012, 375–381 (2012).
  • 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. 12(3), 342–347 (2006).
  • Mingozzi F, Maus MV, Hui DJ et al. CD8+ T-cell responses to adeno-associated virus capsid in humans. Nat. Med. 13(4), 419–422 (2007).
  • Nathwani AC, Tuddenham EG, Rangarajan S et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med. 365(25), 2357–2365 (2011).
  • Finn JD, Nichols TC, Svoronos N et al. The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy. Blood 120(23), 4521–4523 (2012).
  • Arruda VR, Stedman HH, Nichols TC et al. Regional intravascular delivery of AAV-2-FIX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. Blood 105(9), 3458–3464 (2005).
  • Huttner NA, Girod A, Perabo L et al. Genetic modifications of the adeno-associated virus type 2 capsid reduce the affinity and the neutralizing effects of human serum antibodies. Gene Ther. 10(26), 2139–2147 (2003).
  • Donsante A, Vogler C, Muzyczka N et al. Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. Gene Ther. 8(17), 1343–1346 (2001).
  • Finn JD, Ozelo MC, Sabatino DE et al. Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy. Blood 116(26), 5842–5848 (2010).
  • Roth DA, Tawa NE Jr, O’Brien JM, Treco DA, Selden RF; Factor VIII Transkaryotic Therapy Study Group. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N. Engl. J. Med. 344(23), 1735–1742 (2001).
  • Du LM, Nichols TC, Haberichter SL et al. Platelet-targeted expression of human BDD-FVIII reduces bleeding in canine hemophilia A. Presented at: 51st ASH Annual Meeting and Exposition. New Orleans, LA, USA, 5–8 December 2009.
  • Matsui H, Shibata M, Brown B et al. Ex vivo gene therapy for hemophilia A that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors. Stem Cells 25(10), 2660–2669 (2007).
  • Ozelo M, Matsui H, Hegadorn C. Genetically engineered endothelial cells implanted into the omentum of hemophilia A dogs provides long-term circulating FVIII resulting from sustained FVIII expression and persistent cell viability. Blood 114, 1386 (2009).
  • Sun J, Hakobyan N, Valentino LA, Feldman BL, Samulski RJ, Monahan PE. Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX. Blood 112(12), 4532–4541 (2008).
  • Simioni P, Tormene D, Tognin G et al. X-linked thrombophilia with a mutant factor IX (factor IX Padua). N. Engl. J. Med. 361(17), 1671–1675 (2009).
  • Shi Q, Wilcox DA, Fahs SA, Kroner PA, Montgomery RR. Expression of human factor VIII under control of the platelet-specific α IIb promoter in megakaryocytic cell line as well as storage together with vWF. Mol. Genet. Metab. 79(1), 25–33 (2003).
  • Rosenberg JB, Greengard JS, Montgomery RR. Genetic induction of a releasable pool of factor VIII in human endothelial cells. Arterioscler. Thromb. Vasc. Biol. 20(12), 2689–2695 (2000).
  • Shi Q, Wilcox DA, Fahs SA et al. Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies. J. Clin. Invest. 116(7), 1974–1982 (2006).
  • Shi Q, Wilcox D, Fahs S et al. Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A. J. Thromb. Haemost. 5(2), 352–361 (2007).

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