Thrombosis Research
Volume 126, Issue 5 , Pages 365-366 , November 2010

Monkey business for hemophilia management

Received 22 February 2010 ,Revised 25 February 2010 ,Accepted 25 February 2010.

References 

  1. Viiala NO, Larsen SR, Rasko JE. Gene therapy for hemophilia: clinical trials and technical tribulations. Semin Thromb Hemost. 2009;35(1):81–92
  2. Harding TC, Koprivnikar KE, Tu GH, Zayek N, Lew S, Subramanian A, et al. Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia B. Gene Ther. 2004;11:204–213
  3. Hasbrouck NC, High KA. AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects. Gene Ther. 2008;15(11):870–875
  4. Li C, Goudy K, Hirsch M, Asokan A, Fan Y, Alexander J, et al. Cellular immune response to cryptic epitopes during therapeutic gene transfer. Proc Natl Acad Sci U S A. 2009;106(26):10770–10774
  5. Ge Ying, Powell Sandra, Roey Melinda Van, McArthur James G. Factors influencing the development of an anti–factor IX (FIX) immune response following administration of adeno-associated virus–FIX. Blood. 2001;97(12):3733–3737
  6. Ishiwata A, Mimuro J, Mizukami H, Kashiwakura Y, Yasumoto A, Sakata A, et al. Mutant Macaque Factor IX T262A: A Tool for Hemophilia B Gene Therapy Studies in Macaques. Thromb Res. 2010;125(6):533–537
  7. Muramatsu S, Wang L, Ikeguchi K, Fujimoto K, Nakano I, Ozawa K. Recombinant adeno-associated viral vectors bring gene therapy for Parkinson's disease closer to reality. J Neurol. 2002;49(Suppl 2):II36–II40
  8. Favre D, Provost N, Blouin V, Blancho G, Chérel Y, Salvetti A, et al. Immediate and long-term safety of recombinant adeno-associated virus injection into the nonhuman primate muscle. Mol Ther. 2001;4(6):559–566
  9. Lozier JN, Metzger ME, Donahue RE, Morgan RA. The rhesus macaque as an animal model for hemophilia B gene therapy. Blood. 1999;93(6):1875–1881
  10. McCarthy K, Stewart P, Sigman J, Read M, Keith JC, Brinkhous KM, et al. Pharmacokinetics of recombinant factor IX after intravenous and subcutaneous administration in dogs and cynomolgus monkeys. Thromb Haemost. 2002;87(5):824–830
  11. Vandenberghe LH, Wang L, Somanathan S, Zhi Y, Figueredo J, Calcedo R, et al. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med. 2006;12(8):967–971
  12. Li C, Hirsch M, DiPrimio N, Asokan A, Goudy K, Tisch R, et al. Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo. J Virol. 2009;83(13):6817–6824
  13. Chen J, Wu Q, Yang P, Hsu HC, Mountz JD. Determination of specific CD4 and CD8 T cell epitopes after AAV2- and AAV8-hF.IX gene therapy. Mol Ther. 2006;13(2):260–269
  14. Jiang H, Lillicrap D, Patarroyo-White S, Liu T, Qian X, Scallan CD, et al. Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs. Blood. 2006;108(1):107–115
  15. Øvlisen K, Kristensen AT, Tranholm M. Haemophilia. In vivo models of haemophilia - status on current knowledge of clinical phenotypes and therapeutic interventions. Haemophilia. 2008;14(2):248–259

PII: S0049-3848(10)00163-5

doi: 10.1016/j.thromres.2010.02.016

Thrombosis Research
Volume 126, Issue 5 , Pages 365-366 , November 2010

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