SEARCH

SEARCH BY CITATION

References

  • 1
    Lillicrap D, VandenDriessche T, High K. Cellular and genetic therapies for haemophilia. Haemophilia 2006; 12 (Suppl. 3): 3641.
  • 2
    Shi Q, Wilcox DA, Fahs SA, Weiler H, Wells CW, Cooley BC, Desai D, Morateck PA, Gorski J, Montgomery RR. Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies. J Clin Invest 2006; 116: 197482.
  • 3
    Chang AH, Stephan MT, Sadelain M. Stem cell-derived erythroid cells mediate long-term systemic protein delivery. Nat Biotechnol 2006; 24: 101721.
  • 4
    Moayeri M, Hawley TS, Hawley RG. Correction of murine hemophilia A by hematopoietic stem cell gene therapy. Mol Ther 2005; 12: 103442.
  • 5
    Thorrez L, Vandenburgh H, Callewaert N, Mertens N, Shansky J, Wang L, Arnout J, Collen D, Chuah M, Vandendriessche T. Angiogenesis enhances factor IX delivery and persistence from retrievable human bioengineered muscle implants. Mol Ther 2006; 14: 44251.
  • 6
    Xu L, Nichols TC, Sarkar R, McCorquodale S, Bellinger DA, Ponder KP. Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy. Proc Natl Acad Sci U S A 2005; 102: 60805.
  • 7
    VandenDriessche T, Vanslembrouck V, Goovaerts I, Zwinnen H, Vanderhaeghen ML, Collen D, Chuah MK. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice [see comments]. Proc Natl Acad Sci U S A 1999; 96: 1037984.
  • 8
    Kang Y, Xie L, Tran DT, Stein CS, Hickey M, Davidson BL, McCray PB Jr. Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer. Blood 2005; 106: 15528.
  • 9
    Follenzi A, Battaglia M, Lombardo A, Annoni A, Roncarolo MG, Naldini L. Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice. Blood 2004; 103: 37009.
  • 10
    Brown BD, Venneri MA, Zingale A, Sergi LS, Naldini L. Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat Med 2006; 12: 58591.
  • 11
    Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, De Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302: 4159.
  • 12
    Themis M, Waddington SN, Schmidt M, Von Kalle C, Wang Y, Al-Allaf F, Gregory LG, Nivsarkar M, Themis M, Holder MV, Buckley SM, Dighe N, Ruthe AT, Mistry A, Bigger B, Rahim A, Nguyen TH, Trono D, Thrasher AJ, Coutelle C. Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol Ther 2005; 12: 76371.
  • 13
    Olivares EC, Hollis RP, Chalberg TW, Meuse L, Kay MA, Calos MP. Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol 2002; 20: 11248.
  • 14
    Chalberg TW, Portlock JL, Olivares EC, Thyagarajan B, Kirby PJ, Hillman RT, Hoelters J, Calos MP. Integration specificity of phage phiC31 integrase in the human genome. J Mol Biol 2006; 357: 2848.
  • 15
    Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko J, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006; 12: 3427.
  • 16
    Jiang H, Pierce GF, Ozelo MC, De Paula EV, Vargas JA, Smith P, Sommer J, Luk A, Manno CS, High KA, Arruda VR. 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: 4525.
  • 17
    Jiang H, Lillicrap D, Patarroyo-White S, Liu T, Qian X, Scallan CD, Powell S, Keller T, McMurray M, Labelle A, Nagy D, Vargas JA, Zhou S, Couto LB, Pierce GF. delivering factor VIII to hemophilia A mice and dogs. Blood 2, 6, 10715.
  • 18
    Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A 2002; 99: 118549.
  • 19
    Thomas CE, Storm TA, Huang Z, Kay MA. Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J Virol 2004; 78: 311022.
  • 20
    VandenDriessche T, Thorrez L, Acosta-Sanchez A, Petrus I, Wang L, Ma L, De Waele L, Iwasaki Y, Gillijns V, Wilson JM, Collen D, Chuah MK. Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 versus lentiviral vectors for hemophilia B gene therapy. J Thromb Haemost 2006; 5: 1624.
  • 21
    Davidoff AM, Gray JT, Ng CY, Zhang Y, Zhou J, Spence Y, Bakar Y, Nathwani AC. capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models. Mol Ther 2, 5, 87588.
  • 22
    Nathwani AC, Gray JT, Ng CY, Zhou J, Spence Y, Waddington SN, Tuddenham EG, Kemball-Cook G, McIntosh J, Boon-Spijker M, Mertens K, Davidoff AM. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood 2006; 107: 265361.
  • 23
    Jiang H, Couto LB, Patarroyo-White S, Liu T, Nagy D, Vargas JA, Zhou S, Scallan CD, Sommer J, Vijay S, Warren D, Mingozzi F, High KA, Pierce GF. Effects of transient immunosuppression on adeno associated virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 2006; 108: 33213328.
  • 24
    Vandenberghe LH, Wang L, Somanathan S, Zhi Y, Figueredo J, Calcedo R, Sanmiguel J, Desai RA, Chen CS, Johnston J, Grant RL, Gao G, Wilson JM. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med 2006; 12: 96771.
  • 25
    Scallan CD, Jiang H, Liu T, Patarroyo-White S, Sommer JM, Zhou S, Couto LB, Pierce GF. Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice. Blood 2006; 107: 18107.
  • 26
    Arruda VR, Stedman HH, Nichols TC, Haskins ME, Nicholson M, Herzog RW, Couto LB, High KA. Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. Blood 2005; 105: 345864.
  • 27
    Perabo L, Endell J, King S, Lux K, Goldnau D, Hallek M, Buning H. Combinatorial engineering of a gene therapy vector: directed evolution of adeno-associated virus. J Gene Med 2006; 8: 15562.
  • 28
    Huttner NA, Girod A, Perabo L, Edbauer D, Kleinschmidt JA, Buning H, Hallek M. Genetic modifications of the adeno-associated virus type 2 capsid reduce the affinity and the neutralizing effects of human serum antibodies. Gene Ther 2003; 10: 213947.
  • 29
    Lochrie MA, Tatsuno GP, Arbetman AE, Jones K, Pater C, Smith PH, McDonnell JW, Zhou SZ, Kachi S, Kachi M, Campochiaro PA, Pierce GF, Colosi P. Adeno-associated virus (AAV) capsid genes isolated from rat and mouse liver genomic DNA define two new AAV species distantly related to AAV-5. Virology 2006; 353: 6882.
  • 30
    Brown BD, Lillicrap D. Dangerous liaisons: the role of ‘danger’ signals in the immune response to gene therapy. Blood 2002; 100: 113340.
  • 31
    Hausl C, Ahmad RU, Sasgary M, Doering CB, Lollar P, Richter G, Schwarz HP, Turecek PL, Reipert BM. High-dose factor VIII inhibits factor VIII-specific memory B cells in hemophilia A with factor VIII inhibitors. Blood 2005; 106: 341522.
  • 32
    Dobrzynski E, Fitzgerald JC, Cao O, Mingozzi F, Wang L, Herzog RW. Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induced regulatory T cells. Proc Natl Acad Sci U S A 2006; 103: 45927.
  • 33
    Pipe SW. Coagulation factors with improved properties for hemophilia gene therapy. Semin Thromb Hemost 2004; 30: 22737.
  • 34
    Miao HZ, Sirachainan N, Palmer L, Kucab P, Cunningham MA, Kaufman RJ, Pipe SW. Bioengineering of coagulation factor VIII for improved secretion. Blood 2004; 103: 34129.
  • 35
    Gale AJ, Pellequer JL. An engineered interdomain disulfide bond stabilizes human blood coagulation factor VIIIa. J Thromb Haemost 2003; 1: 196671.
  • 36
    Radtke KP, Griffin JH, Riceberg J, Gale AJ. Disulfide bond-stabilized factor VIII has prolonged factor VIIIa activity and improved potency in whole blood clotting assays. J Thromb Haemost 2006; 5: 102108.
  • 37
    Wakabayashi H, Su YC, Ahmad SS, Walsh PN, Fay PJ. A Glu113Ala mutation within a factor VIII Ca2+-binding site enhances cofactor interactions in factor Xase. Biochemistry 2005; 44: 10298304.
  • 38
    Chang JY, Monroe DM, Stafford DW, Brinkhous KM, Roberts HR. Replacing the first epidermal growth factor-like domain of factor IX with that of factor VII enhances activity in vitro and in canine hemophilia B. J Clin Invest 1997; 100: 88692.
  • 39
    Schuettrumpf J, Herzog RW, Schlachterman A, Kaufhold A, Stafford DW, Arruda VR. Factor IX variants improve gene therapy efficacy for hemophilia B. Blood 2005; 105: 231623.
  • 40
    Parker ET, Healey JF, Barrow RT, Craddock HN, Lollar P. Reduction of the inhibitory antibody response to human factor VIII in hemophilia A mice by mutagenesis of the A2 domain B-cell epitope. Blood 2004; 104: 70410.
  • 41
    James PD, Raut S, Rivard GE, Poon MC, Warner M, McKenna S, Leggo J, Lillicrap D. Aminoglycoside suppression of nonsense mutations in severe hemophilia. Blood 2005; 106: 30438.
  • 42
    Baru M, Carmel-Goren L, Barenholz Y, Dayan I, Ostropolets S, Slepoy I, Gvirtzer N, Fukson V, Spira J. Factor VIII efficient and specific non-covalent binding to PEGylated liposomes enables prolongation of its circulation time and haemostatic efficacy. Thromb Haemost 2005; 93: 10618.
  • 43
    Spira J, Plyushch OP, Andreeva TA, Andreev Y. Prolonged bleeding-free period following prophylactic infusion of recombinant factor VIII (Kogenate(R) FS) reconstituted with pegylated liposomes. Blood 2006; 108: 366873.
  • 44
    Negrier C. Gene therapy for hemophilia? Yes. J Thromb Haemost 2004; 2: 12345.
  • 45
    Giangrande PL. Gene therapy for hemophilia? No. J Thromb Haemost 2004; 2: 12367.
  • 46
    Tuddenham EG. Gene therapy for hemophilia? Gene therapy for hemophilia is both desirable and achievable in the near future. J Thromb Haemost 2005; 3: 1314.