메뉴 건너뛰기




Volumn 116, Issue 26, 2010, Pages 5842-5848

Eradication of neutralizing antibodies to factor VIII In canine hemophilia a after liver gene therapy

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; NEUTRALIZING ANTIBODY;

EID: 78650647673     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2010-06-288001     Document Type: Article
Times cited : (128)

References (50)
  • 1
    • 0002930753 scopus 로고    scopus 로고
    • Hemophilia Aand hemophilia B
    • Beutler E. Coller B. Lichtman M. Kipps T. Seligsohn U, eds., 6th ed. New York, NY: McGraw-Hill
    • Roberts HR, Hoffman M. Hemophilia Aand hemophilia B. In: Beutler E. Coller B. Lichtman M. Kipps T. Seligsohn U, eds. Hematology. 6th ed. New York, NY: McGraw-Hill; 2001:1639-1657.
    • (2001) Hematology , pp. 1639-1657
    • Roberts, H.R.1    Hoffman, M.2
  • 2
    • 34250712964 scopus 로고    scopus 로고
    • Immune tolerance therapy for factor VIII inhibitors: Moving from empiricism to an evidence-based approach
    • Dimichele D. Immune tolerance therapy for factor VIII inhibitors: moving from empiricism to an evidence-based approach. J ThrombHaemost. 2007;5(suppl 1):143-150.
    • (2007) J. ThrombHaemost , vol.5 , Issue.1 SUPPL. , pp. 143-150
    • Dimichele, D.1
  • 3
    • 36348975208 scopus 로고    scopus 로고
    • Delays in maturation among adolescents with hemophilia And a history of inhibitors
    • Donfield SM, Lynn HS, Lail AE. Hoots WK. Berntorp E. Gomperts ED. Delays in maturation among adolescents with hemophilia And a history of inhibitors. Blood. 2007;110(10):3656-3661.
    • (2007) Blood , vol.110 , Issue.10 , pp. 3656-3661
    • Donfield, S.M.1    Lynn, H.S.2    Lail, A.E.3    Hoots, W.K.4    Berntorp, E.5    Gomperts, E.D.6
  • 4
    • 65849180700 scopus 로고    scopus 로고
    • Intensive peri-operative use of factor VIII and the Arg593→Cys mutation are risk factors for inhibitor development in mild/moderate hemophilia A
    • Eckhardt CL, Menke LA, van Ommen CH, et al. Intensive peri-operative use of factor VIII and the Arg593→Cys mutation are risk factors for inhibitor development in mild/moderate hemophilia A. J Thromb Haemost. 2009;7(6):930-937.
    • (2009) J. Thromb. Haemost. , vol.7 , Issue.6 , pp. 930-937
    • Eckhardt, C.L.1    Menke, L.A.2    Van Ommen, C.H.3
  • 5
    • 67649863784 scopus 로고    scopus 로고
    • Co-morbidity in the ageing haemophilia patient: The down side of increased life expectancy
    • Mauser-Bunschoten EP, Fransen Van De Putte DE, Schutgens RE. Co-morbidity in the ageing haemophilia patient: the down side of increased life expectancy. Haemophilia. 2009;15(4):853-863.
    • (2009) Haemophilia , vol.15 , Issue.4 , pp. 853-863
    • Mauser-Bunschoten, E.P.1    Fransen Van De Putte, D.E.2    Schutgens, R.E.3
  • 7
    • 69949099131 scopus 로고    scopus 로고
    • Risk factors for inhibitor formation in haemophilia: A prevalent case-control study
    • Ragni MV, Ojeifo O, Feng J, et al. Risk factors for inhibitor formation in haemophilia: a prevalent case-control study. Haemophilia. 2009;15(5):1074-1082.
    • (2009) Haemophilia , vol.15 , Issue.5 , pp. 1074-1082
    • Ragni, M.V.1    Ojeifo, O.2    Feng, J.3
  • 8
    • 64749096663 scopus 로고    scopus 로고
    • Inhibitors of factor VIII in black patients with hemophilia
    • Viel KR, Ameri A, Abshire TC, et al. Inhibitors of factor VIII in black patients with hemophilia. N EnglJ Med. 2009;360(16):1618-1627.
    • (2009) N. EnglJ Med. , vol.360 , Issue.16 , pp. 1618-1627
    • Viel, K.R.1    Ameri, A.2    Abshire, T.C.3
  • 9
    • 0017785868 scopus 로고
    • Massive factor-VIII infusion in haemophiliac with factor-VIII inhibitor, high responder
    • Brackmann HH, Gormsen J. Massive factor-VIII infusion in haemophiliac with factor-VIII inhibitor, high responder. Lancet. 1977;2(8044):933.
    • (1977) Lancet , vol.2 , Issue.8044 , pp. 933
    • Brackmann, H.H.1    Gormsen, J.2
  • 10
    • 33644820684 scopus 로고    scopus 로고
    • Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
    • 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(3):342-347.
    • (2006) Nat. Med. , vol.12 , Issue.3 , pp. 342-347
    • Manno, C.S.1    Pierce, G.F.2    Arruda, V.R.3
  • 11
    • 44249085878 scopus 로고    scopus 로고
    • Safety and efficacy of gene transfer for Leber's congenital amaurosis
    • Maguire AM, Simonelli F, Pierce EA, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N EnglJ Med. 2008;358(21):2240-2248.
    • (2008) N. EnglJ Med. , vol.358 , Issue.21 , pp. 2240-2248
    • Maguire, A.M.1    Simonelli, F.2    Pierce, E.A.3
  • 12
    • 44249120315 scopus 로고    scopus 로고
    • Effect of gene therapy on visual function in Leber's congenital amaurosis
    • Bainbridge JW, Smith AJ, Barker SS, et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med. 2008;358(21):2231-2239.
    • (2008) N. Engl. J. Med. , vol.358 , Issue.21 , pp. 2231-2239
    • Bainbridge, J.W.1    Smith, A.J.2    Barker, S.S.3
  • 13
    • 54949104686 scopus 로고    scopus 로고
    • Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: Short-term results of a phase I trial
    • Hauswirth WW, Aleman TS, Kaushal S, et al. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther. 2008;19(10):979-990.
    • (2008) Hum. Gene Ther. , vol.19 , Issue.10 , pp. 979-990
    • Hauswirth, W.W.1    Aleman, T.S.2    Kaushal, S.3
  • 14
    • 0013442744 scopus 로고    scopus 로고
    • AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
    • 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(8):2963-2972.
    • (2003) Blood , vol.101 , Issue.8 , pp. 2963-2972
    • Manno, C.S.1    Chew, A.J.2    Hutchison, S.3
  • 15
    • 33746909909 scopus 로고    scopus 로고
    • Evidence of multiyear Factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B
    • 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(3):452-455.
    • (2006) Mol. Ther. , vol.14 , Issue.3 , pp. 452-455
    • Jiang, H.1    Pierce, G.F.2    Ozelo, M.C.3
  • 16
    • 17444387438 scopus 로고    scopus 로고
    • Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adenoassociated viral vector
    • Herzog R, Yang E, Couto L, et al. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adenoassociated viral vector. Nature Med. 1999;5:56-63.
    • (1999) Nature Med. , vol.5 , pp. 56-63
    • Herzog, R.1    Yang, E.2    Couto, L.3
  • 17
    • 0037089331 scopus 로고    scopus 로고
    • Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
    • Mount JD, Herzog RW, Tillson DM, et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood. 2002;99(8):2670-2676.
    • (2002) Blood , vol.99 , Issue.8 , pp. 2670-2676
    • Mount, J.D.1    Herzog, R.W.2    Tillson, D.M.3
  • 18
    • 66149110999 scopus 로고    scopus 로고
    • Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency
    • Nichols TC, Dillow AM, Franck HW, et al. Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency. ILAR J. 2009;50(2):144-167.
    • (2009) ILAR J. , vol.50 , Issue.2 , pp. 144-167
    • Nichols, T.C.1    Dillow, A.M.2    Franck, H.W.3
  • 19
    • 33745606715 scopus 로고    scopus 로고
    • Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs
    • Jiang H, Lillicrap D, Patarroyo-White S, 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.
    • (2006) Blood , vol.108 , Issue.1 , pp. 107-115
    • Jiang, H.1    Lillicrap, D.2    Patarroyo-White, S.3
  • 20
    • 33645908797 scopus 로고    scopus 로고
    • Long-term efficacy of adeno-associated virus serotypes 8 and 9 in hemophilia Adogs and mice
    • Sarkar R, Mucci M, Addya S, et al. Long-term efficacy of adeno-associated virus serotypes 8 and 9 in hemophilia Adogs and mice. Hum Gene Ther. 2006;17(4):427-439.
    • (2006) Hum. Gene Ther. , vol.17 , Issue.4 , pp. 427-439
    • Sarkar, R.1    Mucci, M.2    Addya, S.3
  • 21
    • 33846934410 scopus 로고    scopus 로고
    • Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates
    • Nathwani AC, Gray JT, McIntosh J, et al. Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates. Blood. 2007;109(4):1414-1421.
    • (2007) Blood , vol.109 , Issue.4 , pp. 1414-1421
    • Nathwani, A.C.1    Gray, J.T.2    McIntosh, J.3
  • 22
    • 17044394814 scopus 로고    scopus 로고
    • Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy
    • Wang L, Calcedo R, Nichols TC, et al. Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood. 2005;105(8):3079-3086.
    • (2005) Blood , vol.105 , Issue.8 , pp. 3079-3086
    • Wang, L.1    Calcedo, R.2    Nichols, T.C.3
  • 23
    • 65549147773 scopus 로고    scopus 로고
    • Successful treatment of canine hemophilia by continuous expression of canine FVIIa
    • Margaritis P, Roy E, Aljamali MN, et al. Successful treatment of canine hemophilia By continuous expression of canine FVIIa. Blood. 2009;113(16):3682- 3689.
    • (2009) Blood , vol.113 , Issue.16 , pp. 3682-3689
    • Margaritis, P.1    Roy, E.2    Aljamali, M.N.3
  • 24
    • 73349120945 scopus 로고    scopus 로고
    • Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model
    • Sabatino DE, Freguia CF, Toso R, et al. Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model. Blood. 2009;114(20):4562-4565.
    • (2009) Blood , vol.114 , Issue.20 , pp. 4562-4565
    • Sabatino, D.E.1    Freguia, C.F.2    Toso, R.3
  • 25
    • 77952549040 scopus 로고    scopus 로고
    • Anovel monoclonal antibody specific for canine CD25 (P4A10): Selection and evaluation of canine Tregs
    • Abrams VK, Hwang B, Lesnikova M. et al. Anovel monoclonal antibody specific for canine CD25 (P4A10): selection and evaluation of canine Tregs. Vet Immunol Immunopathol. 2010;135(3-4): 257-265.
    • (2010) Vet. Immunol. Immunopathol , vol.135 , Issue.3-4 , pp. 257-265
    • Abrams, V.K.1    Hwang, B.2    Lesnikova, M.3
  • 26
    • 0036212973 scopus 로고    scopus 로고
    • Aberrant splicing and premature termination of transcription of the FVIII gene as a cause of severe canine hemophilia A: Similarities with the intron 22 inversion mutation in human hemophilia
    • Hough C, Kamisue S, Cameron C, et al. Aberrant splicing and premature termination of transcription of the FVIII gene as a cause of severe canine hemophilia A: similarities with the intron 22 inversion mutation in human hemophilia. Thromb Haemost. 2002;87(4):659-665.
    • (2002) Thromb. Haemost. , vol.87 , Issue.4 , pp. 659-665
    • Hough, C.1    Kamisue, S.2    Cameron, C.3
  • 27
    • 0036790813 scopus 로고    scopus 로고
    • The Chapel Hill hemophilia A dog colony exhibits a factor VIII gene inversion
    • Lozier JN, Dutra A, Pak E, et al. The Chapel Hill hemophilia A dog colony exhibits a factor VIII gene inversion. Proc Natl Acad Sci U S A. 2002;99(20):12991-12996.
    • (2002) Proc. Natl. Acad. Sci. U S A , vol.99 , Issue.20 , pp. 12991-12996
    • Lozier, J.N.1    Dutra, A.2    Pak, E.3
  • 28
    • 0029095603 scopus 로고
    • Factor VIII gene inversions in severe hemophilia A: Results of an international consortium study
    • Antonarakis SE, Rossiter JP, Young M. Factor VIII gene inversions in severe hemophilia A: Results of an international consortium study. Blood. 1995;86:2206-2212.
    • (1995) Blood , vol.86 , pp. 2206-2212
    • Antonarakis, S.E.1    Rossiter, J.P.2    Young, M.3
  • 29
    • 0021318961 scopus 로고
    • Development of factor VIII:C antibodies in dogs with hemophilia A (factor VIII:C deficiency)
    • Giles AR, Tinlin S, Hoogendoorn H. Greenwood P. Greenwood R. Development of factor VIII:C antibodies in dogs with hemophilia A (factor VIII:C deficiency). Blood. 1984;63(2):451-456.
    • (1984) Blood , vol.63 , Issue.2 , pp. 451-456
    • Giles, A.R.1    Tinlin, S.2    Hoogendoorn, H.3    Greenwood, P.4    Greenwood, R.5
  • 30
    • 0027389602 scopus 로고
    • The development of homologous (canine/anti-canine) antibodies in dogs with hemophilia A (factor VIII deficiency): A ten year longitudinal study
    • Tinlin S, Webster S, Giles AR. The development of homologous (canine/anti-canine) antibodies in dogs with hemophilia A (factor VIII deficiency): a ten year longitudinal study. Thromb Haemost. 1993;69:21-24.
    • (1993) Thromb. Haemost. , vol.69 , pp. 21-24
    • Tinlin, S.1    Webster, S.2    Giles, A.R.3
  • 31
    • 0036376642 scopus 로고    scopus 로고
    • Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy
    • Herzog RW, Fields PA, Arruda VR, et al. Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy. Hum Gene Ther. 2002;13:1281-1291.
    • (2002) Hum. Gene Ther. , vol.13 , pp. 1281-1291
    • Herzog, R.W.1    Fields, P.A.2    Arruda, V.R.3
  • 32
    • 0036588771 scopus 로고    scopus 로고
    • Inhibitors: Resolving diagnostic and therapeutic dilemmas
    • Dimichele D. Inhibitors: resolving diagnostic and therapeutic dilemmas. Haemophilia. 2002;8(3):280-287.
    • (2002) Haemophilia , vol.8 , Issue.3 , pp. 280-287
    • Dimichele, D.1
  • 33
    • 28844449356 scopus 로고    scopus 로고
    • The inhibitory effects of anticoagulation on in vivo gene transfer by adeno-associated viral or adenoviral vectors
    • Schuettrumpf J, Zou J, Zhang Y, et al. The inhibitory effects of anticoagulation on in vivo gene transfer by adeno-associated viral or adenoviral vectors. Mol Ther. 2006;13(1):88-97.
    • (2006) Mol. Ther. , vol.13 , Issue.1 , pp. 88-97
    • Schuettrumpf, J.1    Zou, J.2    Zhang, Y.3
  • 34
    • 0033607305 scopus 로고    scopus 로고
    • Coexpression of factor VIII heavy and light chain adeno-associated virus vectors produces biologically active protein
    • Burton M, Nakai H, Colosi P. Cunningham J. Mitchell R. Couto L. Coexpression of factor VIII heavy and light chain adeno-associated virus vectors produces biologically active protein. Proc Natl Acad Sci U S A 1999;26:12725-12730.
    • (1999) Proc. Natl. Acad. Sci. U S A , vol.26 , pp. 12725-12730
    • Burton, M.1    Nakai, H.2    Colosi, P.3    Cunningham, J.4    Mitchell, R.5    Couto, L.6
  • 35
    • 10744230709 scopus 로고    scopus 로고
    • Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII
    • Scallan CD, Liu T, Parker AE, et al. Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII. Blood. 2003;102(12):3919-3926.
    • (2003) Blood , vol.102 , Issue.12 , pp. 3919-3926
    • Scallan, C.D.1    Liu, T.2    Parker, A.E.3
  • 36
    • 27744530253 scopus 로고    scopus 로고
    • High-dose factor VIII inhibits factor VIII-specific memory B cells in hemophilia Awith factor VIII inhibitors
    • Hausl C, Ahmad RU, Sasgary M, et al. High-dose factor VIII inhibits factor VIII-specific memory B cells in hemophilia Awith factor VIII inhibitors. Blood. 2005;106(10):3415-3422.
    • (2005) Blood , vol.106 , Issue.10 , pp. 3415-3422
    • Hausl, C.1    Ahmad, R.U.2    Sasgary, M.3
  • 37
    • 34547498762 scopus 로고    scopus 로고
    • Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer
    • Cao O, Dobrzynski E, Wang L, et al. Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer. Blood. 2007;110(4):1132-1140.
    • (2007) Blood , vol.110 , Issue.4 , pp. 1132-1140
    • Cao, O.1    Dobrzynski, E.2    Wang, L.3
  • 38
    • 34948855431 scopus 로고    scopus 로고
    • Modulation of tolerance to the transgene product in anonhuman primate model of AAV-mediated gene transfer to liver
    • Mingozzi F, Hasbrouck NC, Basner-Tschakarjan E, et al. Modulation of tolerance to the transgene product in anonhuman primate model of AAV-mediated gene transfer to liver. Blood. 2007;110(7):2334-2341.
    • (2007) Blood , vol.110 , Issue.7 , pp. 2334-2341
    • Mingozzi, F.1    Hasbrouck, N.C.2    Basner-Tschakarjan, E.3
  • 39
    • 0037988905 scopus 로고    scopus 로고
    • Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
    • Mingozzi F, Liu Y-L, Dobrzynski E, et al. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J Clin Invest. 2003;111:1347-1356.
    • (2003) J. Clin. Invest. , vol.111 , pp. 1347-1356
    • Mingozzi, F.1    Liu, Y.-L.2    Dobrzynski, E.3
  • 40
    • 73949157699 scopus 로고    scopus 로고
    • In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance
    • Annoni A, Brown BD, Cantore A. Sergi LS. Naldini L. Roncarolo MG. In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance. Blood. 2009;114(25):5152-5161.
    • (2009) Blood , vol.114 , Issue.25 , pp. 5152-5161
    • Annoni, A.1    Brown, B.D.2    Cantore, A.3    Sergi, L.S.4    Naldini, L.5    Roncarolo, M.G.6
  • 41
    • 0019973659 scopus 로고
    • Acanine model of hemophilic (factor VIII:C deficiency) bleeding
    • Giles AR, Tinlin S, Greenwood R. Acanine model of hemophilic (factor VIII:C deficiency) bleeding. Blood. 1982;60:727-730.
    • (1982) Blood , vol.60 , pp. 727-730
    • Giles, A.R.1    Tinlin, S.2    Greenwood, R.3
  • 42
    • 69249206732 scopus 로고    scopus 로고
    • The molecular mechanisms of immunomodulation and tolerance induction to factor VIII
    • Waters B, Lillicrap D. The molecular mechanisms of immunomodulation and tolerance induction to factor VIII. J Thromb Haemost. 2009;7(9):1446-1456.
    • (2009) J. Thromb. Haemost. , vol.7 , Issue.9 , pp. 1446-1456
    • Waters, B.1    Lillicrap, D.2
  • 43
    • 33845485057 scopus 로고    scopus 로고
    • Mechanisms of action of immune tolerance induction against factor VIII in patients with congenital haemophilia A and factor VIII inhibitors
    • Reipert BM, van Helden PM, Schwarz HP. Hausl C. Mechanisms of action of immune tolerance induction against factor VIII in patients with congenital haemophilia A and factor VIII inhibitors. Br J Haematol. 2007;136(1):12-25.
    • (2007) Br. J. Haematol. , vol.136 , Issue.1 , pp. 12-25
    • Reipert, B.M.1    Van Helden, P.M.2    Schwarz, H.P.3    Hausl, C.4
  • 44
    • 0037589022 scopus 로고    scopus 로고
    • Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B
    • Xu L, Gao C, Sands MS, et al. Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B. Blood. 2003;101(10):3924-3932.
    • (2003) Blood , vol.101 , Issue.10 , pp. 3924-3932
    • Xu, L.1    Gao, C.2    Sands, M.S.3
  • 45
    • 17844372996 scopus 로고    scopus 로고
    • Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy
    • 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(17):6080-6085.
    • (2005) Proc. Natl. Acad. Sci. U S A , vol.102 , Issue.17 , pp. 6080-6085
    • Xu, L.1    Nichols, T.C.2    Sarkar, R.3    McCorquodale, S.4    Bellinger, D.A.5    Ponder, K.P.6
  • 46
    • 20444476217 scopus 로고    scopus 로고
    • 2 domains presented by B cells as Ig fusion proteins
    • 2 domains presented by B cells as Ig fusion proteins. Blood. 2005;105(12):4865-4870.
    • (2005) Blood , vol.105 , Issue.12 , pp. 4865-4870
    • Lei, T.C.1    Scott, D.W.2
  • 47
    • 73349111285 scopus 로고    scopus 로고
    • 3 antibodies modulate anti-factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy
    • 3 antibodies modulate anti-factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy. Blood. 2009;114(20):4373-4382.
    • (2009) Blood , vol.114 , Issue.20 , pp. 4373-4382
    • Peng, B.1    Ye, P.2    Rawlings, D.J.3    Ochs, H.D.4    Miao, C.H.5
  • 48
    • 35349015845 scopus 로고    scopus 로고
    • Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens
    • Ide LM, Gangadharan B, Chiang KY. Doering CB. Spencer HT. Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens. Blood. 2007;110(8):2855-2863.
    • (2007) Blood , vol.110 , Issue.8 , pp. 2855-2863
    • Ide, L.M.1    Gangadharan, B.2    Chiang, K.Y.3    Doering, C.B.4    Spencer, H.T.5
  • 49
    • 48749103550 scopus 로고    scopus 로고
    • Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I
    • Dickson P, Peinovich M, McEntee M, et al. Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I. J Clin Invest. 2008;118(8):2868-2876.
    • (2008) J. Clin. Invest. , vol.118 , Issue.8 , pp. 2868-2876
    • Dickson, P.1    Peinovich, M.2    McEntee, M.3
  • 50
    • 49449090469 scopus 로고    scopus 로고
    • Neutralizing antibodies to therapeutic enzymes: Considerations for testing, prevention and treatment
    • Wang J, Lozier J, Johnson G, et al. Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment. Nat Biotechnol. 2008;26(8):901-908.
    • (2008) Nat. Biotechnol. , vol.26 , Issue.8 , pp. 901-908
    • Wang, J.1    Lozier, J.2    Johnson, G.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.