-
1
-
-
0002930753
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
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
|