-
1
-
-
0031715429
-
Molecular genetics of coagulation factor VIII gene and haemophilia A
-
Antonarakis S. Molecular genetics of coagulation factor VIII gene and haemophilia A. Haemophilia. 1998;4:1-11.
-
(1998)
Haemophilia
, vol.4
, pp. 1-11
-
-
Antonarakis, S.1
-
2
-
-
0030910204
-
Haemophilia prophylaxis in young patients - A long-term follow-up
-
Lofqvist T, Nilsson I, Berntorp E, Pettersson H. Haemophilia prophylaxis in young patients - a long-term follow-up. J Int Med. 1997;241:395-400.
-
(1997)
J Int Med.
, vol.241
, pp. 395-400
-
-
Lofqvist, T.1
Nilsson, I.2
Berntorp, E.3
Pettersson, H.4
-
3
-
-
0030833362
-
Prophylaxis in haemophilic children
-
Liesner R. Prophylaxis in haemophilic children. Blood Coagul Fibrinolysis. 1997;8:S7-10.
-
(1997)
Blood Coagul Fibrinolysis
, vol.8
-
-
Liesner, R.1
-
4
-
-
0031955272
-
Prophylactic use of factor VIII: An economic evaluation
-
Bohn R, Avorn J, Glynn R, Choodnovskiy I, Haschemeyer R, Aledort L. Prophylactic use of factor VIII: an economic evaluation. Thromb Haemost. 1998;79:932-937.
-
(1998)
Thromb Haemost.
, vol.79
, pp. 932-937
-
-
Bohn, R.1
Avorn, J.2
Glynn, R.3
Choodnovskiy, I.4
Haschemeyer, R.5
Aledort, L.6
-
5
-
-
0030000014
-
Prophylaxis and continuous infusion for hemophilia: Can we afford it?
-
Aledort L, Bohn R. Prophylaxis and continuous infusion for hemophilia: can we afford it? Blood Coagul Fibrinolysis. 1996;7:935-937.
-
(1996)
Blood Coagul Fibrinolysis
, vol.7
, pp. 935-937
-
-
Aledort, L.1
Bohn, R.2
-
6
-
-
0028842288
-
Gene therapy for hemophilia A: Production of therapeutic levels of human factor VIII in vivo in mice
-
Dwarki V, Belloni P, Nijjar T, et al. Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. Proc Natl Acad Sci USA. 1995;92:1023-1027.
-
(1995)
Proc Natl Acad Sci USA
, vol.92
, pp. 1023-1027
-
-
Dwarki, V.1
Belloni, P.2
Nijjar, T.3
-
7
-
-
0032080179
-
Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy
-
Connelly S, Andrews J, Gallo A, et al. Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy. Blood. 1998;91:3273-3281.
-
(1998)
Blood
, vol.91
, pp. 3273-3281
-
-
Connelly, S.1
Andrews, J.2
Gallo, A.3
-
8
-
-
0029943193
-
Sustained expression of therapeutic levels of human factor VIII in mice
-
Connelly S, Gardner J, Lyons R, McClelland A, Kaleko M. Sustained expression of therapeutic levels of human factor VIII in mice. Blood. 1996;87:4671-4677.
-
(1996)
Blood
, vol.87
, pp. 4671-4677
-
-
Connelly, S.1
Gardner, J.2
Lyons, R.3
McClelland, A.4
Kaleko, M.5
-
9
-
-
0029850534
-
Complete short-term correction of canine hemophilia A by in vivo gene therapy
-
Connelly S, Mount J, Mauser A, et al. Complete short-term correction of canine hemophilia A by in vivo gene therapy. Blood. 1996;88:3846-3853.
-
(1996)
Blood
, vol.88
, pp. 3846-3853
-
-
Connelly, S.1
Mount, J.2
Mauser, A.3
-
10
-
-
0032190675
-
Adeno-associated virus expression system for gene transfer
-
Rabinowitz J, Samulski J. Adeno-associated virus expression system for gene transfer. Curr Opin Biotechnol. 1998;9:470-475.
-
(1998)
Curr Opin Biotechnol.
, vol.9
, pp. 470-475
-
-
Rabinowitz, J.1
Samulski, J.2
-
11
-
-
17444372024
-
Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
-
Snyder R, Miao C, Meuse L, et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med. 1999;5:64-70.
-
(1999)
Nat Med.
, vol.5
, pp. 64-70
-
-
Snyder, R.1
Miao, C.2
Meuse, L.3
-
12
-
-
0022411248
-
Distribution of factor VIII mRNA and antigen in human liver and other tissues
-
Wion K, Kelly D, Summertield J, Tuddenham E, Lawn R. Distribution of factor VIII mRNA and antigen in human liver and other tissues. Nature. 1985;317:726-729.
-
(1985)
Nature
, vol.317
, pp. 726-729
-
-
Wion, K.1
Kelly, D.2
Summertield, J.3
Tuddenham, E.4
Lawn, R.5
-
13
-
-
0022401607
-
Ultrastructural localization of factor VIII procoagulant antigen in human liver hepatocytes
-
Zelechowska M, van Mourik J, Brodniewicz-Proba T. Ultrastructural localization of factor VIII procoagulant antigen in human liver hepatocytes. Nature. 1985;317:729-730.
-
(1985)
Nature
, vol.317
, pp. 729-730
-
-
Zelechowska, M.1
Van Mourik, J.2
Brodniewicz-Proba, T.3
-
14
-
-
0029849257
-
Quantitative analysis of the packaging capacity of recombinant adeno-associated virus
-
Dong J, Fan P, Frizzell R. Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum Gene Ther. 1996;7:2101-2112.
-
(1996)
Hum Gene Ther.
, vol.7
, pp. 2101-2112
-
-
Dong, J.1
Fan, P.2
Frizzell, R.3
-
15
-
-
0027319588
-
Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII
-
Pittman D, Alderman E, Tomkinson K, Wang J, Giles A, Kaufman R. Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII Blood. 1993;81:2925-2935.
-
(1993)
Blood
, vol.81
, pp. 2925-2935
-
-
Pittman, D.1
Alderman, E.2
Tomkinson, K.3
Wang, J.4
Giles, A.5
Kaufman, R.6
-
16
-
-
0025724964
-
Characterization of the hepatitis B virus enhl enhancer and X promoter complex
-
Guo W, Bell K, Ou J. Characterization of the hepatitis B virus Enhl enhancer and X promoter complex. J Virol. 1991;65:6686-6692.
-
(1991)
J Virol.
, vol.65
, pp. 6686-6692
-
-
Guo, W.1
Bell, K.2
Ou, J.3
-
17
-
-
0032862984
-
Persistent expression of canine factor IX in Hemophilia B canines
-
Chao H, Samulski R, Bellinger D, Monahan P, Nichols T, Walsh C. Persistent expression of canine factor IX in Hemophilia B canines. Gene Ther. 1999;6:1695-1704.
-
(1999)
Gene Ther.
, vol.6
, pp. 1695-1704
-
-
Chao, H.1
Samulski, R.2
Bellinger, D.3
Monahan, P.4
Nichols, T.5
Walsh, C.6
-
18
-
-
2642642141
-
Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus
-
Xiao X, Li J, Samulski R. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol. 1998;72:2224-2232.
-
(1998)
J Virol.
, vol.72
, pp. 2224-2232
-
-
Xiao, X.1
Li, J.2
Samulski, R.3
-
19
-
-
0029923638
-
Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors
-
Ferrari F, Samulski T, Shenk T, Samulski R, Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J Virol. 1996;70:3227-3234.
-
(1996)
J Virol.
, vol.70
, pp. 3227-3234
-
-
Ferrari, F.1
Samulski, T.2
Shenk, T.3
Samulski, R.4
-
21
-
-
0031775494
-
Adeno-associated virus as a vector for liver-directed gene therapy
-
Xiao W, Berta S, Lu M, Moscioni A, Tazelaar J, Wilson J. Adeno-associated virus as a vector for liver-directed gene therapy. J Virol. 1998;72:10,222-10,226.
-
(1998)
J Virol.
, vol.72
, pp. 10222-10226
-
-
Xiao, W.1
Berta, S.2
Lu, M.3
Moscioni, A.4
Tazelaar, J.5
Wilson, J.6
-
22
-
-
17444387438
-
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated 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 adeno-associated viral vector. Nat Med. 1999;5:56-63.
-
(1999)
Nat Med.
, vol.5
, pp. 56-63
-
-
Herzog, R.1
Yang, E.2
Couto, L.3
-
23
-
-
0031059825
-
Recombinant, B-domain deleted factor VIII (r-VIII SQ): Pharmacokinetics and initial safety aspects in hemophilia A patients
-
Fijnvandraat K, Berntorp E, ten Cate J. et al. Recombinant, B-domain deleted factor VIII (r-VIII SQ): pharmacokinetics and initial safety aspects in hemophilia A patients. Thromb Haemost. 1997;77:298-302.
-
(1997)
Thromb Haemost.
, vol.77
, pp. 298-302
-
-
Fijnvandraat, K.1
Berntorp, E.2
Ten Cate, J.3
-
24
-
-
0032958487
-
Human factor VIII can be packaged and functionally expressed in an adeno-associated virus background: Applicability to haemophilia A gene therapy
-
Gnatenko D, Saenko E, Jesty J, Cao L, Hearing P, Bahou W. Human factor VIII can be packaged and functionally expressed in an adeno-associated virus background: applicability to haemophilia A gene therapy. Br J Haematol. 1999;104:27-36.
-
(1999)
Br J Haematol.
, vol.104
, pp. 27-36
-
-
Gnatenko, D.1
Saenko, E.2
Jesty, J.3
Cao, L.4
Hearing, P.5
Bahou, W.6
-
25
-
-
1342272118
-
Inhibitor antibody development and T cell response to human factor VIII in murine hemophilia A
-
Qian J, Borovok M, Bi L, Kazazian HJ, Hoyer L. Inhibitor antibody development and T cell response to human factor VIII in murine hemophilia A. Thromb Haemost. 1999;81:240-244.
-
(1999)
Thromb Haemost.
, vol.81
, pp. 240-244
-
-
Qian, J.1
Borovok, M.2
Bi, L.3
Kazazian, H.J.4
Hoyer, L.5
-
26
-
-
0032525902
-
Therapeutic levels of human protein C in rats after retroviral vector-mediated hepatic gene therapy
-
Cai S-R, Kennedy S, Bowling W, Flye M, Ponder K. Therapeutic levels of human protein C in rats after retroviral vector-mediated hepatic gene therapy. J Clin Invest. 1998;101:2831-2841.
-
(1998)
J Clin Invest.
, vol.101
, pp. 2831-2841
-
-
Cai, S.-R.1
Kennedy, S.2
Bowling, W.3
Flye, M.4
Ponder, K.5
-
27
-
-
0030997346
-
Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
-
Snyder R, Miao C, Patijn G, et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet. 1997;16:270-276.
-
(1997)
Nat Genet.
, vol.16
, pp. 270-276
-
-
Snyder, R.1
Miao, C.2
Patijn, G.3
-
28
-
-
0033621126
-
Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII- deficient mice
-
VandenDriessche T, Vanslembrouck V, Goovaerts I, et al. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII- deficient mice. Proc Natl Acad Sci USA. 1999;96:10,379-10,384.
-
(1999)
Proc Natl Acad Sci USA
, vol.96
, pp. 10379-10384
-
-
VandenDriessche, T.1
Vanslembrouck, V.2
Goovaerts, I.3
-
29
-
-
0029920320
-
Neutralizing antiidiotypic antibodies to factor VIII inhibitors after desensitization in patients with hemophilia A
-
Gilles J, Desqueper B, Lenk H, Vermylen J, Saint-Remy J. Neutralizing antiidiotypic antibodies to factor VIII inhibitors after desensitization in patients with hemophilia A. J Clin Invest. 1996;97:1382-1388.
-
(1996)
J Clin Invest.
, vol.97
, pp. 1382-1388
-
-
Gilles, J.1
Desqueper, B.2
Lenk, H.3
Vermylen, J.4
Saint-Remy, J.5
-
30
-
-
0028240520
-
Immune tolerance in hemophilia-principal results from the international registry
-
Mariani G, Ghirardini A, Bellocco R. Immune tolerance in hemophilia-principal results from the International Registry. Thromb Haemost. 1994;72:155-158.
-
(1994)
Thromb Haemost.
, vol.72
, pp. 155-158
-
-
Mariani, G.1
Ghirardini, A.2
Bellocco, R.3
-
31
-
-
0026622961
-
Prevalence of inhibitors in a population of 3435 hemophilia patients in France
-
Sultan Y. Prevalence of inhibitors in a population of 3435 hemophilia patients in France. Thromb Haemost. 1992;67:600-602.
-
(1992)
Thromb Haemost.
, vol.67
, pp. 600-602
-
-
Sultan, Y.1
-
32
-
-
0032064006
-
The kinetics of rAAV integration in the liver
-
Miao C, Snyder R, Schowalter D, et al. The kinetics of rAAV integration in the liver. Nat Genet. 1998;19:13-15.
-
(1998)
Nat Genet.
, vol.19
, pp. 13-15
-
-
Miao, C.1
Snyder, R.2
Schowalter, D.3
-
33
-
-
0029655894
-
Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis
-
Fisher K, Gao G, Weitzman M, DeMatteo R, Burda J, Wilson J. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol. 1996;70:520-532.
-
(1996)
J Virol.
, vol.70
, pp. 520-532
-
-
Fisher, K.1
Gao, G.2
Weitzman, M.3
DeMatteo, R.4
Burda, J.5
Wilson, J.6
|