-
1
-
-
0032696798
-
Regulation of factor VIII expression and activity by von Willebrand factor
-
Kaufman, RJ and Pipe, SW (1999). Regulation of factor VIII expression and activity by von Willebrand factor. Thromb Haemost 82: 201-208.
-
(1999)
Thromb Haemost
, vol.82
, pp. 201-208
-
-
Kaufman, R.J.1
Pipe, S.W.2
-
2
-
-
1942521325
-
Bioengineering of coagulation factor VIII for improved secretion
-
Miao, HZ, Sirachainan, N, Palmer, L, Kucab, P, Cunningham, MA, Kaufman, RJ et al. (2004). Bioengineering of coagulation factor VIII for improved secretion. Blood 103: 3412-3419.
-
(2004)
Blood
, vol.103
, pp. 3412-3419
-
-
Miao, H.Z.1
Sirachainan, N.2
Palmer, L.3
Kucab, P.4
Cunningham, M.A.5
Kaufman, R.J.6
-
3
-
-
0030664471
-
Characterization of a genetically engineered inactivation-resistant coagulation factor VIIIa
-
Pipe, SW and Kaufman, RJ (1997). Characterization of a genetically engineered inactivation-resistant coagulation factor VIIIa. Proc Natl Acad Sci USA 94: 11851-11856.
-
(1997)
Proc Natl Acad Sci USA
, vol.94
, pp. 11851-11856
-
-
Pipe, S.W.1
Kaufman, R.J.2
-
4
-
-
0032572579
-
Mistargeting of the lectin ERGIC-53 to the endoplasmic reticulum of HeLa cells impairs the secretion of a lysosomal enzyme
-
Vollenweider, F, Kappeler, F, Itin, C and Hauri, HP (1998). Mistargeting of the lectin ERGIC-53 to the endoplasmic reticulum of HeLa cells impairs the secretion of a lysosomal enzyme. J Cell Biol 142 377-389.
-
(1998)
J Cell Biol
, vol.142
, pp. 377-389
-
-
Vollenweider, F.1
Kappeler, F.2
Itin, C.3
Hauri, H.P.4
-
5
-
-
0031017668
-
Selective packaging of cargo molecules into endoplasmic reticulum-derived COPII vesicles
-
Campbell, JL and Schekman, R (1997). Selective packaging of cargo molecules into endoplasmic reticulum-derived COPII vesicles. Proc Natl Acad Sci USA 94: 837-842.
-
(1997)
Proc Natl Acad Sci USA
, vol.94
, pp. 837-842
-
-
Campbell, J.L.1
Schekman, R.2
-
6
-
-
0031450221
-
The recycling of ERGIC-53 in the early secretary pathway. ERGIC-53 carries a cytosolic endoplasmic reticulum-exit determinant interacting with COPII
-
Kappeler, F, Klopfenstein, DR, Foguet, M, Paccaud, JP and Hauri, HP (1997). The recycling of ERGIC-53 in the early secretary pathway. ERGIC-53 carries a cytosolic endoplasmic reticulum-exit determinant interacting with COPII. J Biol Chem 272: 31801-31808.
-
(1997)
J Biol Chem
, vol.272
, pp. 31801-31808
-
-
Kappeler, F.1
Klopfenstein, D.R.2
Foguet, M.3
Paccaud, J.P.4
Hauri, H.P.5
-
7
-
-
2342542505
-
Coagulation factors with improved properties for hemophilia gene therapy
-
Pipe, SW (2004). Coagulation factors with improved properties for hemophilia gene therapy. Semin Thromb Hemost 30: 227-237.
-
(2004)
Semin Thromb Hemost
, vol.30
, pp. 227-237
-
-
Pipe, S.W.1
-
8
-
-
0031049699
-
Persistence in muscle of an adenoviral vector that lacks all viral genes
-
Chen, HH, Mack, LM, Kelly, R, Ontell, M, Kochanek, S and Clemens, PR (1997). Persistence in muscle of an adenoviral vector that lacks all viral genes. Proc Natl Acad Sci USA 94: 1645-1650.
-
(1997)
Proc Natl Acad Sci USA
, vol.94
, pp. 1645-1650
-
-
Chen, H.H.1
Mack, L.M.2
Kelly, R.3
Ontell, M.4
Kochanek, S.5
Clemens, P.R.6
-
9
-
-
0030809684
-
Complementation of helper-dependent adenoviral vectors: Size effects and titer fluctuations
-
Alemany, R, Dai, Y, Lou, YC, Sethi, E, Prokopenko, E, Josephs, SF et al. (1997). Complementation of helper-dependent adenoviral vectors: size effects and titer fluctuations. J Virol Methods 68: 147-159.
-
(1997)
J Virol Methods
, vol.68
, pp. 147-159
-
-
Alemany, R.1
Dai, Y.2
Lou, Y.C.3
Sethi, E.4
Prokopenko, E.5
Josephs, S.F.6
-
10
-
-
0030900511
-
A helper-dependent system for adenovirus vector production helps define a lower limit for efficient DNA packaging
-
Parks, RJ and Graham, FL (1997). A helper-dependent system for adenovirus vector production helps define a lower limit for efficient DNA packaging. J Virol 71: 3293-3298.
-
(1997)
J Virol
, vol.71
, pp. 3293-3298
-
-
Parks, R.J.1
Graham, F.L.2
-
11
-
-
0036171875
-
Generation of helper-dependent adenoviral vectors by homologous recombination
-
Toietta, G, Pastore, L, Cerullo, V, Finegold, M, Beaudet, AL and Lee, B (2002). Generation of helper-dependent adenoviral vectors by homologous recombination. Mol Ther 5: 204-210.
-
(2002)
Mol Ther
, vol.5
, pp. 204-210
-
-
Toietta, G.1
Pastore, L.2
Cerullo, V.3
Finegold, M.4
Beaudet, A.L.5
Lee, B.6
-
12
-
-
0346872991
-
Improved system for helper-dependent adenoviral vector production
-
Palmer, D and Ng, P (2003). Improved system for helper-dependent adenoviral vector production. Mol Ther 8: 846-852.
-
(2003)
Mol Ther
, vol.8
, pp. 846-852
-
-
Palmer, D.1
Ng, P.2
-
13
-
-
4444333453
-
Long-term correction of ornithine transcarbamylase deficiency by WPRE-mediated overexpression using a helper-dependent adenovirus
-
Mian, A, McCormack, WM Jr., Mane, V, Kleppe, S, Ng, P, Finegold, M et al. (2004). Long-term correction of ornithine transcarbamylase deficiency by WPRE-mediated overexpression using a helper-dependent adenovirus. Mol Ther 10: 492-499.
-
(2004)
Mol Ther
, vol.10
, pp. 492-499
-
-
Mian, A.1
McCormack Jr., W.M.2
Mane, V.3
Kleppe, S.4
Ng, P.5
Finegold, M.6
-
14
-
-
13144283616
-
An adenoviral vector deleted for all viral cooling sequences results in enhanced safety and extended expression of a leptin transgene
-
Morsy, MA, Gu, M, Motzel, S, Zhao, J, Lin, J, Su, Q et al. (1998). An adenoviral vector deleted for all viral cooling sequences results in enhanced safety and extended expression of a leptin transgene. Proc Natl Acad Sci USA 95: 7866-7871.
-
(1998)
Proc Natl Acad Sci USA
, vol.95
, pp. 7866-7871
-
-
Morsy, M.A.1
Gu, M.2
Motzel, S.3
Zhao, J.4
Lin, J.5
Su, Q.6
-
15
-
-
15244353548
-
Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector
-
Toietta, G, Mane, VP, Norona, WS, Finegold, M, Ng, P, McDonagh, AF et al. (2005). Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector. Proc Natl Acad Sci USA 102: 3930-3935.
-
(2005)
Proc Natl Acad Sci USA
, vol.102
, pp. 3930-3935
-
-
Toietta, G.1
Mane, V.P.2
Norona, W.S.3
Finegold, M.4
Ng, P.5
McDonagh, A.F.6
-
16
-
-
13044304200
-
Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons
-
Morral, N, O'Neal, W, Rice, K, Leland, M, Kaplan, J, Piedra, PA et al. (1999). Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons. Proc Natl Acad Sci USA 96: 12816-12821.
-
(1999)
Proc Natl Acad Sci USA
, vol.96
, pp. 12816-12821
-
-
Morral, N.1
O'Neal, W.2
Rice, K.3
Leland, M.4
Kaplan, J.5
Piedra, P.A.6
-
17
-
-
33646806788
-
Helper-dependent adenoviral gene therapy mediates long-term correction of the clotting defect in the canine hemophilia A model
-
McCormack, WM Jr., Seiler, MP, Bertin, TK, Ubhayakar, K, Palmer, DJ, Ng, P et al. (2006). Helper-dependent adenoviral gene therapy mediates long-term correction of the clotting defect in the canine hemophilia A model. J Thromb Haemost 4: 1218-1225.
-
(2006)
J Thromb Haemost
, vol.4
, pp. 1218-1225
-
-
McCormack Jr., W.M.1
Seiler, M.P.2
Bertin, T.K.3
Ubhayakar, K.4
Palmer, D.J.5
Ng, P.6
-
18
-
-
0036145244
-
Lethal toxicity, severe endothelial injury, and a threshold effect with high doses of an adenoviral vector in baboons
-
Morral, N, O'Neal, WK, Rice, K, Leland, MM, Piedra, PA, Aguilar-Cordova, E et al. (2002). Lethal toxicity, severe endothelial injury, and a threshold effect with high doses of an adenoviral vector in baboons. Hum Gene Ther 13: 143-154.
-
(2002)
Hum Gene Ther
, vol.13
, pp. 143-154
-
-
Morral, N.1
O'Neal, W.K.2
Rice, K.3
Leland, M.M.4
Piedra, P.A.5
Aguilar-Cordova, E.6
-
19
-
-
0033543994
-
Gene transfer into the liver of nonhuman primates with E1-deleted recombinant adenoviral vectors: Safety of readministration
-
Nunes, FA, Furth, EE, Wilson, JM and Raper, SE (1999). Gene transfer into the liver of nonhuman primates with E1-deleted recombinant adenoviral vectors: Safety of readministration. Hum Gene Ther 10: 2515-2526.
-
(1999)
Hum Gene Ther
, vol.10
, pp. 2515-2526
-
-
Nunes, F.A.1
Furth, E.E.2
Wilson, J.M.3
Raper, S.E.4
-
20
-
-
0742307422
-
Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates
-
Brunetti-Pierri, N, Palmer, DJ, Beaudet, AL, Carey, KD, Finegold, M and Ng, P (2004). Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates. Hum Gene Ther 15: 35-46.
-
(2004)
Hum Gene Ther
, vol.15
, pp. 35-46
-
-
Brunetti-Pierri, N.1
Palmer, D.J.2
Beaudet, A.L.3
Carey, K.D.4
Finegold, M.5
Ng, P.6
-
21
-
-
0031923328
-
Cytotoxic T-lymphocyte target proteins and their major histocompatibility complex class I restriction in response to adenovirus vectors delivered to mouse liver
-
Jooss, K, Ertl, HC and Wilson, JM (1999). Cytotoxic T-lymphocyte target proteins and their major histocompatibility complex class I restriction in response to adenovirus vectors delivered to mouse liver. J Virol 72: 2945-2954.
-
(1999)
J Virol
, vol.72
, pp. 2945-2954
-
-
Jooss, K.1
Ertl, H.C.2
Wilson, J.M.3
-
22
-
-
33747611692
-
Modulation of TNFalpha, a determinant of acute toxicity associated with systemic delivery of first-generation and helper-dependent adenoviral vectors
-
Mane, VP, Toietta, G, McCormack, WM, Conde, I, Clarke, C, Palmer, D et al. (2006). Modulation of TNFalpha, a determinant of acute toxicity associated with systemic delivery of first-generation and helper-dependent adenoviral vectors. Gene Ther 13: 1272-1280.
-
(2006)
Gene Ther
, vol.13
, pp. 1272-1280
-
-
Mane, V.P.1
Toietta, G.2
McCormack, W.M.3
Conde, I.4
Clarke, C.5
Palmer, D.6
-
23
-
-
0032506791
-
High doses of a helper-dependent adenoviral vector yield supraphysiological levels of alpha1-antitrypsin with negligible toxicity
-
Morral, N, Parks, RJ, Zhou, H, Langston, C, Schiedner, G, Quinones J, et al. (1998). High doses of a helper-dependent adenoviral vector yield supraphysiological levels of alpha1-antitrypsin with negligible toxicity. Hum Gene Ther 9: 2709-2716.
-
(1998)
Hum Gene Ther
, vol.9
, pp. 2709-2716
-
-
Morral, N.1
Parks, R.J.2
Zhou, H.3
Langston, C.4
Schiedner, G.5
Quinones, J.6
-
24
-
-
0027049847
-
Cell-specific expression of cytosolic phosphoenolpyruvate carboxykinase in transgenic mice
-
Beale, EG, Clouthier, DE and Hammer, RE (1992). Cell-specific expression of cytosolic phosphoenolpyruvate carboxykinase in transgenic mice. FASEB J 6: 3330-3337.
-
(1992)
FASEB J
, vol.6
, pp. 3330-3337
-
-
Beale, E.G.1
Clouthier, D.E.2
Hammer, R.E.3
-
25
-
-
0027319588
-
Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII
-
Pittman, DD, Alderman, EM, Tomkinson, KN, Wang, JH, Giles, AR and Kaufman, RJ, (1993). Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII. Blood 81: 2925-2935.
-
(1993)
Blood
, vol.81
, pp. 2925-2935
-
-
Pittman, D.D.1
Alderman, E.M.2
Tomkinson, K.N.3
Wang, J.H.4
Giles, A.R.5
Kaufman, R.J.6
-
26
-
-
0022760260
-
A large region (approximately equal to 95 kDa) of human factor VIII is dispensable for in vitro procoagulant activity
-
Toole, JJ, Pittman, DD, Orr, EC, Murtha, P, Wasley, LC and Kaufman, RJ (1986). A large region (approximately equal to 95 kDa) of human factor VIII is dispensable for in vitro procoagulant activity. Proc Natl Acad Sci USA 83: 5939-5942.
-
(1986)
Proc Natl Acad Sci USA
, vol.83
, pp. 5939-5942
-
-
Toole, J.J.1
Pittman, D.D.2
Orr, E.C.3
Murtha, P.4
Wasley, L.C.5
Kaufman, R.J.6
-
27
-
-
0030821993
-
Mutagenesis of a potential immunoglobulin-binding protein-binding site enhances secretion of coagulation factor VIII
-
Swaroop, M, Moussalli, M, Pipe, SW and Kaufman, RJ (1997). Mutagenesis of a potential immunoglobulin-binding protein-binding site enhances secretion of coagulation factor VIII. J Biol Chem 272: 24121-24124.
-
(1997)
J Biol Chem
, vol.272
, pp. 24121-24124
-
-
Swaroop, M.1
Moussalli, M.2
Pipe, S.W.3
Kaufman, R.J.4
-
28
-
-
0028937341
-
A 110-amino acid region within the A1-domain of coagulation factor VIII inhibits secretion from mammalian cells
-
Marquette, KA, Pittman, DD and Kaufman, RJ (1995). A 110-amino acid region within the A1-domain of coagulation factor VIII inhibits secretion from mammalian cells. J Biol Chem 270: 10297-10303.
-
(1995)
J Biol Chem
, vol.270
, pp. 10297-10303
-
-
Marquette, K.A.1
Pittman, D.D.2
Kaufman, R.J.3
-
29
-
-
0029011723
-
Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A
-
Bi, L, Lawler, AM, Antonarakis, SE, High, KA, Gearhart, JD and Kazazian, HH Jr. (1995). Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet 10: 119-121.
-
(1995)
Nat Genet
, vol.10
, pp. 119-121
-
-
Bi, L.1
Lawler, A.M.2
Antonarakis, S.E.3
High, K.A.4
Gearhart, J.D.5
Kazazian Jr., H.H.6
-
30
-
-
0031424112
-
Biosynthesis, assembly and secretion of coagulation factor VIII
-
Kaufman, RJ, Pipe, SW, Tagliavacca, L, Swaroop, M and Moussalli, M (1997). Biosynthesis, assembly and secretion of coagulation factor VIII. Blood Coagul Fibrinolysis 8: S3-S14.
-
(1997)
Blood Coagul Fibrinolysis
, vol.8
-
-
Kaufman, R.J.1
Pipe, S.W.2
Tagliavacca, L.3
Swaroop, M.4
Moussalli, M.5
-
31
-
-
0034728357
-
ATP-dependent dissociation of non-disulfide-linked aggregates of coagulation factor VIII is a rate-limiting step for secretion
-
Tagliavacca, L, Wang, Q and Kaufman, RJ (2000). ATP-dependent dissociation of non-disulfide-linked aggregates of coagulation factor VIII is a rate-limiting step for secretion. Biochemistry 39: 1973-1981.
-
(2000)
Biochemistry
, vol.39
, pp. 1973-1981
-
-
Tagliavacca, L.1
Wang, Q.2
Kaufman, R.J.3
-
32
-
-
0036154968
-
Sustained human factor VIII expression in hemophilia A mice following systemic delivery of a gutless adenoviral vector
-
Reddy, PS, Sakhuja, K, Ganesh, S, Yang, L, Kayda, D, Brann, T et al. (2002). Sustained human factor VIII expression in hemophilia A mice following systemic delivery of a gutless adenoviral vector. Mol Ther 5: 63-73.
-
(2002)
Mol Ther
, vol.5
, pp. 63-73
-
-
Reddy, P.S.1
Sakhuja, K.2
Ganesh, S.3
Yang, L.4
Kayda, D.5
Brann, T.6
-
33
-
-
0037372398
-
Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors
-
Chuah, MK, Schiedner, G, Thorrez, L, Brown, B, Johnston, M, Gillijns, V et al. (2003). Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors. Blood 101: 1734-1743.
-
(2003)
Blood
, vol.101
, pp. 1734-1743
-
-
Chuah, M.K.1
Schiedner, G.2
Thorrez, L.3
Brown, B.4
Johnston, M.5
Gillijns, V.6
-
34
-
-
0141593513
-
A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia
-
Ehrhardt, A, Xu, H, Dillow, AM, Bellinger, DA, Nichols, TC and Kay, MA (2003). A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia. Blood 102: 2403-2411.
-
(2003)
Blood
, vol.102
, pp. 2403-2411
-
-
Ehrhardt, A.1
Xu, H.2
Dillow, A.M.3
Bellinger, D.A.4
Nichols, T.C.5
Kay, M.A.6
-
35
-
-
1642459412
-
Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A
-
Brown, BD, Shi, CX, Powell, S, Hurlbut, D, Graham, FL and Lillicrap, D (2004). Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A. Blood 103: 804-810.
-
(2004)
Blood
, vol.103
, pp. 804-810
-
-
Brown, B.D.1
Shi, C.X.2
Powell, S.3
Hurlbut, D.4
Graham, F.L.5
Lillicrap, D.6
-
36
-
-
1342272118
-
Inhibitor antibody development and T cell response to human factor VIII in murine hemophilia A
-
Qian, J, Borovok, M, Bi, L, Kazazian, HH Jr. and Hoyer, LW (1999). Inhibitor antibody development and T cell response to human factor VIII in murine hemophilia A. Thromb Haemost 81: 240-244.
-
(1999)
Thromb Haemost
, vol.81
, pp. 240-244
-
-
Qian, J.1
Borovok, M.2
Bi, L.3
Kazazian Jr., H.H.4
Hoyer, L.W.5
-
37
-
-
0016853978
-
Proceedings: A more uniform measurement of factor VIII inhibitors
-
612
-
Kasper, CK, Aledort, L, Aronson, D, Counts, R, Edson, JR, van Eys, J, et al. (1975). Proceedings: A more uniform measurement of factor VIII inhibitors. Thromb Diath Haemorrh 34: 612.
-
(1975)
Thromb Diath Haemorrh
, vol.34
-
-
Kasper, C.K.1
Aledort, L.2
Aronson, D.3
Counts, R.4
Edson, J.R.5
van Eys, J.6
-
38
-
-
33846309241
-
Toll-like receptor 9 triggers an innate immune response to helper-dependent adenoviral vectors
-
Cerullo, V, Seiler, MP, Mane, V, Brunetti-Pierri, N, Clarke, C, Bertin, TK et al. (2007). Toll-like receptor 9 triggers an innate immune response to helper-dependent adenoviral vectors. Mol Ther 15 378-385.
-
(2007)
Mol Ther
, vol.15
, pp. 378-385
-
-
Cerullo, V.1
Seiler, M.P.2
Mane, V.3
Brunetti-Pierri, N.4
Clarke, C.5
Bertin, T.K.6
|