-
1
-
-
12944317288
-
Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector
-
Balague C, Zhou J, Dai Y, Alemany R, et al. (2000). Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector. Blood 95: 820-828.
-
(2000)
Blood
, vol.95
, pp. 820-828
-
-
Balague, C.1
Zhou, J.2
Dai, Y.3
Alemany, R.4
-
3
-
-
0034192227
-
Correction of the coagulation defect in hemophilia A mice through factor VIII expression in skin
-
Fakharzadeh SS, Zhang Y, Sarkar R and Kazazian HH Jr (2000). Correction of the coagulation defect in hemophilia A mice through factor VIII expression in skin. Blood 95: 2799-2805.
-
(2000)
Blood
, vol.95
, pp. 2799-2805
-
-
Fakharzadeh, S.S.1
Zhang, Y.2
Sarkar, R.3
Kazazian Jr., H.H.4
-
4
-
-
0035411087
-
High level expression of human factor VIII in mammalian cells after retroviral-mediated gene transfer
-
Guo X, Wang H, Chu H, Wang X, et al. (2001). High level expression of human factor VIII in mammalian cells after retroviral-mediated gene transfer. Chin. Med. J. 114: 690-693.
-
(2001)
Chin. Med. J
, vol.114
, pp. 690-693
-
-
Guo, X.1
Wang, H.2
Chu, H.3
Wang, X.4
-
5
-
-
37549030199
-
Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin
-
Hanna J, Wernig M, Markoulaki S, Sun CW, et al. (2007). Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin. Science 318: 1920-1923.
-
(2007)
Science
, vol.318
, pp. 1920-1923
-
-
Hanna, J.1
Wernig, M.2
Markoulaki, S.3
Sun, C.W.4
-
6
-
-
30744476548
-
Efficient AAV1-AAV2 hybrid vector for gene therapy of hemophilia
-
Hauck B, Xu RR, Xie J, Wu W, et al. (2006). Efficient AAV1-AAV2 hybrid vector for gene therapy of hemophilia. Hum. Gene Ther. 17: 46-54.
-
(2006)
Hum. Gene Ther
, vol.17
, pp. 46-54
-
-
Hauck, B.1
Xu, R.R.2
Xie, J.3
Wu, W.4
-
7
-
-
33749822056
-
Evidence for the benefits of prophylaxis in the management of hemophilia A
-
Hoots WK and Nugent DJ (2006). Evidence for the benefits of prophylaxis in the management of hemophilia A. Thromb. Haemost. 96: 433-440.
-
(2006)
Thromb. Haemost
, vol.96
, pp. 433-440
-
-
Hoots, W.K.1
Nugent, D.J.2
-
8
-
-
12144286557
-
Evidence of a pluripotent human embryonic stem cell line derived from a cloned blastocyst
-
Hwang WS, Ryu YJ, Park JH, Park ES, et al. (2004). Evidence of a pluripotent human embryonic stem cell line derived from a cloned blastocyst. Science 303: 1669-1674.
-
(2004)
Science
, vol.303
, pp. 1669-1674
-
-
Hwang, W.S.1
Ryu, Y.J.2
Park, J.H.3
Park, E.S.4
-
9
-
-
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, et al. (2007). Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens. Blood 110: 2855-2863.
-
(2007)
Blood
, vol.110
, pp. 2855-2863
-
-
Ide, L.M.1
Gangadharan, B.2
Chiang, K.Y.3
Doering, C.B.4
-
10
-
-
0033200188
-
Advances toward gene therapy for hemophilia at the millennium
-
Kaufman RJ (1999). Advances toward gene therapy for hemophilia at the millennium. Hum. Gene Ther. 10: 2091-2107.
-
(1999)
Hum. Gene Ther
, vol.10
, pp. 2091-2107
-
-
Kaufman, R.J.1
-
12
-
-
0037853196
-
Efficient production of human FVIII in hemophilic mice using lentiviral vectors
-
Kootstra NA, Matsumura R and Verma IM (2003). Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol. Ther. 7: 623-631.
-
(2003)
Mol. Ther
, vol.7
, pp. 623-631
-
-
Kootstra, N.A.1
Matsumura, R.2
Verma, I.M.3
-
13
-
-
76749158991
-
-
Zhonghua Yi. Xue. Yi. Chuan Xue. Za Zhi
-
Kuang Y, Wang J, Lu X, Lu S, et al. (2010). Generation of factor VIII gene knockout mouse by tetraploid embryo complementation technology. Zhonghua Yi. Xue. Yi. Chuan Xue. Za Zhi. 27: 1-6.
-
(2010)
Generation of Factor VIII Gene Knockout Mouse By Tetraploid Embryo Complementation Technology
, vol.27
, pp. 1-6
-
-
Kuang, Y.1
Wang, J.2
Lu, X.3
Lu, S.4
-
14
-
-
36148960428
-
Reprogramming somatic cells towards pluripotency by defined factors
-
Lewitzky M and Yamanaka S (2007). Reprogramming somatic cells towards pluripotency by defined factors. Curr. Opin. Biotechnol. 18: 467-473.
-
(2007)
Curr. Opin. Biotechnol
, vol.18
, pp. 467-473
-
-
Lewitzky, M.1
Yamanaka, S.2
-
15
-
-
35348867348
-
Ex vivo gene therapy for hemophilia A that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors
-
Matsui H, Shibata M, Brown B, Labelle A, et al. (2007). Ex vivo gene therapy for hemophilia A that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors. Stem. Cells 25: 2660-2669.
-
(2007)
Stem. Cells
, vol.25
, pp. 2660-2669
-
-
Matsui, H.1
Shibata, M.2
Brown, B.3
Labelle, A.4
-
16
-
-
0014207488
-
Intrinsic:Immunological tolerance in allophenic mice
-
Mintz B and Silvers WK (1967). Intrinsic:immunological tolerance in allophenic mice. Science 158: 1484-1486.
-
(1967)
Science
, vol.158
, pp. 1484-1486
-
-
Mintz, B.1
Silvers, W.K.2
-
17
-
-
28444459497
-
Correction of murine hemophilia A by hematopoietic stem cell gene therapy
-
Moayeri M, Hawley TS and Hawley RG (2005). Correction of murine hemophilia A by hematopoietic stem cell gene therapy. Mol. Ther. 12: 1034-1042.
-
(2005)
Mol. Ther.
, vol.12
, pp. 1034-1042
-
-
Moayeri, M.1
Hawley, T.S.2
Hawley, R.G.3
-
18
-
-
77955030405
-
Unique strategies for therapeutic gene transfer in haemophilia A and haemophilia BWFH State-of-the-Art Session on Therapeutic Gene Transfer Buenos Aires
-
Argentina,Suppl
-
Montgomery RR, Monahan PE and Ozelo MC (2010). Unique strategies for therapeutic gene transfer in haemophilia A and haemophilia BWFH State-of-the-Art Session on Therapeutic Gene Transfer Buenos Aires, Argentina. Haemophilia (Suppl 16) 5: 29-34.
-
(2010)
Haemophilia
, vol.5
, Issue.16
, pp. 29-34
-
-
Montgomery, R.R.1
Monahan, P.E.2
Ozelo, M.C.3
-
19
-
-
34249880066
-
Generation of germline-competent induced pluripotent stem cells
-
Okita K, Ichisaka T and Yamanaka S (2007). Generation of germline-competent induced pluripotent stem cells. Nature 448: 313-317.
-
(2007)
Nature
, vol.448
, pp. 313-317
-
-
Okita, K.1
Ichisaka, T.2
Yamanaka, S.3
-
20
-
-
38049187707
-
Reprogramming of human somatic cells to pluripotency with defined factors
-
Park IH, Zhao R, West JA, Yabuuchi A, et al. (2008). Reprogramming of human somatic cells to pluripotency with defined factors. Nature 451: 141-146.
-
(2008)
Nature
, vol.451
, pp. 141-146
-
-
Park, I.H.1
Zhao, R.2
West, J.A.3
Yabuuchi, A.4
-
21
-
-
34247862426
-
Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment
-
Pierce GF, Lillicrap D, Pipe SW and Vandendriessche T (2007). Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment. J. Thromb. Haemost. 5: 901-906.
-
(2007)
J. Thromb. Haemost
, vol.5
, pp. 901-906
-
-
Pierce, G.F.1
Lillicrap, D.2
Pipe, S.W.3
Vandendriessche, T.4
-
22
-
-
0020644686
-
X-chromosome instability in pluripotential stem cell lines derived from parthenogenetic embryos
-
Robertson EJ, Evans MJ and Kaufman MH (1983). X-chromosome instability in pluripotential stem cell lines derived from parthenogenetic embryos. J. Embryol. Exp. Morphol. 74: 297-309.
-
(1983)
J. Embryol. Exp. Morphol
, vol.74
, pp. 297-309
-
-
Robertson, E.J.1
Evans, M.J.2
Kaufman, M.H.3
-
23
-
-
0038170070
-
Development of improved factor VIII molecules and new gene transfer approaches for hemophilia A
-
Saenko EL, Ananyeva NM, Moayeri M, Ramezani A, et al. (2003). Development of improved factor VIII molecules and new gene transfer approaches for hemophilia A. Curr. Gene Ther. 3: 27-41.
-
(2003)
Curr. Gene Ther
, vol.3
, pp. 27-41
-
-
Saenko, E.L.1
Ananyeva, N.M.2
Moayeri, M.3
Ramezani, A.4
-
24
-
-
0033936079
-
Properties of anti-factor VIII inhibitor antibodies in hemophilia A patients
-
Scandella DH (2000). Properties of anti-factor VIII inhibitor antibodies in hemophilia A patients. Semin. Thromb. Hemost. 26: 137-142.
-
(2000)
Semin. Thromb. Hemost
, vol.26
, pp. 137-142
-
-
Scandella, D.H.1
-
25
-
-
36248966518
-
Induction of pluripotent stem cells from adult human fibroblasts by defined factors
-
Takahashi K, Tanabe K, Ohnuki M, Narita M, et al. (2007). Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131: 861-872.
-
(2007)
Cell
, vol.131
, pp. 861-872
-
-
Takahashi, K.1
Tanabe, K.2
Ohnuki, M.3
Narita, M.4
-
26
-
-
0141962306
-
Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction
-
Tiede A, Eder M, von Depka M, Battmer K, et al. (2003). Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction. Gene Ther. 10: 1917-1925.
-
(2003)
Gene Ther
, vol.10
, pp. 1917-1925
-
-
Tiede, A.1
Eder, M.2
von Depka, M.3
Battmer, K.4
-
27
-
-
58849113643
-
Phenotypic correction of murine hemophilia A using an iPS cellbased therapy
-
Xu D, Alipio Z, Fink LM, Adcock DM, et al. (2009). Phenotypic correction of murine hemophilia A using an iPS cellbased therapy. Proc. Natl. Acad. Sci. U. S. A. 106: 808-813.
-
(2009)
Proc. Natl. Acad. Sci. U. S. A
, vol.106
, pp. 808-813
-
-
Xu, D.1
Alipio, Z.2
Fink, L.M.3
Adcock, D.M.4
-
28
-
-
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, et al. (2005). 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. 102: 6080-6085.
-
(2005)
Proc. Natl. Acad. Sci. U. S. A
, vol.102
, pp. 6080-6085
-
-
Xu, L.1
Nichols, T.C.2
Sarkar, R.3
McCorquodale, S.4
-
29
-
-
34548073293
-
High expression reduces an antibody response after neonatal gene therapy with B domain-deleted human factor VIII in mice
-
Xu L, Mei M, Ma X and Ponder KP (2007). High expression reduces an antibody response after neonatal gene therapy with B domain-deleted human factor VIII in mice. J. Thromb. Haemost. 5: 1805-1812.
-
(2007)
J. Thromb. Haemost
, vol.5
, pp. 1805-1812
-
-
Xu, L.1
Mei, M.2
Ma, X.3
Ponder, K.P.4
-
30
-
-
79952115967
-
The use of PEGylated liposomes in the development of drug delivery applications for the treatment of hemophilia
-
Yatuv R, Robinson M, Dayan-Tarshish I and Baru M (2010). The use of PEGylated liposomes in the development of drug delivery applications for the treatment of hemophilia. Int. J. Nanomedicine 5: 581-591.
-
(2010)
Int. J. Nanomedicine
, vol.5
, pp. 581-591
-
-
Yatuv, R.1
Robinson, M.2
Dayan-Tarshish, I.3
Baru, M.4
-
31
-
-
3042815553
-
Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice
-
Ye P, Thompson AR, Sarkar R, Shen Z, et al. (2004). Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice. Mol. Ther. 10: 117-126.
-
(2004)
Mol. Ther
, vol.10
, pp. 117-126
-
-
Ye, P.1
Thompson, A.R.2
Sarkar, R.3
Shen, Z.4
-
32
-
-
36749043230
-
Induced pluripotent stem cell lines derived from human somatic cells
-
Yu J, Vodyanik MA, Smuga-Otto K, Antosiewicz-Bourget J, et al. (2007). Induced pluripotent stem cell lines derived from human somatic cells. Science 318: 1917-1920.
-
(2007)
Science
, vol.318
, pp. 1917-1920
-
-
Yu, J.1
Vodyanik, M.A.2
Smuga-Otto, K.3
Antosiewicz-Bourget, J.4
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