-
1
-
-
0017151562
-
Ultrastructure of human bone marrow cell maturation
-
Breton-Gorius J, Reyes F. Ultrastructure of human bone marrow cell maturation. Int Rev Cytol. 1976;46:251-321.
-
(1976)
Int Rev Cytol
, vol.46
, pp. 251-321
-
-
Breton-Gorius, J.1
Reyes, F.2
-
2
-
-
0016910150
-
Biology of megakaryocytes
-
Ebbe S. Biology of megakaryocytes. Prog Hemost Thromb. 1976;3:211-229.
-
(1976)
Prog Hemost Thromb
, vol.3
, pp. 211-229
-
-
Ebbe, S.1
-
3
-
-
0033552631
-
Blood platelets are assembled principally at the ends of proplatelet processes produced by differentiated megakaryocytes
-
Italiano JE Jr, Lecine P, Shivdasani RA, Hartwig JH. Blood platelets are assembled principally at the ends of proplatelet processes produced by differentiated megakaryocytes. J Cell Biol. 1999; 147(6):1299-1312.
-
(1999)
J Cell Biol
, vol.147
, Issue.6
, pp. 1299-1312
-
-
Italiano, J.E.1
Lecine, P.2
Shivdasani, R.A.3
Hartwig, J.H.4
-
4
-
-
31044443970
-
The biogenesis of platelets from megakaryocyte proplatelets
-
Patel SR, Hartwig JH, Italiano JE Jr. The biogenesis of platelets from megakaryocyte proplatelets. J Clin Invest. 2005;115(12): 3348-3354.
-
(2005)
J Clin Invest
, vol.115
, Issue.12
, pp. 3348-3354
-
-
Patel, S.R.1
Hartwig, J.H.2
Italiano, J.E.3
-
5
-
-
2142699977
-
Megakaryocytes and beyond: The birth of platelets
-
Italiano JE Jr, Shivdasani RA. Megakaryocytes and beyond: the birth of platelets. J Thromb Haemost. 2003;1(6):1174-1182.
-
(2003)
J Thromb Haemost
, vol.1
, Issue.6
, pp. 1174-1182
-
-
Italiano, J.E.1
Shivdasani, R.A.2
-
6
-
-
84905252442
-
Platelet membrane glycoproteins: A historical review
-
Nurden AT. Platelet membrane glycoproteins: a historical review. Semin Thromb Hemost. 2014; 40(5):577-584.
-
(2014)
Semin Thromb Hemost
, vol.40
, Issue.5
, pp. 577-584
-
-
Nurden, A.T.1
-
7
-
-
0027937953
-
Characterization of regulatory elements in the 59-flanking region of the rat GPIIb gene by studies in a primary rat marrow culture system
-
Block KL, Ravid K, Phung QH, Poncz M. Characterization of regulatory elements in the 59-flanking region of the rat GPIIb gene by studies in a primary rat marrow culture system. Blood. 1994;84(10):3385-3393.
-
(1994)
Blood
, vol.84
, Issue.10
, pp. 3385-3393
-
-
Block, K.L.1
Ravid, K.2
Phung, Q.H.3
Poncz, M.4
-
8
-
-
0026355641
-
Longterm improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits
-
Chowdhury JR, Grossman M, Gupta S, Chowdhury NR, Baker JR Jr, Wilson JM. Longterm improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits. Science. 1991;254(5039):1802-1805.
-
(1991)
Science
, vol.254
, Issue.5039
, pp. 1802-1805
-
-
Chowdhury, J.R.1
Grossman, M.2
Gupta, S.3
Chowdhury, N.R.4
Baker, J.R.5
Wilson, J.M.6
-
9
-
-
0025874746
-
Expression of human factor IX in rat capillary endothelial cells: Toward somatic gene therapy for hemophilia B
-
Yao SN, Wilson JM, Nabel EG, Kurachi S, Hachiya HL, Kurachi K. Expression of human factor IX in rat capillary endothelial cells: toward somatic gene therapy for hemophilia B. Proc Natl Acad Sci USA. 1991;88(18):8101-8105.
-
(1991)
Proc Natl Acad Sci USA
, vol.88
, Issue.18
, pp. 8101-8105
-
-
Yao, S.N.1
Wilson, J.M.2
Nabel, E.G.3
Kurachi, S.4
Hachiya, H.L.5
Kurachi, K.6
-
10
-
-
0022271481
-
Attempts at gene therapy in beta-thalassemic mice
-
Anderson WF, Goldberg S, Kantoff P, Berg P, Eglitis M, Humphries RK. Attempts at gene therapy in beta-thalassemic mice. Ann N Y Acad Sci. 1985;445:445-451.
-
(1985)
Ann N Y Acad Sci
, vol.445
, pp. 445-451
-
-
Anderson, W.F.1
Goldberg, S.2
Kantoff, P.3
Berg, P.4
Eglitis, M.5
Humphries, R.K.6
-
11
-
-
0022418843
-
Immune response restored by gene therapy in mice
-
Steinmetz M. Immune response restored by gene therapy in mice. Nature. 1985;316(6023):14-15.
-
(1985)
Nature
, vol.316
, Issue.6023
, pp. 14-15
-
-
Steinmetz, M.1
-
12
-
-
0028807727
-
T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years
-
Blaese RM, Culver KW, Miller AD, et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science. 1995;270(5235):475-480.
-
(1995)
Science
, vol.270
, Issue.5235
, pp. 475-480
-
-
Blaese, R.M.1
Culver, K.W.2
Miller, A.D.3
-
13
-
-
0033578285
-
Integrin alphaIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus-transduced human hematopoietic cells
-
Wilcox DA, Olsen JC, Ishizawa L, Griffith M, White GC II. Integrin alphaIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus-transduced human hematopoietic cells. Proc Natl Acad Sci USA. 1999;96(17):9654-9659.
-
(1999)
Proc Natl Acad Sci USA
, vol.96
, Issue.17
, pp. 9654-9659
-
-
Wilcox, D.A.1
Olsen, J.C.2
Ishizawa, L.3
Griffith, M.4
White GC, I.I.5
-
14
-
-
0033611139
-
Primary megakaryocytes reveal a role for transcription factor NF-E2 in integrin a IIb b 3 signaling
-
Shiraga M, Ritchie A, Aidoudi S, et al. Primary megakaryocytes reveal a role for transcription factor NF-E2 in integrin a IIb b 3 signaling. J Cell Biol. 1999;147(7):1419-1430.
-
(1999)
J Cell Biol
, vol.147
, Issue.7
, pp. 1419-1430
-
-
Shiraga, M.1
Ritchie, A.2
Aidoudi, S.3
-
15
-
-
13044312102
-
Ex vivo cultured megakaryocytes express functional glycoprotein IIb-IIIa receptors and are capable of adenovirus-mediated transgene expression
-
Faraday N, Rade JJ, Johns DC, et al. Ex vivo cultured megakaryocytes express functional glycoprotein IIb-IIIa receptors and are capable of adenovirus-mediated transgene expression. Blood. 1999;94(12):4084-4092.
-
(1999)
Blood
, vol.94
, Issue.12
, pp. 4084-4092
-
-
Faraday, N.1
Rade, J.J.2
Johns, D.C.3
-
16
-
-
0028928884
-
Platelets generated in vitro from proplatelet-displaying human megakaryocytes are functional
-
Choi ES, Nichol JL, Hokom MM, Hornkohl AC, Hunt P. Platelets generated in vitro from proplatelet-displaying human megakaryocytes are functional. Blood. 1995;85(2):402-413.
-
(1995)
Blood
, vol.85
, Issue.2
, pp. 402-413
-
-
Choi, E.S.1
Nichol, J.L.2
Hokom, M.M.3
Hornkohl, A.C.4
Hunt, P.5
-
17
-
-
0028302409
-
Stimulation of megakaryocytopoiesis and thrombopoiesis by the c-Mpl ligand
-
de Sauvage FJ, Hass PE, Spencer SD, et al. Stimulation of megakaryocytopoiesis and thrombopoiesis by the c-Mpl ligand. Nature. 1994; 369(6481):533-538.
-
(1994)
Nature
, vol.369
, Issue.6481
, pp. 533-538
-
-
De Sauvage, F.J.1
Hass, P.E.2
Spencer, S.D.3
-
18
-
-
84879867061
-
Lentiviral hematopoietic stem cell gene therapy in patients\ with Wiskott-Aldrich syndrome
-
Aiuti A, Biasco L, Scaramuzza S, et al. Lentiviral hematopoietic stem cell gene therapy in patients\ with Wiskott-Aldrich syndrome. Science. 2013; 341(6148):1233151.
-
(2013)
Science
, vol.341
, Issue.6148
, pp. 1233151
-
-
Aiuti, A.1
Biasco, L.2
Scaramuzza, S.3
-
19
-
-
84879873039
-
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
-
Biffi A, Montini E, Lorioli L, et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science. 2013; 341(6148):1233158.
-
(2013)
Science
, vol.341
, Issue.6148
, pp. 1233158
-
-
Biffi, A.1
Montini, E.2
Lorioli, L.3
-
20
-
-
77951866417
-
Cell biology Beyond clotting: The powers of platelets
-
Leslie M. Cell biology. Beyond clotting: the powers of platelets. Science. 2010;328(5978):562-564.
-
(2010)
Science
, vol.328
, Issue.5978
, pp. 562-564
-
-
Leslie, M.1
-
21
-
-
84880010050
-
The incredible journey: From megakaryocyte development to platelet formation
-
Machlus KR, Italiano JE Jr. The incredible journey: from megakaryocyte development to platelet formation. J Cell Biol. 2013;201(6): 785-796.
-
(2013)
J Cell Biol
, vol.201
, Issue.6
, pp. 785-796
-
-
Machlus, K.R.1
Italiano, J.E.2
-
22
-
-
0030307624
-
The platelet glycoprotein Ib-V-IX system: Regulation of gene expression
-
Roth GJ, Yagi M, Bastian LS. The platelet glycoprotein Ib-V-IX system: regulation of gene expression. Stem Cells. 1996;14(suppl 1): 188-193.
-
(1996)
Stem Cells
, vol.14
, pp. 188-193
-
-
Roth, G.J.1
Yagi, M.2
Bastian, L.S.3
-
23
-
-
0025740866
-
Tissue-specific expression of the platelet GPIIb gene
-
Uzan G, Prenant M, Prandini MH, Martin F, Marguerie G. Tissue-specific expression of the platelet GPIIb gene. J Biol Chem. 1991;266(14): 8932-8939.
-
(1991)
J Biol Chem
, vol.266
, Issue.14
, pp. 8932-8939
-
-
Uzan, G.1
Prenant, M.2
Prandini, M.H.3
Martin, F.4
Marguerie, G.5
-
24
-
-
0037073715
-
Cloning and analysis of the thrombopoietininduced megakaryocyte-specific glycoprotein VI promoter and its regulation by GATA-1, Fli-1, and Sp1
-
Holmes ML, Bartle N, Eisbacher M, Chong BH. Cloning and analysis of the thrombopoietininduced megakaryocyte-specific glycoprotein VI promoter and its regulation by GATA-1, Fli-1, and Sp1. J Biol Chem. 2002;277(50):48333-48341.
-
(2002)
J Biol Chem
, vol.277
, Issue.50
, pp. 48333-48341
-
-
Holmes, M.L.1
Bartle, N.2
Eisbacher, M.3
Chong, B.H.4
-
25
-
-
0037071389
-
The molecular and cellular biology of thrombopoietin: The primary regulator of platelet production
-
Kaushansky K, Drachman JG. The molecular and cellular biology of thrombopoietin: the primary regulator of platelet production. Oncogene. 2002; 21(21):3359-3367.
-
(2002)
Oncogene
, vol.21
, Issue.21
, pp. 3359-3367
-
-
Kaushansky, K.1
Drachman, J.G.2
-
26
-
-
0025975187
-
Selective targeting of gene products with the megakaryocyte platelet factor 4 promoter
-
Ravid K, Beeler DL, Rabin MS, Ruley HE, Rosenberg RD. Selective targeting of gene products with the megakaryocyte platelet factor 4 promoter. Proc Natl Acad Sci USA. 1991; 88(4):1521-1525.
-
(1991)
Proc Natl Acad Sci USA
, vol.88
, Issue.4
, pp. 1521-1525
-
-
Ravid, K.1
Beeler, D.L.2
Rabin, M.S.3
Ruley, H.E.4
Rosenberg, R.D.5
-
27
-
-
0025271480
-
Megakaryocytic and erythrocytic lineages share specific transcription factors
-
Romeo PH, Prandini MH, Joulin V, et al. Megakaryocytic and erythrocytic lineages share specific transcription factors. Nature. 1990; 344(6265):447-449.
-
(1990)
Nature
, vol.344
, Issue.6265
, pp. 447-449
-
-
Romeo, P.H.1
Prandini, M.H.2
Joulin, V.3
-
28
-
-
0036792665
-
Control of megakaryocytespecific gene expression by GATA-1 and FOG-1: Role of Ets transcription factors
-
Wang X, Crispino JD, Letting DL, Nakazawa M, Poncz M, Blobel GA. Control of megakaryocytespecific gene expression by GATA-1 and FOG-1: role of Ets transcription factors. EMBO J. 2002; 21(19):5225-5234.
-
(2002)
EMBO J
, vol.21
, Issue.19
, pp. 5225-5234
-
-
Wang, X.1
Crispino, J.D.2
Letting, D.L.3
Nakazawa, M.4
Poncz, M.5
Blobel, G.A.6
-
29
-
-
0023098325
-
Structure of the rat platelet factor 4 gene: A marker for megakaryocyte differentiation
-
Doi T, Greenberg SM, Rosenberg RD. Structure of the rat platelet factor 4 gene: a marker for megakaryocyte differentiation. Mol Cell Biol. 1987;7(2):898-904.
-
(1987)
Mol Cell Biol
, vol.7
, Issue.2
, pp. 898-904
-
-
Doi, T.1
Greenberg, S.M.2
Rosenberg, R.D.3
-
30
-
-
0035437186
-
Localization of distal regulatory domains in the megakaryocyte-specific platelet basic protein platelet factor 4 gene locus
-
Zhang C, Thornton MA, Kowalska MA, et al. Localization of distal regulatory domains in the megakaryocyte-specific platelet basic protein platelet factor 4 gene locus. Blood. 2001;98(3): 610-617.
-
(2001)
Blood
, vol.98
, Issue.3
, pp. 610-617
-
-
Zhang, C.1
Thornton, M.A.2
Kowalska, M.A.3
-
31
-
-
84893419411
-
Platelettargeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A
-
Du LM, Nurden P, Nurden AT, et al. Platelettargeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A. Nat Commun. 2013;4:2773.
-
(2013)
Nat Commun
, vol.4
, pp. 2773
-
-
Du, L.M.1
Nurden, P.2
Nurden, A.T.3
-
32
-
-
84897024164
-
Highlevel transgene expression in induced pluripotent stem cell-derived megakaryocytes: Correction of Glanzmann thrombasthenia
-
Sullivan SK, Mills JA, Koukouritaki SB, et al. Highlevel transgene expression in induced pluripotent stem cell-derived megakaryocytes: correction of Glanzmann thrombasthenia. Blood. 2014;123(5): 753-757.
-
(2014)
Blood
, vol.123
, Issue.5
, pp. 753-757
-
-
Sullivan, S.K.1
Mills, J.A.2
Koukouritaki, S.B.3
-
33
-
-
23944447124
-
Conditional overexpression of transgenes in megakaryocytes and platelets in vivo
-
Nguyen HG, Yu G, Makitalo M, et al. Conditional overexpression of transgenes in megakaryocytes and platelets in vivo. Blood. 2005;106(5): 1559-1564.
-
(2005)
Blood
, vol.106
, Issue.5
, pp. 1559-1564
-
-
Nguyen, H.G.1
Yu, G.2
Makitalo, M.3
-
34
-
-
4444281228
-
Gene therapy for platelet disorders: Studies with Glanzmann's thrombasthenia
-
Wilcox DA, White GC II. Gene therapy for platelet disorders: studies with Glanzmann's thrombasthenia. J Thromb Haemost. 2003;1(11): 2300-2311.
-
(2003)
J Thromb Haemost
, vol.1
, Issue.11
, pp. 2300-2311
-
-
Wilcox, D.A.1
White GC, I.I.2
-
35
-
-
84898058464
-
Congenital platelet disorders and understanding of platelet function
-
Nurden AT, Nurden P. Congenital platelet disorders and understanding of platelet function. Br J Haematol. 2014;165(2):165-178.
-
(2014)
Br J Haematol
, vol.165
, Issue.2
, pp. 165-178
-
-
Nurden, A.T.1
Nurden, P.2
-
36
-
-
84931430100
-
Inherited disorders of platelet function: Selected updates
-
Nurden AT, Nurden P. Inherited disorders of platelet function: selected updates. J Thromb Haemost. 2015;13(suppl 1):S2-S9.
-
(2015)
J Thromb Haemost
, vol.13
, pp. S2-S9
-
-
Nurden, A.T.1
Nurden, P.2
-
37
-
-
84911451470
-
NovoSeven (recombinant factor VIIa) for the treatment of bleeding episodes and perioperative management in patients with Glanzmann's thrombasthenia
-
Franchini M, Lippi G. NovoSeven (recombinant factor VIIa) for the treatment of bleeding episodes and perioperative management in patients with Glanzmann's thrombasthenia. Expert Rev Hematol. 2014;7(6):733-740.
-
(2014)
Expert Rev Hematol
, vol.7
, Issue.6
, pp. 733-740
-
-
Franchini, M.1
Lippi, G.2
-
38
-
-
31444437760
-
Congenital and acquired platelet disorders: Current dilemmas and treatment strategies
-
White GC II. Congenital and acquired platelet disorders: current dilemmas and treatment strategies. Semin Hematol. 2006;43(1 suppl 1): S37-S41.
-
(2006)
Semin Hematol
, vol.43
, Issue.1
, pp. S37-S41
-
-
White GC, I.I.1
-
39
-
-
27644524098
-
Sustained engraftment post bone marrow transplant despite anti-platelet antibodies in Glanzmann thrombasthenia
-
Flood VH, Johnson FL, Boshkov LK, et al. Sustained engraftment post bone marrow transplant despite anti-platelet antibodies in Glanzmann thrombasthenia. Pediatr Blood Cancer. 2005;45(7):971-975.
-
(2005)
Pediatr Blood Cancer
, vol.45
, Issue.7
, pp. 971-975
-
-
Flood, V.H.1
Johnson, F.L.2
Boshkov, L.K.3
-
40
-
-
0345097306
-
Correction of a large animal model of type I Glanzmann's thrombasthenia by nonmyeloablative bone marrow transplantation
-
Niemeyer GP, Boudreaux MK, Goodman-Martin SA, Monroe CM, Wilcox DA, Lothrop CD Jr. Correction of a large animal model of type I Glanzmann's thrombasthenia by nonmyeloablative bone marrow transplantation. Exp Hematol. 2003;31(12):1357-1362.
-
(2003)
Exp Hematol
, vol.31
, Issue.12
, pp. 1357-1362
-
-
Niemeyer, G.P.1
Boudreaux, M.K.2
Goodman-Martin, S.A.3
Monroe, C.M.4
Wilcox, D.A.5
Lothrop, C.D.6
-
41
-
-
84888426066
-
Indication for allogeneic stem cell transplantation in Glanzmann's thrombasthenia
-
Wiegering V, Sauer K, Winkler B, Eyrich M, Schlegel PG. Indication for allogeneic stem cell transplantation in Glanzmann's thrombasthenia. Hamostaseologie. 2013;33(4):305-312.
-
(2013)
Hamostaseologie
, vol.33
, Issue.4
, pp. 305-312
-
-
Wiegering, V.1
Sauer, K.2
Winkler, B.3
Eyrich, M.4
Schlegel, P.G.5
-
42
-
-
79956114852
-
Allogeneic hematopoietic stem cell transplantation in Glanzmann thrombasthenia complicated by platelet alloimmunization
-
Wiegering V, Winkler B, Langhammer F, et al. Allogeneic hematopoietic stem cell transplantation in Glanzmann thrombasthenia complicated by platelet alloimmunization. Klin Padiatr. 2011;223(3):173-175.
-
(2011)
Klin Padiatr
, vol.223
, Issue.3
, pp. 173-175
-
-
Wiegering, V.1
Winkler, B.2
Langhammer, F.3
-
43
-
-
0034660309
-
Megakaryocyte-targeted synthesis of the integrin b(3)-subunit results in the phenotypic correction of Glanzmann thrombasthenia
-
Wilcox DA, Olsen JC, Ishizawa L, et al. Megakaryocyte-targeted synthesis of the integrin b(3)-subunit results in the phenotypic correction of Glanzmann thrombasthenia. Blood. 2000;95(12): 3645-3651.
-
(2000)
Blood
, vol.95
, Issue.12
, pp. 3645-3651
-
-
Wilcox, D.A.1
Olsen, J.C.2
Ishizawa, L.3
-
44
-
-
27144537145
-
Therapeutic expression of the platelet-specific integrin, alphaIIbbeta3, in a murine model for Glanzmann thrombasthenia
-
Fang J, Hodivala-Dilke K, Johnson BD, et al. Therapeutic expression of the platelet-specific integrin, alphaIIbbeta3, in a murine model for Glanzmann thrombasthenia. Blood. 2005;106(8): 2671-2679.
-
(2005)
Blood
, vol.106
, Issue.8
, pp. 2671-2679
-
-
Fang, J.1
Hodivala-Dilke, K.2
Johnson, B.D.3
-
45
-
-
79959329020
-
Platelet gene therapy improves hemostatic function for integrin alphaIIbbeta3-deficient dogs
-
Fang J, Jensen ES, Boudreaux MK, et al. Platelet gene therapy improves hemostatic function for integrin alphaIIbbeta3-deficient dogs. Proc Natl Acad Sci USA. 2011;108(23):9583-9588.
-
(2011)
Proc Natl Acad Sci USA
, vol.108
, Issue.23
, pp. 9583-9588
-
-
Fang, J.1
Jensen, E.S.2
Boudreaux, M.K.3
-
46
-
-
13244262627
-
Targeting platelet GPIbalpha transgene expression to human megakaryocytes and forming a complete complex with endogenous GPIbbeta and GPIX
-
Shi Q, Wilcox DA, Morateck PA, Fahs SA, Kenny D, Montgomery RR. Targeting platelet GPIbalpha transgene expression to human megakaryocytes and forming a complete complex with endogenous GPIbbeta and GPIX. J Thromb Haemost. 2004;2(11):1989-1997.
-
(2004)
J Thromb Haemost
, vol.2
, Issue.11
, pp. 1989-1997
-
-
Shi, Q.1
Wilcox, D.A.2
Morateck, P.A.3
Fahs, S.A.4
Kenny, D.5
Montgomery, R.R.6
-
47
-
-
84857781525
-
Correction of murine Bernard-Soulier syndrome by lentivirusmediated gene therapy
-
Kanaji S, Kuether EL, Fahs SA, et al. Correction of murine Bernard-Soulier syndrome by lentivirusmediated gene therapy. Mol Ther. 2012;20(3): 625-632.
-
(2012)
Mol Ther
, vol.20
, Issue.3
, pp. 625-632
-
-
Kanaji, S.1
Kuether, E.L.2
Fahs, S.A.3
-
48
-
-
0041743223
-
Antithrombotic thrombocytes: Ectopic expression of urokinasetype plasminogen activator in platelets
-
Kufrin D, Eslin DE, Bdeir K, et al. Antithrombotic thrombocytes: ectopic expression of urokinasetype plasminogen activator in platelets. Blood. 2003;102(3):926-933.
-
(2003)
Blood
, vol.102
, Issue.3
, pp. 926-933
-
-
Kufrin, D.1
Eslin, D.E.2
Bdeir, K.3
-
49
-
-
73949126440
-
A study of variations in the reported haemophilia A prevalence around the world
-
Stonebraker JS, Bolton-Maggs PH, Soucie JM, Walker I, Brooker M. A study of variations in the reported haemophilia A prevalence around the world. Haemophilia. 2010;16(1):20-32.
-
(2010)
Haemophilia
, vol.16
, Issue.1
, pp. 20-32
-
-
Stonebraker, J.S.1
Bolton-Maggs, P.H.2
Soucie, J.M.3
Walker, I.4
Brooker, M.5
-
50
-
-
84859596442
-
Modern haemophilia care
-
Berntorp E, Shapiro AD. Modern haemophilia care. Lancet. 2012;379(9824):1447-1456.
-
(2012)
Lancet
, vol.379
, Issue.9824
, pp. 1447-1456
-
-
Berntorp, E.1
Shapiro, A.D.2
-
52
-
-
0035822005
-
Factor VIII transkaryotic therapy study group. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A
-
Roth DA, Tawa NE Jr, O'Brien JM, Treco DA, Selden RF; Factor VIII Transkaryotic Therapy Study Group. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med. 2001; 344(23):1735-1742.
-
(2001)
N Engl J Med
, vol.344
, Issue.23
, pp. 1735-1742
-
-
Roth, D.A.1
Tawa, N.E.2
O'Brien, J.M.3
Treco, D.A.4
Selden, R.F.5
-
53
-
-
0141679053
-
Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion
-
Powell JS, Ragni MV, White GC II, et al. Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood. 2003; 102(6):2038-2045.
-
(2003)
Blood
, vol.102
, Issue.6
, pp. 2038-2045
-
-
Powell, J.S.1
Ragni, M.V.2
White GC, I.I.3
-
54
-
-
84855161388
-
Adenovirus-associated virus vectormediated gene transfer in hemophilia B
-
Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vectormediated gene transfer in hemophilia B. N Engl J Med. 2011;365(25):2357-2365.
-
(2011)
N Engl J Med
, vol.365
, Issue.25
, pp. 2357-2365
-
-
Nathwani, A.C.1
Tuddenham, E.G.2
Rangarajan, S.3
-
55
-
-
0027319588
-
Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII
-
Pittman DD, Alderman EM, Tomkinson KN, Wang JH, Giles AR, Kaufman RJ. Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII. Blood. 1993;81(11):2925-2935.
-
(1993)
Blood
, vol.81
, Issue.11
, 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
-
56
-
-
3042815553
-
Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice
-
Ye P, Thompson AR, Sarkar R, et al. Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice. Mol Ther. 2004;10(1): 117-126.
-
(2004)
Mol Ther
, vol.10
, Issue.1
, pp. 117-126
-
-
Ye, P.1
Thompson, A.R.2
Sarkar, R.3
-
57
-
-
79952194475
-
Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors
-
Sabatino DE, Lange AM, Altynova ES, et al. Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors. Mol Ther. 2011;19(3): 442-449.
-
(2011)
Mol Ther
, vol.19
, Issue.3
, pp. 442-449
-
-
Sabatino, D.E.1
Lange, A.M.2
Altynova, E.S.3
-
58
-
-
84870302675
-
A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model
-
Kitazawa T, Igawa T, Sampei Z, et al. A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model. Nat Med. 2012;18(10):1570-1574.
-
(2012)
Nat Med
, vol.18
, Issue.10
, pp. 1570-1574
-
-
Kitazawa, T.1
Igawa, T.2
Sampei, Z.3
-
59
-
-
84877704167
-
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
-
McIntosh J, Lenting PJ, Rosales C, et al. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood. 2013;121(17):3335-3344.
-
(2013)
Blood
, vol.121
, Issue.17
, pp. 3335-3344
-
-
McIntosh, J.1
Lenting, P.J.2
Rosales, C.3
-
60
-
-
84864364642
-
Recent progress in gene therapy for hemophilia
-
Chuah MK, Nair N, VandenDriessche T. Recent progress in gene therapy for hemophilia. Hum Gene Ther. 2012;23(6):557-565.
-
(2012)
Hum Gene Ther
, vol.23
, Issue.6
, pp. 557-565
-
-
Chuah, M.K.1
Nair, N.2
VandenDriessche, T.3
-
61
-
-
84880427173
-
Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype
-
Siner JI, Iacobelli NP, Sabatino DE, et al. Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype. Blood. 2013;121(21):4396-4403.
-
(2013)
Blood
, vol.121
, Issue.21
, pp. 4396-4403
-
-
Siner, J.I.1
Iacobelli, N.P.2
Sabatino, D.E.3
-
63
-
-
84882263725
-
Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A
-
Johnston JM, Denning G, Doering CB, Spencer HT. Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A. Gene Ther. 2013;20(6):607-615.
-
(2013)
Gene Ther
, vol.20
, Issue.6
, pp. 607-615
-
-
Johnston, J.M.1
Denning, G.2
Doering, C.B.3
Spencer, H.T.4
-
64
-
-
1542544992
-
Induction of megakaryocytes to synthesize and store a releasable pool of human factor VIII
-
Wilcox DA, Shi Q, Nurden P, et al. Induction of megakaryocytes to synthesize and store a releasable pool of human factor VIII. J Thromb Haemost. 2003;1(12):2477-2489.
-
(2003)
J Thromb Haemost
, vol.1
, Issue.12
, pp. 2477-2489
-
-
Wilcox, D.A.1
Shi, Q.2
Nurden, P.3
-
65
-
-
33745864802
-
Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies
-
Shi Q, Wilcox DA, Fahs SA, et al. Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies. J Clin Invest. 2006;116(7):1974-1982.
-
(2006)
J Clin Invest
, vol.116
, Issue.7
, pp. 1974-1982
-
-
Shi, Q.1
Wilcox, D.A.2
Fahs, S.A.3
-
66
-
-
33846453333
-
Lentivirusmediated platelet-derived factor VIII gene therapy in murine haemophilia A
-
Shi Q, Wilcox DA, Fahs SA, et al. Lentivirusmediated platelet-derived factor VIII gene therapy in murine haemophilia A. J Thromb Haemost. 2007;5(2):352-361.
-
(2007)
J Thromb Haemost
, vol.5
, Issue.2
, pp. 352-361
-
-
Shi, Q.1
Wilcox, D.A.2
Fahs, S.A.3
-
67
-
-
10744231807
-
Factor VIII ectopically expressed in platelets: Efficacy in hemophilia A treatment
-
Yarovoi HV, Kufrin D, Eslin DE, et al. Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatment. Blood. 2003;102(12): 4006-4013.
-
(2003)
Blood
, vol.102
, Issue.12
, pp. 4006-4013
-
-
Yarovoi, H.V.1
Kufrin, D.2
Eslin, D.E.3
-
68
-
-
84925547246
-
Gene therapy: Genie in a vector
-
Gould J. Gene therapy: genie in a vector. Nature. 2014;515(7528):S160-S161.
-
(2014)
Nature
, vol.515
, Issue.7528
, pp. S160-S161
-
-
Gould, J.1
-
69
-
-
0036075304
-
The von Willebrand factor propeptide (VWFpp) traffics an unrelated protein to storage
-
Haberichter SL, Jozwiak MA, Rosenberg JB, Christopherson PA, Montgomery RR. The von Willebrand factor propeptide (VWFpp) traffics an unrelated protein to storage. Arterioscler Thromb Vasc Biol. 2002;22(6):921-926.
-
(2002)
Arterioscler Thromb Vasc Biol
, vol.22
, Issue.6
, pp. 921-926
-
-
Haberichter, S.L.1
Jozwiak, M.A.2
Rosenberg, J.B.3
Christopherson, P.A.4
Montgomery, R.R.5
-
70
-
-
0037441597
-
Critical independent regions in the VWF propeptide and mature VWF that enable normal VWF storage
-
Haberichter SL, Jacobi P, Montgomery RR. Critical independent regions in the VWF propeptide and mature VWF that enable normal VWF storage. Blood. 2003;101(4):1384-1391.
-
(2003)
Blood
, vol.101
, Issue.4
, pp. 1384-1391
-
-
Haberichter, S.L.1
Jacobi, P.2
Montgomery, R.R.3
-
71
-
-
20444417088
-
Intracellular interaction of von Willebrand factor and factor VIII depends on cellular context: Lessons from platelet-expressed factor VIII
-
Yarovoi H, Nurden AT, Montgomery RR, Nurden P, Poncz M. Intracellular interaction of von Willebrand factor and factor VIII depends on cellular context: lessons from platelet-expressed factor VIII. Blood. 2005;105(12):4674-4676.
-
(2005)
Blood
, vol.105
, Issue.12
, pp. 4674-4676
-
-
Yarovoi, H.1
Nurden, A.T.2
Montgomery, R.R.3
Nurden, P.4
Poncz, M.5
-
72
-
-
84926973426
-
Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A
-
Wang X, Shin SC, Chiang AF, et al. Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A. Mol Ther. 2015;23(4):617-626.
-
(2015)
Mol Ther
, vol.23
, Issue.4
, pp. 617-626
-
-
Wang, X.1
Shin, S.C.2
Chiang, A.F.3
-
73
-
-
84899072656
-
Platelets in tumor progression: A host factor that offers multiple potential targets in the treatment of cancer
-
Sharma D, Brummel-Ziedins KE, Bouchard BA, Holmes CE. Platelets in tumor progression: a host factor that offers multiple potential targets in the treatment of cancer. J Cell Physiol. 2014;229(8): 1005-1015.
-
(2014)
J Cell Physiol
, vol.229
, Issue.8
, pp. 1005-1015
-
-
Sharma, D.1
Brummel-Ziedins, K.E.2
Bouchard, B.A.3
Holmes, C.E.4
-
74
-
-
84933278691
-
Selective targeting of bioengineered platelets to prostate cancer vasculature: New paradigm for therapeutic modalities
-
Montecinos VP, Morales CH, Fischer TH, et al. Selective targeting of bioengineered platelets to prostate cancer vasculature: new paradigm for therapeutic modalities. J Cell Mol Med. 2015; 19(7):1530-1537.
-
(2015)
J Cell Mol Med
, vol.19
, Issue.7
, pp. 1530-1537
-
-
Montecinos, V.P.1
Morales, C.H.2
Fischer, T.H.3
-
75
-
-
84906217464
-
Overexpression of tumstatin in genetically modified megakaryocytes changes the proangiogenic effect of platelets
-
Li J, Luo J, Luo YQ, et al. Overexpression of tumstatin in genetically modified megakaryocytes changes the proangiogenic effect of platelets. Transfusion. 2014;54(8):2106-2117.
-
(2014)
Transfusion
, vol.54
, Issue.8
, pp. 2106-2117
-
-
Li, J.1
Luo, J.2
Luo, Y.Q.3
-
76
-
-
84960939219
-
Platelets engineered to store interleukin-24 inhibited melanoma growth in mice [abstract]
-
Abstract OR342
-
Fang J, Yao M, Jing W, Sun B, Johnson BD, Wilcox DA. Platelets engineered to store interleukin-24 inhibited melanoma growth in mice [abstract]. J Thromb Haemost. 2015; 13(suppl S2):225. Abstract OR342.
-
(2015)
J Thromb Haemost
, vol.13
, pp. 225
-
-
Fang, J.1
Yao, M.2
Jing, W.3
Sun, B.4
Johnson, B.D.5
Wilcox, D.A.6
-
77
-
-
84922534997
-
Scalable generation of universal platelets from human induced pluripotent stem cells
-
Feng Q, Shabrani N, Thon JN, et al. Scalable generation of universal platelets from human induced pluripotent stem cells. Stem Cell Rep. 2014;3(5):817-831.
-
(2014)
Stem Cell Rep
, vol.3
, Issue.5
, pp. 817-831
-
-
Feng, Q.1
Shabrani, N.2
Thon, J.N.3
-
78
-
-
84879373708
-
Challenges and promises for the development of donor-independent platelet transfusions
-
Lambert MP, Sullivan SK, Fuentes R, French DL, Poncz M. Challenges and promises for the development of donor-independent platelet transfusions. Blood. 2013;121(17):3319-3324.
-
(2013)
Blood
, vol.121
, Issue.17
, pp. 3319-3324
-
-
Lambert, M.P.1
Sullivan, S.K.2
Fuentes, R.3
French, D.L.4
Poncz, M.5
-
79
-
-
84930644370
-
Comparative analysis of human ex vivo-generated platelets vs megakaryocyte-generated platelets in mice: A cautionary tale
-
Wang Y, Hayes V, Jarocha D, et al. Comparative analysis of human ex vivo-generated platelets vs megakaryocyte-generated platelets in mice: a cautionary tale. Blood. 2015;125(23): 3627-3636.
-
(2015)
Blood
, vol.125
, Issue.23
, pp. 3627-3636
-
-
Wang, Y.1
Hayes, V.2
Jarocha, D.3
-
80
-
-
84916619967
-
Apoptotic effects of platelet factor VIII on megakaryopoiesis: Implications for a modified human FVIII for platelet-based gene therapy
-
Greene TK, Lyde RB, Bailey SC, et al. Apoptotic effects of platelet factor VIII on megakaryopoiesis: implications for a modified human FVIII for platelet-based gene therapy. J Thromb Haemost. 2014;12(12):2102-2112.
-
(2014)
J Thromb Haemost
, vol.12
, Issue.12
, pp. 2102-2112
-
-
Greene, T.K.1
Lyde, R.B.2
Bailey, S.C.3
-
81
-
-
0037129435
-
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
-
Hacein-Bey-Abina S, Le Deist F, Carlier F, et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med. 2002;346(16): 1185-1193.
-
(2002)
N Engl J Med
, vol.346
, Issue.16
, pp. 1185-1193
-
-
Hacein-Bey-Abina, S.1
Le Deist, F.2
Carlier, F.3
-
82
-
-
10944271807
-
Efficacy of gene therapy for SCID is being confirmed
-
Cavazzana-Calvo M, Fischer A. Efficacy of gene therapy for SCID is being confirmed. Lancet. 2004;364(9452):2155-2156.
-
(2004)
Lancet
, vol.364
, Issue.9452
, pp. 2155-2156
-
-
Cavazzana-Calvo, M.1
Fischer, A.2
-
83
-
-
0034724857
-
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
-
Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 2000;288(5466):669-672.
-
(2000)
Science
, vol.288
, Issue.5466
, pp. 669-672
-
-
Cavazzana-Calvo, M.1
Hacein-Bey, S.2
De Saint Basile, G.3
-
84
-
-
14844339323
-
Gene therapy.Panel urges limits on X-SCID trials
-
Kaiser J. Gene therapy. Panel urges limits on X-SCID trials. Science. 2005;307(5715):1544-1545.
-
(2005)
Science
, vol.307
, Issue.5715
, pp. 1544-1545
-
-
Kaiser, J.1
-
85
-
-
0037448352
-
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
-
Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 2003;348(3): 255-256.
-
(2003)
N Engl J Med
, vol.348
, Issue.3
, pp. 255-256
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
-
86
-
-
0037462590
-
Gene therapy.Seeking the cause of induced leukemias in X-SCID trial
-
Kaiser J. Gene therapy. Seeking the cause of induced leukemias in X-SCID trial. Science. 2003; 299(5606):495.
-
(2003)
Science
, vol.299
, Issue.5606
, pp. 495
-
-
Kaiser, J.1
-
87
-
-
0030914867
-
Quiescence, cycling, and turnover in the primitive hematopoietic stem cell compartment
-
Bradford GB, Williams B, Rossi R, Bertoncello I. Quiescence, cycling, and turnover in the primitive hematopoietic stem cell compartment. Exp Hematol. 1997;25(5):445-453.
-
(1997)
Exp Hematol
, vol.25
, Issue.5
, pp. 445-453
-
-
Bradford, G.B.1
Williams, B.2
Rossi, R.3
Bertoncello, I.4
-
88
-
-
79954733266
-
Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection
-
Biffi A, Bartolomae CC, Cesana D, et al. Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection. Blood. 2011;117(20):5332-5339.
-
(2011)
Blood
, vol.117
, Issue.20
, pp. 5332-5339
-
-
Biffi, A.1
Bartolomae, C.C.2
Cesana, D.3
-
89
-
-
53449090226
-
Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity
-
Shi Q, Fahs SA, Wilcox DA, et al. Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity. Blood. 2008; 112(7):2713-2721.
-
(2008)
Blood
, vol.112
, Issue.7
, pp. 2713-2721
-
-
Shi, Q.1
Fahs, S.A.2
Wilcox, D.A.3
-
90
-
-
51649110213
-
Analysis of the spatial and temporal characteristics of plateletdelivered factor VIII-based clots
-
Neyman M, Gewirtz J, Poncz M. Analysis of the spatial and temporal characteristics of plateletdelivered factor VIII-based clots. Blood. 2008; 112(4):1101-1108.
-
(2008)
Blood
, vol.112
, Issue.4
, pp. 1101-1108
-
-
Neyman, M.1
Gewirtz, J.2
Poncz, M.3
|