Platelet Functional Genomics

Platelet Proteomics: Principles, Analysis, and Applications

Volumn , Issue , 2011, Pages 253-284

  Salles, Isabelle I ^a   O'Connor, Marie N ^b   Thijssen Timmer, Daphne C ^c   Broos, Katleen ^d   Deckmyn, Hans ^d  

^a IMPERIAL COLLEGE LONDON   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c UNIVERSITY OF AMSTERDAM   (Netherlands)

^d UNIVERSITY OF LEUVEN   (Belgium)

Author keywords

Genetic manipulation of mammalian megakaryocytes MK and platelets, in vitro and in vivo approaches; Platelet functional genomics, functional roles of candidate genes and proteins, and megakaryocytes; Promise, of human platelet production in experimental animals entailing new perspectives

Indexed keywords

EID: 84857489459     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/9780470940297.ch11     Document Type: Chapter

References (185)

1. Watkins NA, Gusnanto A, de Bono B, De S, Miranda-Saavedra D, Hardie DL, et al., A HaemAtlas: Characterizing gene expression in differentiated human blood cells, Blood, 113(19):e1-9 (2009).
2. O'Connor MN, Salles, II, Cvejic A, Watkins NA, Walker A, Garner SF, et al., Functional genomics in zebrafish permits rapid characterization of novel platelet membrane proteins, Blood, 113(19):4754-4762 (2009).
3. Crozatier M, Meister M, Drosophila haematopoiesis, Cell. Microbiol., 9(5):1117-1126 (2007).
4. Davidson CJ, Tuddenham EG, McVey JH, 450 million years of hemostasis, J.Thromb. Haemost., 1(7):1487-1494 (2003).
5. Grecchi R, Saliba AM, Mariano M, Morphological changes, surface receptors andphagocytic potential of fowl mono-nuclear phagocytes and thrombocytes in vivo andin vitro, J. Pathol., 130(1):23-31 (1980).
6. Daimon T, Mizuhira V, Uchida K, Fine structural distribution of the surfaceconnectedcanalicular system in frog thrombocytes, Cell Tissue Res., 201(3):431-439 (1979).
7. Jagadeeswaran P, Sheehan JP, Craig FE, Troyer D, Identification and characterizationof zebrafish thrombocytes, Br. J. Haematol., 107(4):731-738 (1999).
8. Lin HF, Traver D, Zhu H, Dooley K, Paw BH, Zon LI, et al., Analysis of thrombocytedevelopment in CD41-GFP transgenic zebrafish, Blood, 106(12):3803-3810 (2005).
9. Jagadeeswaran P, Gregory M, Day K, Cykowski M, Thattaliyath B, Zebrafish: Agenetic model for hemostasis and thrombosis, J. Thromb. Haemost., 3(1):46-53 (2005).
10. Carradice D, Lieschke GJ, Zebrafish in hematology: Sushi or science? Blood, 111(7):3331-3342 (2008).
11. Streisinger G, Walker C, Dower N, Knauber D, Singer F, Production of clonesof homozygous diploid zebra fish (Brachydanio rerio), Nature , 291(5813):293-296 (1981).
12. Lieschke GJ, Currie PD, Animal models of human disease: Zebrafish swim intoview, Nat. Rev. Genet., 8(5):353-367 (2007).
13. Davidson AJ, Zon LI, The "definitive" (and "primitive") guide to zebrafishhematopoiesis, Oncogene, 23(43):7233-7246 (2004).
14. Hanumanthaiah R, Day K, Jagadeeswaran P, Comprehensive analysis of blood coagulationpathways in teleostei: Evolution of coagulation factor genes and identificationof zebrafish factor VIIi, Blood Cells Mol. Dis., 29(1):57-68 (2002).
15. Jagadeeswaran P, Sheehan JP, Analysis of blood coagulation in the zebrafish, BloodCells Mol. Dis. 25(3-4):239-249 (1999).
16. Traver D, Herbomel P, Patton EE, Murphey RD, Yoder JA, Litman GW, et al., Thezebrafish as a model organism to study development of the immune system, Adv.Immunol., 81:253-330 (2003).
17. Gering M, Rodaway AR, Gottgens B, Patient RK, Green AR, The SCL gene specifieshaemangioblast development from early mesoderm, EMBO J., 17(14):4029-4045 (1998).
18. Gottgens B, Barton LM, Chapman MA, Sinclair AM, Knudsen B, Grafham D, et al., Transcriptional regulation of the stem cell leukemia gene (SCL)-comparativeanalysis of five vertebrate SCL loci, Genome Res., 12(5):749-759 (2002).
19. Detrich HW 3rd, Kieran MW, Chan FY, Barone LM, Yee K, Rundstadler JA, et al., Intraembryonic hematopoietic cell migration during vertebrate development, Proc.Natl. Acad. Sci. USA, 92(23):10713-10717 (1995).
20. Kalev-Zylinska ML, Horsfield JA, Flores MV, Postlethwait JH, Vitas MR, BaasAM, et al., Runx1 is required for zebrafish blood and vessel development andexpression of a human RUNX1-CBF2T1 transgene advances a model for studiesof leukemogenesis, Development, 129(8):2015-2030 (2002).
21. Thompson MA, Ransom DG, Pratt SJ, MacLennan H, Kieran MW, Detrich HW3rd, et al., The cloche and spadetail genes differentially affect hematopoiesis andvasculogenesis, Dev. Biol., 197(2):248-269 (1998).
22. Berman J, Hsu K, Look AT, Zebrafish as a model organism for blood diseases, Br.J. Haematol., 123(4):568-576 (2003).
23. de Jong JL, Zon LI, Use of the zebrafish system to study primitive and definitivehematopoiesis, Annu. Rev. Genet., 39:481-501 (2005).
24. Willett CE, Cortes A, Zuasti A, Zapata AG, Early hematopoiesis and developinglymphoid organs in the zebrafish, Dev. Dyn., 214(4):323-336 (1999).
25. Kataoka H, Ochi M, Enomoto K, Yamaguchi A, Cloning and embryonic expressionpatterns of the zebrafish Runt domain genes, runxa and runxb, Mech. Dev.98(1-2):139-143 (2000).
26. Burns CE, DeBlasio T, Zhou Y, Zhang J, Zon L, Nimer SD, Isolation and characterizationof runxa and runxb, zebrafish members of the runt family of transcriptionalregulators, Exp. Hematol., 30(12):1381-1389 (2002).
27. Bertrand JY, Kim AD, Teng S, Traver D, CD41 + cmyb + precursors colonizethe zebrafish pronephros by a novel migration route to initiate adult hematopoiesis, Development, 135(10):1853-1862 (2008).
28. Palis J, Yoder MC, Yolk-sac hematopoiesis: The first blood cells of mouse and man, Exp. Hematol., 29(8):927-936 (2001).
29. Day K, Krishnegowda N, Jagadeeswaran P, Knockdown of prothrombin in zebrafish, Blood Cells Mol. Dis., 32(1):191-198 (2004).
30. Sheehan J, Templer M, Gregory M, Hanumanthaiah R, Troyer D, Phan T, et al., Demonstration of the extrinsic coagulation pathway in teleostei: Identification ofzebrafish coagulation factor VII, Proc. Natl. Acad. Sci. USA , 98(15):8768-8773 (2001).
31. Gregory M, Hanumanthaiah R, Jagadeeswaran P, Genetic analysis of hemostasis andthrombosis using vascular occlusion, Blood Cells Mol. Dis., 29(3):286-295 (2002).
32. Amores A, Force A, Yan YL, Joly L, Amemiya C, Fritz A, et al., Zebrafish hoxclusters and vertebrate genome evolution, Science, 282(5394):1711-1714 (1998).
33. Postlethwait JH, Yan YL, Gates MA, Horne S, Amores A, Brownlie A, et al., Vertebrate genome evolution and the zebrafish gene map, Nat. Genet., 18(4):345-349 (1998).
34. Wilming LG, Gilbert JG, Howe K, Trevanion S, Hubbard T, Harrow JL, Thevertebrate genome annotation (Vega) database, Nucleic Acids Res. 36(databaseissue):D753-D760 (2008).
35. Brown LA, Rodaway AR, Schilling TF, Jowett T, Ingham PW, Patient RK, et al., Insights into early vasculogenesis revealed by expression of the ETS-domaintranscription factor Fli-1 in wild-type and mutant zebrafish embryos, Mech. Dev., 90(2):237-252 (2000).
36. Nishikawa K, Kobayashi M, Masumi A, Lyons SE, Weinstein BM, Liu PP, et al., Self-association of Gata1 enhances transcriptional activity in vivo in zebra fishembryos, Mol. Cell. Biol., 23(22):8295-8305 (2003).
37. Pratt SJ, Drejer A, Foott H, Barut B, Brownlie A, Postlethwait J, et al., Isolationand characterization of zebrafish NFE2, Physiol. Genom., 11(2):91-98 (2002).
38. Thattaliyath B, Cykowski M, Jagadeeswaran P, Young thrombocytes initiate theformation of arterial thrombi in zebrafish, Blood, 106(1):118-124 (2005).
39. Jagadeeswaran P, Liu YC, Sheehan JP, Analysis of hemostasis in the zebrafish, Meth. Cell. Biol., 59:337-357 (1999).
40. Denis MM, Tolley ND, Bunting M, Schwertz H, Jiang H, Lindemann S, et al., Escaping the nuclear confines: Signal-dependent pre-mRNA splicing in anucleateplatelets, Cell, 122(3):379-391 (2005).
41. Goessling W, North TE, Zon LI, New waves of discovery: Modeling cancer inzebrafish, J. Clin. Oncol., 25(17):2473-2479 (2007).
42. Chico TJ, Ingham PW, Crossman DC, Modeling cardiovascular disease in thezebrafish, Trends Cardiovasc. Med., 18(4):150-155 (2008).
43. Pelster B, Burggren WW, Disruption of hemoglobin oxygen transport does notimpact oxygen-dependent physiological processes in developing embryos of zebrafish (Danio rerio), Circ. Res., 79(2):358-362 (1996).
44. Grillitsch S, Medgyesy N, Schwerte T, Pelster B, The influence of environmentalP(O(2)) on hemoglobin oxygen saturation in developing zebrafish Danio rerio, J. Exp. Biol. 208(Pt. 2):309-316 (2005).
45. Ekker SC, Stemple DL, Clark M, Chien CB, Rasooly RS, Javois LC, Zebrafishgenome project: Bringing new biology to the vertebrate genome field, Zebrafish, 4(4):239-251 (2007).
46. DrieverW, Solnica-Krezel L, Schier AF, Neuhauss SC, Malicki J, Stemple DL, et al., A genetic screen for mutations affecting embryogenesis in zebrafish, Development123:37-46 (1996).
47. Haffter P, Granato M, Brand M, Mullins MC, Hammerschmidt M, Kane DA, et al., The identification of genes with unique and essential functions in the developmentof the zebrafish, Danio rerio, Development, 123:1-36 (1996).
48. Sivasubbu S, Balciunas D, Amsterdam A, Ekker SC, Insertional mutagenesis strategiesin zebrafish, Genome Biol. 8(Suppl. 1):S9 (2007).
49. Wang D, Jao LE, Zheng N, Dolan K, Ivey J, Zonies S, et al., Efficient genome-widemutagenesis of zebrafish genes by retroviral insertions, Proc. Natl. Acad. Sci. USA, 104(30):12428-12433 (2007).
50. Kawakami K, Takeda H, Kawakami N, Kobayashi M, Matsuda N, Mishina M, Atransposon-mediated gene trap approach identifies developmentally regulated genesin zebrafish, Dev. Cell, 7(1):133-144 (2004).
51. McCallum CM, Comai L, Greene EA, Henikoff S, Targeted screening for inducedmutations, Nat. Biotechnol., 18(4):455-457 (2000).
52. Wienholds E, Schulte-Merker S, Walderich B, Plasterk RH, Target-selected inactivationof the zebrafish rag1 gene, Science, 297(5578):99-102 (2002).
53. Wienholds E, van Eeden F, Kosters M, Mudde J, Plasterk RH, Cuppen E, Efficienttarget-selected mutagenesis in zebrafish, Genome Res., 13(12):2700-2707 (2003).
54. Skromne I, Prince VE, Current perspectives in zebrafish reverse genetics: Movingforward, Dev. Dyn., 237(4):861-882 (2008).
55. Stemple DL, TILLING-a high-throughput harvest for functional genomics, Nat.Rev. Genet., 5(2):145-150 (2004).
56. Nasevicius A, Ekker SC, Effective targeted gene "knockdown" in zebrafish, Nat.Genet., 26(2):216-220 (2000).
57. Summerton J, Weller D, Morpholino antisense oligomers: Design, preparation, andproperties, Antisense Nucleic Acid Drug Dev., 7(3):187-195 (1997).
58. Heasman J, Morpholino oligos: Making sense of antisense? Dev. Biol., 243(2):209-214 (2002).
59. Long Q, Meng A, Wang H, Jessen JR, Farrell MJ, Lin S, GATA-1 expressionpattern can be recapitulated in living transgenic zebrafish using GFP reporter gene, Development, 124(20):4105-4111 (1997).
60. Lawson ND, Weinstein BM, In vivo imaging of embryonic vascular developmentusing transgenic zebrafish, Dev. Biol., 248(2):307-318 (2002).
61. Kissa K, Murayama E, Zapata A, Cortes A, Perret E, Machu C, et al., Live imagingof emerging hematopoietic stem cells and early thymus colonization, Blood, 111(3):1147-1156 (2008).
62. Welcome Trust Case Control Consortium et al., Genome-wide association studyof 14, 000 cases of seven common diseases and 3, 000 shared controls, Nature, 447(7145):661-678 (2007).
63. Ouwehand WH, Platelet genomics and the risk of atherothrombosis, J. Thromb.Haemost. 5(Suppl. 1):188-195 (2007).
64. Samani NJ, Erdmann J, Hall AS, Hengstenberg C, Mangino M, Mayer B, et al., Genomewide association analysis of coronary artery disease, N. Engl. J. Med., 357(5):443-453 (2007).
65. Kelly GM, Erezyilmaz DF, Moon RT, Induction of a secondary embryonic axisin zebrafish occurs following the overexpression of beta-catenin, Mech. Dev., 53(2):261-273 (1995).
66. Toyama R, O'Connell ML, Wright CV, Kuehn MR, Dawid IB, Nodal inducesectopic goosecoid and lim1 expression and axis duplication in zebrafish, Development, 121(2):383-391 (1995).
67. Nikaido M, Tada M, Saji T, Ueno N, Conservation of BMP signaling in zebrafish mesoderm patterning, Mech. Dev. 61(1-2):75-88 (1997).
68. Koos DS, Ho RK, The nieuwkoid gene characterizes and mediates a Nieuwkoopcenter-like activity in the zebrafish, Curr. Biol., 8(22):1199-1206 (1998).
69. Halloran MC, Sato-Maeda M, Warren JT, Su F, Lele Z, Krone PH, et al., Laserinducedgene expression in specific cells of transgenic zebrafish, Development, 127(9):1953-1960 (2000).
70. Hardy ME, Ross LV, Chien CB, Focal gene misexpression in zebrafish embryosinduced by local heat shock using a modified soldering iron, Dev. Dyn., 236(11):3071-3076 (2007).
71. Skromne I, Thorsen D, Hale M, Prince VE, Ho RK, Repression of the hindbraindevelopmental program by Cdx factors is required for the specification of the vertebratespinal cord, Development, 134(11):2147-2158 (2007).
72. Paulus H, Protein splicing and related forms of protein autoprocessing, Annu. Rev.Biochem., 69:447-496 (2000).
73. Zeidler MP, Tan C, Bellaiche Y, Cherry S, Hader S, Gayko U, et al., Temperaturesensitivecontrol of protein activity by conditionally splicing inteins, Nat. Biotechnol., 22(7):871-876 (2004).
74. Liu KJ, Arron JR, Stankunas K, Crabtree GR, Longaker MT, Chemical rescueof cleft palate and midline defects in conditional GSK-3beta mice, Nature, 446(7131):79-82 (2007).
75. Taneyhill LA, Coles EG, Bronner-Fraser M, Snail2 directly represses cadherin6Bduring epithelial-to-mesenchymal transitions of the neural crest, Development, 134(8):1481-1490 (2007).
76. Pei DS, Sun YH, Long Y, Zhu ZY, Inhibition of no tail (ntl) gene expressionin zebrafish by external guide sequence (EGS) technique, Mol. Biol. Rep., 35(2):139-143 (2008).
77. Murphey RD, Stern HM, Straub CT, Zon LI, A chemical genetic screen for cell cycleinhibitors in zebrafish embryos, Chem. Biol. Drug Des., 68(4):213-219 (2006).
78. North TE, Goessling W, Walkley CR, Lengerke C, Kopani KR, Lord AM, et al., Prostaglandin E2 regulates vertebrate haematopoietic stem cell homeostasis, Nature, 447(7147):1007-1011 (2007).
79. Tran TC, Sneed B, Haider J, Blavo D, White A, Aiyejorun T, et al., Automated, quantitativescreening assay for antiangiogenic compounds using transgenic zebrafish, Cancer Res., 67(23):11386-11392 (2007).
80. Burns CG, Milan DJ, Grande EJ, Rottbauer W, MacRae CA, Fishman MC, Highthroughputassay for small molecules that modulate zebrafish embryonic heart rate, Nat. Chem. Biol., 1(5):263-264 (2005).
81. Peterson RT, Shaw SY, Peterson TA, Milan DJ, Zhong TP, Schreiber SL, et al., Chemical suppression of a genetic mutation in a zebrafish model of aortic coarctation, Nat. Biotechnol., 22(5):595-599 (2004).
82. Sun L, Bradford CS, Ghosh C, Collodi P, Barnes DW, ES-like cell cultures derivedfrom early zebrafish embryos, Mol. Mar. Biol. Biotechnol., 4(3):193-199 (1995).
83. Lee KY, Huang H, Ju B, Yang Z, Lin S, Cloned zebrafish by nuclear transfer fromlong-term-cultured cells, Nat. Biotechnol., 20(8):795-799 (2002).
84. Moehle EA, Rock JM, Lee YL, Jouvenot Y, DeKelver RC, Gregory PD, et al., Targeted gene addition into a specified location in the human genome using designedzinc finger nucleases, Proc. Natl. Acad. Sci. USA, 104(9):3055-3060 (2007).
85. Ekker SC, Zinc finger-based knockout punches for zebrafish genes, Zebrafish, 5(2):121-123 (2008).
86. Foley JE, Yeh JR, Maeder ML, Reyon D, Sander JD, Peterson RT, et al., Rapid mutationof endogenous zebrafish genes using zinc finger nucleases made by OligomerizedPool ENgineering (OPEN), PLoS ONE, 4(2):e4348 (2009).
87. Junt T, Schulze H, Chen Z, Massberg S, Goerge T, Krueger A, et al., Dynamicvisualization of thrombopoiesis within bone marrow, Science , 317(5845):1767-1770 (2007).
88. Italiano JE Jr., Patel-Hett S, Hartwig JH, Mechanics of proplatelet elaboration, J. Thromb. Haemost. 5(Suppl. 1):18-23 (2007).
89. Guerriero R, Testa U, Gabbianelli M, Mattia G, Montesoro E, Macioce G, et al., Unilineage megakaryocytic proliferation and differentiation of purified hematopoieticprogenitors in serum-free liquid culture, Blood, 86(10):3725-3736 (1995).
90. Debili N, Wendling F, Cosman D, Titeux M, Florindo C, Dusanter-Fourt I, et al., The Mpl receptor is expressed in the megakaryocytic lineage from late progenitorsto platelets, Blood, 85(2):391-401 (1995).
91. Christensen JL, Weissman IL, Flk-2 is a marker in hematopoietic stem cell differentiation:A simple method to isolate long-term stem cells, Proc. Natl. Acad. Sci.USA, 98(25):14541-14546 (2001).
92. Uchida N, Weissman IL, Searching for hematopoietic stem cells: evidence that Thy-1.1lo Lin- Sca-1+ cells are the only stem cells in C57BL/Ka-Thy-1.1 bone marrow, J. Exp. Med., 175(1):175-184 (1992).
93. Manz MG, Miyamoto T, Akashi K, Weissman IL, Prospective isolation ofhuman clonogenic common myeloid progenitors, Proc. Natl. Acad. Sci. USA, 99(18):11872-11877 (2002).
94. Chang Y, Bluteau D, Debili N, Vainchenker W, From hematopoietic stem cells toplatelets, J. Thromb. Haemost. 5(Suppl. 1):318-327 (2007).
95. Richardson JL, Shivdasani RA, Boers C, Hartwig JH, Italiano JE Jr., Mechanisms oforganelle transport and capture along proplatelets during platelet production, Blood, 106(13):4066-4075 (2005).
96. Cramer EM, Norol F, Guichard J, Breton-Gorius J, Vainchenker W, Masse JM, et al., Ultrastructure of platelet formation by human megakaryocytes cultured withthe Mpl ligand, Blood, 89(7):2336-2346 (1997).
97. Flaumenhaft R, Dilks JR, Richardson J, Alden E, Patel-Hett SR, Battinelli E, et al., Megakaryocyte-derived microparticles: direct visualization and distinction fromplatelet-derived microparticles, Blood, 113(5):1112-1121 (2009).
98. Ungerer M, Peluso M, Gillitzer A, Massberg S, Heinzmann U, Schulz C, et al., Generation of functional culture-derived platelets from CD34+ progenitor cells tostudy transgenes in the platelet environment, Circ. Res., 95(5):e36-e44 (2004).
99. Fujimoto TT, Kohata S, Suzuki H, Miyazaki H, Fujimura K, Production offunctional platelets by differentiated embryonic stem (ES) cells in vitro, Blood, 102(12):4044-4051 (2003).
100. van Hensbergen Y, Schipper LF, Brand A, Slot MC, Welling M, Nauta AJ, et al., Ex vivo culture of human CD34+ cord blood cells with thrombopoietin (TPO)accelerates platelet engraftment in a NOD/SCID mouse model, Exp. Hematol., 34(7):943-950 (2006).
101. van den Oudenrijn S, de Haas M, Calafat J, van der Schoot CE, von dem Borne AE, A combination of megakaryocyte growth and development factor and interleukin-1is sufficient to culture large numbers of megakaryocytic progenitors and megakaryocytesfor transfusion purposes, Br. J. Haematol., 106(2):553-563 (1999).
102. Cortin V, Garnier A, Pineault N, Lemieux R, Boyer L, Proulx C, Efficient in vitromegakaryocyte maturation using cytokine cocktails optimized by statistical experimentaldesign, Exp. Hematol., 33(10):1182-1191 (2005).
103. Balduini A, d'Apolito M, Arcelli D, Conti V, Pecci A, Pietra D, et al., Cord bloodin vitro expanded CD41 cells: Identification of novel components of megakaryocytopoiesis, J. Thromb. Haemost., 4(4):848-860 (2006).
104. Chen TW, Yao CL, Chu IM, Chuang TL, Hsieh TB, Hwang SM, Large generation ofmegakaryocytes from serum-free expanded human CD34+ cells, Biochem. Biophys.Res. Commun., 378(1):112-117 (2009).
105. Boyer L, Robert A, Proulx C, Pineault N, Increased production of megakaryocytesnear purity from cord blood CD34+ cells using a short two-phase culture system, J. Immunol. Meth. 332(1-2):82-91 (2008).
106. Matsunaga T, Tanaka I, Kobune M, Kawano Y, Tanaka M, Kuribayashi K, et al., Ex vivo large-scale generation of human platelets from cord blood CD34+ cells, Stem Cells, 24(12):2877-2887 (2006).
107. Sullenbarger B, Bahng JH, Gruner R, Kotov N, Lasky LC, Prolonged continuousin vitro human platelet production using three-dimensional scaffolds, Exp. Hematol., 37(1):101-110 (2009).
108. Proulx C, Dupuis N, St-Amour I, Boyer L, Lemieux R, Increased megakaryopoiesisin cultures of CD34-enriched cord blood cells maintained at 39 degrees C, Biotechnol.Bioeng., 88(6):675-680 (2004).
109. Mostafa SS, Miller WM, Papoutsakis ET, Oxygen tension influences the differentiation, maturation and apoptosis of human megakaryocytes, Br. J. Haematol., 111(3):879-889 (2000).
110. Cheng L, Qasba P, Vanguri P, Thiede MA, Human mesenchymal stem cells supportmegakaryocyte and pro-platelet formation from CD34(+) hematopoietic progenitorcells, J. Cell. Physiol., 184(1):58-69 (2000).
111. Avecilla ST, Hattori K, Heissig B, Tejada R, Liao F, Shido K, et al., Chemokinemediatedinteraction of hematopoietic progenitors with the bone marrow vascularniche is required for thrombopoiesis, Nat. Med., 10(1):64-71 (2004).
112. Jeanpierre S, Nicolini FE, Kaniewski B, Dumontet C, Rimokh R, Puisieux A, et al., BMP4 regulation of human megakaryocytic differentiation is involved in thrombopoietinsignaling, Blood, 112(8):3154-3163 (2008).
113. O'Brien JJ, Spinelli SL, Tober J, Blumberg N, Francis CW, Taubman MB, et al., 15-Deoxy-delta12, 14-PGJ2 enhances platelet production from megakaryocytes, Blood, 112(10):4051-4060 (2008).
114. Gandhi MJ, Drachman JG, Reems JA, Thorning D, Lannutti BJ, A novel strategyfor generating platelet-like fragments from megakaryocytic cell lines and humanprogenitor cells, Blood Cells Mol. Dis., 35(1):70-73 (2005).
115. Nishikii H, Eto K, Tamura N, Hattori K, Heissig B, Kanaji T, et al., Metalloproteinaseregulation improves in vitro generation of efficacious platelets from mouseembryonic stem cells, J. Exp. Med., 205(8):1917-1927 (2008).
116. Gaur M, Kamata T, Wang S, Moran B, Shattil SJ, Leavitt AD, Megakaryocytesderived from human embryonic stem cells: a genetically tractable system to studymegakaryocytopoiesis and integrin function, J. Thromb. Haemost., 4(2):436-442 (2006).
117. Takayama N, Nishikii H, Usui J, Tsukui H, Sawaguchi A, Hiroyama T, et al., Generation of functional platelets from human embryonic stem cells in vitro via ESsacs, VEGF-promoted structures that concentrate hematopoietic progenitors, Blood, 111(11):5298-5306 (2008).
118. Choi ES, Nichol JL, Hokom MM, Hornkohl AC, Hunt P, Platelets generatedin vitro from proplatelet-displaying human megakaryocytes are functional, Blood, 85(2):402-413 (1995).
119. Cortin V, Pineault N, Garnier A, Ex vivo megakaryocyte expansion and platelet productionfrom human cord blood stem cells, Meth. Mol. Biol., 482:109-126 (2009).
120. Gillitzer A, Peluso M, Laugwitz KL, Munch G, Massberg S, Konrad I, et al., Retroviralinfection and selection of culture-derived platelets allows study of the effectof transgenes on platelet physiology ex vivo and on thrombus formation in vivo, Arterioscler. Thromb. Vasc. Biol., 25(8):1750-1755 (2005).
121. Lapidot T, Fajerman Y, Kollet O, Immune-deficient SCID and NOD/SCIDmice models as functional assays for studying normal and malignant humanhematopoiesis, J. Mol. Med., 75(9):664-673 (1997).
122. Shultz LD, Schweitzer PA, Christianson SW, Gott B, Schweitzer IB, Tennent B, et al., Multiple defects in innate and adaptive immunologic function in NOD/LtSzscidmice, J. Immunol., 154(1):180-191 (1995).
123. Shultz LD, Ishikawa F, Greiner DL, Humanized mice in translational biomedicalresearch, Nat. Rev. Immunol., 7(2):118-130 (2007).
124. Ueda T, Yoshino H, Kobayashi K, Kawahata M, Ebihara Y, Ito M, et al., Hematopoieticrepopulating ability of cord blood CD34(+) cells in NOD/Shi-scid mice, StemCells, 18(3):204-213 (2000).
125. Perez LE, Rinder HM, Wang C, Tracey JB, Maun N, Krause DS, Xenotransplantationof immunodeficient mice with mobilized human blood CD34+ cells providesan in vivo model for human megakaryocytopoiesis and platelet production, Blood, 97(6):1635-1643 (2001).
126. Angelopoulou M, Novelli E, Grove JE, Rinder HM, Civin C, Cheng L, et al., Cotransplantation of human mesenchymal stem cells enhances human myelopoiesisand megakaryocytopoiesis in NOD/SCID mice, Exp. Hematol., 31(5):413-420 (2003).
127. Angelopoulou MK, Rinder H, Wang C, Burtness B, Cooper DL, Krause DS, Apreclinical xenotransplantation animal model to assess human hematopoietic stemcell engraftment, Transfusion, 44(4):555-566 (2004).
128. Tijssen MR, van Hennik PB, di Summa F, Zwaginga JJ, van der Schoot CE, Voermans C, Transplantation of human peripheral blood CD34-positive cells in combinationwith ex vivo generated megakaryocytes results in fast platelet formation inNOD/SCID mice, Leukemia, 22(1):203-208 (2008).
129. Feng Y, Zhang L, Xiao ZJ, Li B, Liu B, Fan CG, et al., An effective and simpleexpansion system for megakaryocyte progenitor cells using a combination of heparinwith thrombopoietin and interleukin-11, Exp. Hematol., 33(12):1537-1543 (2005).
130. Pick M, Perry C, Lapidot T, Guimaraes-Sternberg C, Naparstek E, Deutsch V, et al., Stress-induced cholinergic signaling promotes inflammation-associated thrombopoiesis, Blood, 107(8):3397-3406 (2006).
131. Mattia G, Milazzo L, Vulcano F, Pascuccio M, Macioce G, Hassan HJ, et al., Long-term platelet production assessed in NOD/SCID mice injected with cord bloodCD34(+) cells, thrombopoietin-amplified in clinical grade serum-free culture, Exp.Hematol., 36(2):244-252 (2008).
132. Bruno S, Gunetti M, Gammaitoni L, Dane A, Cavalloni G, Sanavio F, et al., In vitro and in vivo megakaryocyte differentiation of fresh and ex-vivo expandedcord blood cells: Rapid and transient megakaryocyte reconstitution, Haematologica, 88(4):379-387 (2003).
133. Bruno S, Gunetti M, Gammaitoni L, Perissinotto E, Caione L, Sanavio F, et al., Fast but durable megakaryocyte repopulation and platelet production in NOD/SCIDmice transplanted with ex-vivo expanded human cord blood CD34+ cells, Stem Cells, 22(2):135-143 (2004).
134. Perez LE, Alpdogan O, Shieh JH, Wong D, Merzouk A, Salari H, et al., Increasedplasma levels of stromal-derived factor-1 (SDF-1/CXCL12) enhance human thrombopoiesisand mobilize human colony-forming cells (CFC) in NOD/SCID mice, ExpHematol., 32(3):300-307 (2004).
135. Suzuki K, Hiramatsu H, Fukushima-ShintaniM, Heike T, Nakahata T, Efficient assayfor evaluating human thrombopoiesis using NOD/SCID mice transplanted with cordblood CD34+ cells, Eur. J. Haematol., 78(2):123-130 (2006).
136. Schipper LF, van Hensbergen Y, Fibbe WE, Brand A, A sensitive quantitative singleplatformflow cytometry protocol to measure human platelets in mouse peripheralblood, Transfusion, 47(12):2305-2314 (2007).
137. Nakamura T, Miyakawa Y, Miyamura A, Yamane A, Suzuki H, Ito M, et al., Anovel nonpeptidyl human c-Mpl activator stimulates human megakaryopoiesis andthrombopoiesis, Blood, 107(11):4300-4307 (2006).
138. Tse WW, Zang SL, Bunting KD, Laughlin MJ, Umbilical cord blood transplantationin adult myeloid leukemia, Bone Marrow Transpl., 41(5):465-472 (2008).
139. Salles II, Thijs T, Brunaud C, De Meyer SF, Thys J, Vanhoorelbeke K, Deckmyn H, Human platelets produced in nonobese diabetic/severe combinedimmunodeficient (NOD/SCID) mice upon transplantation of human cord bloodCD34(+) cells are functionally active in an ex vivo flow model of thrombosis, Blood, 114(24):5044-5051 (2009).
140. Salmon P, Kindler V, Ducrey O, Chapuis B, Zubler RH, Trono D, High-level transgeneexpression in human hematopoietic progenitors and differentiated blood lineagesafter transduction with improved lentiviral vectors, Blood , 96(10):3392-3398 (2000).
141. Barraza RA, Poeschla EM, Human gene therapy vectors derived from felinelentiviruses, Vet. Immunol. Immunopathol. 123(1-2):23-31 (2008).
142. Philpott NJ, Thrasher AJ, Use of nonintegrating lentiviral vectors for gene therapy, Hum Gene Ther., 18(6):483-489 (2007).
143. Santoni de Sio F, Naldini L, Short-term culture of human CD34+ cells for lentiviralgene transfer, Meth. Mol. Biol., 506:59-70 (2009).
144. Santoni de Sio FR, Gritti A, Cascio P, Neri M, Sampaolesi M, Galli C, et al., Lentiviral vector gene transfer is limited by the proteasome at postentry steps invarious types of stem cells, Stem Cells, 26(8):2142-2152 (2008).
145. Raslova H, Komura E, Le Couedic JP, Larbret F, Debili N, Feunteun J, et al., FLI1 monoallelic expression combined with its hemizygous loss underlies Paris-Trousseau/Jacobsen thrombopenia, J. Clin. Invest., 114(1):77-84 (2004).
146. Shi Q, Wilcox DA, Morateck PA, Fahs SA, Kenny D, Montgomery RR, Targeting platelet GPIbalpha transgene expression to human megakaryocytes and forminga complete complex with endogenous GPIbbeta and GPIX, J. Thromb. Haemost., 2(11):1989-1997 (2004).
147. Shi Q, Wilcox DA, Fahs SA, Fang J, Johnson BD, Du LM, et al., Lentivirusmediatedplatelet-derived factor VIII gene therapy in murine haemophilia A, J. Thromb. Haemost., 5(2):352-361 (2007).
148. Fang J, Hodivala-Dilke K, Johnson BD, Du LM, Hynes RO, White GC 2nd, WilcoxDA, et al., Therapeutic expression of the platelet-specific integrin, alphaIIbbeta3, ina murine model for Glanzmann thrombasthenia, Blood, 106(8):2671-2679 (2005).
149. Reiser J, Lai Z, Zhang XY, Brady RO, Development of multigene and regulatedlentivirus vectors, J. Virol., 74(22):10589-10599 (2000).
150. Wilcox DA, Olsen JC, Ishizawa L, Griffith M, White GC 2nd, Integrin alphaIIbpromoter-targeted expression of gene products in megakaryocytes derived fromretrovirus-transduced human hematopoietic cells, Proc. Natl. Acad. Sci. USA, 96(17):9654-9659 (1999).
151. Wilcox DA, Olsen JC, Ishizawa L, Bray PF, French DL, Steeber DA, et al., Megakaryocyte-targeted synthesis of the integrin beta(3)-subunit results in thephenotypic correction of Glanzmann thrombasthenia, Blood , 95(12):3645-3651 (2000).
152. Lavenu-Bombled C, Izac B, Legrand F, Cambot M, Vigier A, Masse JM, et al., Glycoprotein Ibalpha promoter drives megakaryocytic lineage-restricted expressionafter hematopoietic stem cell transduction using a self-inactivating lentiviral vector, Stem Cells, 25(6):1571-1577 (2007).
153. Gillitzer A, Peluso M, Bultmann A, Munch G, Gawaz M, Ungerer M, Effect ofdominant negative SNAP-23 expression on platelet function, J. Thromb. Haemost., 6(10):1757-1763 (2008).
154. Briquet-Laugier V, Lavenu-Bombled C, Schmitt A, Leboeuf M, Uzan G, Dubart-Kupperschmitt A, et al., Probing platelet factor 4 alpha-granule targeting, J. Thromb.Haemost., 2(12):2231-2240 (2004).
155. Yan XQ, Lacey DL, Saris C, Mu S, Hill D, Hawley RG, et al., Ectopic overexpressionof c-mpl by retroviral-mediated gene transfer suppressed megakaryopoiesis butenhanced erythropoiesis in mice, Exp. Hematol., 27(9):1409-1417 (1999).
156. Fock EL, Yan F, Pan S, Chong BH, NF-E2-mediated enhancement of megakaryocyticdifferentiation and platelet production in vitro and in vivo, Exp. Hematol., 36(1):78-92 (2008).
157. Li Z, Fehse B, Schiedlmeier B, Dullmann J, Frank O, Zander AR, et al., Persistingmultilineage transgene expression in the clonal progeny of a hematopoietic stemcell, Leukemia, 16(9):1655-1663 (2002).
158. Klug CA, Cheshier S, Weissman IL, Inactivation of a GFP retrovirus occurs atmultiple levels in long-term repopulating stem cells and their differentiated progeny, Blood, 96(3):894-901 (2000).
159. Ghevaert C, Wilcox DA, Fang J, Armour KL, Clark MR, Ouwehand WH, et al., Developing recombinant HPA-1a-specific antibodies with abrogated Fcgamma receptorbinding for the treatment of fetomaternal alloimmune thrombocytopenia, J. Clin.Invest., 118(8):2929-2938 (2008).
160. Dupre L, Marangoni F, Scaramuzza S, Trifari S, Hernandez RJ, Aiuti A, et al., Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WASpromoter/cDNA-containing lentiviral vector and nonlethal irradiation, Hum GeneTher., 17(3):303-313 (2006).
161. Orange JS, Stone KD, Turvey SE, Krzewski K, The Wiskott-Aldrich syndrome, Cell. Mol. Life Sci., 61(18):2361-2385 (2004).
162. Horn PA, Keyser KA, Peterson LJ, Neff T, Thomasson BM, Thompson J, et al., Efficient lentiviral gene transfer to canine repopulating cells using an overnighttransduction protocol, Blood, 103(10):3710-3716 (2004).
163. Kootstra NA, Matsumura R, Verma IM, Efficient production of human FVIII inhemophilic mice using lentiviral vectors, Mol. Ther. 7(5Pt 1):623-631 (2003).
164. Tiede A, Eder M, von Depka M, Battmer K, Luther S, Kiem HP, et al., Recombinantfactor VIII expression in hematopoietic cells following lentiviral transduction, GeneTher., 10(22):1917-1925 (2003).
165. Wilcox DA, Shi Q, Nurden P, Haberichter SL, Rosenberg JB, Johnson BD, et al., Induction of megakaryocytes to synthesize and store a releasable pool of humanfactor VIII, J. Thromb. Haemost., 1(12):2477-2489 (2003).
166. Yarovoi HV, Kufrin D, Eslin DE, Thornton MA, Haberichter SL, Shi Q, et al., Factor VIII ectopically expressed in platelets: Efficacy in hemophilia A treatment, Blood, 102(12):4006-4013 (2003).
167. Shi Q, Wilcox DA, Fahs SA, Weiler H, Wells CW, Cooley BC, et al., FactorVIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titerinhibitory antibodies, J. Clin. Invest., 116(7):1974-1982 (2006).
168. Shi Q, Fahs SA, Wilcox DA, Kuether EL, Morateck PA, Mareno N, et al., Syngeneictransplantation of hematopoietic stem cells that are genetically modified to expressfactor VIII in platelets restores hemostasis to hemophilia A mice with preexistingFVIII immunity, Blood, 112(7):2713-2721 (2008).
169. Wilcox DA, Du LM, Haberichter SL, Jacobi PM, Fang J, Jensen ES, et al., Platelet targeted expression of coagulation factor VIII (FVIII) shows efficacy for using thedog as a large animal model for gene therapy of hemophilia A, Proc. 50th Meeting American Society of Hematology, San Francisco, Dec. 6-9, 2008.
170. Kikuchi J, Mimuro J, Ogata K, Tabata T, Ueda Y, Ishiwata A, Sakata Y, et al., Sustained transgene expression by human cord blood derived CD34+ cellstransduced with simian immunodeficiency virus agmTYO1-based vectors carryingthe human coagulation factor VIII gene in NOD/SCID mice, J. Gene Med., 6(10):1049-1060 (2004).
171. Ohmori T, Mimuro J, Takano K, Madoiwa S, Kashiwakura Y, Ishiwata A, et al., Efficient expression of a transgene in platelets using simian immunodeficiencyvirus-based vector harboring glycoprotein Ibalpha promoter: In vivo model forplatelet-targeting gene therapy, FASEB J., 20(9):1522-1524 (2006).
172. Ohmori T, Ishiwata A, Kashiwakura Y, Madoiwa S, Mitomo K, Suzuki H, et al., Phenotypic correction of hemophilia A by ectopic expression of activated factor VIIin platelets, Mol. Ther., 16(8):1359-1365 (2008).
173. Labbaye C, Spinello I, Quaranta MT, Pelosi E, Pasquini L, Petrucci E, et al., Athree-step pathway comprising PLZF/miR-146a/CXCR4 controls megakaryopoiesis, Nat. Cell. Biol, 10(7):788-801 (2008).
174. Gushiken FC, Patel V, Liu Y, Pradhan S, Bergeron AL, Peng Y, et al., Proteinphosphatase 2A negatively regulates integrin alpha(IIb)beta(3) signaling, J. Biol.Chem., 283(19):12862-12869 (2008).
175. Jung YJ, Chae HC, Seoh JY, Ryu KH, Park HK, Kim YJ, et al., Pim-1 induced polyploidybut did not affect megakaryocytic differentiation of K562 cells and CD34+cells from cord blood, Eur. J. Haematol., 78(2):131-138 (2007).
176. Schulze H, Dose M, Korpal M, Meyer I, Italiano JE, Jr., Shivdasani RA, RanBP10 is a cytoplasmic guanine nucleotide exchange factor that modulates noncentrosomal microtubules, J. Biol. Chem., 283(20):14109-14119 (2008).
177. Bouilloux F, Juban G, Cohet N, Buet D, Guyot B, Vainchenker W, et al., EKLF restricts megakaryocytic differentiation at the benefit of erythrocytic differentiation, Blood, 112(3):576-584 (2008).
178. Levay K, Slepak VZ, Tescalcin is an essential factor in megakaryocytic differentiationassociated with Ets family gene expression, J. Clin. Invest., 117(9):2672-2683 (2007).
179. Gurbuxani S, Xu Y, Keerthivasan G, Wickrema A, Crispino JD, Differential requirementsfor survivin in hematopoietic cell development, Proc. Natl. Acad. Sci. USA, 102(32):11480-11485 (2005).
180. Olthof SG, Fatrai S, Drayer AL, Tyl MR, Vellenga E, Schuringa JJ, Down regulation of signal transducer and activator of transcription 5 (STAT5) in CD34+ cells promotes megakaryocytic development, whereas activation of STAT5 drives erythropoiesis, Stem Cells, 26(7):1732-1742 (2008).
181. Berthebaud M, Riviere C, Jarrier P, Foudi A, Zhang Y, Compagno D, et al., RGS16 is a negative regulator of SDF-1-CXCR4 signaling in megakaryocytes, Blood, 106(9):2962-2968 (2005).
182. Eto K, Nishikii H, Ogaeri T, Suetsugu S, Kamiya A, Kobayashi T, et al., TheWAVE2/Abi1 complex differentially regulates megakaryocyte development andspreading: Implications for platelet biogenesis and spreading machinery, Blood, 110(10):3637-3647 (2007).
183. Ohmori T, Kashiwakura Y, Ishiwata A, Madoiwa S, Mimuro J, Sakata Y, Silencingof a targeted protein in in vivo platelets using a lentiviral vector delivering shorthairpin RNA sequence, Arterioscler. Thromb. Vasc. Biol., 27(10):2266-2272 (2007).
184. Petrich BG, Marchese P, Ruggeri ZM, Spiess S, Weichert RA, Ye F, et al., Talinis required for integrin-mediated platelet function in hemostasis and thrombosis, J. Exp. Med., 204(13):3103-3111 (2007).
185. Nieswandt B, Moser M, Pleines I, Varga-Szabo D, Monkley S, Critchley D, et al., Loss of talin1 in platelets abrogates integrin activation, platelet aggregation, andthrombus formation in vitro and in vivo, J. Exp. Med., 204(13):3113-3118 (2007).