Factor VIII-eGFP fusion proteins with preserved functional activity for the analysis of the early secretory pathway of factor VIII

Thrombosis and Haemostasis

Volumn 102, Issue 5, 2009, Pages 925-935

  Heinz, Stefan ^a   Schüttrumpf, Jörg ^a   Simpson, Jeremy C ^b   Pepperkok, Rainer ^b   Nicolaes, Gerry A ^c   Abriss, Daniela ^a   Milanov, Peter ^a   Roth, Stefanie ^a   Seifried, Erhard ^a   Tonn, Torsten ^a  

^a Inst Transfus Med I   (Germany)

^b EUROPEAN MOLECULAR BIOLOGY LABORATORY   (Germany)

^c MAASTRICHT UNIVERSITY   (Netherlands)

Author keywords

Coagulation; eGFP; Factor VIII; Intracellular transport; Live cell imaging

Indexed keywords

BLOOD CLOTTING FACTOR 8; ENHANCED GREEN FLUORESCENT PROTEIN;

AMINO ACID SEQUENCE; ANIMAL CELL; ARTICLE; BLOOD CLOTTING; CARBOXY TERMINAL SEQUENCE; ENDOPLASMIC RETICULUM; GOLGI COMPLEX; HUMAN; HUMAN CELL; NONHUMAN; PRIORITY JOURNAL; PROTEIN FUNCTION; PROTEIN LOCALIZATION; PROTEIN PROCESSING; PROTEIN SECRETION; PROTEIN STRUCTURE; PROTEIN TRANSPORT;

ANIMALS; BLOOD COAGULATION; CELL LINE; CERCOPITHECUS AETHIOPS; CHO CELLS; COS CELLS; CRICETINAE; CRICETULUS; ENDOPLASMIC RETICULUM; FACTOR VIII; FACTOR VIIIA; GOLGI APPARATUS; GREEN FLUORESCENT PROTEINS; HUMANS; MODELS, MOLECULAR; PEPTIDE FRAGMENTS; PROTEIN CONFORMATION; PROTEIN STRUCTURE, TERTIARY; PROTEIN TRANSPORT; RECOMBINANT FUSION PROTEINS;

EID: 70449565560     PISSN: 03406245     EISSN: None     Source Type: Journal    
DOI: 10.1160/TH08-12-0807     Document Type: Article

References (52)

1. Hoyer LW. Hemophilia A. N Engl J Med 1994; 330: 38-47.
2. Soucie JM, Evatt B, Jackson D. Occurrence of hemophilia in the United States. The Hemophilia Surveillance System Project Investigators. Am J Hematol 1998; 59: 288-294.
3. Pipe SW. Recombinant clotting factors. Thromb Haemost 2008; 99: 840-850.
4. Powell J. The next generation of anti-haemophilia factor, factor VIII. Long-lasting protection from spontaneous bleeding, are we there yet? Thromb Haemost 2008; 100: 365-366.
5. Musso R, Santagostino E, Faradji A, et al. Safety and efficacy of sucrose-formulated full-length recombinant factor VIII: experience in the standard clinical setting. Thromb Haemost 2008 ; 99: 52-58.
6. Chudakov DM, Lukyanov S, Lukyanov KA. Fluorescent proteins as a toolkit for in vivo imaging. Trends Biotechnol 2005; 23: 605-613. (Pubitemid 41682374)
7. Giepmans BN, Adams SR, Ellisman MH, et al. The fluorescent toolbox for assessing protein location and function. Science 2006; 312: 217-224.
8. Wessels E, Simpson JC. Impact of live cell imaging on coated vesicle research. Semin Cell Dev Biol 2007; 18: 412-423. (Pubitemid 47369233)
9. Furie B, Furie BC. In vivo thrombus formation. J Thromb Haemost 2007; 5 (Suppl. 1): 12-17.
10. Dorner AJ, Kaufman RJ. The levels of endoplasmic reticulum proteins and ATP affect folding and secretion of selective proteins. Biologicals 1994; 22: 103-112.
11. Kaufman RJ, Wasley LC, Dorner AJ. Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells. J Biol Chem 1988; 263: 6352-6362.
12. Pittman DD, Kaufman RJ. Structure-function relationships of factor VIII elucidated through recombinant DNA technology. Thromb Haemost 1989; 61: 161-165.
13. Lynch CM, Israel DI, Kaufman RJ, et al. Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production. Hum Gene Ther 1993; 4: 259-272. (Pubitemid 23209559)
14. Soukharev S, Hammond D, Ananyeva NM, et al. Expression of factor VIII in recombinant and transgenic systems. Blood Cells Mol Dis 2002; 28: 234-248. (Pubitemid 34743757)
15. van den BM, Bierings R, Storm G, et al. Requirements for cellular co-trafficking of factor VIII and von Willebrand factor to Weibel-Palade bodies. J Thromb Haemost 2007; 5: 2235-2242. (Pubitemid 47617876)
16. Morris JA, Dorner AJ, Edwards CA, et al. Immunoglobulin binding protein (BiP) function is required to protect cells from endoplasmic reticulum stress but is not required for the secretion of selective proteins. J Biol Chem 1997; 272: 4327-4334.
17. Nichols WC, Ginsburg D. From the ER to the golgi: insights from the study of combined factors V and VIII deficiency. Am J Hum Genet 1999; 64: 1493-1498. (Pubitemid 30481501)
18. Pittman DD, Tomkinson KN, Kaufman RJ. Post-translational requirements for functional factor V and factor VIII secretion in mammalian cells. J Biol Chem 1994; 269: 17329-17337.
19. Zhang B, McGee B, Yamaoka JS, et al. Combined deficiency of factor V and factor VIII is due to mutations in either LMAN1 or MCFD2. Blood 2006; 107: 1903-1907. (Pubitemid 43289370)
20. Becker S, Simpson JC, Pepperkok R, et al. Confocal microscopy analysis of native, full length and B-domain deleted coagulation factor VIII trafficking in mammalian cells. Thromb Haemost 2004; 92: 23-35. (Pubitemid 39010865)
21. Pipe SW. Coagulation factors with improved properties for hemophilia gene therapy. Semin Thromb Hemost 2004; 30: 227-237.
22. Ngo JC, Huang M, Roth DA, et al. Crystal structure of human factor VIII: implications for the formation of the factor IXa-factor VIIIa complex. Structure 2008; 16: 597-606.
23. Griesbeck O, Baird GS, Campbell RE, et al. Reducing the environmental sensitivity of yellow fluorescent protein. Mechanism and applications. J Biol Chem 2001; 276: 29188-29194. (Pubitemid 37452008)
24. Krieger E, Darden T, Nabuurs SB, et al. Making optimal use of empirical energy functions: force-field parameterization in crystal space. Proteins 2004; 57: 678-683. (Pubitemid 39626989)
25. Toole JJ, Knopf JL, Wozney JM, et al. Molecular cloning of a cDNA encoding human antihaemophilic factor. Nature 1984; 312: 342-347. (Pubitemid 15209311)
26. Tonn T, Herder C, Becker S, et al. Generation and characterization of human hematopoietic cell lines expressing factor VIII. J Hematother Stem Cell Res 2002; 11: 695-704. (Pubitemid 34851801)
27. Mitta B, Rimann M, Ehrengruber MU, et al. Advanced modular self-inactivating lentiviral expression vectors for multigene interventions in mammalian cells and in vivo transduction. Nucleic Acids Res 2002; 30: e113.
28. Beaudouin J, Mora-Bermudez F, Klee T, et al. Dissecting the contribution of diffusion and interactions to the mobility of nuclear proteins. Biophys J 2006; 90: 1878-1894.
29. Karges B, Karges W, Mine M, et al. Mutation Ala(171)Thr stabilizes the gonadotropin-releasing hormone receptor in its inactive conformation, causing familial hypogonadotropic hypogonadism. J Clin Endocrinol Metab 2003; 88: 1873-1879.
30. Kuzhikandathil EV, Oxford GS. Dominant-negative mutants identify a role for GIRK channels in D3 dopamine receptor-mediated regulation of spontaneous secretory activity. J Gen Physiol 2000; 115: 697-706.
31. de Groot BL, van Aalten DM, Scheek RM, et al. Prediction of protein conformational freedom from distance constraints. Proteins 1997; 29: 240-251.
32. van den BM, Bierings R, Storm G, et al. Requirements for cellular co-trafficking of factor VIII and von Willebrand factor to Weibel-Palade bodies. J Thromb Haemost 2007; 5: 2235-2242. (Pubitemid 47617876)
33. Dorner AJ, Bole DG, Kaufman RJ. The relationship of N-linked glycosylation and heavy chain-binding protein association with the secretion of glycoproteins. J Cell Biol 1987; 105(6 Pt 1): 2665-2674.
34. Chalfie M, Tu Y, Euskirchen G, et al. Green fluorescent protein as a marker for gene expression. Science 1994; 263: 802-805. (Pubitemid 24093206)
35. Miao HZ, Sirachainan N, Palmer L, et al. Bioengineering of coagulation factor VIII for improved secretion. Blood 2004; 103: 3412-3419. (Pubitemid 38525672)
36. Pittman DD, Tomkinson KN, Kaufman RJ. Posttranslational requirements for functional factor V and factor VIII secretion in mammalian cells. J Biol Chem 1994; 269: 17329-17337.
37. Dominguez M, Dejgaard K, Fullekrug J, et al. gp25L/emp24/p24 protein family members of the cis-Golgi network bind both COP I and II coatomer. J Cell Biol 1998; 140: 751-765. (Pubitemid 28141215)
38. Fiedler K, Rothman JE. Sorting determinants in the transmembrane domain of p24 proteins. J Biol Chem 1997; 272: 24739-24742. (Pubitemid 27415638)
39. Lowe M, Kreis TE. Regulation of membrane traffic in animal cells by COPI. Biochim Biophys Acta 1998; 1404: 53-66. (Pubitemid 28375705)
40. Saraste J, Kuismanen E. Pre- and post-Golgi vacuoles operate in the transport of Semliki Forest virus membrane glycoproteins to the cell surface. Cell 1984; 38: 535-549. (Pubitemid 14022046)
41. Blum R, Stephens DJ, Schulz I. Lumenal targeted GFP, used as a marker of soluble cargo, visualises rapid ERGIC to Golgi traffic by a tubulo-vesicular network. J Cell Sci 2000; 113(Pt 18): 3151-3159. (Pubitemid 30793924)
42. Kuismanen E, Jantti J, Makiranta V, et al. Effect of caffeine on intracellular transport of Semliki Forest virus membrane glycoproteins. J Cell Sci 1992; 102( Pt 3): 505-513.
43. Simpson JC, Nilsson T, Pepperkok R. Biogenesis of tubular ER-to-Golgi transport intermediates. Mol Biol Cell 2006; 17(2): 723-737. (Pubitemid 43191107)
44. Griffiths G, Pfeiffer S, Simons K, et al. Exit of newly synthesized membrane proteins from the trans cisterna of the Golgi complex to the plasma membrane. J Cell Biol 1985; 101: 949-964. (Pubitemid 16258222)
45. Matlin KS, Simons K. Reduced temperature prevents transfer of a membrane glycoprotein to the cell surface but does not prevent terminal glycosylation. Cell 1983; 34: 233-243.
46. Duden R, Griffiths G, Frank R, et al. Beta-COP, a 110 kd protein associated with non-clathrin-coated vesicles and the Golgi complex, shows homology to beta-adaptin. Cell 1991; 64: 649-665. (Pubitemid 121002980)
47. Griffiths G, Pepperkok R, Locker JK, et al. Immunocytochemical localization of beta-COP to the ER-Golgi boundary and the TGN. J Cell Sci 1995; 108(Pt 8): 2839-2856.
48. Sachs UJ, Nieswandt B. In vivo thrombus formation in murine models. Circ Res 2007; 100: 979-991.
49. van den BM, Bierings R, Storm G, et al. Requirements for cellular co-trafficking of factor VIII and von Willebrand factor to Weibel-Palade bodies. J Thromb Haemost 2007; 5: 2235-2242. (Pubitemid 47617876)
50. Kaufman RJ, Pipe SW, Tagliavacca L, et al. Biosynthesis, assembly and secretion of coagulation factor VIII. Blood Coagul Fibrinolysis 1997; 8 (Suppl. 2): 3-14. (Pubitemid 28140908)
51. Bolt G, Kristensen C, Steenstrup TD. More than one intracellular processing bottleneck delays the secretion of coagulation factor VII. Thromb Haemost 2008 Aug; 100: 204-210.
52. Malhotra JD, Miao H, Zhang K, et al. Antioxidants reduce endoplasmic reticulum stress and improve protein secretion. Proc Natl Acad Sci U S A 2008; 105: 18525-18530.