Thrombosis and Haemostasis, Table of Contents Thromb Haemost 1997; 78(01): 532-536DOI: 10.1055/s-0038-1657583 Transgenic production of hemostasis proteins Schattauer GmbH Stuttgart Vitamin K-Dependent Protein Production in Transgenic Animals Henryk Luboń J. Holland Laboratory, American Red Cross, Rockville, MD, USA , Rekha K Paleyanda J. Holland Laboratory, American Red Cross, Rockville, MD, USA › Author Affiliations Recommend Article Abstract PDF Download Buy Article PDF (1071 kb) References References 1 Furie B, Furie BC. Molecular and cellular biology of blood coagulation. N Engl J Med 1992; 326: 800-805 2 D’Angelo SV, Comp PC, Esmon CT, D’Angelo A. Relationship between protein C antigen and anticoagulant activity during oral anticoagulation and in selected disease states. J Clin Invest 1986; 77: 416-425 3 Robertson LE, Wasserstrum N, Banez E, Vasquez M, Sears DA. Hereditary factor VII deficiency in pregnancy: Peripartum treatment with factor VII concentrate. Am J Hematol 1992; 40: 38-41 4 Aiach M, Gandrille S, Emmerich J. A review of mutations causing deficiencies of antithrombin, Protein C and Protein S. Thromb Haemost 1995; 74: 81-89 5 Tuddenham EGD, Pemberton S, Cooper DN. Inherited factor VII deficiency: Genetics and molecular pathology. Thromb Haemost 1995; 74: 313-321 6 Bauer KA. Management of patients with hereditary defects predisposing to thrombosis including pregnant women. Thromb Haemost 1995; 74: 94-100 7 Pabinger I, Schneider B. Thrombotic risk in hereditary anti-thrombin III, protein C, or protein S deficiency. A cooperative, retrospective study. Arterioscler Thromb Vase Biol 1996; 16: 742-748 8 Stirling D, Ludlam CA. Therapeutic concentrates for the treatment of congenital deficiencies of factors VII, XI and XIII. Semin Thromb Hemost 1993; 19: 48-53 9 Thompson AR. Factor IX concentrates for clinical use. Semin Thromb Hemost 1993; 19: 25-35 10 Hirsh J, Prins MH, Samama M. Approach to the thrombophilic patient for hemostasis and thrombosis: Basic Principles and Clinical Practice. In: Colman RW, Hirsh J, Marder VJ, Salzman EW. (eds) Hemostasis and Thrombosis: Basic Principles and Clinical Practice. 03. rd ed Philadelphia: Lippincott; 1994. pp. 1543-1561 11 Andrew M, Brooker LA. Blood component therapy in neonatal hemostatic disorders. Transfusion Med Rev 1995; 09: 231-250 12 Hedner U, Bjoem S, Bemvil SS, Tengbom L, Stigendahl L. Clinical experience with human plasma-derived factor VIIa in patients with hemophilia A and high titer inhibitors. Hemostasis 1989; 19: 335-343 13 Kim HC, McMillan CW, White GC, Bergman GE, Horton M, Saidi P. Purified factor IX using monoclonal immunoaffinity technique: Clinical trials in hemophilia B and comparison to prothrombin complex concentrates. Blood 1992; 79: 568-575 14 Dreyfus M, Magny JF, Bridey F, Schwarz HP, Panche C, Dehan M, Tchemia G. Treatment of homozygous protein C deficiency and neonatal purpura fulminans with a purified protein C concentrate. N Eng J Med 1991; 325: 1565-1568 15 De Stefano V, Mastrangelo S, Schwarz HP, Pola P, Flore B, Bizzi B, Leone G. Replacement therapy with a purified protein C concentrate during of initiation of oral anticoagulation in severe protein C congenital deficiency. Thromb Haemostas 1993; 70: 247-249 16 Schramm W, Spannagl M, Bauer KA, Rosenberg RD, Birkner B, Linnau Y, Schwarz HP. Treatment of coumarin-induced skin necrosis with a monoclonal antibody purified protein C concentrate. Arch Dermat 1993; 12: 753-756 17 Bemtrop E. Why prescribe highly purified factor VIII and IX concentrates. Vox Sang 1996; 70: 61-68 18 Schmidt ML, Smith HE, Gamerman S, DiMichele D, Glazer S, Scott JP. Prolonged recombinant activated factor VII (rFVIIa) treatment for severe bleeding in a factor IX-deficient patient with an inhibitor. Br J Haematol 1991; 78: 460-463 19 Busby S, Kumar A, Joseph M, Halfjpap L, Insley M, Berkner K, Kurachi K, Woodbury R. Expression of active human factor IX in transfected cells. Nature 1985; 316: 271-273 20 Anson DS, Qusten DE, Brownlee GG. Expression of active human clotting factor IX from recombinant DNA clones in mammalian cells. Nature 1985; 315: 683-685 21 de laSalle H, Altenbuiger W, Elkaim R, Dott K, Dieterle A, Drillien R, Cazenave JP, Tolstoshev P, Lecocq PL. Active gamma-carboxylated human factor IX expressed using recombinant DNA techniques. Nature 1985; 316: 268-270 22 Kaufman R, Wasley L, Fune BC, Furie B, Shoemaker C. Expression, purification, and characterization of recombinant γ-carboxylated human factor IX synthesized in Chinese hamster ovary cells. J Biol Chem 1986; 261: 9622-9628 23 Yan BS, Grinnell BW, Wold F. Post-translational modification of protein: Some problems left to solve. Trends in Biological Sci 1989; 14: 264-268 24 Yan SB, Razzano P, Chao YB, Walls JD, Berg DT, McClure DB, Grinell BW. Characterization and novel purification of recombinant human protein C from three mammalian cell lines. Bio/Technology 1990; 8: 655-661 25 Grinnell BW, Walls JD, Berg DT, Boston J, McClure DB, Yan SB. Expression, characterization, and processing of recombinant human protein C from adenovirus-transformed cell lines. In: Hershberger CL, Queener SW, Hegeman G. (eds) Genetics and Molecular Biology of Industrial Microorganisms, American Society of Microbiology. Washington, DC: 1989. pp. 226-237 26 Grinnell BW, Walls JD, Gerlitz B, Berg DT, McClure DB, Ehrlich H, Bang NU, Yan SB. Native and modified recombinant human protein C: Function, secretion, and posttranslational modifications. In: Biuley DF, Drohan WN. (eds) Protein C and Related Anticoagulants. Houston, TX: Gulf; 1990. pp. 29-63 27 Berkner KK. Expression of recombinant vitamin K-dependent proteins in mammalian cells. Methods Enzymol 1993; 222: 450-477 28 Macik BG, Hohneker J, Roberts HR, Griffen AM. Use of recombinant activated factor VII for treatment of a retro-pharyngeal hemorrhage in a hemophilic patient with a high titer inhibitor. Am J Hematol 1989; 32: 232-234 29 Hedner U, Glazer S, Falch J. Recombinant activated factor VII in the treatment of bleeding episodes in patients with inherited and acquired bleeding disorders. Transfusion Med Rev 1993; 7: 78-83 30 Ragni M, White G, Pasi J, Garzone P, Courter S, Goodfellow J. Dose response relationship of rFIX in the surgical setting. Blood 1996; 88: 329a (abstract) 31 White G, Lusher J, Shapiro A, Tubridy K, Courter S. Recombinant factor IX in the treatment of previously-treated patients. Blood 1996; 88: 327a (abstract) 32 DiMichele D. Hemophilia 1996: New approach to an old disease. Pediat Clin North Am 1996; 43: 709-736 33 Roberts HR, Eberst ME. Current management of hemophilia B. Hematol Oncol Clin North Am 1993; 7: 1269-1280 34 Hennighausen L. The mammary gland as a bioreactor: Production of foreign proteins in milk. Prot Exp Purif 1990; 01: 3-8 35 Choo KH, Raphael K, McAdam W, Peterson MG. Expression of active human clotting factor IX in transgenic mice: Use of cDNA with complete mRNA sequence. Nucl Acids Res 1987; 15: 871-884 36 Jallat S, Perraud F, Dalemans W, Balland A, Dieterle A, Faure T, Meulien P, Pavirani A. Characterization of recombinant human factor IX expressed in transgenic mice and in derived trans-immortalized hepatic cell lines. EMBO J 1990; 09: 3295-3301 37 Simons JP, Wilmut I, Clark AJ, Archibald AL, Bishop JO, Lathe R. Gene transfer into sheep. Bio/Technology 1987; 6: 179-183 38 Clark AJ, Bessos H, Bishop JO, Brown P, Harris S, Lathe R, McClenaghan M, Prowse C, Simons JP, Whitelaw CBA, Wilmut I. Expression of human anti-hemophilic factor IX in the milk of transgenic sheep. Bio/Technology 1989; 07: 487-892 39 Whitelaw CBA, Archibald AL, Harris S, McClenaghan M, Simons JP, Clark AJ. Targeting expression to the mammary gland: Intronic sequences can enhance the efficiency of gene expression in transgenic mice. Transgenic Res 1991; 01: 3-13 40 Clark AJ, Cowper A, Wallace R, Wright G, Simons JP. Rescuing transgene expression by co-integration. Bio/Technology 1992; 10: 1450-1454 41 Yuli F, Binas B, Harold G, Wallace R, Clark AJ. Transgene rescue in the mammary gland is associated with transcription but does not require translation of BLG transgenes. Transgenic Res 1997; 06: 11-17 42 Yuli F, Harold G, Wallace R, Cowper A, Percy J, Cottingham I, Clark A. Fixing human factor IX (fIX): Correction of a ciyptic RNA splice enables the production of biologically active fIX in the mammary gland of transgenic mice. Proc Natl Acad Sci USA 1995; 92: 10899-10903 43 Velander WH, Page RL, Morcol T, Russell CG, Canseco R, Young JM, Drohan WN, Gwazdauskas FC, Wilkins TD, Johnson JL. Production of biologically active protein C in the milk of transgenic mice. Ann NY Acad Sci 1992; a 665: 391-403 44 Hu Y, Qiu X, Xue J, (Hsueh LL) Liu Z. Regulation of human clotting factor IX cDNA expression in transgenic mice. Science In China(B) 1995; 38: 825-833 45 Paleyanda RK, Russell CG, Chang RR, Johnson J, Velander W, Drohan WN, Lubon H. γ-Carboxylation of human vitamin K-dependent proteins in the mouse mammary gland. In: Proc. of the 1995 Miami Bio/Technology Winter Symposium, Advances in Gene Technology, IRL Press at Oxford University Press, Miami Bio/Technology Short Reports. 1995. 6. 108 46 Foster DC, Yoshitake S, Davie EW. The nucleotide sequence of the gene for human protein C. Proc Natl Acad Sci 1985; 82: 4673-4677 47 Paleyanda RK, Zhang D-W, Hennighausen L, McKnight R, Drohan WN, Lubon H. Regulation of human protein C gene expression by the mouse WAP promoter. Transgenic Res 1994; 03: 335-343 48 Drohan WN, Zhang D-W, Paleyanda RK, Chang R, Wroble M, Velander W, Lubon H. Inefficient processing of human protein C in the mouse mammary gland. Transgenic Res 1994; a 03: 355-364 49 Suttie JW. Report of workshop on expression of vitamin K-dependent proteins in bacterial and mammalian cells. Thromb Res 1986; 44: 129-134 50 Paleyanda RK. The expression of recombinant human protein C in the transgenic mammary gland. Ph.D. Thesis. 1994. b 51 Yan BS, Chao YB, van HalbeekH. Novel Asn-linked oligosaccharides terminating in GalNAcβ(l→4)[Fucα(l→3)] GlcNAcβ(l→•) are present in recombinant human protein C expressed in human kidney 293 cells. Glycobiology 1993; 03: 597-608 52 Velander WH, Johnson JL, Page RL, Russell CG, Subramanian A, Wilkins TD, Gwazdauskas FC, Pittius C, Drohan WN. High-level expression of a heterologous protein in the milk of transgenic swine using the cDNA encoding human protein C. Proc Nad Acad Sci USA 1992; b 89: 12003-12007 53 Van CottKE, Lubon H, Russell CG, Butler SP, Gwazdauskas FC, Knight J, Drohan NW, Velander WH. Phenotypic and genotypic stability of multiple lines of transgenic pigs expressing recombinant protein C. Transgenic Res. in press 54 Van Cott KE, Williams BL, Gwazdauskas F, Lee TK, Lubon H, Drohan WN, Velander WH. Affinity purification of biologically active and inactive forms of recombinant human protein C produced in the porcine mammary gland. J Mol Recognition. in press 55 Lee TK, Drohan WN, Lubon H. Proteolytic processing of human protein C in swine mammary gland. J Biochem 1995; 118: 81-87 56 Lee TK, Bangalore N, Velander W, Drohan WN, Lubon H. Activation of recombinant human protein C. Thromb Res 1996; 82: 225-234 57 Drohan WN, Wilkins TD, Latimer E, Zhou D, Velander W, Lee TK, Lubon H. Production and purification of recombinant human protein C from the milk of transgenic swine. In: Galindo E, Ramirez OT. (eds). Advances in Bioprocess Engineering. Kluwer Netherlands: Academic; 1994. b pp. 501-507 58 Medved LV, Orthner CL, Lubon H, Lee TK, Drohan WN, Ingham KC. Thermal stability and domain-domain interactions in natural and recombinant protein C. J Biol Chem 1995; 270: 13652-13659 59 Colman A, Gamer I. The transgenic mammary gland as a bioreactor: Expectations and realizations. In: Proceedings of the 1995 Miami Bio/Technology Winter Symposium, Advances in Gene Technology, IRL Press at Oxford University Press, Miami Bio/Technology Short Reports. 1995. a 06. 107 60 van de Ven WJM, Roebroek AJM, van Duijnhoven HLP. Structure and function of eukaryotic proprotein processing enzymes of the subtilisin family of serine proteases. Crit Rev Oncog 1993; 04: 115-136 61 Colman A. Production of vitamin K-dependent proteins in the milk of transgenic mammals. In: Exploiting transgenic technology for commercial development. IBC Meeting San Diego, CA: 1995. a (abstract) 62 Lin SW, Smith KJ, Welsch D, Stafford DW. Expression and characterization of human factor IX and factor IX-factor X chimeras in mouse C127 cells. J Biol Chem 1990; 256: 144-150 63 Hatsuzawa K, Hosaka M, Nakagawa T, Nagase M, Shoda A, Murakami K, Nakayama K. Structure and expression of mouse furin, a yeast kex2-related protease. J Biol Chem 1990; 265: 22075-22078 64 Rehemtulla A, Roth DA, Wasley LC, Kuliopulos A, Walsh CT, Furie B, Furie BC, Kaufman RJ. In vitro and in vivo functional characterization of bovine vitamin K-dependent γ-carboxylase expressed in Chinese hamster ovary cells. Proc Natl Acad Sci USA 1993; 90: 4611-4615 65 Drews R, Paleyanda RK, Lee TK, Chang RR, Rehemtulla A, Kaufman RJ, Drohan WN, Lubon H. Proteolytic maturation of protein C upon engineering the mouse mammary gland to express furin. Proc Natl Acad Sci USA 1995; 92: 10462-10466
