Establishment of embryonic stem cells secreting human factor VIII for cell-based treatment of hemophilia A

Journal of Thrombosis and Haemostasis

Volumn 6, Issue 8, 2008, Pages 1352-1359

  Kasuda, S ^a,b   Kubo, Atsushi ^a   Sakurai, Y ^a   Irion, S ^c   Ohashi, K ^a   Tatsumi, K ^a   Nakajima, Y ^a   Saito, Y ^a   Hatake, K ^a   Pipe, S W ^d   Shima, M ^a   Yoshioka, A ^a  

^a NARA MEDICAL UNIVERSITY   (Japan)

^b HYOGO COLLEGE OF MEDICINE   (Japan)

^c MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^d UNIVERSITY OF MICHIGAN HEALTH SYSTEM   (United States)

Author keywords

Cell based therapy; Embryonic stem cells; Factor VIII; Hemophilia A

Indexed keywords

BLOOD CLOTTING FACTOR 8; DNA; DOXYCYCLINE; GREEN FLUORESCENT PROTEIN; STEM CELL FACTOR RECEPTOR; TETRACYCLINE;

ANTICOAGULATION; ARTICLE; CELL CULTURE; CELL DIFFERENTIATION; CELL LINE; CELL SELECTION; CONTROLLED STUDY; EMBRYO; EMBRYONIC STEM CELL; ENDODERM; GENE EXPRESSION; GENE INDUCTION; HEMOPHILIA A; HUMAN; HUMAN CELL; PRIORITY JOURNAL; PROTEIN SECRETION; STEM CELL GENE THERAPY;

CELL CULTURE TECHNIQUES; CELL DIFFERENTIATION; DOXYCYCLINE; EMBRYONIC STEM CELLS; FACTOR VIII; GENE EXPRESSION; GENETIC VARIATION; GREEN FLUORESCENT PROTEINS; HEMOPHILIA A; HUMANS; RECOMBINANT PROTEINS; RNA, MESSENGER;

EID: 47649083489     PISSN: 15387933     EISSN: 15387836     Source Type: Journal    
DOI: 10.1111/j.1538-7836.2008.03022.x     Document Type: Article

References (28)

1. Antonarakis SE. Molecular genetics of coagulation factor VIII gene and haemophilia A. Haemophilia 1998; 4(Suppl. 2): 1-11.
2. Manco-Johnson M. Hemophilia management: Optimizing treatment based on patient needs. Curr Opin Pediatr 2005; 17: 3-6.
3. Lillicrap D, Vandendriessche T, High K. Cellular and genetic therapies for haemophilia. Haemophilia 2006; 12(Suppl. 3): 36-41.
4. Sarkar R, Tetreault R, Gao G, Wang L, Bell P, Chandler R, Wilson JM, Kazazian HH Jr. Total correction of hemophilia.A mice with canine FVIII using an AAV. 8 serotype. Blood 2004; 103: 1253-60.
5. Kaiser J. Gene therapy. Side effects sideline hemophilia trial. Science 2004; 304: 1423-5.
6. Schuettrumpf J, Liu JH, Couto LB, Addya K, Leonard DG, Zhen Z, Sommer J, Arruda VR. Inadvertent germline transmission of AAV2 vector: Findings in a rabbit model correlate with those in a human clinical trial. Mol Ther 2006; 13: 1064-73.
7. Ko S, Tanaka I, Kanehiro H, Kanokogi H, Ori J, Shima M, Yoshioka A, Giles A, Nakajima Y. Preclinical experiment of auxiliary partial orthotopic liver transplantation as a curative treatment for hemophilia. Liver Transpl 2005; 11: 579-84.
8. Ohashi K, Waugh JM, Dake MD, Yokoyama T, Kuge H, Nakajima Y, Yamanouchi M, Naka H, Yoshioka A, Kay MA. Liver tissue engineering at extrahepatic sites in mice as a potential new therapy for genetic liver diseases. Hepatology 2005; 41: 132-40.
9. Kumaran V, Benten D, Follenzi A, Joseph B, Sarkar R, Gupta S. Transplantation of endothelial cells corrects the phenotype in hemophilia A mice. J Thromb Haemost 2005; 3: 2022-31.
10. Kyba M, Perlingeiro RC, Daley GQ. HoxB4 confers definitive lymphoid-myeloid engraftment potential on embryonic stem cell and yolk sac hematopoietic progenitors. Cell 2002; 109: 29-37.
11. Kubo A, Chen V, Kennedy M, Zahradka E, Daley GQ, Keller G. The homeobox gene HEX regulates proliferation and differentiation of hemangioblasts and endothelial cells during ES cell differentiation. Blood 2005; 105: 4590-7.
12. Shibata M, Shima M, Morichika S, McVey J, Tuddenham EG, Tanaka I, Suzuki H, Nogami K, Minamoto Y, Hato T, Saenko EL, Scandella D, Yoshioka A. An alloantibody recognizing the FVIII A1 domain in a patient with CRM reduced haemophilia A due to deletion of a large portion of the A1 domain DNA sequence. Thromb Haemost 2000; 84: 442-8.
13. Miao HZ, Sirachainan N, Palmer L, Kucab P, Cunningham MA, Kaufman RJ, Pipe SW. Bioengineering of coagulation factor VIII for improved secretion. Blood 2004; 103: 3412-19.
14. Fehling HJ, Lacaud G, Kubo A, Kennedy M, Robertson S, Keller G, Kouskoff V. Tracking mesoderm induction and its specification to the hemangioblast during embryonic stem cell differentiation. Development 2003; 130: 4217-27.
15. Kubo A, Shinozaki K, Shannon JM, Kouskoff V, Kennedy M, Woo S, Fehling HJ, Keller G. Development of definitive endoderm from embryonic stem cells in culture. Development 2004; 131: 1651-62.
16. Kaufman RJ, Wasley LC, Dorner AJ. Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells. J Biol Chem 1988; 263: 6352-62.
17. Tada S, Era T, Furusawa C, Sakurai H, Nishikawa S, Kinoshita M, Nakao K, Chiba T, Nishikawa S. Characterization of mesendoderm: A diverging point of the definitive endoderm and mesoderm in embryonic stem cell differentiation culture. Development 2005; 132: 4363-74.
18. 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: 2925-35.
19. Toole JJ, Pittman DD, Orr EC, Murtha P, Wasley LC, Kaufman RJ. A large region (approximately equal to 95kDa) of human factor VIII is dispensable for in vitro procoagulant activity. Proc Natl Acad Sci USA 1986; 83: 5939-42.
20. Gouon-Evans V, Boussemart L, Gadue P, Nierhoff D, Koehler CI, Kubo A, Shafritz DA, Keller G. BMP-4 is required for hepatic specification of mouse embryonic stem cell-derived definitive endoderm. Nat Biotechnol 2006; 24: 1402-11.
21. Barbera JPM, Clements M, Thomas P, Rodriguez T, Meloy D, Kioussis D, Beddington RSP. The homeobox gene Hex is required in definitive endoderm tissues for normal forebrain, liver and thyroid formation. Development 2000; 127: 2433-45.
22. Pittman DD, Marquette KA, Kaufman RJ. Role of the B domain for factor VIII and factor V expression and function. Blood 1994; 84: 4214-25.
23. Pipe SW, Morris JA, Shah J, Kaufman RJ. Differential interaction of coagulation factor VIII and factor V with protein chaperones calnexin and calreticulin. J Biol Chem 1998; 273: 8537-44.
24. Shi Q, Wilcox DA, Fahs SA, Fang J, Johnson BD, Du LM, Desai D, Montgomery RR. Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A. J Thromb Haemost 2007; 5: 352-61.
25. Pipe SW, Saint-Remy JM, Walsh CE. New high-technology products for the treatment of haemophilia. Haemophilia 2004; 10(Suppl. 4): 55-63.
26. Hubbard AR, Bevan SA, Weller LJ. Potency estimation of recombinant factor VIII: Effect of assay method and standard. Br J Haematol 2001; 113: 533-6.
27. Fujikawa T, Oh SH, Pi L, Hatch HM, Shupe T, Petersen BE. Teratoma formation leads to failure of treatment for type I diabetes using embryonic stem cell-derived insulin-producing cells. Am J Pathol 2005; 166: 1781-91.
28. Kolossov E, Bostani T, Roell W, Breitbach M, Pillekamp F, Nygren JM, Sasse P, Rubenchik O, Fries JW, Wenzel D, Geisen C, Xia Y, Lu Z, Duan Y, Kettenhofen R, Jovinge S, Bloch W, Bohlen H, Welz A, Hescheler J, et.al. Engraftment of engineered ES cell-derived cardiomyocytes but not BM cells restores contractile function to the infarcted myocardium. J Exp Med 2006; 203: 2315-27.