Acknowledgement
Supported by : Korea University, Korea Hemophilia Foundation
References
- Burns, J., Friedmann, T., Driever, W., Burrascano, M., and Yee, J.-K. (1993) Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells. Proc. Natl. Acad. Sci. USA 90, 8033-8037
- Chang, L. J. and He, J. (2001) Retroviral vectors for gene therapy of AIDS and cancer. Curr. Opin. Mol. Ther. 3. 468-475
- Chuah, M. K., Collen, D., and VandenDriessche, T. (2004) Clinical gene transfer studies for hemophilia A. Semin. Thromb. Hemost. 30, 249-256
- De Meyer, D. F., Vanhoorelbeke, K., Chuah, M. K., Pareyn, I., Gillijns, V., et al. (2006) Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor. Blood 107, 4728-4736 https://doi.org/10.1182/blood-2005-09-3605
- Doering, C., Parker, E. T., Healey, J. F., Craddock, H. N., Barrow, R. T., et al. (2002) Expression and characterization of recombinant murine factor VIII. Thromb. Haemost. 88, 450-458 https://doi.org/10.1055/s-0037-1613237
- Eum, W. S., Jang, S. H., Kim, D. W., Choi, H. S., Choi, S. H., et al. (2005) Enhanced transduction of Cu, Zn-superoxide dismutase with HIV-1 Tat protein transduction domains at both termini. Mol. Cells 19, 191-197
- Fay, P. J., Coumans, J. V., and Walker, F. J. (1991) von Willebrand factor mediates protection of factor VIII from activated protein C-catalyzed inactivation. J. Biol. Chem. 266, 2172-2177
- Federici, A. B. (2003) The factor VIII/von Willebrand factor complex: basic and clinical issues. Haematologica 88, EREP02
- Gangadharan, B., Parker, E. T., Ide, L. M., Spencer, H. T., and Doering, C. B. (2006) High-level expression of porcine factor VIII from genetically modified bone marrow-derived stem cells. Blood 107, 3859-3864 https://doi.org/10.1182/blood-2005-12-4961
- Hofmann, W., Schubert, D., LaBonte, J., Munson, L., Gibson, S., et al. (1999) Species-specific, postentry barriers to primate immunodeficiency virus infection. J. Virol. 12, 10010-10018
- Kaufman, R. J., Pipe, S. W., Tagliavacca, L., Swaroop, M., and Moussalli, M. (1997) Biosynthesis, assembly and secretion of coagulation factor VIII. Blood 8, S3-14
- Kaufman, R. J., Wasley, L. C., and Dorner, A. J. (1988) Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells. J. Biol. Chem. 263, 6352-6362
- Keeney, S. and Cumming, A. M. (2001) The molecular biology of von Willebrand disease. Clin. Lab. Haematol. 23, 209-230 https://doi.org/10.1046/j.1365-2257.2001.00400.x
- Kohn, D. (2001) Gene therapy for genetic haematological disorders and immunodeficiencies. J. Intern. Med. 249, 379-390 https://doi.org/10.1046/j.1365-2796.2001.00803.x
- Lewis, P., Hensel, M., and Emerman, M. (1992) Human immunodeficiency virus infection of cells arrested in the cell cycle. EMBO J. 11, 3053-3058
- Naldini, L., Blomer, U., Gallay, P., Ory, D., Mulligan, R. et al. (1996) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263-269 https://doi.org/10.1126/science.272.5259.263
- Over, J., Sixma, J. J., Bruine, M. H., Trieschnigg, M. C., Vlooswijk, R. A., et al. (1978) Survival of 125iodine-labeled Factor VIII in normals and patients with classic hemophilia. Observations on the heterogeneity of human Factor VIII. J. Clin. Invest. 62, 223-234
- Park, S. W. and Choi, S. Y. (2004) A stable gene transfer system for hematopoietic progenitor cells from human bone marrow using pseudotyped retroviral vectors. Mol. Cells 17, 297-303
- Parolin, C., Taddeo, B., Palu, G., and Sodroski, J. (1996) Use of cis- and trans-acting viral regulatory sequences to improve expression of human immunodeficiency virus vectors in human lymphocytes. Virology 222, 415-422 https://doi.org/10.1006/viro.1996.0438
- Rosenberg, J. B., Foster, P. A., Kaufman, R. J., Vokac, E. A., Moussalli, M., et al. (1998) Intracellular trafficking of Factor VIII to von Willebrand factor storage granules. J. Clin. Invest. 101, 613-624 https://doi.org/10.1172/JCI1250
- Powell, J. S., Ragni, M. V., White, G. C. Lusher, J. M., Hillman- Wiseman, C., et al. (2003) Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood 102, 2038-2045 https://doi.org/10.1182/blood-2003-01-0167
- Sadler, J. E. (1998) Biochemistry and genetics of von Willebrand factor. Annu. Rev. Biochem. 67, 395-424 https://doi.org/10.1146/annurev.biochem.67.1.395
- Sadler, J. E. and Davie, E. W. (1994) In the molecular basis of blood disease, Stamatoyannopoulos, G., Nienhuis, A. W., Majerus, P. W., and Varmus, H. (eds.), pp. 657-700, Philadelphia, WB Saunders
- Tuddenham, E. G., Lane, R. S., Rotblat, F., Johnson, A. J., Snape, T. J., et al. (1982) Response to infusions of polyelectrolyte fractionated human factor VIII concentrate in human haemophilia A and von Willebrand's disease. Br. J. Haematol. 52, 259-267 https://doi.org/10.1111/j.1365-2141.1982.tb03888.x
- Yee, J., Miyanohara, A., LaPorte, P., Bouic, K., Burns, J., et al. (1994) A general method for the generation of high-titer, pantropic retroviral vectors: highly efficient infection of primary hepatocytes. Proc. Natl. Acad. Sci. USA 91, 9564-9568
- Zennou, V., Petit, C., Guetard, D., Nerhbass, U., Montagnier, L., et al. (2000) HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 101, 173-185 https://doi.org/10.1016/S0092-8674(00)80828-4