Preloading potential of retroviral vectors is packaging cell clone dependent and centrifugation onto CH-296 ensures highest transduction efficiency

Human Gene Therapy

Volumn 20, Issue 4, 2009, Pages 337-349

  Sondergaard, Claus S ^a,b   Haldrup, Christa ^a   Beer, Christiane ^a   Andersen, Bente ^a   Kohn, Donald B ^c,d   Pedersen, Lene ^a,b  

^a AARHUS UNIVERSITY   (Denmark)

^b AARHUS UNIVERSITY HOSPITAL   (Denmark)

^c UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^d CHILDREN S HOSPITAL LOS ANGELES   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

FIBRONECTIN; PEPTIDE CH 296; RETROVIRUS VECTOR; UNCLASSIFIED DRUG;

ANIMAL CELL; ARTICLE; CELL CLONE; CELL LINE; CENTRIFUGATION; COMPLEX FORMATION; CONTROLLED STUDY; ENZYME LINKED IMMUNOSORBENT ASSAY; GENETIC TRANSDUCTION; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; MATERIAL COATING; MOLONEY LEUKEMIA ONCOVIRUS; NONHUMAN; PACKAGING CELL LINE; PROTEIN AGGREGATION; VIRAL GENE DELIVERY SYSTEM; VIRUS LOAD;

ANIMALS; CELL LINE; CENTRIFUGATION; CHROMATOGRAPHY, AFFINITY; CLONE CELLS; FIBRONECTINS; GENETIC VECTORS; HUMANS; MICE; RETROVIRIDAE; TIME FACTORS; TISSUE CULTURE TECHNIQUES; TRANSDUCTION, GENETIC; VIRION; VIRUS ASSEMBLY;

MOLONEY MURINE LEUKEMIA VIRUS;

EID: 64049106511     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2008.140     Document Type: Article

References (35)

1. Aiuti, A., Slavin, S., Aker, M., Ficara, F., Deola, S., Mortellaro, A., Morecki, S., Andolfi, G., Tabucchi, A., Carlucci, F., Marinello, E., Cattaneo, F., Vai, S., Servida, P., Miniero, R., Roncarolo, M.G., and Bordignon, C. (2002). Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296, 2410-2413.
2. Ayuk, F.A., Li, Z., Kühlcke, K., Lindemann, C., Schade, U.M., Ecker, H.-G., Zander, A.R., and Fehse, B. (1999). Establishment of an optimised gene transfer protocol for human primary T lymphocytes according to clinical requirements. Gene Ther. 6, 1788-1792.
3. Beer, C., and Pedersen, L. (2007). Matrix fibronectin binds gammaretrovirus and assists in entry: New light on viral infections. J. Virol. 81, 8247-8257.
4. Beer, C., Andersen, D.S., Rojek, A., and Pedersen, L. (2005). Caveola-dependent endocytic entry of amphotropic murine leukemia virus. J. Virol. 79, 10776-10787.
5. Bunnell, B.A., Muul, L.M., Donahue, R.E., Blaese, R.M., and Morgan, R.A. (1995). High-efficiency retroviral-mediated gene transfer into human and nonhuman primate peripheral blood lymphocytes. Proc. Natl. Acad. Sci. U.S.A. 92, 7739-7743.
6. Cavazzana-Calvo, M., Hacein-Bey, S., De Saint Basile, G., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J.L., Bousso, P., Deist, F.L., and Fischer, A. (2000). Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288, 669-672.
7. + cells. J. Gene Med. 3, 219-227.
8. Dao, M.A., Hashino, K., Kato, I., and Nolta, J.A. (1998). Adhesion to fibronectin maintains regenerative capacity during ex vivo culture and transduction of human hematopoietic stem and progenitor cells. Blood 92, 4612-4621.
9. Evans, L.H., Morrison, R.P., Malik, F.G., Portis, J., and Britt, W.J. (1990). A neutralizable epitope common to the envelope glycoproteins of ecotropic, polytropic, xenotropic, and amphotropic murine leukemia viruses. J. Virol. 64, 6176-6183.
10. Farrell, K.B., and Eiden, M.V. (2005). Dissection of gammaretroviral receptor function by using type III phosphate transporters as models. J. Virol. 79, 9332-9336.
11. Fehse, B. (2008). Clinical application of a retroviral gene transfer protocol based on centrifugation-mediated vector preloading. Hum. Gene Ther. 19, 655-656; author reply, 657-658.
12. + - enriched blood stem cells. Blood 104, 3408-3409.
13. Forestell, S.P., Bohnlein, E., and Rigg, R.J. (1995). Retroviral endpoint titer is not predictive of gene transfer efficiency: Implications for vector production. Gene Ther. 2, 723-730.
14. Gaspar, H.B., Parsley, K.L., Howe, S., King, D., Gilmour, K.C., Sinclair, J., Brouns, G., Schmidt, M., von Kalle, C., and Barington, T. (2004). Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 364, 2181-2187.
15. Hacein-Bey-Abina, S., Le Deist, F., Carlier, F., Bouneaud, C., Hue, C., De Villartay, J.P., Thrasher, A.J., Wulffraat, N., Sorensen, R., Dupuis-Girod, S., Fischer, A., Davies, E.G., Kuis, W., Leiva, L., and Cavazzana-Calvo, M. (2002). Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N. Engl. J. Med. 346, 1185-1193.
16. Halene, S., Wang, L., Cooper, R.M., Bockstoce, D.C., Robbins, P.B., and Kohn, D.B. (1999). Improved expression in hematopoietic and lymphoid cells in mice after transplantation of bone marrow transduced with a modified retroviral vector. Blood 94, 3349-3357.
17. Hanenberg, H., Xiao, X.L., Dilloo, D., Hashino, K., Kato, I., and Williams, D.A. (1996). Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells. Nat. Med. 2, 876-882.
18. + hematopoietic cells. Hum. Gene Ther. 8, 2193-2206.
19. Hughes, C., Galea-Lauri, J., Farzaneh, F., and Darling, D. (2001). Streptavidin paramagnetic particles provide a choice of three affinity-based capture and magnetic concentration strategies for retroviral vectors. Mol. Ther. 3, 623-630.
20. Kotani, H., Newton, P.B., III, Zhang, S., Chiang, Y.L., Otto, E., Weaver, L., Blaese, R.M., Anderson, W.F., and McGarrity, G.J. (1994). Improved methods of retroviral vector transduction and production for gene therapy. Hum. Gene Ther. 5, 19-28.
21. Kühlcke, K., Fehse, B., Schilz, A., Loges, S., Lindemann, C., Ayuk, F., Lehmann, F., Stute, N., Fauser, A.A., Zander, A.R., and Eckert, H.G. (2002). Highly efficient retroviral gene transfer based on centrifugation-mediated vector preloading of tissue culture vessels. Mol. Ther. 5, 473-478.
22. Kustikova, O.S., Wahlers, A., Kühlcke, K., Stahle, B., Zander, A.R., Baum, C., and Fehse, B. (2003). Dose finding with retroviral vectors: Correlation of retroviral vector copy numbers in single cells with gene transfer efficiency in a cell population. Blood 102, 3934-3937.
23. Miller, A.D., and Buttimore, C. (1986). Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production. Mol. Cell. Biol. 6, 2895-2902.
24. Miller, A.D., and Chen, F. (1996). Retrovirus packaging cells based on 10A1 murine leukemia virus for production of vectors that use multiple receptors for cell entry. J. Virol. 70, 5564-5571.
25. Miller, A.D., and Rosman, G.J. (1989). Improved retroviral vectors for gene transfer and expression. Biotechniques 7, 989-990.
26. Miller, A.D., Garcia, J.V., von Suhr, N., Lynch, C.M., Wilson, C., and Eiden, M.V. (1991). Construction and properties of retrovirus packaging cells based on gibbon ape leukemia virus. J. Virol. 65, 2220-2224.
27. Miller, D.G., Adam, M.A., and Miller, A.D. (1990). Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol. Cell. Biol. 10, 4239-4242.
28. Ott, M.G., Schmidt, M., Schwarzwaelder, K., Stein, S., Siler, U., Koehl, U., Glimm, H., Kühlcke, K., Schilz, A., Kunkel, H., Naundorf, S., Brinkmann, A., Deichmann, A., Fischer, M., Ball, C., Pilz, I., Dunbar, C., Du, Y., Jenkins, N.A., Copeland, N.G., Luthi, U., Hassan, M., Thrasher, A.J., Hoelzer, D., von Kalle, C., Seger, R., and Grez, M. (2006). Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 12, 401-409.
29. Pedersen, L., Johann, S.V., van Zeijl, M., Pedersen, F.S., and O'Hara, B. (1995). Chimeras of receptors for gibbon ape leukemia virus/feline leukemia virus B and amphotropic murine leukemia virus reveal different modes of receptor recognition by retrovirus. J. Virol. 69, 2401-2405.
30. Quintás-Cardama, A., Yeh, R.K., Hollyman, D., Stefanski, J., Taylor, C., Nikhamin, Y., Imperato, G., Sadelain, M., Riviere, I., and Brentjens, R.J. (2007). Multifactorial optimization of gammaretroviral gene transfer into human T lymphocytes for clinical application. Hum. Gene Ther. 18, 1253-1260.
31. Robbins, P.B., Yu, X.J., Skelton, D.M., Pepper, K.A., Wasserman, R.M., Zhu, L., and Kohn, D.B. (1997). Increased probability of expression from modified retroviral vectors in embryonal stem cells and embryonal carcinoma cells. J. Virol. 71, 9466-9474.
32. Robbins, P.B., Skelton, D.C., Yu, X.J., Halene, S., Leonard, E.H., and Kohn, D.B. (1998). Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells. Proc. Natl. Acad. Sci. U.S.A. 95, 10182-10187.
33. Soneoka, Y., Cannon, P.M., Ramsdale, E.E., Griffiths, J.C., Romano, G., Kingsman, S.M., and Kingsman, A.J. (1995). A transient three-plasmid expression system for the production of high titer retroviral vectors. Nucleic Acids Res. 23, 628-633.
34. + T lymphocytes by MuLV-10A1 retrovirus pseudotype. Hum. Gene Ther. 11, 1005-1014.
35. van Lunzen, J., Glaunsinger, T., Stahmer, I., von Baehr, V., Baum, C., Schilz, A., Kuehlcke, K., Naundorf, S., Martinius, H., Hermann, F., Giroglou, T., Newrzela, S., Muller, I., Brauer, F., Brandenburg, G., Alexandrov, A., and von Laer, D. (2007). Transfer of autologous gene-modified T cells in HIV-infected patients with advanced immunodeficiency and drug-resistant virus. Mol. Ther. 15, 1024-1033.