Rapid generation of stable cell lines expressing high levels of erythropoietin, factor VIII, and an antihuman CD20 antibody using lentiviral vectors

Human Gene Therapy Methods

Volumn 24, Issue 4, 2013, Pages 214-227

  Baranyi, Lajos ^a   Doering, Christopher B ^b   Denning, Gabriella ^c   Gautney, Richard E ^c   Harris, Kyle T ^c   Trent Spencer, H ^b   Roy, Andre ^a   Zayed, Hatem ^a   Dropulic, Boro ^a  

^a Lentigen Corporation   (United States)

^b EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c Expression Therapeutics   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CD20 ANTIBODY; ERYTHROPOIETIN; LENTIVIRUS VECTOR; RECOMBINANT BLOOD CLOTTING FACTOR 8;

AMINO ACID SEQUENCE; ARTICLE; CELL CLONE; CELL LINE; FLUORESCENCE IN SITU HYBRIDIZATION; GENE AMPLIFICATION; GENE ISOLATION; GENETIC SCREENING; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN GLYCOSYLATION; STABLE TRANSFECTION; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; ANTIBODIES, MONOCLONAL, MURINE-DERIVED; CHO CELLS; CRICETINAE; CRICETULUS; ERYTHROPOIETIN; FACTOR VIII; GENETIC VECTORS; HEK293 CELLS; HUMANS; LENTIVIRUS; TRANSFECTION; TRANSGENES;

EID: 84883042346     PISSN: 19466536     EISSN: 19466544     Source Type: Journal    
DOI: 10.1089/hgtb.2013.002     Document Type: Article

References (45)

1. Bebbington, C.R., Renner, G., Thomson, S., et al. (1992). Highlevel expression of a recombinant antibody from myeloma cells using a glutamine synthetase gene as an amplifiable selectable marker. Biotechnology 10, 169-175.
2. Beyer, T., Lohse, S., Berger, S., et al. (2009). Serum-free production and purification of chimeric IgA antibodies. J. Immunol. Methods 346, 26-37.
3. Boissel, J.P., Lee, W.R., Presnell, S.R., et al. (1993). Erythropoietin structure-function relationships. Mutant proteins that test a model of tertiary structure. J. Biol. Chem. 268, 15983-15993. (Pubitemid 23222105)
4. Butler, M. (2006). Optimisation of the cellular metabolism of glycosylation for recombinant proteins produced by mammalian cell systems. Cytotechnology 50, 57-76.
5. Cumming, D.A. (1991). Structural heterogeneity and functional differentiation: a rationale for glycosylation analysis of recombinant therapeutics. Semin. Cell. Biol. 2, 273-279.
6. Delorme, E., Lorenzini, T., Giffin, J., et al. (1992). Role of glycosylation on the secretion and biological activity of erythropoietin. Biochemistry 31, 9871-9876.
7. Doering, C.B., Healey, J.F., Parker, E.T., et al. (2002). High level expression of recombinant porcine coagulation factor VIII. J. Biol. Chem. 277, 38345-38349. (Pubitemid 35154689)
8. Dropulic, B. (2011). Lentiviral vectors: their molecular design, safety, and use in laboratory and preclinical research. Hum. Gene Ther. 22, 649-657.
9. Dunker, A.K., Oldfield, C.J., Meng, J., et al. (2008). The unfoldomics decade: an update on intrinsically disordered proteins. BMC Genomics 9 Suppl 2, S1.
10. Fang, J., Yi, S., Simmons, A., et al. (2007). An antibody delivery system for regulated expression of therapeutic levels of monoclonal antibodies in vivo. Mol. Ther. 15, 1153-1159. (Pubitemid 46813618)
11. Flesher, A.R., Marzowski, J., Wang, W.C., and Raff, H.V. (1995). Fluorophore-labeled carbohydrate analysis of immunoglobulin fusion proteins: correlation of oligosaccharide content with in vivo clearance profile. Biotechnol. Bioeng. 47, 405.
12. Freire, T., Bay, S., Vichier-Guerre, S., et al. (2006). Carbohydrate antigens: synthesis aspects and immunological applications in cancer. Mini Rev. Med. Chem. 6, 1357-1373. (Pubitemid 44890543)
13. Funk, W.D., MacGillivray, R.T., Mason, A.B., et al. (1990). Expression of the amino-terminal half-molecule of human serum transferrin in cultured cells and characterization of the recombinant protein. Biochemistry 29, 1654-1660. (Pubitemid 20063402)
14. Grillberger, L., Kreil, T.R., Nasr, S., and Reiter, M. (2009). Emerging trends in plasma-free manufacturing of recombinant protein therapeutics expressed in mammalian cells. Biotechnol. J. 4, 186-201.
15. Grund, N., Maier, P., Giordano, F.A., et al. (2010). Analysis of self-inactivating lentiviral vector integration sites and flanking gene expression in human peripheral blood progenitor cells after alkylator chemotherapy. Hum. Gene Ther. 21, 943-956.
16. Heinrich, H.W. (1990). Recombinant virus vaccines-present status and future prospects. Arch. Exp. Veterinarmed.44, 849-853.
17. Hooker, A., and James, D. (1998). The glycosylation heterogeneity of recombinant human IFN-gamma. J. Interferon Cytokine Res. 18, 287-295. (Pubitemid 28226173)
18. Jenkins, N. (1995). Monitoring and control of recombinant glycoprotein heterogeneity in animal cell cultures. Biochem. Soc. Trans. 23, 171-175.
19. Jenkins, N., Murphy, L., and Tyther, R. (2008). Post-translational modifications of recombinant proteins: significance for biopharmaceuticals. Mol. Biotechnol. 39, 113-118.
20. Joung, J., Robertson, J.S., Griffiths, E., and Knezevic, I. (2008). WHO informal consultation on regulatory evaluation of therapeutic biological medicinal products held at WHO Headquarters, Geneva, 19-20 April 2007. Biologicals 36, 269-276.
21. Jurado Garcia, J.M., Torres, S.E., Olmos, H.D., and Alba, C.E. (2007). Erythropoietin pharmacology. Clin. Transl. Oncol. 9, 715-722.
22. Kanda, Y., Yamane-Ohnuki, N., Sakai, N., et al. (2006). Comparison of cell lines for stable production of fucose-negative antibodies with enhanced ADCC. Biotechnol. Bioeng. 94, 680-688. (Pubitemid 44014199)
23. Kelley, B. (2009). Industrialization of mAb production technology: the bioprocessing industry at a crossroads. MAbs 1, 443-452.
24. Kresse, G.B. (2009). Biosimilars-science, status, and strategic perspective. Eur. J. Pharm. Biopharm. 72, 479-486.
25. Larrick, J.W., and Thomas, D.W. (2001). Producing proteins in transgenic plants and animals. Curr. Opin. Biotechnol. 12, 411-418. (Pubitemid 32718797)
26. Laubach, V.E., Garvey, E.P., and Sherman, P.A. (1996). Highlevel expression of human inducible nitric oxide synthase in Chinese hamster ovary cells and characterization of the purified enzyme. Biochem. Biophys. Res. Commun. 218, 802-807. (Pubitemid 26050744)
27. Lifely, M.R., Hale, C., Boyce, S., et al. (1995). Glycosylation and biological activity of CAMPATH-1H expressed in different cell lines and grown under different culture conditions. Glycobiology 5, 813-822. (Pubitemid 26021983)
28. Liu, T., Li, J., Zeng, R., et al. (2009). Capillary electrophoresiselectrospray mass spectrometry for characterisation of hihg-mannose-type N-glycosylation and differential oxidation in glycoproteins by charge differential oxidation and protease-glycosidase digestion. Anal. Chem. 73, 5875-5885.
29. Llop, E., Gutierrez-Gallego, R., Segura, J., et al. (2008). Structural analysis of the glycosylation of gene-activated erythropoietin (epoetin delta, Dynepo). Anal. Biochem. 383, 243-254.
30. Lusher, J.M., Arkin, S., Abildgaard, C.F., and Schwartz, R.S. (1993). Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. Kogenate Previously Untreated Patient Study Group. N. Engl. J. Med. 328, 453-459. (Pubitemid 23056434)
31. Macdougall, I.C., and Ashenden, M. (2009). Current and upcoming erythropoiesis-stimulating agents, iron products, and other novel anemia medications. Adv. Chronic Kidney Dis. 16, 117-130.
32. Maiese, K., Chong, Z.Z., and Shang, Y.C. (2008). Raves and risks for erythropoietin. Cytokine Growth Factor Rev. 19, 145-155.
33. Naldini L, Blömer U, Gallay P, Ory D, Mulligan R, Gage FH, Verma IM, Trono D. (1996). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science. 272, 263-267. (Pubitemid 26119138)
34. O'Connell, B.C., and Tabak, L.A. (1993). A comparison of serine and threonine O-glycosylation by UDP-GalNAc:polypeptide N- acetylgalactosaminyltransferase. J. Dent. Res. 72, 1554-1558.
35. Robinson, D.K., Chan, C.P., Yu, L.C., et al. (1994). Characterization of a recombinant antibody produced in the course of a high yield fed-batch process. Biotechnol. Bioeng. 44, 727-735. (Pubitemid 24277100)
36. Ronco, C. (2008). Is the advent of biosimilars affecting the practice of nephrology and the safety of patients? Contrib. Nephrol. 161, 261-270.
37. Schellekens, H. (2008). Recombinant human erythropoietins, biosimilars and immunogenicity. J. Nephrol. 21, 497-502.
38. Schmidt, F.R. (2004). Recombinant expression systems in the pharmaceutical industry. Appl. Microbiol. Biotechnol. 65, 363-372. (Pubitemid 39281088)
39. Schroder, A.R., Shinn, P., Chen, H., et al. (2002). HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110, 521-529. (Pubitemid 35232295)
40. Simerska, P., Moyle, P.M., and Toth, I. (2009). Modern lipid-, carbohydrate-, and peptide-based delivery systems for peptide, vaccine, and gene products. Med. Res. Rev. 31, 520-547.
41. Wandinger, S.K., Richter, K., and Buchner, J. (2008). The Hsp90 chaperone machinery. J. Biol. Chem. 283, 18473-18477.
42. Wurm, F.M. (2004). Production of recombinant protein therapeutics in cultivated mammalian cells. Nat. Biotechnol. 22, 1393-1398. (Pubitemid 39482858)
43. Wurm, F.M., Pallavicini, M.G., and Arathoon, R. (1992). Integration and stability of CHO amplicons containing plasmid sequences. Dev. Biol. Stand. 76, 69-82.
44. Yuen, C.T., Storring, P.L., Tiplady, R.J., et al. (2003). Relationships between the N-glycan structures and biological activities of recombinant human erythropoietins produced using different culture conditions and purification procedures. Br. J. Haematol. 121, 511-526. (Pubitemid 36628347)
45. Zheng, Y., Guo, Z., and Cai, Z. (2009). Combination of betaelimination and liquid chromatography/quadrupole time-offlight mass spectrometry for the determination of O-glycosylation sites. Talanta 78, 358-363.