Large-scale production of lentiviral vector in a closed system hollow fiber bioreactor

Molecular Therapy Methods and Clinical Development

Volumn 2, Issue , 2015, Pages 15020-

  Sheu, Jonathan ^a   Beltzer, Jim ^b   Fury, Brian ^a   Wilczek, Katarzyna ^a   Tobin, Steve ^a   Falconer, Danny ^a   Nolta, Jan ^a   Bauer, Gerhard ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b Terumo BCT Inc ^*  (United States)

Author keywords

[No Author keywords available]

Indexed keywords

LENTIVIRUS VECTOR; REAGENT; RNA; ADENOSINE TRIPHOSPHATASE (CALCIUM MAGNESIUM);

ARTICLE; CELL EXPANSION; CELL GROWTH; CONTROLLED STUDY; GENE THERAPY; GENETIC TRANSFECTION; HOLLOW FIBER REACTOR; HUMAN; HUMAN CELL; INDUCED PLURIPOTENT STEM CELL; LARGE SCALE PRODUCTION; NONHUMAN; PRIORITY JOURNAL; DOWNSTREAM PROCESSING; GENE TARGETING; HEK293 CELL LINE; HUMAN CELL CULTURE;

EID: 85015226333     PISSN: None     EISSN: 23290501     Source Type: Journal    
DOI: 10.1038/mtm.2015.20     Document Type: Article

References (31)

1. 1 Reiffers, J, Bernard, P, David, B, Vezon, G, Sarrat, A, Marit, G, et al. Successful autologous transplantation with peripheral blood hemopoietic cells in a patient with acute leukemia. Exp Hematol 14 (1986), 312–315.
2. 2 Kessinger, A, Armitage, JO, Landmark, JD, Smith, DM, Weisenburger, DD, Autologous peripheral hematopoietic stem cell transplantation restores hematopoietic function following marrow ablative therapy. Blood 71 (1988), 723–727.
3. 3 Buckley, RH, Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Eng J Med 340 (1999), 508–516.
4. 4 Rosenberg, SA, Aebersold, P, Cornetta, K, Kasid, A, Morgan, RA, Moen, R, et al. Gene transfer into humans—immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Eng J Med 323 (1990), 570–578.
5. 5 Blaese, RM, Culver, KW, Miller, AD, Carter, CS, Fleisher, T, Clerici, M, et al. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science 270 (1995), 475–480.
6. 6 Felgner, PL, Gadek, TR, Holm, M, Roman, R, Chan, HW, Wenz, M, et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 84 (1987), 7413–7417.
7. 7 Van Tendeloo, VF, Ponsaerts, P, Lardon, F, Nijs, G, Lenjou, M, Van Broeckhoven, C, et al. Highly efficient gene delivery by mRNA electroporation in human hematopoietic cells: superiority to lipofection and passive pulsing of mRNA and to electroporation of plasmid cDNA for tumor antigen loading of dendritic cells. Blood 98 (2001), 49–56.
8. 8 Tsurumi, Y, Takeshita, S, Chen, D, Kearney, M, Rossow, ST, Passeri, J, et al. Direct intramuscular gene transfer of naked DNA encoding vascular endothelial growth factor augments collateral development and tissue perfusion. Circulation 94 (1996), 3281–3290.
9. 9 Edelstein, ML, Mohammad, RA, Jo, W, Gene therapy clinical trials worldwide to 2007—an update. J Gene Med 9 (2007), 833–842.
10. 10 Bordignon, C, Yu, SF, Smith, CA, Hantzopoulos, P, Ungers, GE, Keever, CA, et al. Retroviral vector-mediated high-efficiency expression of adenosine deaminase (ADA) in hematopoietic long-term cultures of ADA-deficient marrow cells. Proc Natl Acad Sci USA 86 (1989), 6748–6752.
11. 11 Kohli, M, Rago, C, Lengauer, C, Kinzler, KW, Vogelstein, B, Facile methods for generating human somatic cell gene knockouts using recombinant adeno associated viruses. Nucleic Acids Res, 32, 2004, e3.
12. 12 Candotti, F, Shaw, KL, Muul, L, Carbonaro, D, Sokolic, R, Choi, C, et al. Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood 120 (2012), 3635–3646.
13. 13 Hacein-Bey-Abina, S, Pai, SY, Gaspar, HB, Armant, M, Berry, CC, Blanche, S, et al. “A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency.”. N Eng J Med 371 (2014), 1407–1417.
14. 14 Takahashi, K, Shinya, Y, Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 126 (2006), 663–676.
15. 15 Smith, AG, Culture and differentiation of embryonic stem cells. J Tissue Culture Methods 13 (1991), 89–94.
16. 16 Byrne, JA, Simonsson, S, Western, PS, Gurdon, JB, Nuclei of adult mammalian somatic cells are directly reprogrammed to oct-4 stem cell gene expression by amphibian oocytes. Current Biol 13 (2003), 1206–1213.
17. 17 Chin, MH, Mason, MJ, Xie, W, Volinia, S, Singer, M, Peterson, C, et al. Induced pluripotent stem cells and embryonic stem cells are distinguished by gene expression signatures. Cell Stem Cell 5 (2009), 111–123.
18. 18 Kim, K, Doi, A, Wen, B, Ng, K, Zhao, R, Cahan, P, et al. Epigenetic memory in induced pluripotent stem cells. Nature 467 (2010), 285–290.
19. 19 Raya, A, Rodríguez-Pizà, I, Guenechea, G, Vassena, R, Navarro, S, Barrero, MJ, et al. Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells. Nature 460 (2009), 53–59.
20. 20 Yusa, K, Rashid, ST, Strick-Marchand, H, Varela, I, Liu, PQ, Paschon, DE, et al. Targeted gene correction of [agr] 1-antitrypsin deficiency in induced pluripotent stem cells. Nature 478 (2011), 391–394.
21. 21 Zhao, J, Lever, AM, Lentivirus-mediated gene expression. Methods Mol Biol 366 (2007), 343–355.
22. 22 Grieger, JC, Richard, JS, “Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps.”. J Virol 79 (2005), 9933–9944.
23. 23 Wu, Z, Hongyan, Y, Peter, C, “Effect of genome size on AAV vector packaging.”. Mol Ther 18 (2010), 80–86.
24. 24 Sauer, B, Site-specific DNA recombination in mammalian cells by the Cre recombinase of bacteriophage P1. Proc Natl Acad Sci USA 85 (1988), 5166–5170.
25. 25 Sebastiano, V, Zhen, HH, Haddad, B, Bashkirova, E, Melo, SP, Wang, P, et al. Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa. Sci Transl Med, 6, 2014, 264ra163.
26. 26 Hanley, PJ, Mei, Z, Durett, AG, Cabreira-Harrison Mda, G, Klis, M, Li, W, et al. Efficient manufacturing of therapeutic mesenchymal stromal cells with the use of the Quantum Cell Expansion System. Cytotherapy 16 (2014), 1048–1058.
27. 27 Roberts, I, Baila, S, Rice, RB, Janssens, ME, Nguyen, K, Moens, N, et al. Scale-up of human embryonic stem cell culture using a hollow fibre bioreactor. Biotechnol Lett 34 (2012), 2307–2315.
28. 28 Hassell, T, Gleave, S, Butler, M, Growth inhibition in animal cell culture. Appl Biochem Biotechnol 30 (1991), 29–41.
29. 29 Merten, OW, Charrier, S, Laroudie, N, Fauchille, S, Dugué, C, Jenny, C, et al. Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application. Hum Gene Ther 22 (2010), 343–356.
30. 30 Ausubel, LJ, Hall, C, Sharma, A, Shakeley, R, Lopez, P, Quezada, V, et al. Production of CGMP-grade lentiviral vectors. BioProcess Int 10 (2012), 32–43.
31. 31 Boussif, O, Lezoualc'h, F, Zanta, MA, Mergny, MD, Scherman, D, Demeneix, B, et al. “A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine.”. Proc Natl Acad Sci USA 92 (1995), 7297–7301.