Development and Optimization of AAV hFIX Particles by Transient Transfection in an iCELLis ® Fixed-Bed Bioreactor

Human Gene Therapy Methods

Volumn 27, Issue 3, 2016, Pages 112-121

  Powers, Alicia D ^a   Piras, Bryan A ^a   Clark, Robert K ^a   Lockey, Timothy D ^a   Meagher, Michael M ^a  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9; CARBON DIOXIDE; DNA; GREEN FLUORESCENT PROTEIN; OXYGEN; PARVOVIRUS VECTOR; POLYETHYLENEIMINE;

ANIMAL EXPERIMENT; ARTICLE; CONCENTRATION (PARAMETERS); CONTROLLED STUDY; CULTURE MEDIUM; DISPOSABLE EQUIPMENT; DNA TRANSFECTION; FEASIBILITY STUDY; FILM; FIXED BED REACTOR; HEK293 CELL LINE; HUMAN; HUMAN CELL; LARGE SCALE PRODUCTION; MOUSE; NONHUMAN; PARTICLE SIZE; PRIORITY JOURNAL; PROCESS OPTIMIZATION; SCALE UP; TRANSIENT TRANSFECTION; VIRAL GENE DELIVERY SYSTEM; BIOREACTOR; BIOSYNTHESIS; DEPENDOPARVOVIRUS; GENE THERAPY; GENE VECTOR; GENETIC TRANSFECTION; GENETICS; THERAPEUTIC USE;

BIOREACTORS; DEPENDOVIRUS; FACTOR IX; GENETIC THERAPY; GENETIC VECTORS; HEK293 CELLS; HUMANS; TRANSFECTION;

EID: 84975744802     PISSN: 19466536     EISSN: 19466544     Source Type: Journal    
DOI: 10.1089/hgtb.2016.021     Document Type: Article

References (32)

1. Luo J, Luo Y, Sun J, et al. Adeno-associated virus-mediated cancer gene therapy: Current status. Cancer Lett 2015;356:347-356.
2. Basner-Tschakarjan E, Mingozzi F. Cell-mediated immunity to AAV vectors, evolving concepts and potential solutions. Front Immunol 2014;5:350.
3. Aucoin MG, Perrier M, Kamen AA. Critical assessment of current adeno-associated viral vector production and quantification methods. Biotechnol Adv 2008;26:73-88.
4. Kaplitt MG, Feigin A, Tang C, et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: An open label, phase I trial. Lancet 2007;369:2097-2105.
5. Bainbridge JW, Smith AJ, Barker SS, et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 2008;358:2231-2239.
6. Bowles DE, McPhee SW, Li C, et al. Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. Mol Ther 2012;20:443-455.
7. High KH, Nathwani A, Spencer T, et al. Current status of haemophilia gene therapy. Haemophilia 2014;20 Suppl 4:43-49.
8. Liu YL, Wagner K, Robinson N, et al. Optimized production of high-titer recombinant adeno-associated virus in roller bottles. Biotechniques 2003;34:184-189.
9. Drittanti L, Jenny C, Poulard K, et al. Optimised helper virus-free production of high-quality adeno-associated virus vectors. J Gene Med 2001;3:59-71.
10. Chahal PS, Schulze E, Tran R, et al. Production of adeno-associated virus (AAV) serotypes by transient transfection of HEK293 cell suspension cultures for gene delivery. J Virol Methods 2014;196:163-173.
11. Hambor JE. Bioreactor design and bioprocess controls for industrialized cell processing. Bio-process Technol 2012;10:22-33.
12. Merten OW, Cruz PE, Rochette C, et al. Comparison of different bioreactor systems for the production of high titer retroviral vectors. Biotechnol Prog 2001;17:326-335.
13. Merten OW. State-of-the-art of the production of retroviral vectors. J Gene Med 2004;6 Suppl 1:S105-S124.
14. Racher AJ, Fooks AR, Griffiths JB. Culture of 293 cells in different culture systems: Cell growth and recombinant adenovirus production. Biotechnol Tech 1995;9:169-174.
15. Van Der Loo J, Swaney W, Grassman E, et al. Scale-up and manufacturing of clinical-grade self-inactivating y-retroviral vectors by transient transfection. Gene Ther 2012;19:246-254.
16. Gerlach JC, Lubberstedt M, Edsbagge J, et al. Interwoven four-compartment capillary membrane technology for three-dimensional perfusion with decentralized mass exchange to scale up embryonic stem cell culture. Cells Tissues Organs 2010; 192:39-49.
17. Azarin SM, Palecek SP. Development of scalable culture systems for human embryonic stem cells. Biochem Eng J 2010;48:378-384.
18. Havelange N, Marigliano M, Sainte-Marie M, et al. Poxvirus production on chicken embryo fibroblasts in iCELLis™ disposable fixed-bed bioreactor. In: Proceedings of the 21st Annual Meeting of the European Society for Animal Cell Technology (ESACT), Dublin, Ireland, June 7-10, 2009. (Springer, New York). 2012; pp. 719-722.
19. Lennaertz A, Knowles S, Drugmand J-C, et al. Viral vector production in the integrity® iCELLis® single-use fixed-bed bioreactor, from bench-scale to industrial scale. BMC Proc 2013;7:P59.
20. Drugmand J-C, Esteban G, Alaoui N, et al. On-line monitoring: Animal cell cultivation in iCELLis™ fixed-bed reactor using dielectric measurements. In: Proceedings of the 21st Annual Meeting of the European Society for Animal Cell Technology (ESACT), Dublin, Ireland, June 7-10, 2009. (Springer, New York). 2012; pp. 395-399.
21. Wang X, Olszewska M, Qu J, et al. Large-scale clinical-grade retroviral vector production in a fixed-bed bioreactor. J Immunother 2015;38:127-135.
22. Lesch HP, Heikkila KM, Lipponen EM, et al. Process development of adenoviral vector production in fixed bed bioreactor: From bench to commercial scale. Hum Gene Ther 2015;26:560-571.
23. Drugmand J-C, Havelange N, Collignon F, et al. 4g/L-day: Monoclonal antibody volumetric productivity in the iCELLisTM disposable fixed-bed bioreactor. In: Proceedings of the 21st Annual Meeting of the European Society for Animal Cell Technology (ESACT), Dublin, Ireland, June 7-10, 2009. (Springer, New York). 2012; pp. 375-378.
24. Rajendran R, Lingasa R, Vuppu SV, et al. Assessment of packed bed bioreactor systems in the production of viral vaccines. AMB Express 2014; 4:25.
25. Allay JA, Sleep S, Long S, et al. Good manufacturing practice production of self-complementary sero-type 8 adeno-associated viral vector for a hemophilia B clinical trial. Hum Gene Ther 2011;22: 595-604.
26. Nathwani AC, Gray JT, Ng CY, et al. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient trans-duction of murine and nonhuman primate liver. Blood 2006;107:2653-2661.
27. Lorber B, Fischer F, Bailly M, et al. Protein analysis by dynamic light scattering: Methods and techniques for students. Biochem Mol Biol Educ 2012;40:372-382.
28. Fagone P, Wright JF, Nathwani AC, et al. Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods. Hum Gene Ther Methods 2011;23:1-7.
29. Huang X, Hartley AV, Yin Y, et al. AAV2 production with optimized N/P ratio and PEI-mediated transfection results in low toxicity and high titer for in vitro and in vivo applications. J Virol Methods 2013;193:270-277.
30. Ahn HH, Lee MS, Cho MH, et al. DNA/PEI nano-particles for gene delivery of rat bone marrow stem cells. Colloids Surfaces A Physicochem Eng Aspects 2008;313:116-120.
31. Doria M, Ferrara A, Auricchio A. AAV2/8 vectors purified from culture medium with a simple and rapid protocol transduce murine liver, muscle, and retina efficiently. Hum Gene Ther Methods 2013;24:392-398.
32. Reed SE, Staley EM, Mayginnes JP, et al. Transfection of mammalian cells using linear polyethylenimine is a simple and effective means of producing recombinant adeno-associated virus vectors. J Virol Methods 2006;138:85-98.