Rational plasmid design and bioprocess optimization to enhance recombinant adeno-associated virus (AAV) productivity in mammalian cells

Biotechnology Journal

Volumn 11, Issue 2, 2016, Pages 290-297

  Emmerling, Verena V ^a,b   Pegel, Antje ^b   Milian, Ernest G ^c   Venereo Sanchez, Alina ^c   Kunz, Marion ^b   Wegele, Jessica ^b   Kamen, Amine A ^d   Kochanek, Stefan ^a   Hoerer, Markus ^b  

^a UNIVERSITY OF ULM   (Germany)

^b Rentschler Biotechnologie GmbH   (Germany)

^c NATIONAL RESEARCH COUNCIL CANADA   (Canada)

^d MCGILL UNIVERSITY   (Canada)

Author keywords

Adeno associated virus; HEK293T; ICELLis; Recombinant vector; Vector production

Indexed keywords

BIOREACTORS; BODY FLUIDS; DNA; GENE THERAPY; MAMMALS; MANUFACTURE; PRODUCTIVITY; VIRUSES;

ADENO-ASSOCIATED VIRUS; BIOPROCESS OPTIMIZATION; HEK293T; ICELLIS; MANUFACTURING STRATEGY; RECOMBINANT ADENO-ASSOCIATED VIRUS; STIRRED TANK BIOREACTORS; VOLUMETRIC PRODUCTIVITY;

VECTORS;

PARVOVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ARTICLE; BIOPROCESS; CELL SUSPENSION; CONTROLLED STUDY; DNA TRANSFECTION; HEK293 CELL LINE; HELA CELL LINE; HUMAN; HUMAN ADENOVIRUS C; HUMAN CELL; LARGE SCALE PRODUCTION; MAMMAL CELL; NONHUMAN; PLASMID; PRIORITY JOURNAL; PROCESS OPTIMIZATION; START CODON; SUSPENSION CELL CULTURE; TRANSIENT TRANSFECTION; VIRUS RECOMBINANT; BIOENGINEERING; DEPENDOPARVOVIRUS; GENETICS; PHYSIOLOGY; PROCEDURES; VIRUS REPLICATION;

BIOENGINEERING; DEPENDOVIRUS; HEK293 CELLS; HELA CELLS; HUMANS; PLASMIDS; VIRUS REPLICATION;

EID: 84957441838     PISSN: 18606768     EISSN: 18607314     Source Type: Journal    
DOI: 10.1002/biot.201500176     Document Type: Article

References (17)

1. Moran, N., First gene therapy nears landmark European market authorization. Nature Biotechnol. 2012, 30, 807-809.
2. Miyake, K., Miyake, N., Yamazaki, Y., Shimada, T., Hirai, Y., Serotype-independent method of recombinant adeno-associated virus (AAV) vector production and purification. J. Nippon Med. Sch 2012, 79, 394-402.
3. Weitzman, M. D., Linden, R. M., Adeno-associated virus biology. Methods Mol. Biol. 2011, 807, 1-23.
4. Chahal, P. S., Schulze, E., Tran, R., Montes, J., Kamen, A. A., 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.
5. Ayuso, E., Mingozzi, F., Bosch, F., Production, purification and characterization of adeno-associated vectors. Curr. Gene Ther. 2010, 10, 423-436.
6. Aucoin, M. G., Perrier, M., Kamen, A. A., Critical assessment of current adeno-associated viral vector production and quantification methods. Biotechnol. Adv. 2008, 26, 73-88.
7. Merten, O.-W., Advances in cell culture: Anchorage dependence. Philos. Trans. R. Soc. Lond. B Biol. Sci. 2015, 370, 20140040.
8. Hoerer, M., Dubielzig, R., Ries, S., Kosfeld-Bergauer, M., Helper constructs for producing hybrid rAAV particles of various AAV serotypes. WO Patent Application 2003/074686A1, 2003.
9. Mayginnes, J. P., Reed, S. E., Berg, H. G., Staley, E. M. et al., Quantitation of encapsidated recombinant adeno-associated virus DNA in crude cell lysates and tissue culture medium by quantitative, real-time PCR. J. Virol. Methods 2006, 137, 193-204.
10. Aurnhammer, C., Haase, M., Muether, N., Hausl, M. et al., Universal real-time PCR for the detection and quantification of adeno-associated virus serotype 2-derived inverted terminal repeat sequences. Hum. Gene Ther. Methods 2012, 23, 18-28.
11. Rohr, U.-P., Wulf, M.-A., Stahn, S., Steidl, U. et al., Fast and reliable titration of recombinant adeno-associated virus type-2 using quantitative real-time PCR. J. Virol. Methods 2002, 106, 81-88.
12. Grimm, D., Kern, A., Rittner, K., Kleinschmidt, J. A., Novel tools for production and purification of recombinant adenoassociated virus vectors. Hum. Gene Ther. 1998, 9, 2745-2760.
13. Steinbach, S., Wistuba, A., Bock, T., Kleinschmidt, J. A., Assembly of adeno-associated virus type 2 capsids in vitro. The J. Gen. Virol. 1997, 78, 1453-1462.
14. Giacca, M., Zacchigna, S., Virus-mediated gene delivery for human gene therapy. J. Controlled Release 2012, 161, 377-388.
15. Jenny, C., Toublanc, E., Danos, O., Merten, O. W., Evaluation of a serum-free medium for the production of rAAV-2 using HeLa derived producer cells. Cytotechnology 2005, 49, 11-23.
16. Mezzina, M., Merten, O. W., Adeno-associated viruses. Methods Mol. Biol. 2011, 737, 211-234.
17. van Oers, M. M., Pijlman, G. P., Vlak, J. M., Thirty years of baculovirus-insect cell protein expression: From dark horse to mainstream technology. J. Gen. Virol. 2015, 96, 6-23.