Characterization of a recombinant adeno-associated virus type 2 reference standard material

Human Gene Therapy

Volumn 21, Issue 10, 2010, Pages 1273-1285

  Lock, Martin ^a   McGorray, Susan ^b   Auricchio, Alberto ^c,d   Ayuso, Eduard ^e   Beecham, E Jeffrey ^f   Blouin Tavel, Véronique ^g   Bosch, Fatima ^e   Bose, Mahuya ^h   Byrne, Barry J ^b,h   Caton, Tina ^h   Chiorini, John A ⁱ   Chtarto, Abdelwahed ^j   Clark, K Reed ^k,l   Conlon, Thomas ^h   Darmon, Christophe ^g   Doria, Monica ^d   Douar, Anne ^m   Flotte, Terence R ⁿ   Francis, Joyce D ^h   Francois, Achille ^g   Giacca, Mauro ^o   Korn, Michael T ^a   Korytov, Irina ^h   Leon, Xavier ^e   Leuchs, Barbara ^p   Lux, Gabriele ^q   Melas, Catherine ^j   Mizukami, Hiroaki ^r   Moullier, Philippe ^g   Müller, Marcus ^p   Ozawa, Keiya ^r   Philipsberg, Tina ^h   Poulard, Karine ^m   Raupp, Christina ^p   Rivière, Christel ^m   Roosendaal, Sigrid D ^s   Samulski, R Jude ^f   Soltys, Steven M ^f   Surosky, Richard ^t   Tenenbaum, Liliane ^j   Thomas, Darby L ^u   Van Montfort, Bart ^s   Veres, Gabor ^u   Wright, J Fraser ^v,w   Xu, Yili ^t   Zelenaia, Olga ^v   Zentilin, Lorena ^o   Snyder, Richard O ^b,h   more..

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^c UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^d TELETHON INSTITUTE OF GENETICS AND MEDICINE TIGEM   (Italy)

^e UNIVERSITAT AUTÒNOMA DE BARCELONA   (Spain)

^f UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^g NANTES UNIVERSITY HOSPITAL   (France)

^h UNIVERSITY OF FLORIDA   (United States)

ⁱ NATIONAL INSTITUTE OF DENTAL AND CRANIOFACIAL RESEARCH   (United States)

^j FREE UNIVERSITY OF BRUSSELS   (Belgium)

^k THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN S HOSPITAL   (United States)

^l OHIO STATE UNIVERSITY   (United States)

^m GÉNÉTHON   (France)

ⁿ UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^o INTERNATIONAL CENTRE FOR GENETIC ENGINEERING AND BIOTECHNOLOGY   (Italy)

^p GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

^q Dr Reckeweg and Co   (Germany)

^r JICHI MEDICAL UNIVERSITY   (Japan)

^s Amsterdam Molecular Therapeutics   (Netherlands)

^t SANGAMO BIOSCIENCES INC   (United States)

^u APPLIED GENETIC TECHNOLOGIES CORPORATION   (United States)

^v CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^w UNIVERSITY OF PENNSYLVANIA   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; BETA GALACTOSIDASE; GREEN FLUORESCENT PROTEIN;

ADENOVIRUS 2; ANIMAL CELL; ARTICLE; CONTROLLED STUDY; DRUG PURITY; DRUG STERILITY; ENZYME LINKED IMMUNOSORBENT ASSAY; GENETIC TRANSFECTION; MICROBIAL CONTAMINATION; NONHUMAN; QUANTITATIVE ANALYSIS; VIRUS PARTICLE;

BIOLOGICAL ASSAY; DEPENDOVIRUS; DNA, VIRAL; ELECTROPHORESIS, POLYACRYLAMIDE GEL; ENZYME-LINKED IMMUNOSORBENT ASSAY; GENETIC VECTORS; GENOME, VIRAL; HELPER VIRUSES; POLYMERASE CHAIN REACTION; REFERENCE STANDARDS; TRANSDUCTION, GENETIC; VIRUS REPLICATION;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS - 2; MIRIDAE;

EID: 77958099436     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2009.223     Document Type: Article

References (26)

1. Brantly, M.L., Chulay, J.D., Wang, L., Mueller, C., Humphries, M., Spencer, L.T., Rouhani, F., Conlon, T.J., Calcedo, R., Betts, M.R., Spencer, C., Byrne, B.J., Wilson, J.M., and Flotte, T.R. (2009). Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc. Natl. Acad. Sci. U.S.A. 106, 16363-16368.
2. Burger, C., Gorbatyuk, O.S., Velardo, M.J., Peden, C.S., Williams, P., Zolotukhin, S., Reier, P.J., Mandel, R.J., and Muzyczka, N. (2004). Recombinant AAV viral vectors pseu-dotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system. Mol. Ther. 10, 302-317.
3. CBER/FDA (Center for Biologics Evaluation and Research, U.S. Food and Drug Administration). (1993). Points to consider in the characterization of cell lines used to produce biologicals. Available at http://www.fda.gov/downloads/ BiologicsBlood Vaccines/GuidanceComplianceRegulatoryInformation/Other RecommendationsforManufacturers/UCM062745.pdf (accessed July 2010).
4. Chadeuf, G., Favre, D., Tessier, J., Provost, N., Nony, P., Kleinschmidt, J., Moullier, P., and Salvetti, A. (2000). Efficient recombinant adeno-associated virus production by a stable rep-cap HeLa cell line correlates with adenovirus-induced am-plification of the integrated rep-cap genome. J. Gene Med. 2, 260-268.
5. Croyle, M.A., Cheng, X., and Wilson, J.M. (2001). Development of formulations that enhance physical stability of viral vectors for gene therapy. Gene Ther. 8, 1281-1290.
6. Grimm, D., Kern, A., Rittner, K., and Kleinschmidt, J.A. (1998). Novel tools for production and purification of recombinant adenoassociated virus vectors. Hum. Gene Ther. 9, 2745-2760.
7. Hutchins, B. (2002). Development of a reference material for characterizing adenovirus vectors. BioProcess. J. 1, 25-28.
8. Kärber, G. (1931). 50% end-point calculation. Arch. Exp. Pathol. Pharmak. 162, 480-483.
9. Littell, R.C., Milliken, G.A., Stroup, W.W., and Wolfinger, R.E. (2006). SASforMixed Models, 2nd ed. (SAS Institute, Cary, NC).
10. Maguire, A.M., Simonelli, F., Pierce, E.A., Pugh, E.N., Jr., Min-gozzi, F., Bennicelli, J., Banfi, S., Marshall, K.A., Testa, F., Surace, E.M., Rossi, S., Lyubarsky, A., Arruda, V.R., Konkle, B., Stone, E., Sun, J., Jacobs, J., Dell'Osso, L., Hertle, R., Ma, J.X., Redmond, T.M., Zhu, X., Hauck, B., Zelenaia, O., Shindler, K.S., Maguire, M.G., Wright, J.F., Volpe, N.J., McDonnell, J.W., Auricchio, A., High, K.A., and Bennett, J. (2008). Safety and efficacy of gene transfer for Leber's con-genital amaurosis. N. Engl. J. Med. 358, 2240-2248.
11. McCarty, D.M., Fu, H., Monahan, P.E., Toulson, C.E., Naik, P., and Samulski, R.J. (2003). Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther. 10, 2112-2118.
12. Moss, R.B., Rodman, D., Spencer, L.T., Aitken, M.L., Zeitlin, P.L., Waltz, D., Milla, C., Brody, A.S., Clancy, J.P., Ramsey, B., Hamblett, N., and Heald, A.E. (2004). Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: A multicenter, double-blind, placebo-controlled trial. Chest 125, 509-521.
13. Moullier, P., and Snyder, R.O. (2008). International efforts for recombinant adeno-associated viral vector reference stan-dards. Mol. Ther. 16, 1185-1188.
14. Mueller, C., and Flotte, T.R. (2008). Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther. 15, 858-863.
15. Nienhuis, A. (2009). Dose-escalation study of a self comple-mentary adeno-associated viral vector for gene transfer in hemophilia B. Clinical trial NCT00979238. Available at http://clinicaltrials.gov/ct2/show/NCT00979238 (accessed July 2010).
16. Potter, M., Phillipsberg, G., Phillipsberg, T., Pettersen, M., San-ders, D., Korytov, I., Fife, J., Zolotukhin, S., Byrne, B.J., and Muzyczka, N. (2008). Manufacture and stability study of the recombinant adeno-associated virus serotype 2 vector refer-ence standard. Bioprocess. J. 7, 8-14.
17. Rabilloud, T., Strub, J.M., Luche, S., van Dorsselaer, A., and Lunardi, J. (2001). A comparison between SYPRO Ruby and ruthenium II tris(bathophenanthroline disulfonate) as fluorescent stains for protein detection in gels. Proteomics 1, 699-704.
18. Salvetti, A., Oreve, S., Chadeuf, G., Favre, D., Cherel, Y., Champion-Arnaud, P., David-Ameline, J., and Moullier, P. (1998). Factors influencing recombinant adeno-associated virus production. Hum. Gene Ther. 9, 695-706.
19. Snyder, R.O., and Flotte, T.R. (2002). Production of clinical-grade recombinant adeno-associated virus vectors. Curr. Opin. Biotechnol. 13, 418-423.
20. Sommer, J.M., Smith, P.H., Parthasarathy, S., Isaacs, J., Vijay, S., Kieran, J., Powell, S.K., McClelland, A., and Wright, J.F. (2003). Quantification of adeno-associated virus particles and empty capsids by optical density measurement. Mol. Ther. 7, 122-128.
21. Van Vliet, K., Blouin, V., Agbandje-McKenna, M., and Snyder, R.O. (2006). Proteolytic mapping of the adeno-associated virus capsid. Mol. Ther. 14, 809-821.
22. Warrington, K.H., Jr., and Herzog, R.W. (2006). Treatment of human disease by adeno-associated viral gene transfer. Hum. Genet. 119, 571-603.
23. Weiss, W., Weiland, F., and Gorg, A. (2009). Protein detection and quantitation technologies for gel-based proteome analysis. Methods Mol. Biol. 564, 59-82.
24. Wright, J.F., Qu, G., Tang, C., and Sommer, J.M. (2003). Recombinant adeno-associated virus: Formulation challenges and strategies for a gene therapy vector. Curr. Opin. Drug Discov. Dev. 6, 174-178.
25. Zen, Z., Espinoza, Y., Bleu, T., Sommer, J.M., and Wright, J.F. (2004). Infectious titer assay for adeno-associated virus vectors with sensitivity sufficient to detect single infectious events. Hum. Gene Ther. 15, 709-715.
26. Zolotukhin, S., Byrne, B.J., Mason, E., Zolotukhin, I., Potter, M., Chesnut, K., Summerford, C., Samulski, R.J., and Muzyczka, N. (1999). Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther. 6, 973-985