Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses

Human Gene Therapy

Volumn 10, Issue 6, 1999, Pages 1031-1039

  Clark, K Reed ^a,d   Liu, Xinglou ^a   Mcgrath, John P ^b   Johnson, Philip R ^a,c  

^a OHIO STATE UNIVERSITY   (United States)

^b ALKERMES INC   (United States)

^c THE OHIO STATE UNIVERSITY COLLEGE OF MEDICINE   (United States)

^d NATIONWIDE CHILDREN S HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

HEPARIN; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENOVIRUS; AFFINITY CHROMATOGRAPHY; ARTICLE; HELPER VIRUS; NONHUMAN; POLYMERASE CHAIN REACTION; VIRUS ISOLATION; VIRUS RECOMBINANT;

CHROMATOGRAPHY, AFFINITY; CHROMATOGRAPHY, HIGH PRESSURE LIQUID; CYTOPATHOGENIC EFFECT, VIRAL; DEPENDOVIRUS; ELECTROPHORESIS, POLYACRYLAMIDE GEL; GENETIC VECTORS; HELPER VIRUSES; HUMANS; POLYMERASE CHAIN REACTION; RECOMBINATION, GENETIC; TRANSDUCTION, GENETIC;

EID: 0033541640     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950018427     Document Type: Article

References (35)

1. ALEXANDER, I.E., RUSSELL, D.W., and MILLER, A.D. (1994). DNA-damaging agents greatly increase the transduction of nondviding cells by adeno-associated virus vectors. J. Virol. 68, 1911-1920.
2. ALEXANDER, I.E., RUSSELL, D.W., and MILLER, A.D. (1997). Transfer of contaminants in adeno-associated virus vector stocks can mimic transduction and lead to artifactual results. Hum. Gene Ther. 8, 1911-1920.
3. ALLEN, J.M., DEBELAK, D.J., REYNOLDS, T.C., and MILLER, A.D. (1997). Identification and elimination of replication-competent adeno-associated virus (AAV) that arise by nonhomologous recombination during AAV vector production. J. Virol. 71, 6816-6822.
4. CARTER, B.J., LAUGHLIN, C.A., DE LA MAZA, L.M., and MYERS, M. (1979). Adeno-associated virus autointerference. Virology 92, 449-462.
5. CLARK, K.R., VOULGAROPOULOU, F., FRALEY, D.M., and JOHNSON, P.R. (1995). Cell line for the production of recombinant adeno-associated virus. Hum. Gene Ther. 6, 1329-1341.
6. CLARK, K.R., VOULGAROPOULOU, F., and JOHNSON, P.R. (1996). A stable cell line carrying adenovirus inducible rep and cap genes allows for infectivity titration of adeno-associated virus vectors. Gene Ther. 3, 1124-1132.
7. CLARK, K.R., SFERRA, T.J., and JOHNSON, P.R. (1997). Recombinant adeno-associated virus vectors mediate long-term transgene expression in muscle. Hum. Gene Ther. 8, 659-669.
8. FERRARI, F.K., SAMULSKI, T., SHENK T., and SAMULSKI, R.J. (1996). Second-strand synthesis is a rate limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 70, 3227-3234.
9. FISHER, K.J., GAO, G.-P., WEITZMAN, M.D., DEMATTEO, R., BURDA, J.F., and WILSON, J.M. (1996). Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70, 520-532.
10. FISHER, K.J., JOOSS, K., ALSTON, K., YANG, Y., HAECHKER, S.E., HIGH, K., PATHAK, R., RAPER, S.E., and WILSON, J.M. (1997). Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med. 3, 306-312.
11. FLANNERY, J.G., ZOLOTUKHIN, S., VAQUERO, M.I., LAVAIL, M.M., MUZYCZKA, N., and HAUSWIRTH, W.W. (1997). Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Proc. Natl. Acad. Sci. U.S.A. 94, 6916-6921.
12. FLOTTE, T.R., AFIONE, S.A., CONRAD, C., McGRATH, S.A., SOLOW, R., OKA, H., ZEITLIN, P.L., GUGGINO, W.B., and CARTER, B.J. (1993). Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc. Natl. Acad. Sci. U.S.A. 90, 10613-10617.
13. GAO, G.-P., QU, G., FAUST, L.Z., ENGDAHL, R.K., XIAO, W., HUGHES, J.V., ZOLTICK, P.W., and WILSON, J.M. (1998). High-titer adeno-associated viral vectors from a rep/cap cell line and hybrid shuttle virus. Hum. Gene Ther. 9, 2353-2362.
14. GIBSON, U.E, HEID, C.A., and WILLIAMS, P.M. (1996). A novel method for real time quantitative RT-PCR. Genome Res. 6, 995-1001.
15. GRAHAM, F.L., and PREVEC, L. (1991). Manipulation of adenovirus vectors. Methods Mol. Biol. 7, 109-128.
16. HALBERT, C.L., ALEXANDER, I.E., WOLGAMOT, G.M., and MILLER, A.D. (1995). Adeno-associated virus vectors transduce primary cells much less efficiently than immortalized cells. J. Virol. 69, 1473-1479.
17. HOLLAND, P.M., ABRAMSON, R.D., WATSON, R., and GELFAND, D.H. (1991). Detection of specific polymerase chain reaction product by utilizing the 5′-3′ exonuclease activity of Thermus aquaticus DNA polymerase. Proc. Natl. Acad. Sci. U.S.A. 88, 7276-7280.
18. KAPLITT, M.G., LEONE, P., SAMULSKI, R.J., XIAO, X., PFAFF, D.W., O'MALLEY, K.L., and DURING, M.J. (1994). Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nature Genet. 8, 148-154.
19. KATAYAMA, S., YOSHIKI, K., OKADA, N., KOKUBU, T., and SHIMIZU, Y. (1998). Efficacy of a mixed glycoprotein vaccine against pseudorabies in pregnant sows. J. Vet. Med. Sci. 60, 23-27.
20. KESSLER, P.D., PODSAKOFF, G.M., CHEN, X., MCQUISTON, S.A., COLOSI, P.C., MATELIS, L.A., KURTZMAN, G.J., and BYRNE, B.J. (1996). Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc. Natl. Acad. Sci. U.S.A. 93, 14082-14087.
21. KOEBERL, D.D., ALEXANDER, I.E., HALBERT, C.L., RUSSELL, D.W., and MILLER, A.D. (1997). Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc. Natl. Acad. Sci. U.S.A. 94, 1426-1431.
22. LAUGHLlN, C.A., MYERS, M.W., RISIN, D.L., and CARTER, B.J. (1979). Defective-interfering particles of the human parvovirus adeno-associated virus. Virology 94, 162-174.
23. LIU, X., CLARK, K.R., and JOHNSON, P.R. (1999). Production of recombinant adeno-associated virus vectors using a packaging cell line and a hybrid recombinant adenovirus. Gene Ther. 6, 293-299.
24. MENDELSON, E., TREMPE, J.P., and CARTER, B.J. (1986). Identification of the trans-acting Rep proteins of adeno-associated virus by antibodies to a synthetic oligopeptide. J. Virol. 60, 823-832.
25. MIAO, C.H., SNYDER, R.O., SCHOWALTER, D.B., PATIJN, G.A., DONAHUE, B., WINTHER, B., and KAY, M.A. (1998). The kinetics of rAAV integration in the liver. Nature Genet. 19, 13-15.
26. NAVARRO DEL CANIZO, A.A., MAZZA, M., BELLINZONI, R., and CASCONE, O. (1996). Foot and mouth disease virus concentration and purification by affinity chromatography. Appl. Biochem. Biotechnol. 61, 399-409.
27. RUSSELL, D.W., ALEXANDER, I.E., and MILLER, A.D. (1995). DNA synthesis and topoisomerase inhibitors increase transduction by adeno-associated virus vectors. Proc. Natl. Acad. Sci. U.S.A. 92, 5719-5723.
28. 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.
29. SNYDER, R.O., MIAO, C.H., PATIJN, G.A., SPRATT, S.K., DANOS, O., NAGY, D., GOWN, A.M., WINTHER, B., MEUSE, L., COHEN, L.K., THOMPSON, A.R., and KAY, M.A. (1997). Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nature Genet. 16, 270-276.
30. SUMMERFORD, C., and SAMULSKI, R.J. (1998). Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J. Virol. 72, 1438-1445.
31. TAMAYOSE, K., HIRAI, Y., and SHIMADA, T. (1996). A new strategy for large-scale preparation of high-titer recombinant adeno-associated virus vectors by using packaging cell lines and sulfonated cellulose column chromatography. Hum. Gene Ther. 7, 507-513.
32. VINCENT, K.A., PIRAINO, S.T., and WADSWORTH, S.C. (1997). Analysis of recombinant adeno-associated virus packaging and requirements for rep and cap gene products. J. Virol. 71, 1897-1905.
33. WANG, X.S., KHUNTIRAT, B., QING, K., PONNAZHAGAN, S., KUBE, D.M., ZHOU, S., DWARKI, V.J., and SRIVASTAVA, A. (1998). Characterization of wild-type adeno-associated virus type 2-like particles generated during recombinant viral vector production and strategies for their elimination. J. Virol. 72, 5474-5480.
34. XIAO, X., LI, J., and SAMULSKI, R.J. (1996). Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70, 8098-8108.
35. XIAO, X., LI, J., and SAMULSKI, R.J. (1998). Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol. 72, 2224-2232.