High-titer recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H

Human Gene Therapy

Volumn 10, Issue 15, 1999, Pages 2527-2537

  Zhang, Xiaoliu ^a   De Alwis, Mahesh ^b   Hart, Stephen L ^b   Fitzke, Frederick W ^c   Inglis, Stephen C ^a   Boursnell, Mike E G ^a   Levinsky, Roland J ^b   Kinnon, Christine ^b   Ali, Robin R ^b,c   Thrasher, Adrian J ^b  

^a Cantab Pharmaceuticals Ltd   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

GLYCOPROTEIN; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ARTICLE; CONTAMINATION; FACILITATION; GENE DELETION; GENETIC COMPLEMENTATION; HELPER CELL; IMMUNE RESPONSE; NONHUMAN; PROTEIN EXPRESSION; VIROGENESIS; VIRUS GENOME; VIRUS RECOMBINANT; VIRUS REPLICATION;

ADENO-ASSOCIATED VIRUS; ANIMALIA; DNA VIRUSES; HERPES; SIMPLEXVIRUS;

EID: 0033544027     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950016861     Document Type: Article

References (44)

1. ALI, R.R., REICHEL, M.B., THRASHER, A.J., LEVINSKY, R.J., KINNON, C., KANUGA, N., HUNT, D.M., and BHATTACHARYA, S.S. (1996). Gene transfer into the mouse retina mediated by an adeno-associated viral vector. Hum. Mol. Genet. 5, 591-594.
2. ALI, R.R., REICHEL, M.B., DE, A.M., KANUGA, N., KINNON, C., LEVINSKY, R.J., HUNT, D.M., BHATTACHARYA, S.S., and THRASHER, A.J. (1998). Adeno-associated virus gene transfer to mouse retina. Hum. Gene Ther. 9, 81-86.
3. ALLEN, J.M., DEBELAK, D.M., REYNOLDS, T.C., and MILLER, A.D. (1997). Identification and elimination of replication-competent adeno-associated virus (AAV) that can arise by nonhomologous recombination during AAV vector production. J. Virol. 71, 6816-6822.
4. BENNETT, J., DUAN, D., ENGELHARDT, J.F., and MAGUIRE, A.M. (1997). Real-time, noninvasive in vivo assessment of adeno-associated virus-mediated retinal transduction. Invest. Ophthalmol. Vis. Sci. 38, 2857-2863.
5. BOURSNELL, M.E., ENTWISLE, C., BLAKELY, D., ROBERTS, C., DUNCAN, I.A., CHISHOLD, S.E., MARTIN, G.M., JENNINGS, R., NI, C.D., SOBEK, I., INGLIS, S.C., and McLEAN, C.S. (1997). A genetically inactivated herpes simplex virus type 2 (HSV-2) vaccine provides effective protection against primary and recurrent HSV-2 disease. J. Infect. Dis. 175, 16-25.
6. BULLER, R.M., JANIK, J.E., SEBRING, E.D., and ROSE, J.A. (1981). Herpes simplex virus types 1 and 2 completely help adenovirus-associated virus replication. J. Virol. 40, 241-247.
7. CHIORINI, J.A., WENDTNER, C.M., URCELAY, E., SAFER, B., HALLEK, M., and KOTIN, R.M. (1995). High-efficiency transfer of the T cell costimulatory molecule B7-2 to lymphoid cells using high-titer recombinant adeno-associated virus vectors. Hum. Gene Ther. 6, 1531-1541.
8. CLARK, K.R., SFERRA, T.J., and JOHNSON, P.R. (1997) Recombinant adeno-associated viral vectors mediate long term transgen expression in muscle. Hum Gene Ther. 8, 659-669.
9. CONWAY, J.E., ZOLOTUKHIN, S., MUZYCZKA, N., HAYWARD, G.S., and BYRNE, B.J. (1997). Recombinant adeno-associated virus type 2 replication and packaging is entirely supported by a herpes simplex virus type 1 amplicon expressing Rep and Cap. J. Virol. 71, 8780-8789.
10. FAN, P.D. and DONG, J.Y. (1997). Replication of rep-cap genes is essential for the high-efficiency production of recombinant AAV. Hum. Gene Ther. 8, 87-98.
11. 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, 3227-3334.
12. 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.
13. FISHER, K.J., JOOSS, K., ALSTON, J., YANG, Y., HAECKER, 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.
14. FLANNERY, J.G., ZOLOTUKHIN, S., VAQUERO, M.I., LAVAIL, M.M., MUZYCZK, 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.
15. FLOTTE, T.R. and CARTER, B.J. (1995). Adeno-associated virus vectors for gene therapy. Gene Ther. 2, 357-362.
16. FLOTTE, T.R., BARRAZA, O.X., SOLOW, R., AFIONE, S.A., CARTER, B.J., and GUGGINO, W.B. (1995). An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction. Gene Ther. 2, 29-37.
17. FORRESTER, A., FARRELL, H., WILKINSON, G., KAYE, J., DAVIS, P.N., and MINSON, T. (1992). Construction of properties of a mutant of herpes simplex virus type 1 with glycoprotein H coding sequences deleted. J. Virol. 66, 341-348.
18. GOSSEN, M.S., FREUNDLIEB, S., BENDER, G., MULLER, G., HILLEN, W., and BUJARD, H. (1995). Transcriptional activation by tetracycles in mammalian cells. Science 268, 1766-1769.
19. HART, S.L., ARANCIBIA, C.C., WOLFERT, M.A., MAILHOS, C., O'REILLY, N.J., ALI, R.R., COUTELLE, C., GEORGE, A.J., HARBOTTLE, R.P., KNIGHT, A.M., LARKIN, D.F., LEVINSKY, R.J., SEYMOUR, L.W., THRASHER, A.M. and KINNON, C. (1998). Lipid-mediated enhancement of transfection by a nonviral integrin-targeting vector. Hum. Gene Ther. 9, 575-585.
20. HERZOG, R.W., HAGSTROM, J.N., KUNG, S.H., TAI, S.J., WILSON, J.M., FISHER, K.J., and HIGH, K.A. (1997). Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc. Natl. Acad. Sci. U.S.A. 94, 5804-5809.
21. HILL, T.J., FIELD, H.J., and BLYTH, W.A. (1975). Acute and recurrent infection with herpes simplex virus in the mouse: A model for studying latency and recurrent disease. J. Gen. Virol. 28, 341-353.
22. INOUE, N., and RUSSELL, D.W. (1998). Packaging cells based on inducible gene amplication for the production of adeno-associated virus vectors. J. Virol. 72, 7024-7031.
23. JOHNSTON, K.M., JACOBY, D., PECHAN, P.A., FRAEFEL, C., BORGHESANI, P., SCHUBACK, D., DUNN, R.J., SMITH, F.I., and BREAKEFIELD, X.O. (1997). HSV/AAV hybrid amplicon vectors extend transgene expression in human glioma cells. Hum. Gene Ther. 8, 359-370.
24. JOMARY, C., VINCENT, K.A., GRIST, J., NEAL, M.J., and JONES, S.E. (1997). Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration. Gene Ther. 4, 683-690.
25. 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.
26. 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.
27. 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.
28. KOTIN, R.M. (1994). Prospects for the use of adeno-associated virus as a vector for human gene therapy. Hum. Gene Ther. 5, 793-801.
29. LAUGHLIN, C.A., TRATSCHIN, J.D., COON, H., and CARTER, B.J. (1983). Cloning of infectious adeno-associated virus genomes in bacterial plasmids. Gene 23, 65-73.
30. LI, J., SAMULSKI, R.J., and XIAO, X. (1997). Rose for highly regulated rep gene expression in adeno-associated virus vector production. J. Virol. 71, 5236-5243.
31. MATSUSHITA, T., ELLIGER, S., ELLIGER, C., PODSAKOFF, G., VILLAREAL, L., KURTZMAN, G.J., IWAKI, Y., and COLOSI, P. (1998). Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther. 5, 938-945.
32. MCPHERSON, R.A., ROSENTHAL, L.J., and ROSE, J.A. (1985). Human cytamegalovirus completely helps adeno-associated virus replication. Virology 147, 217-222.
33. MIAO, C.H., SNYDER, R.O., SCHOWALTER, D.B., PATJIN, G.A., DONAHUE, B., WINTHER, B., and KAY, M.A. (1998). The kinetics of rAAV integration in the liver. Nature Genet. 19, 13-15.
34. MONAHAN, P.E., SAMULSKI, R.J., TAZELAAR, J., XIAO, X., NICHOLS, T.C., BELLINGER, D.A., READ, M.S., and WALSH, C.E. (1998). Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Ther. 5, 40-49.
35. SMITH, I.L., HARDWICKE, M.A., and SANDRI, G.R. (1992). Evidence that the herpes simplex virus immediate early protein ICP27 acts posttranscriptionally during infection to regulate gene expression. Virology 186, 74-86.
36. THOMSON, B.J., WEINDLER, F.W., GRAY, D., SCHWAAB, V. and HEILBRONN, R. (1994). Human herpesvirus 6 (HHV-6) is a helper virus for adeno-associated virus type 2 (AAV-2) and the AAV-2 rep gene homologue in HHV-6 can mediate AAV-2 DNA replication and regulate gene expression. Virology 204, 304-311.
37. 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.
38. 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, 5472-5480.
39. WEINDLER, F.W. and HEILBRONN, R. (1991). A subset of herpes simplex virus replication genes provides helper functions for productive adeno-associated virus replication. J. Virol. 65, 2476-2483.
40. 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. Vol. 70, 8098-8108.
41. XIAO, X., LI, W., and SAMULSKI, R.J. (1997). A novel 165-base-pair terminal repeat sequence is the sole cis requirement for the adeno-associated virus life cycle. J. Virol. 71, 941-948.
42. 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.
43. ZHANG, X., O'SHEA, H., ENTWISLE, C., BOURSNELL, M., EFSTATHIOU, S., and INGLIS, S. (1998). An efficient selection system for packaging herpes simplex virus amplicons. J. Gen. Virol. 79, 125-131
44. ZOLOTUKHIN, S., POTTER, M., HAUSWIRTH, W.W., GUY, J., and MUZYCZKA, N. (1996). A "humanized" green fluorescent protein cDNA adapted for high-level expression in mammalian cells. J. Virol. 70, 4646-4654.