High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type I vector expressing AAV-2 rep and cap

Gene Therapy

Volumn 6, Issue 6, 1999, Pages 986-993

  Conway, J E ^a   Ap Rhys, C M J ^a   Zolotukhin, I ^b,c   Zolotukhin, S ^b   Muzyczka, N ^b   Hayward, G S ^a   Byrne, B J ^b,c  

^a JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF FLORIDA   (United States)

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

Author keywords

Adeno associated virus; Gene therapy; Herpes simplex virus; rAAV; Vector production

Indexed keywords

CAPSID PROTEIN; HELICASE; PLASMID DNA; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ARTICLE; CONTROLLED STUDY; DNA TRANSFECTION; HELPER VIRUS; HERPES SIMPLEX VIRUS 1; NONHUMAN; PRIORITY JOURNAL; PROVIRUS; VIRUS PURIFICATION; VIRUS RECOMBINANT; VIRUS TITRATION;

BLOTTING, WESTERN; CELL LINE; DEPENDOVIRUS; FLUORESCENT ANTIBODY TECHNIQUE; GENE THERAPY; GENETIC VECTORS; HUMANS; SIMPLEXVIRUS; TRANSFECTION; VIROLOGY; VIRUS REPLICATION;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS 2; ANIMALIA; HERPES; HUMAN HERPESVIRUS 1; MIRIDAE; SIMPLEXVIRUS;

EID: 0033054126     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3300937     Document Type: Article

References (52)

1. Kessler PD et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 1996; 93: 14082-14087.
2. Herzog RW et al. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc Natl Acad Sci USA 1997; 94: 5804-5809.
3. Koeberl DD et al. Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc Natl Acad Sci USA 1997; 94: 1426-1431.
4. Nakai H et al. Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood 1998; 91: 4600-4607.
5. Monahan PE et al. Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Therapy 1998; 5: 40-49.
6. Song S et al. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci USA 1998; 95: 14384-14388.
7. Kaplitt MG et al. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet 1994; 8: 148-154.
8. Peel AL et al. Efficient transduction of green fluorescent protein in spinal cord neurons using adeno-associated virus vectors containing cell type-specific promoters. Gene Therapy 1997; 4: 16-24.
9. Xiao X et al. Adeno-associated virus (AAV) vector antisense gene transfer in vivo decreases GABA(A) alpha1 containing receptors and increases inferior collicular seizure sensitivity. Brain Res 1997; 756: 76-83.
10. Flotte TR, Carter BJ. Adeno-associated virus vectors for gene therapy of cystic fibrosis. Meth Enzymol 1998; 292: 717-732.
11. Muzyczka N. Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr Top Microbiol Immunol 1992; 158: 97-129.
12. Samulski RJ et al. Rescue of adeno-associated virus from recombinant plasmids: gene correction within the terminal repeats of AAV. Cell 1983; 33: 135-143.
13. Hermonat PL, Muzyczka N. Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells. Proc Natl Acad Sci USA 1984; 81: 6466-6470.
14. Berns KI. The Parvoviruses. Plenum Press: New York, 1984.
15. Chejanovsky N, Carter BJ. Mutagenesis of an AUG codon in the adeno-associated virus rep gene: effects on viral DNA replication. Virology 1989; 173: 120-128.
16. Samulski RJ, Chang LS, Shenk T. A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J Virol 1987; 61: 3096-3101.
17. Carter BJ. The growth of adeno-associated virus. In: Tijssen P (ed). Handbook of Parvoviruses. CRC Press: Boca Raton, 1990, pp 155-168.
18. Huang MM, Hearing P. Adenovirus early region 4 encodes two gene products with redundant effects in lytic infection. J Virol 1989; 63: 2605-2615.
19. Samulski RJ, Shenk T. Adenovirus E1B 55-Mr polypeptide facilitates timely cytoplasmic accumulation of adeno-associated virus mRNAs. J Virol 1988; 62: 206-210.
20. Xiao X, Li J, Samulski RJ. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol 1998; 72: 2224-2232.
21. Matsushita T et al. Adeno-associated virus vectors can be efficienty produced without helper virus. Gene Therapy 1998; 5: 938-945.
22. Clark KR et al. Cell lines for the production of recombinant adeno-associated virus. Hum Gene Ther 1995; 6: 1329-1341.
23. Vincent AJ et al. Preclinical testing of recombinant adenoviral herpes simplex virus-thymidine kinase gene therapy for central nervous system malignancies. Neurosurgery 1997; 41: 442-451.
24. Li J, Samulski RJ, Xiao X. Role for highly regulated rep gene expression in adeno-associated virus vector production. J Virol 1997; 71: 5236-5243.
25. Rose JA, Koczot F. Adenoviurs-associated virus multiplication VII. Helper requirement for viral deoxyribonucleic acid and ribonucleic acid systhesis. J Virol 1972; 10: 1-8.
26. Buller RML. Herpes simplex virus types 1 and 2 completely help adnovirus-associated virus replication. J Virol 1981; 40: 241-247.
27. Mishra L, Rose JA. Adeno-associated virus DNA replication is induced by genes that are essential for HSV-1 DNA synthesis. Virology 1990; 179: 632-639.
28. Weindler FW, Heilbronn R. A subset of herpes simplex virus replication genes provides helper functions for productive adeno-associated virus replication. J Virol 1991; 65: 2476-2483.
29. Knipe DM. The role of viral and cellular nuclear proteins in herpes simplex virus replication (review). Adv Virus Res 1989; 37: 85-123.
30. Weller SK. Genetic analysis of HSV-1 gene required for genome replication. In: Wagner EK (ed). Herpes Virus Transcription and its Regulation. CRC Press: Boca Raton, 1991, pp 105-136.
31. Rice SA, Knipe DM. Genetic evidence for two distinct transactivation functions of the herpes simplex virus alpha protein ICP27. J Virol 1990; 64: 1704-1715.
32. Conway JE et al. Recombinant adeno-associated virus replication and packaging is supported by an HSV amplicon expressing Rep and Cap. J Virol 1997; 71: 8780-8789.
33. Carter BJ et al. Properties of an adenovirus type 2 mutant, Ad2dl807, having a deletion near the right-hand genome terminus: failure to help AAV replication. Virology 1983; 126: 505-516.
34. Green MR, Roeder RG. Transcripts of the adeno-associated virus genome: mapping of the major RNAs. J Virol 1980; 36: 79-92.
35. Laughlin CA et al. Defective-interfering particles of the human parvovirus adeno-associated virus. Virology 1979; 94: 162-174.
36. Lusby E, Fife KH, Berns KI. Nucleotide sequence of the inverted terminal repetition in adeno-associated virus DNA. J Virol 1980; 34: 402-409.
37. Marcus CJ, Laughlin CA, Carter BJ. Adeno-associated virus RNA transcription in vivo. Eur J Biochem 1981; 121: 147-154.
38. Khleif SN et al. Inhibition of cellular transformation by the adeno-associated virus rep gene. Virology 1991; 181: 738-741.
39. Heilbronn R et al. The adeno-associated virus rep gene suppresses herpes simplex virus-induced DNA amplification. J Virol 1990; 64: 3012-3018.
40. Weitzman MD et al. Interaction of wild-type and mutant adenoassociated virus (AAV) Rep proteins on AAV hairpin DNA. J Virol 1996; 70: 2440-2448.
41. Weitzman MD, Fisher KJ, Wilson JM. Recruitment of wild-type and recombinant adeno-associated virus into adenovirus replication centers. J Virol 1996; 70: 1845-1854.
42. Quinlan MP, Chen LB, Knipe DM. The intranuclear location of a herpes simplex virus DNA binding protein is determined by the status of viral DNA replication. Cell 1984; 36: 857-868.
43. Liptak LM, Uprichard SL, Knipe DM. Functional order of assembly of herpes simplex virus DNA replication proteins into prereplicative site structures. J Virol 1996; 70: 1759-1767.
44. Lukonis CJ, Weller SK. Characterization of nuclear structures in cells infected with herpes simplex virus type 1 in the absence of viral DNA replication. J Virol 1996; 70: 1751-1758.
45. Zhong L, Hayward GS. Assembly of complete functionally active herpes simplex virus DNA replication compartements and recruitement of associated viral and cellular proteins in transient cotransfection assays. J Virol 1997; 71: 3146-3160.
46. Zolotukhin S et al. A 'humanized' green fluorescent protein cDNA adapted for high-level expression in mammalian cells. J Virol 1996; 70: 4646-4654.
47. Sandri-Goldin RM, Mendoza GE. A herpesvirus regulatory protein appears to act post-transcriptionally by affecting mRNA processing. Genes Dev 1992; 6: 848-863.
48. McLauchlan J et al. Herpes simplex virus IE63 acts at the post-transcriptional level to stimulate viral mRNA 3′ processing. J Virol 1992; 66: 6939-6945.
49. Johnson LG et al. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat Genet 1992; 2: 21-25.
50. Johnson NW, Curtis MA. Preventive therapy for periodontal diseases (review). Adv Dental Res 1994; 8: 337-348.
51. Weindler FW, Heilbronn R. A subset of herpes simplex virus replication genes provides helper functions for productive adeno-associated virus replication. J Virol 1991; 65: 2476-2483.
52. Clark KR, Voulgaropoulou F, Johnson PR. A stable cell line carrying adenovirus-inducible rep and cap genes allows for infectivity titration of adeno-associated virus vectors. Gene Therapy 1996; 3: 1124-1132.