Clearance and characterization of residual HSV DNA in recombinant adeno-associated virus produced by an HSV complementation system

Gene Therapy

Volumn 18, Issue 2, 2011, Pages 135-144

  Ye, G J ^a   Scotti, M M ^a   Liu, J ^a   Wang, L ^a   Knop, D R ^a   Veres, G ^a  

^a APPLIED GENETIC TECHNOLOGIES CORPORATION   (United States)

Author keywords

adeno associated virus; DNA packaging; gene transfer; herpes simplex virus; rAAV production

Indexed keywords

DEOXYRIBONUCLEASE; DNA FRAGMENT; SINGLE STRANDED DNA; VIRUS DNA; VIRUS PROTEIN;

ADENO ASSOCIATED VIRUS; ADENOVIRUS; ARTICLE; CELL STRAIN HEK293; CHROMATOGRAPHY; COMPLEMENT SYSTEM; DNA FLANKING REGION; DNA SEQUENCE; GENETIC COMPLEMENTATION; HERPES SIMPLEX VIRUS; HUMAN; NONHUMAN; PRIORITY JOURNAL; VIRAL CLEARANCE; VIRUS CHARACTERIZATION; VIRUS PURIFICATION;

ANIMALS; CELL LINE; CRICETINAE; DEPENDOVIRUS; DNA, VIRAL; ENDODEOXYRIBONUCLEASES; ENDORIBONUCLEASES; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEK293 CELLS; HUMANS; RECOMBINATION, GENETIC; SIMPLEXVIRUS; TRANSDUCTION, GENETIC;

ADENO-ASSOCIATED VIRUS; SIMPLEXVIRUS;

EID: 79951512054     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2010.102     Document Type: Article

References (27)

1. Muzyczka N, Berns KI. Parvoviridae: the viruses and their replication. In: Knipe DM, Howley PM (eds). Fields Virology. Lippincott Williams & Wilkins: Philadelphia, 2001, pp 2327-2359.
2. Jiang H, Pierce GF, Ozelo MC, De Paula EV, Vargas JA, Smith P et al. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual wit (Pubitemid 44184967)
3. Kaplitt MG, Feigin A, Tang C, Fitzsimons HL, Mattis P, Lawlor PA et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 2007; 369: 2097-2105. (Pubitemid 46935946)
4. Worgall S, Sondhi D, Hackett NR, Kosofsky B, Kekatpure MV, Neyzi N et al. Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum Gene Ther 2008; 19: 463-474. (Pubitemid 351748791)
5. Brantly ML, Spencer LT, Humphries M, Conlon TJ, Spencer CT, Poirier A et al. Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alpha-1 antitrypsin (AAT) vector in AAT-deficient adults. Hum Gene Ther 2006; 17: 1177-1186. (Pubitemid 44975135)
6. Samulski RJ, Chang LS, Shenk T. Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J Virol 1989; 63: 3822-3828. (Pubitemid 19205454)
7. Nony P, Chadeuf G, Tessier J, Moullier P, Salvetti A. Evidence for packaging of rep-cap sequences into adeno-associated virus (AAV) type 2 capsids in the absence of inverted terminal repeats: a model for generation of rep-positive AAV particles. J Virol 2003; 77: 776-781. (Pubitemid 36005019)
8. Chadeuf G, Ciron C, Moullier P, Salvetti A. Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery. Mol Ther 2005; 12: 744-753. (Pubitemid 41350156)
9. Hauck B, Murphy SL, Smith PH, Qu G, Liu X, Zelenaia O et al. Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses. Mol Ther 2009; 17: 144-152.
10. Levy HC, Bowman VD, Govindasamy L, McKenna R, Nash K, Warrington K et al. Heparin binding induces conformational changes in Adeno-associated virus serotype 2. J Struct Biol 2009; 165: 146-156.
11. Kang W, Wang L, Harrell H, Liu J, Thomas DL, Mayfield TL et al. An efficient rHSV-based complementation system for the production of multiple rAAV vector serotypes. Gene Ther 2009; 16: 229-239.
12. Thomas DL, Wang L, Niamke J, Liu J, Kang W, Scotti MM et al. Scalable rAAV production using rHSV co-infection of suspension adapted mammalian cells. Hum Gene Ther 2009; 20: 861-870.
13. Chang YE, Van Sant C, Krug PW, Sears AE, Roizman B. The null mutant of the U(L)31 gene of herpes simplex virus 1: construction and phenotype in infected cells. J Virol 1997; 71: 8307-8315. (Pubitemid 27446407)
14. Roizman BKDM (ed). Herpes Simplex Viruses and Their Replication. Lippincott Williams & Wilkins: Philadelphia, 2001, pp 2399-2459.
15. Wright JF. Transient transfection methods for clinical adeno-associated viral vector production. Hum Gene Ther 2009; 20: 698-706.
16. Thorne BA, Takeya RK, Peluso RW. Manufacturing recombinant adeno-associated viral vectors from producer cell clones. Hum Gene Ther 2009; 20: 707-714.
17. Virag T, Cecchini S, Kotin RM. Producing recombinant adeno-associated virus in foster cells: overcoming production limitations using a baculovirus-insect cell expression strategy. Hum Gene Ther 2009; 20: 807-817.
18. Clement N, Knop DR, Byrne BJ. Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies. Hum Gene Ther 2009; 20: 796-806.
19. Thomas DL, Wang L, Niamke J, Liu J, Kang W, Scotti MM et al. Scalable recombinant adeno-associated virus production using recombinant herpes simplex virus type 1 coinfection of suspension-adapted mammalian cells. Hum Gene Ther 2009; 20: 861-870.
20. Grimm D, Kern A, Rittner K, Kleinschmidt JA. Novel tools for production and purification of recombinant adenoassociated virus vectors. Hum Gene Ther 1998; 9: 2745-2760. (Pubitemid 29027018)
21. McCarthy AM, McMahan L, Schaffer PA. Herpes simplex virus type 1 ICP27 deletion mutants exhibit altered patterns of transcription and are DNA deficient. J Virol 1989; 63: 18-27. (Pubitemid 19028077)
22. Uprichard SL, Knipe DM. Herpes simplex ICP27 mutant viruses exhibit reduced expression of specific DNA replication genes. J Virol 1996; 70: 1969-1980. (Pubitemid 26062962)
23. De Bruyn G, Vargas-Cortez M, Warren T, Tyring SK, Fife KH, Lalezari J et al. A randomized controlled trial of a replication defective (gH deletion) herpes simplex virus vaccine for the treatment of recurrent genital herpes among immunocompetent subjects. Vaccine 2006; 24: 914-920. (Pubitemid 43185307)
24. Duff R, Rapp F. Oncogenic transformation of hamster embryo cells after exposure to inactivated herpes simplex virus type 1. J Virol 1973; 12: 209-217.
25. 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.
26. Hoggan MD, Blacklow NR, Rowe WP. Studies of small DNA viruses found in various adenovirus preparations: physical, biological, and immunological characteristics. Proc Natl Acad Sci USA 1966; 55: 1467-1474.
27. De la Maza LM, Carter BJ. Heavy and light particles of adeno-associated virus. J Virol 1980; 33: 1129-1137. (Pubitemid 10148587)