Site-specific integration of functional transgenes into the human genome by adeno/AAV hybrid vectors

Molecular Therapy

Volumn 10, Issue 4, 2004, Pages 660-670

  Recchia, Alessandra ^a   Perani, Laura ^a   Sartori, Daniela ^a   Olgiati, Clelia ^a   Mavilio, Fulvio ^b,c  

^a SAN RAFFAELE HOSPITAL   (Italy)

^b UNIVERSITY OF MODENA AND REGGIO EMILIA   (Italy)

^c MOLMED S P A   (Italy)

Author keywords

Adeno associated virus; Adenovirus; Gene transfer; Site specific integration; Viral vectors

Indexed keywords

ADENOVIRUS VECTOR; INTEGRASE; PARVOVIRUS VECTOR; RNA DIRECTED RNA POLYMERASE; SPACER DNA; VIRUS VECTOR;

ANIMAL MODEL; ARTICLE; CHROMOSOME 19; DNA INTEGRATION; DNA SEQUENCE; DRUG SAFETY; DRUG USE; EX VIVO STUDY; FEMALE; GENE CASSETTE; GENE FUNCTION; GENE INSERTION; GENE TRANSFER; HUMAN; HUMAN CELL; HUMAN CHROMOSOME; HUMAN GENOME; IN VIVO STUDY; INVERTED TERMINAL REPEAT; MALE; MOUSE; NONHUMAN; PROTEIN DNA BINDING; PROTEIN EXPRESSION; TRANSGENE; TRANSGENIC MOUSE; VIRAL GENE THERAPY; VIRUS GENOME; VIRUS HYBRID; WILD TYPE;

ADENOVIRIDAE; ANIMALS; BASE SEQUENCE; CELL LINE; CHROMOSOMES, HUMAN, PAIR 19; DEPENDOVIRUS; DNA-BINDING PROTEINS; FEMALE; GENE EXPRESSION REGULATION; GENE TARGETING; GENE TRANSFER TECHNIQUES; GENES, REPORTER; GENETIC VECTORS; GENOME, HUMAN; GREEN FLUORESCENT PROTEINS; HUMANS; KERATINOCYTES; MALE; MICE; MICE, TRANSGENIC; MOLECULAR SEQUENCE DATA; RNA, MESSENGER; TERMINAL REPEAT SEQUENCES; TRANSGENES; VIRAL PROTEINS;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; ANIMALIA; INSERTION SEQUENCES; MUS MUSCULUS;

EID: 4644285407     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.07.003     Document Type: Article

References (34)

1. A.R. Schroder P. Shinn H. Chen C. Berry J.R. Ecker F. Bushman HIV-1 integration in the human genome favors active genes and local hotspots Cell 110 2002 521-529
2. X. Wu Y. Li B. Crise S.M. Burgess Transcription start regions in the human genome are favored targets for MLV integration Science 300 2003 1749-1751
3. C.E. Thomas A. Ehrhardt M.A. Kay Progress and problems with the use of viral vectors for gene therapy Nat. Rev. Genet. 4 2003 346-358
4. H.H. Chen L.M. Mack R. Kelly M. Ontell S. Kochanek P.R. Clemens Persistence in muscle of an adenoviral vector that lacks all viral genes Proc. Natl. Acad. Sci. USA 94 1997 1645-1650
5. G. Schiedner et al. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity Nat. Genet. 18 1998 180-183
6. M.A. Morsy et al. An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene Proc. Natl. Acad. Sci. USA 95 1998 7866-7871
7. N. Morral et al. High doses of a helper-dependent adenoviral vector yield supraphysiological levels of alpha1-antitrypsin with negligible toxicity Hum. Gene Ther. 9 1998 2709-2716
8. N. Morral et al. Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons Proc. Natl. Acad. Sci. USA 96 1999 12816-12821
9. C.E. Thomas G. Schiedner S. Kochanek M.G. Castro P.R. Lowenstein Peripheral infection with adenovirus causes unexpected long-term brain inflammation in animals injected intracranially with first-generation, but not with high-capacity, adenovirus vectors: toward realistic long-term neurological gene therapy for chronic diseases Proc. Natl. Acad. Sci. USA 97 2000 7482-7487
10. I.H. Kim A. Jozkowicz P.A. Piedra K. Oka L. Chan Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector Proc. Natl. Acad. Sci. USA 98 2001 13282-13287
11. K.I. Berns R.M. Linden The cryptic life style of adeno-associated virus Bioessays 17 1995 237-245
12. R.M. Kotin et al. Site-specific integration by adeno-associated virus Proc. Natl. Acad. Sci. USA 87 1990 2211-2215
13. R.J. Samulski et al. Targeted integration of adeno-associated virus (AAV) into human chromosome 19 10 1991 3941-3950
14. R.M. Linden P. Ward C. Giraud E. Winocour K.I. Berns Site-specific integration by adeno-associated virus Proc. Natl. Acad. Sci. USA 93 1996 11288-11294
15. C.H. Miao et al. The kinetics of rAAV integration in the liver Nat. Genet. 19 1998 13-15
16. S. Ponnazhagan et al. Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells Hum. Gene Ther. 8 1997 275-284
17. C.C. Yang et al. Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro J. Virol. 71 1997 9231-9247
18. D.G. Miller E.A. Rutledge D.W. Russell Chromosomal effects of adeno-associated virus vector integration Nat. Genet. 30 2002 147-148
19. F. Palombo A. Monciotti A. Recchia R. Cortese G. Ciliberto N. La Monica Site-specific integration in mammalian cells mediated by a new hybrid baculovirus-adeno-associated virus vector J. Virol. 72 1998 5025-5034
20. + hybrid amplicon vector improves the stability of transgene expression in human cells by site-specific integration J. Virol. 76 2002 7150-7162
21. T. Heister I. Heid M. Ackermann C. Fraefel Herpes simplex virus type 1/adeno-associated virus hybrid vectors mediate site-specific integration at the adeno-associated virus preintegration site, AAVS1, on human chromosome 19 J. Virol. 76 2002 7163-7173
22. A. Recchia et al. Site-specific integration mediated by a hybrid adenovirus/adeno-associated virus vector Proc. Natl. Acad. Sci. USA 96 1999 2615-2620
23. B.A. Antoni A.B. Rabson I.L. Miller J.P. Trempe N. Chejanovsky B.J. Carter Adeno-associated virus Rep protein inhibits human immunodeficiency virus type 1 production in human cells J. Virol. 65 1991 396-404
24. P.L. Hermonat Inhibition of bovine papillomavirus plasmid DNA replication by adeno-associated virus Virology 189 1992 329-333
25. M.D. Weitzman K.J. Fisher J.M. Wilson Recruitment of wild-type and recombinant adeno-associated virus into adenovirus replication centers J. Virol. 70 1996 1845-1854
26. S. Freundlieb C. Schirra-Muller H. Bujard A tetracycline controlled activation/repression system with increased potential for gene transfer into mammalian cells J. Gene Med. 1 1999 4-12
27. G. Rizzuto et al. Development of animal models for adeno-associated virus site-specific integration J. Virol. 73 1999 2517-2526
28. S. Hacein-Bey-Abina et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 Science 302 2003 415-419
29. H. Nakai Y. Iwaki M.A. Kay L.B. Couto Isolation of recombinant adeno-associated virus vector - cellular DNA junctions from mouse liver J. Virol. 73 1999 5438-5447
30. H. Nakai E. Montini S. Fuess T.A. Storm M. Grompe M.A. Kay AAV serotype 2 vectors preferentially integrate into active genes in mice Nat. Genet. 34 2003 297-302
31. D.W. Russell AAV loves an active genome Nat. Genet. 34 2003 241-242
32. M. Schmidt S. Afione R.M. Kotin Adeno-associated virus type 2 Rep78 induces apoptosis through caspase activation independently of p53 J. Virol. 74 2000 9441-9450
33. S.M. Young Jr. R.J. Samulski Adeno-associated virus (AAV) site-specific recombination does not require a Rep-dependent origin of replication within the AAV terminal repeat Proc. Natl. Acad. Sci. USA 98 2001 13525-13530
34. D. Huser S. Weger R. Heilbronn Kinetics and frequency of adeno-associated virus site-specific integration into human chromosome 19 monitored by quantitative real-time PCR J. Virol. 76 2002 7554-7559