Robust, persistent transgene expression in human embryonic stem cells is achieved with AAVS1-targeted integration

Stem Cells

Volumn 26, Issue 2, 2008, Pages 496-504

  Smith, Joseph R ^a,b,e   Maguire, Sean ^c   Davis, Lesley A ^a,b   Alexander, Morgan ^a,b   Yang, Fentang ^c   Chandran, Siddharthan ^d   Ffrench Constant, Charles ^a,d   Pedersen, Roger A ^a,b  

^a Wellcome Trust Genome Campus ^*  (United Kingdom)

^b UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^c WELLCOME TRUST SANGER INSTITUTE   (United Kingdom)

^d UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^e ADDENBROOKE S HOSPITAL   (United Kingdom)

Author keywords

Adeno associated virus; Human embryonic stem cells; Stable Gene Expression; Targeting

Indexed keywords

ENHANCED GREEN FLUORESCENT PROTEIN; OCTAMER TRANSCRIPTION FACTOR 4; STAGE SPECIFIC EMBRYO ANTIGEN 3;

ADENOVIRUS 2; ANALYTIC METHOD; ARTICLE; CELL CLONE; CELL DIFFERENTIATION; CHROMOSOME 19; COMPARATIVE STUDY; CONTROLLED STUDY; EMBRYONIC STEM CELL; GENE LOCUS; GENE SILENCING; HUMAN; HUMAN CELL; POLYMERASE CHAIN REACTION; TRANSGENE;

CELL DIFFERENTIATION; CELL LINE; CHROMOSOMES, HUMAN, PAIR 19; DEPENDOVIRUS; EMBRYONIC STEM CELLS; GENE EXPRESSION; GENE SILENCING; GENES, REPORTER; GREEN FLUORESCENT PROTEINS; HUMANS; IN SITU HYBRIDIZATION, FLUORESCENCE; PHENOTYPE; RECOMBINANT PROTEINS; TRANSFECTION; VIRUS INTEGRATION;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS - 2;

EID: 40949126488     PISSN: 10665099     EISSN: None     Source Type: Journal    
DOI: 10.1634/stemcells.2007-0039     Document Type: Article

References (30)

1. Vallier L, Rugg-Gunn PJ, Bouhon IA et al. Enhancing and diminishing gene function in human embryonic stem cells. STEM CELLS 2004;22:2-11.
2. Vallier L, Reynolds D, Pedersen RA. Nodal inhibits differentiation of human embryonic stem cells along the neuroectodermal default pathway. Dev Biol 2004;275:403-421.
3. Smith JR, Vallier L, Lupo G et al. Inhibition of Activin/Nodal signaling promotes specification of human embryonic stem cells into neuroectoderm. Dev Biol 2008;318:107-117.
4. Cherry SR, Biniszkiewicz D, van Parijs L et al. Retroviral expression in embryonic stem cells and hematopoietic stem cells. Mol Cell Biol 2000;20:7419-7426.
5. Laker C, Meyer J, Schopen A et al. Host cis-mediated extinction of a retrovirus permissive for expression in embryonal stem cells during differentiation. J Virol 1998;72:339-348.
6. Yao S, Sukonnik T, Kean T et al. Retrovirus silencing, variegation, extinction, and memory are controlled by a dynamic interplay of multiple epigenetic modifications. Mol Ther 2004;10:27-36.
7. He J, Yang Q, Chang LJ. Dynamic DNA methylation and histone modifications contribute to lentiviral transgene silencing in murine embryonic carcinoma cells. J Virol 2005;79:13497-13508.
8. Zwaka T, Thomson JA. Homologous recombination in human embryonic stem cells. Nat Biotechnol 2003;21:319-321.
9. Urbach A, Schuldiner M, Benvenisty N. Modeling for Lesch-Nyhan disease by gene targeting in human embryonic stem cells. STEM CELLS 2004;22:635-641.
10. Ellis J. Silencing and variegation of gammaretrovirus and lentivirus vectors. Hum Gene Ther 2005;16:1241-1246.
11. Liew CG, Draper JS, Walsh J et al. Transient and stable transgene expression in human embryonic stem cells. STEM CELLS 2007;25:1521-1528.
12. Gropp M, Itsykson P, Singer O et al. Stable genetic modification of human embryonic stem cells by lentiviral vectors. Mol Ther 2003;7:281-287.
13. Ma Y, Ramezani A, Lewis R et al. High level sustained transgene expression in human embryonic stem cells using lentiviral vectors. STEM CELLS 2003;21:111-117.
14. Wu X, Li Y, Crise B et al. Transcription start regions in the human genome are favored targets for MLV integration. Science 2003;300:1749-1751.
15. Hacein-Bey-Abina S, Von Kalle C, Schmidt M et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302:415-419.
16. Ledermann B. Embryonic stem cells and gene targeting. Exp Physiol 2000;85:603-613.
17. Cheung AK, Hoggan MD, Hauswirth WW et al. Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells. J Virol 1980;33:739-748.
18. Weitzman MD, Kyöstiö SR, Kotin RM et al. Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA. Proc Natl Acad Sci U S A 1994;91:5808-5812.
19. Philpott NJ, Gomos J, Berns KI et al. A p5 integration efficiency element mediates Rep-dependent integration into AAVS1 at chromosome 19. Proc Natl Acad Sci U S A 2002;99:12381-12385.
20. McCarty DM, Young SM Jr, Samulski RJ. Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet 2004;38:819-845.
21. Philpott NJ, Gomos J, Falck-Pedersen E. Transgene expression after rep-mediated site-specific integration into chromosome 19. Hum Gene Ther 2004;15:47-61.
22. Fu Y, Wang Y, Evans SM. Viral sequences enable efficient and tissue-specific expression of transgenes in Xenopus. Nat Biotechnol 1998;16:253-257.
23. Hsiao CD, Hsieh FJ, Tsai HJ. Enhanced expression and stable transmission of transgenes flanked by inverted terminal repeats from adeno-associated virus in zebrafish. Dev Dyn 2001;220:323-336.
24. Ogata T, Kozuka T, Kanda T. Identification of an insulator in AAVS1, a preferred region for integration of adeno-associated virus DNA. J Virol 2003;77:9000-9007.
25. Schatten G, Smith J, Navara C et al. Culture of human embryonic stem cells. Nat Methods 2005;2:455-463.
26. Recchia A, Perani L, Sartori D et al. Site-specific integration of functional transgenes into the human genome by adeno/AAV hybrid vectors. Mol Ther 2004;10:660-670.
27. Yue Y, Grossmann B, Ferguson-Smith M et al. Comparative cytogenetics of human chromosome 3q21.3 reveals a hot spot for ectopic recombination in hominoid evolution. Genomics 2005;85:36-47.
28. Huser D, Weger S, Heilbronn R. et al. Kinetics and frequency of adeno-associated virus site-specific integration into human chromosome 19 monitored by quantitative real-time PCR. J Virol 2002;76:7554-7559.
29. Costa M, Dottori M, Ng E et al. The hESC line Envy expresses high levels of GFP in all differentiated progeny. Nat Methods 2005;2:259-260.
30. Vroemen M, Weidner N, Blesch A. Loss of gene expression in lentivirus- and retrovirus-transduced neural progenitor cells is correlated to migration and differentiation in the adult spinal cord. Exp Neurol 2005;195:127-139.