Episomal Transgene Expression in Pluripotent Stem Cells

Methods in Molecular Biology

Volumn 767, Issue , 2011, Pages 369-387

  Lufino, Michele M P ^a   Popplestone, Anna R ^a   Cowley, Sally A ^b   Edser, Pauline A H ^a   James, William S ^b   Wade Martins, Richard ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

Extrachromosomal vector; Gene expression vector; HSV 1 amplicon; iBAC; Stem cells

Indexed keywords

DNA;

ANIMAL; ARTICLE; CELL CLONE; CELL DIFFERENTIATION; CELL LINE; CYTOLOGY; DENSITY GRADIENT CENTRIFUGATION; DOPAMINERGIC NERVE CELL; EMBRYONIC STEM CELL; FILTRATION; GENE EXPRESSION; GENE TRANSFER; GENE VECTOR; GENETICS; HERPES SIMPLEX VIRUS 1; HUMAN; METABOLISM; MOUSE; PHYSIOLOGY; PLASMID; PLURIPOTENT STEM CELL; TITRIMETRY; TRANSGENE; VIRION; VIROLOGY; VIRUS ASSEMBLY;

ANIMALS; CELL DIFFERENTIATION; CELL LINE; CENTRIFUGATION, DENSITY GRADIENT; CLONE CELLS; DNA; DOPAMINERGIC NEURONS; EMBRYONIC STEM CELLS; FILTRATION; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HERPESVIRUS 1, HUMAN; HUMANS; MICE; PLASMIDS; PLURIPOTENT STEM CELLS; TITRIMETRY; TRANSGENES; VIRION; VIRUS ASSEMBLY;

HERPES; HUMAN HERPESVIRUS 1;

EID: 80054732670     PISSN: 10643745     EISSN: 19406029     Source Type: Book Series    
DOI: 10.1007/978-1-61779-201-4_27     Document Type: Chapter

References (16)

1. Spaete, R. R., and Frenkel, N. (1982) The herpes simplex virus amplicon: a new eucary-otic defective-virus cloning-amplifying vector Cell 30, 295–304.
2. Fraefel, C., Song, S., Lim, F., Lang, P., Yu, L., Wang, Y., Wild, P., and Geller, A. I. (1996) Helper virus-free transfer of herpes simplex virus type 1 plasmid vectors into neural cells J Virol 70, 7190–7.
3. Saeki, Y., Fraefel, C., Ichikawa, T., Breakefield, X. O., and Chiocca, E. A. (2001) Improved helper virus-free packaging system for HSV amplicon vectors using an ICP27-deleted, oversized HSV-1 DNA in a bacterial artificial chromosome Mol Ther 3, 591–601.
4. Wade-Martins, R., Smith, E. R., Tyminski, E., Chiocca, E. A., and Saeki, Y. (2001) An infectious transfer and expression system for genomic DNA loci in human and mouse cells Nat Biotechnol 19, 1067–70.
5. Wang, S., and Vos, J. M. (1996) A hybrid her-pesvirus infectious vector based on Epstein-Barr virus and herpes simplex virus type 1 for gene transfer into human cells in vitro and in vivo J Virol 70, 8422–30.
6. Piechaczek, C., Fetzer, C., Baiker, A., Bode, J., and Lipps, H. J. (1999) A vector based on the SV40 origin of replication and chromosomal S/MARs replicates episomally in CHO cells Nucleic Acids Res 27, 426–8.
7. Lufino, M. M., Manservigi, R., and Wade-Martins, R. (2007) An S/MAR-based infectious episomal genomic DNA expression
8. Moralli, D., Simpson, K. M., Wade-Martins, R., and Monaco, Z. L. (2006) A novel human artificial chromosome gene expression system using herpes simplex virus type 1 vectors EMBO Rep 7, 911–8.
9. Hacein-Bey-Abina, S., von Kalle, C., Schmidt, M., Le Deist, F., Wulffraat, N., McIntyre, E., Radford, I., Villeval, J. L., Fraser, C. C., Cavazzana-Calvo, M., and Fischer, A. (2003) A serious adverse event after successful gene therapy for X-linked severe combined immu-nodeficiency N Engl J Med 348, 255–6.
10. Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., McCormack, M. P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C. S., Pawliuk, R., Morillon, E., Sorensen, R., Forster, A., Fraser, P., Cohen, J. I., de Saint Basile, G., Alexander, I., Wintergerst, U., Frebourg, T., Aurias, A., Stoppa-Lyonnet, D., Romana, S., Radford-Weiss, I., Gross, F., Valensi, F., Delabesse, E., Macintyre, E., Sigaux, F., Soulier, J., Leiva, L. E., Wissler, M., Prinz, C., Rabbitts, T. H., Le Deist, F., Fischer, A., and Cavazzana-Calvo, M. (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 Science 302, 415–9.
11. Hibbitt, O. C., Harbottle, R. P., Waddington, S. N., Bursill, C. A., Coutelle, C., Channon, K. M., and Wade-Martins, R. (2007) Delivery and long-term expression of a 135 kb LDLR genomic DNA locus in vivo by hydrodynamic tail vein injection J Gene Med 9, 488–97.
12. Wade-Martins, R., Saeki, Y., and Chiocca, E. A. (2003) Infectious delivery of a 135-kb LDLR genomic locus leads to regulated complementa-tion of low-density lipoprotein receptor deficiency in human cells Mol Ther 7, 604–12.
13. Bowers, W. J., and Federoff, H. J. (2006) Herpes simplex virus type 1-derived amplicon vectors, Gene Transfer: Delivery and Expression of DNA and RNA, A Laboratory Manual, pg. 227–254, Cold Spring Harbor Press, Cold Spring Harbor, NY.
14. Watanabe, K., Ueno, M., Kamiya, D., Nishiyama, A., Matsumura, M., Wataya, T., Takahashi, J. B., Nishikawa, S., Nishikawa, S., Muguruma, K., and Sasai, Y. (2007) A ROCK inhibitor permits survival of dissociated human embryonic stem cells Nat Biotechnol 25, 681–6.
15. Ludwig, T. E., Bergendahl, V., Levenstein, M. E., Yu, J., Probasco, M. D., and Thomson, J. A. (2006) Feeder-independent culture of human embryonic stem cells Nat Methods 3, 637–46.
16. Lufino, M. M., Edser, P. A., and Wade-Martins, R. (2008) Advances in high-capac-ity extrachromosomal vector technology: episomal maintenance, vector delivery, and transgene expression Mol Ther 16, 1525–38.