Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters

Nature Biotechnology

Volumn 23, Issue 1, 2005, Pages 108-116

  Amendola, Mario ^a,b   Venneri, Mary Anna ^a,c   Biffi, Alessandra ^a   Vigna, Elisa ^c   Naldini, Luigi ^a,b  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^c UNIVERSITY OF TURIN   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

BIOTECHNOLOGY; GENETIC ENGINEERING; VECTORS;

MULTISUBUNIT COMPONENTS; TRANSGENESIS;

GENES;

GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR; CD34 ANTIGEN; LUCIFERASE; MESSENGER RNA;

ANIMAL CELL; ARTICLE; GENE EXPRESSION; GENE FUNCTION; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETIC TRANSCRIPTION; GENOME; HELA CELL; HUMAN; HUMAN CELL; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; PLASMID; PRIORITY JOURNAL; PROMOTER REGION; RNA TRANSCRIPTION; TRANSGENE; TRANSGENICS; BIOSYNTHESIS; CELL CULTURE; CELL LINE; CELL SEPARATION; CONFOCAL MICROSCOPY; FLOW CYTOMETRY; FLUORESCENCE MICROSCOPY; GENETICS; LENTIVIRINAE; METABOLISM; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SOUTHERN BLOTTING; WESTERN BLOTTING;

ANIMALIA; LENTIVIRUS;

ANTIGENS, CD34; BLOTTING, SOUTHERN; BLOTTING, WESTERN; CELL LINE; CELL SEPARATION; CELLS, CULTURED; FLOW CYTOMETRY; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HELA CELLS; HUMANS; LENTIVIRUS; LUCIFERASES; MICROSCOPY, CONFOCAL; MICROSCOPY, FLUORESCENCE; PLASMIDS; PROMOTER REGIONS (GENETICS); REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA, MESSENGER; TRANSCRIPTION, GENETIC; TRANSGENES;

EID: 15244351083     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt1049     Document Type: Article

References (32)

1. Kay, M.A., Glorioso, J.C. & Naldini, L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat. Med. 7, 33-40 (2001).
2. Klump, H. et al. Retroviral vector-mediated expression of HoxB4 in hematopoietic cells using a novel coexpression strategy. Gene Ther. 8, 811-817 (2001).
3. Emerman, M. & Temin, H.M. Quantitative analysis of gene suppression in integrated retrovirus vectors. Mol. Cell Biol. 6, 792-800 (1986).
4. Miller, A.D. in Retroviruses (eds. Coffin, J., Hughes, S.H. & Varmus, H.E.) 437-474 (Cold Spring Harbor Laboratory Press, Cold Spring Harbor, NY, 2000) 10.
5. Furler, S., Paterna, J.C., Weibel, M. & Bueler, H. Recombinant AAV vectors containing the foot and mouth disease virus 2A sequence confer efficient bicistronic gene expression in cultured cells and rat substantia nigra neurons. Gene Ther. 8, 864-873 (2001).
6. Martinez-Salas, E. Internal ribosome entry site biology and its use in expression vectors. Curr. Opin. Biotechnol. 10, 458-464 (1999).
7. Sokolic, R.A. et al. A bicistronic retrovirus vector containing a picornavirus internal ribosome entry site allows for correction of X-linked CGD by selection for MDR1 expression. Blood 87, 42-50 (1996).
8. Mizuguchi, H., Xu, Z., Ishii-Watabe, A., Uchida, E. & Hayakawa, T. IRES-dependent second gene expression is significantly lower than cap-dependent first gene expression in a bicistronic vector. Mol. Ther. 1, 376-382 (2000).
9. Hennecke, M. et al. Composition and arrangement of genes define the strength of IRES-driven translation in bicistronic mRNAs. Nucleic Acids Res. 29, 3327-3334 (2001).
10. Kozak, M. Alternative ways to think about mRNA sequences and proteins that appear to promote internal initiation of translation. Gene 318, 1-23 (2003).
11. Follenzi, A., Ailles, L.E., Bakovic, S., Geuna, M. & Naldini, L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat. Genet. 25, 217-222 (2000).
12. Lois, C., Hong, E.J., Pease, S., Brown, E.J. & Baltimore, D. Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors. Science 295, 868-872 (2002).
13. Baron, U., Freundlieb, S., Gossen, M. & Bujard, H. Co-regulation of two gene activities by tetracycline via a bidirectional promoter. Nucleic Acids Res. 23, 3605-3606 (1995).
14. Bray, M. et al. A small element from the Mason-Pfizer monkey virus genome makes human immunodeficiency virus type 1 expression and replication Rev-independent. Proc. Natl. Acad. Sci. USA 91, 1256-1260 (1994).
15. Qiao, J., Roy, V., Girard, M.H. & Caruso, M. High translation efficiency is mediated by the encephalomyocarditis virus internal ribosomal entry sites if the natural sequence surrounding the eleventh AUG is retained. Hum. Gene Ther. 13, 881-887 (2002).
16. Szymczak, A.L. et al. Correction of multi-gene deficiency in vivo using a single 'self-cleaving' 2A peptide-based retroviral vector. Nat. Biotechnol. 22, 589-594 (2004).
17. Ailles, L. et al. Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells. Mol. Ther. 6, 615-626 (2002).
18. Cavalieri, S. et al. Human T lymphocytes transduced by lentiviral vectors in the absence of TCR activation maintain an intact immune competence. Blood 102, 497-505 (2003).
19. Biffi, A. et al. Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. J. Clin. Invest. 113, 1118-1129 (2004).
20. de Felipe, P. & Ryan, M.D. Targeting of proteins derived from self-processing polyproteins containing multiple signal sequences. Traffic 5, 616-626 (2004).
21. Gardiner-Garden, M. & Frommer, M. CpG islands in vertebrate genomes. J. Mol. Biol. 196, 261-282 (1987).
22. Lavia, P., Macleod, D. & Bird, A. Coincident start sites for divergent transcripts at a randomly selected CpG-rich island of mouse. EMBO J. 6, 2773-2779 (1987).
23. Johnson, P. & Friedmann, T. Limited bidirectional activity of two housekeeping gene promoters: human HPRT and PGK. Gene 88, 207-213 (1990).
24. Scacheri, P.C. et al. Bidirectional transcriptional activity of PGK-neomycin and unexpected embryonic lethality in heterozygote chimeric knockout mice. Genesis 30, 259-263 (2001).
25. Taveau, M. et al. Bidirectional transcriptional activity of the Pgk1 promoter and transmission ratio distortion in Capn3-deficient mice. Genomics 84, 592-595 (2004).
26. Unsinger, J., Kroger, A., Hauser, H. & Wirth, D. Retroviral vectors for the transduction of autoregulated, bidirectional expression cassettes. Mol. Ther. 4, 484-489 (2001).
27. Xie, M., He, Y. & Gan, S. Bidirectionalization of polar promoters in plants. Nat. Biotechnol. 19, 677-679 (2001).
28. Trinklein, N.D. et al. An abundance of bidirectional promoters in the human genome. Genome Res. 14, 62-66 (2004).
29. Takai, D. & Jones, P.A. Origins of bidirectional promoters: computational analyses of intergenic distance in the human genome. Mol. Biol. Evol. 21, 463-467 (2004).
30. Adachi, N. & Lieber, M.R. Bidirectional gene organization: a common architectural feature of the human genome. Cell 109, 807-809 (2002).
31. Cawley, S. et al. Unbiased mapping of transcription factor binding sites along human chromosomes 21 and 22 points to widespread regulation of noncoding RNAs. Cell 116, 499-509 (2004).
32. De Palma, M., Venneri, M.A., Roca, C. & Naldini, L. Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells. Nat. Med. 9, 789-795 (2003).