Regulated gene expression systems

Gene Therapy

Volumn 7, Issue 2, 2000, Pages 120-125

  Clackson, T ^a  

^a ARIAD PHARMACEUTICALS INC   (United States)

Author keywords

Drug regulated transcription; Inducible expression; Regulated vectors; Secretion

Indexed keywords

DOXYCYCLINE; ECDYSONE; MIFEPRISTONE; PROTEIN; RAPAMYCIN; TETRACYCLINE;

CLINICAL TRIAL; GENE EXPRESSION REGULATION; GENE TARGETING; GENE THERAPY; GENETIC ENGINEERING; GENETIC STABILITY; HUMAN; IMMUNOGENICITY; NONHUMAN; PRIORITY JOURNAL; REVIEW; TARGET CELL; TRANSCRIPTION REGULATION;

EID: 0033981631     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3301120     Document Type: Review

References (30)

1. Varley AW, Munford RS. Physiologically responsive gene therapy. Molec Med Today 1998; 4: 445-451.
2. Clackson T. Controlling mammalian gene expression with small molecules. Curr Opin Chem Biol 1997; 1: 210-218.
3. Gossen M, Bujard H. Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc Natl Acad Sci USA 1992; 89: 5547-5551.
4. Gossen M et al. Transcriptional activation by tetracyclines in mammalian cells. Science 1995; 268: 1766-1769.
5. No D, Yao TP, Evans RM. Ecdysone-inducible gene expression in mammalian cells and transgenic mice. Proc Natl Acad Sci USA 1996; 93: 3346-3351.
6. Wang Y, O'Malley BW Jr, Tsai SY, O'Malley BW. A regulatory system for use in gene transfer. Proc Natl Acad Sci USA 1994; 91: 8180-8184.
7. Rivera VM et al. A humanized system for pharmacologic control of gene expression. Nature Med 1996; 2: 1028-1032.
8. Ho SN et al. Dimeric ligands define a role for transcriptional activation domains in reinitiation. Nature 1996; 382: 822-826.
9. Amara JF et al. A versatile synthetic dimerizer for the regulation of protein-protein interactions. Proc Natl Acad Sci USA 1997; 94: 10618-10623.
10. Magari SR et al. Pharmacologic control of a humanized gene therapy system implanted into nude mice. J Clin Invest 1997; 100: 2865-2872.
11. Spencer DM. Creating conditional mutations in mammals. Trends Genet 1996; 12: 181-187.
12. Pomerantz JL, Sharp PA, Pabo CO. Structure-based design of transcription factors. Science 1995; 267: 93-96.
13. Liberles SD, Diver ST, Austin DJ, Schreiber SL. Inducible gene expression and protein translocation using non toxic ligands identified by a mammalian three-hybrid screen. Proc Natl Acad Sci USA 1997; 94: 7825-7830.
14. Clackson T et al. Inducible gene expression in vivo using non-immunosuppressive analogs of rapamycin. (Submitted).
15. Bohl D, Heard JM. Modulation of erythropoietin delivery from engineered muscles in mice. Hum Gene Ther 1997; 8: 195-204.
16. Bohl D, Naffakh N, Heard JM. Long-term control of erythropoietin secretion by doxycycline in mice transplanted with engineered primary myoblasts. Nature Med 1997; 3: 299-305.
17. Rizzuto G et al. Efficient and regulated erythropoietin production by naked DNA injection and muscle electroporation. Proc Natl Acad Sci USA 1999; 96: 6417-6422.
18. Ho DY, McLaughlin JR, Sapolsky RM. Inducible gene expression from defective herpes simplex virus vectors using the tetracycline-responsive promoter system. Mol Brain Res 1996; 41: 200-209.
19. Burcin MM et al. Adenovirus-mediated regulable target gene expression in vivo. Proc Natl Acad Sci USA 1999; 96: 355-360.
20. Rendahl KG et al. Regulation of gene expression in vivo following transduction by two separate rAAV vectors. Nat Biotechnol 1998; 16: 757-761.
21. Bohl D, Salvetti A, Mouiller P, Heard JM. Control of erythropoietin delivery by doxycycline in mice after intramuscular injection of adeno-associated vector. Blood 1998; 95: 1512-1517.
22. Rivera VM et al. Long-term regulated expression of growth hormone in mice after intramuscular gene transfer. Proc Natl Acad Sci USA 1999; 96: 8657-8662.
23. Ye X et al. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science 1999; 283: 88-91.
24. Natesan S et al. A general strategy to enhance the potency of chimeric activation activators. Proc Natl Acad Sci USA 1999; 96: 13898-13903.
25. Gossen M, Bujard H. Anhydrotetracycline, a novel effector for tetracycline controlled gene expression systems in eukaryotic cells. Nucleic Acids Res 1993; 21: 4411-4412.
26. Riddell SR et al. T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients. Nature Med 1996; 2: 216-223.
27. White S et al. Recognition of the four Watson-Crick base pairs in the DNA minor groove by synthetic ligands. Nature 1998; 391: 468-471.
28. Nyanguile O, Uesugi M, Austin DJ, Verdine GL. A nonnatural transcriptional coactivator. Proc Natl Acad Sci USA 1997; 94: 13402-13406.
29. Werstuck G, Green MR. Controlling gene expression in living cells through small molecule-RNA interactions. Science 1998; 282: 296-298.
30. Rivera VM et al. Pharmacological regulation of therapeutic protein secretion in vivo through controlled aggregation in the endoplasmic reticulum. Science 2000 (in press).