Pharmacologic control of a humanized gene therapy system implanted into nude mice

Journal of Clinical Investigation

Volumn 100, Issue 11, 1997, Pages 2865-2872

  Magari, Shannon R ^a   Rivera, Victor M ^a   Iuliucci, John D ^a   Gilman, Michael ^a   Cerasoli Jr , Franklin ^a  

^a ARIAD PHARMACEUTICALS INC   (United States)

Author keywords

Dimerizer; Human growth hormone; Rapamycin; Transcription factor

Indexed keywords

GROWTH HORMONE; RAPAMYCIN; TRANSCRIPTION FACTOR;

ALTERNATIVE RNA SPLICING; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BINDING SITE; CONTROLLED STUDY; DIMERIZATION; DNA BINDING; GENE EXPRESSION REGULATION; GENE THERAPY; GENETIC REGULATION; IMPLANTATION; MOUSE; NONHUMAN; NUDE MOUSE; PRIORITY JOURNAL; PROTEIN PROTEIN INTERACTION; TRANSCRIPTION REGULATION;

EID: 0031441285     PISSN: 00219738     EISSN: None     Source Type: Journal    
DOI: 10.1172/JCI119835     Document Type: Article

References (31)

1. Isgaard, J., L. Carlsson, O. Isaksson, and J. Jansson. 1988. Pulsatile intravenous growth hormone (GH) infusion to hypophysectomized rats increases insulin-like growth factor I messenger ribonucleic acid in skeletal tissues more effectively than continuous GH infusion. Endocrinology. 123:2605-2610.
2. Wolf, E., E. Kahnt, J. Ehrlein, W. Hermanns, G. Brem, and R. Wanke. 1993. Effects of long-term elevated serum levels of growth hormone on life expectancy of mice: lessons learned from transgenic animal models. Mech. Ageing Dev. 68:71-87.
3. Rivera, V., T. Clackson, S. Natesan, R. Pollock, J. Amara, T. Keenan, S. Magari, T. Phillips, N. Courage, F. Cerasoli, et al. 1996. A humanized system for pharmacologic control of gene expression. Nat. Med. 2:1028-103.
4. Chen, J., X. Zheng, E. Brown, and S. Schreiber. 1995. Identification of an 11 kDa hFKBP12-rapamycin binding domain within the 289-kDa hFKBP-rapamycin-associated protein and characterization of a critical serine residue. Proc. Natl. Acad. Sci. USA. 92:4947-4951.
5. Pomerantz, J., P. Sharp, and C. Pabo. 1995. Structure-based design of transcription factors. Science. 267:93-96.
6. Standaert, R., A. Galat, G. Verdine, and S. Schreiber. 1990. Molecular cloning and overexpression of the human FK506-binding protein hFKBP. Nature. 346:671-674.
7. Riddell, S. 1996. T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients. Nat. Med. 2:216-223.
8. Wang, Y., B.W. O'Malley, Jr., S. Tsai, and B.W. O'Malley. 1994. A regulatory system for use in gene transfer. Proc. Natl. Acad. Sci. USA. 91:8180-8184.
9. Bohl, D., N. Naffakh, and J. Heard. 1997. Long-term control of erythropoietin secretion by doxycycline in mice transplanted with engineered primary myoblast. Nat. Med. 3:299-305.
10. Dhawan, J., T. Rando, S. Elson, H. Bujard, and H. Blau. 1995. Tetracycline-regulated gene expression following direct gene transfer into mouse skeletal muscle. Som. Cell Mol. Genet. 21:233-240.
11. Schmitz, M., and P. Baeuerle. 1991. The p65 subunit is responsible for the strong trascription activating potential of NF-kappa B. EMBO (Eur. Mol. Biol. Organ.) J. 10:3805-3817.
12. Heartlein, M., V. Roman, J. Jtang, J. Sellers, A. Zuliani, D. Treco, and R. Selden. 1994. Long-term production and delivery of human growth hormone. Proc. Natl. Acad. Sci. USA. 91:10968-10971.
13. Kahan, B., J. Chang, and S. Sehgal. 1991. Preclinical evaluation of a new potent immunosuppressive agent, rapamycin. Transplantation (Baltimore). 52: 185-191.
14. Kay, J., L. Kromwel, S. Doe, and M. Denyer. 1991. Inhibition of T and B lymphocyte proliferation by rapamycin. Immunology. 72:544-549.
15. Sehgal, S., K. Molnar-Kimbar, T. Ocain, and B. Weichman. 1994. Rapamycin: a novel immunosuppressive macrolide. Med. Res. Rev. 14:1-22.
16. Stepkowski, S., H. Chen, P. Daloze, and B. Kahan. 1991. Rapamycin, a potent immunosuppressive drug for vascularized heart, kidney, and small bowel Transplantation in the rat. Transplantation (Baltimore). 51:22-26.
17. Abraham, R.T., and G.J. Wiederrecht. 1996. Immunopharmacology of rapamycin. Ann. Rev. Immunol. 14:483-510.
18. Hindmarsh, P., and C. Brook. 1996. Final height of short normal children treated with growth hormone. Lancet (N. Am. Ed.). 348:13-16.
19. Amato, G., G. Izzo, G. La Montagna, and A. Bellastella. 1996. Low dose recombinant human growth hormone normalizes bone metabolism and cortical bone density and improves trabecular bone density in growth hormone deficient adults without causing adverse effects. Clin. Endocrinol. 45:27-32.
20. Oscarsson, J., G. Lindstedt, P. Lundberg, and S. Eden. 1996. Continuous subcutaneous infusion of low dose growth hormone decreases serum sex-hormone binding globulin and testosterone concentrations in moderately obese middle-aged men. Clin. Endocrinol. 44:23-29.
21. Yatscoff, R. 1996. Pharmacokinetics of rapamycin. Transplant. Proc. 28: 970-973.
22. Baker, H., S. Sehgal, and C. Vezna. 1978. Rapamycin (AY-22,989), a new antifungal antibiotic. III. In vitro and in vivo evaluation. J. Antibiot. (Tokyo). 31:539-545.
23. Granger, D., J. Cromwell, S. Chen, J. Goswitz, D. Morrow, F. Beierle, S. Sehgal, D. Canafax, and A. Matas. 1995. Prolongation of renal allograft survival in a large animal model by oral rapamycin monotherapy. Transplantation (Baltimore). 59:183-186.
24. Dias, V., and R. Yatscoff. 1994. Investigation of rapamycin transport and uptake across absorptive human intestinal cell monolayers. Clin. Biochem. 27:31-36.
25. Helms, S., and F. Rottman. 1990. Characterization of an inducible promoter system to investigate decay of stable mRNA molecules. Nucleic Acids Res. 18:255-259.
26. Shyu, A.-B., J. Belasco, and M. Greenberg. 1991. Two distinct destabilizing elements in the c-fos message trigger deadenylation as a first step in rapid mRNA decay. Genes Dev. 5:221-231.
27. Carlson, R.P., and S.N. Sehgal. 1996. Effect of rapamycin in animal models of arthritis and other autoimmune diseases. In Principles of Drug Development in Transplantation and Autoimmunity. R.G. Landes Co., Austin, TX. 303-308.
28. Chen, H.F., S.J. Qi, D.S. Xu, J.P. Wu, and P. Daloze. 1996. The immunosuppressive effect of rapamycin on mouse small bowel transplantation. Transplantation (Baltimore). 61:523-526.
29. Christians, U., and K.F. Sewing. 1996. Pharmacokinetics, metabolism and therapeutic drug monitoring of the macrolide immunosuppressants tacrolimus (FK506) and sirolimus (rapamycin). In Principles of Drug Development in Transplantation and Autoimmunity. R.G. Landes Co., Austin, TX. 483-505.
30. Belshaw, P., J. Schoepfer, K. Liu, K. Morrison, and S. Schreiber. 1995. Rational design of orthogonal receptor-ligand combinations. Angew. Chem. Int. Ed. Eng. 34:2129-2132.
31. Gibaldi, M., and D. Perrier. 1982. Pharmacokinetics. Marcel Dekker, Inc., New York. pp. 145-198.