Exploring strategies for the design of artificial transcription factors: Targeting sites proximal to known regulatory regions for the induction of γ-globin expression and the treatment of sickle cell disease

Journal of Biological Chemistry

Volumn 280, Issue 5, 2005, Pages 3707-3714

  Gräslund, Torbjörn ^a   Li, Xuelin ^a   Magnenat, Laurent ^a   Popkov, Mikhail ^a   Barbas III, Carlos F ^a  

^a SCRIPPS RESEARCH INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DISEASES; GENES; GENETIC ENGINEERING; HEMOGLOBIN; MUTAGENESIS; ZINC;

ARTIFICIAL TRANSCRIPTION FACTORS; GENE EXPRESSION; SICKLE CELL DISEASE;

DNA SEQUENCES;

GAMMA GLOBIN; HEMOGLOBIN F; TRANSCRIPTION FACTOR;

ARTICLE; BINDING SITE; CHROMATIN; CONTROLLED STUDY; DNA BINDING; DNA SEQUENCE; DRUG DESIGN; DRUG EFFICACY; DRUG MECHANISM; ERYTHROLEUKEMIA CELL; HUMAN; HUMAN CELL; LEUKEMIA CELL LINE; PRIORITY JOURNAL; REGULATORY MECHANISM; REPORTER GENE; SICKLE CELL ANEMIA; TRANSCRIPTION INITIATION; TRANSCRIPTION REGULATION; ZINC FINGER MOTIF;

ANEMIA, SICKLE CELL; BASE SEQUENCE; CHROMATIN IMMUNOPRECIPITATION; DEOXYRIBONUCLEASE I; DNA FOOTPRINTING; FLOW CYTOMETRY; GENE THERAPY; GENES, REPORTER; GLOBINS; HUMANS; K562 CELLS; MICROSCOPY, FLUORESCENCE; MOLECULAR SEQUENCE DATA; PROMOTER REGIONS (GENETICS); RETROVIRIDAE; TRANSCRIPTION FACTORS; TRANSFECTION;

EID: 13544277695     PISSN: 00219258     EISSN: None     Source Type: Journal    
DOI: 10.1074/jbc.M406809200     Document Type: Article

References (49)

1. Kollias, G., Wrighton, N., Hurst, J., and Grosveld, F. (1986) Cell 46, 89-94
2. Chada, K., Magram, J., Raphael, K., Radice, G., Lacy, E., and Costantini, F. (1985) Nature 314, 377-380
3. Chada, K., Magram, J., and Costantini, F. (1986) Nature 319, 685-689
4. Rutherford, T., and Nienhuis, A. W. (1987) Mol. Cell. Biol. 7, 398-402
5. Peterson, K. R. (2003) Curr. Opin. Hematol. 10, 123-129
6. Wijgerde, M., Grosveld, F., and Fraser, P. (1995) Nature 377, 209-213
7. Li, Q., Peterson, K. R., Fang, X., and Stamatoyannopoulos, G. (2002) Blood 100, 3077-3086
8. Stamatoyannopoulos, G., and Grosveld, F. (2001) in The Molecular Basis of Blood Diseases (Stamatoyannopoulos, G., Majerus, P., Perlmutter, R., and Varmus, H., eds) pp. 135-182, W. B. Saunders Co., Philadelphia, PA
9. Murray, N., Serjeant, B. E., and Serjeant, G. R. (1988) Br. J. Haematol. 69, 89-92
10. Swank, R. A., and Stamatoyannopoulos, G. (1998) Curr. Opin. Genet. Dev. 8, 366-370
11. Blouin, M.-J., Beauchemin, H., Wright, A., De Paepe, M., Sorette, M., Bleau, A.-M., Nakamoto, B., Ou, C.-N., Stamatoyannopoulos, G., and Trudel, M. (2000) Nat. Med. 6, 177-182
12. May, C., Rivella, S., Callegari, J., Heller, G., Gaensler, K. M. L., Luzzatto, L., and Sadelain, M. (2000) Nature 406, 82-86
13. Desjarlais, J. R., and Berg, J. M. (1992) Proc. Natl. Acad. Sci. U. S. A. 89, 7345-7349
14. Wu, H., Yang, W. P., and Barbas, C. F., III (1995) Proc. Natl. Acad. Sci. U. S. A. 92, 344-348
15. Greisman, H. A., and Pabo, C. O. (1997) Science 275, 657-661
16. Segal, D. J., Dreier, B., Beerli, R. R., and Barbas, C. F., III (1999) Proc. Natl. Acad. Sci. U. S. A. 96, 2758-2763
17. Dreier, B., Beerli, R. R., Segal, D. J., Flippin, J. D., and Barbas, C. F., III (2001) J. Biol. Chem. 276, 29466-29478
18. Dreier, B., Segal, D. J., and Barbas, C. F., III (2000) J. Mol. Biol. 303, 489-502
19. Seipel, K., Georgiev, O., and Schaher, W. (1992) EMBO J. 11, 4961-4968
20. Beerli, R. R., Dreier, B., and Barbas, C. F., III (2000) Proc. Natl. Acad. Sci. U. S. A. 97, 1495-1500
21. Tan, S., Guschin, D., Davalos, A., Lee, Y. L., Snowden, A. W., Jouvenot, Y., Zhang, H. S., Howes, K., McNamara, A. R., Lai, A., Ullman, C., Reynolds, L., Moore, M., Isalan, M., Berg, L. P., Campos, B., Qi, H., Spratt, S. K., Case, C. C., Pabo, C. O., Campisi, J., and Gregory, P. D. (2003) Proc. Natl. Acad. Sci. U. S. A. 100, 11997-12002
22. Rebar, E. J., Huang, Y., Hickey, R., Nath, A. K., Meoli, D., Nath, S., Chen, B., Xu, L., Liang, Y., Jamieson, A. C., Zhang, L., Spratt, S. K., Case, C. C., Wolffe, A., and Giordano, F. J. (2002) Nat. Med. 8, 1427-1432
23. Xu, D., Ye, D., Fisher, M., and Juliano, R. L. (2002) J. Pharmacol. Exp. Ther. 302, 963-971
24. Beerli, R. R., and Barbas III, C. F. (2002) Nat. Biotechnol. 20, 135-141
25. Magnenat, L., Biancafort, P., and Barbas, C. F., III (2004) J. Mol. Biol. 341, 635-649
26. Zhang, L., Spratt, S. K., Liu, Q., Johnstone, B., Qi, H., Raschke, E. E., Jamieson, A. C., Rebar, E. J., Wolffe, A. P., and Case, C. C. (2000) J. Biol. Chem. 275, 33850-33860
27. Liu, P. Q., Rebar, E. J., Zhang, L., Liu, Q., Jamieson, A. C., Liang, Y., Qi, H., Li, P. X., Chen, B., Mendel, M. C., Zhong, X., Lee, Y. L., Eisenberg, S. P., Spratt, S. K., Case, C. C., and Wolffe, A. P. (2001) J. Biol. Chem. 276, 11323-11334
28. Mannervik, M., Nibu, Y., Zhang, H., and Levine, M. (1999) Science 284, 606-609
29. Beerli, R. R., Segal, D. J., Dreier, B., and Barbas, C. F., III (1998) Proc. Natl. Acad. Sci. U. S. A. 95, 14628-14633
30. Segal, D. J., Beerli, R. R., Blancafort, P., Dreier, B., Effertz, K., Huber, A., Koksch, B., Lund, C. V., Magnenat, L., Valente, D., and Barbas, C. F., 3rd. (2003) Biochemistry 42, 2137-2148
31. Laniel, M. A., Beliveau, A., and Guerin, S. L. (2001) Methods Mol. Biol. 148, 13-30
32. Skarpidi, E., Vassilopoulos, G., Li, Q., and Stamatoyannopoulos, G. (2000) Blood 96, 321-326
33. Boyd, K. E., Wells, J., Gutman, J., Bartley, S. M., and Farnham, P. J. (1998) Proc. Natl. Acad. Sci. U. S. A. 95, 13887-13892
34. Collis, P., Antoniou, M., and Grosveld, F. (1990) EMBO J. 9, 233-240
35. Margolin, J. F., Friedman, J. R., Meyer, W. K., Vissing, H., Thiesen, H. J., and Rauscher, F. J., III (1994) Proc. Natl. Acad. Sci. U. S. A. 91, 4509-4513
36. Asano, H., Li, X. S., and Stamatoyannopoulos, G. (1999) Mol. Cell. Biol. 19, 3571-3579
37. Asano, H., Li, X. S., and Stamatoyannopoulos, G. (2000) Blood 95, 3578-3584
38. Gumucio, D. L., Rood, K. L., Blanchard-McQuate, K. L., Gray, T. A., Saulino, A., and Collins, F. S. (1991) Blood 78, 1853-1863
39. Anagnou, N. P., Karlsson, S., Moulton, A. D., Keller, G., and Nienhuis, A. W. (1986) EMBO J. 5, 121-126
40. Langdon, S. D., and Kaufman, R. E. (1998) Blood 91, 309-318
41. Collins, F. S., Metherall, J. E., Yamakawa, M., Pan, J., Weissman, S. M., and Forget, B. G. (1985) Nature 313, 325-326
42. Gelinas, R., Endlich, B., Pfeiffer, C., Yagi. M., and Stamatoyannopoulos, G. (1985) Nature 313, 323-325
43. Berry, M., Grosveld, F., and Dillon, N. (1992) Nature 358, 499-502
44. Ikuta, T., and Kan, Y. W. (1991) Proc. Natl. Acad. Sci. U. S. A. 88, 10188-10192
45. Segal, D. J., Goncalves, J., Eberhardy, S., Swan, C. H., Torbett, B. E., Li, X., and Barbas, C. F., III (2004) J. Biol. Chem. 279, 14509-14519
46. Haley, J. D., Smith, D. E., Schwedes, J., Brennan, R., Pearce, C., Moore, C., Wang, F., Petti, F., Grosveld, F., Jane, S. M., Noguchi, C. T., and Schechter, A. N. (2003) Biochem. Pharmacol. 66, 1755-1768
47. Powars, D. R., Weiss, J. N., Chan, L. S., and Schroeder, W. A. (1984) Blood 63, 921-926
48. Powars, D. R., Chan, L., and Schroeder, W. A. (1989) Ann. N. Y. Acad. Sci. 565, 262-278
49. Xu, L., Zerby, D., Huang, Y., Ji, H., Nyanguile, O. F., de los Angeles, J. E., and Kadan, M. J. (2001) Mol. Ther. 3, 262-273