The artificial zinc finger coding gene 'Jazz' binds the utrophin promoter and activates transcription

Gene Therapy

Volumn 7, Issue 12, 2000, Pages 1076-1083

  Corbi, N ^a   Libri, V ^a   Fanciulli, M ^b   Tinsley, J M ^c   Davies, K E ^c   Passananti, C ^a  

^a CNR   (Italy)

^b REGINA ELENA NATIONAL CANCER INSTITUTE   (Italy)

^c UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

DMD; Dystrophin; Gene therapy; Protein design; Utrophin; Zinc finger

Indexed keywords

DNA BINDING PROTEIN; DOUBLE STRANDED DNA; TRANSCRIPTION FACTOR SP1; UTROPHIN; ZINC FINGER PROTEIN;

ALPHA HELIX; AMINO ACID SEQUENCE; ANIMAL CELL; ARTICLE; DISSOCIATION CONSTANT; DUCHENNE MUSCULAR DYSTROPHY; GENE THERAPY; GENETIC ENGINEERING; HUMAN; HUMAN CELL; MOLECULAR RECOGNITION; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; TRANSCRIPTION REGULATION;

ANIMALS; CYTOSKELETAL PROTEINS; HUMANS; MEMBRANE PROTEINS; MICE; PROMOTER REGIONS (GENETICS); TRANS-ACTIVATION (GENETICS); TRANSCRIPTION FACTORS; TRANSFECTION; TUMOR CELLS, CULTURED; UTROPHIN; ZINC FINGERS;

ANIMALIA;

EID: 0034089616     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3301204     Document Type: Article

References (44)

1. Choo Y, Sánchez-García I, Klug A. In vivo repression by a site-specific DNA-binding protein designed against an oncogenic sequence. Nature 1994; 372: 642-645.
2. Pomerantz JL, Sharp PA, Pabo CO. Structure-based design of transcription factors. Science 1995; 267: 93-96.
3. Wu H, Yang WP, Barbas III CF. Building zinc fingers by selection: Toward a therapeutic application. Proc Natl Acad Sci USA 1995; 92: 344-348.
4. Bryson JW et al. Protein design: A hierarchic approach. Science 1995; 270: 935-940.
5. Corbi N, Perez M, Maione R, Passananti C. Synthesis of a new zinc finger peptide; comparison of its 'code' deduced and 'CASTing' derived binding sites. FEBS Lett 1997; 417: 71-74.
6. Kim JS, Pabo CO. Transcriptional repression by zinc finger peptides. Exploring the potential for applications in gene therapy. J Biol Chem 1997; 272: 29795-29800.
7. Corbi N, Libri V, Fanciulli M, Passananti C. Binding properties of the artificial zinc fingers coding gene Sintl. Biochem Biophys Res Commun 1998; 253: 686-692.
8. Desjarlais JR, Berg JM. Length-encoded multiplex binding site determination: Application to zinc finger proteins. proc Natl Acad Sci USA 1994; 91: 11099-11103.
9. Choo Y, Klug A. Selection of DNA binding sites for zinc fingers using rationally randomized DNA reveals coded interactions. Proc Natl Acad Sci USA 1994; 91: 11168-11172.
10. Rebar EJ, Pabo CO. Zinc finger phage: Affinity selection of fingers with new DNA-binding specificities. Science 1994; 263: 671-674.
11. Choo Y, Klug A. Physical basis of a protein-DNA recognition code. Curr Opin Struct Biol 1997; 7: 117-125.
12. Gogos JA et al. Recognition of diverse sequences by class I zinc fingers: Asymmetries and indirect effects on specificity in the interaction between CF2II and A+T-rich elements. Proc Natl Acad Sci USA 1996; 93: 2159-2164.
13. Kim JS et al. Design of TATA box-binding protein/zinc finger fusions for targeted regulation of gene expression. Proc Natl Acad Sci USA 1997; 94: 3616-3620.
14. Jamieson AC, Wang H, Kim SH. A zinc finger directory for high-affinity DNA recognition. Proc Natl Acad Sci USA 1996; 93: 12834-12839.
15. Greisman AH, Pabo CO. A general strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites. Science 1997; 275: 657-661.
16. Isalan M, Choo Y, Klug A. Synergy between adjacent zinc fingers in sequence-specific DNA recognition. Proc Natl Acad Sci USA 1997; 94: 5617-5621.
17. Choo Y. End effects in DNA recognition by zinc finger arrays. Nucleic Acid Res 1998; 26: 554-557.
18. Mandel-Gutfreund Y, Margalit H. Quantitative parameters for amino acid-base interaction: Implications for prediction of protein-DNA binding sites. Nucleic Acid Res 1998; 26: 2306-2312.
19. Liu Q, Segal DJ, Ghiara JB, Barbas III CF. Design of polydactyl zinc-finger proteins for unique addressing within complex genomes. Proc Natl Acad Sci USA 1997; 94: 5525-5530.
20. Kamiuchi T et al. Artificial nine zinc-finger peptide with 30 base pair binding sites. Biochemistry 1998; 37: 13827-13834.
21. Love D et al. Dystrophin and dystrophin-related proteins: A review of protein and RNA studies. Neuromusc Disord 1993; 3: 5-21.
22. Pons F et al. Dystrophin and dystrophin-related protein (utrophin) distribution in normal and dystrophin-deficient skeletal muscles. Neuromusc Disord 1993; 3: 507-514.
23. Pearce M et al. The utrophin and dystrophin genes share similarities in genomic structure. Hum Molec Genet 1993; 2: 1765-1772.
24. Winder SJ, Gibson TJ, Kendrick-Jones J. Dystrophin and utrophin: The missing links! FEBS Lett 1995; 369: 27-33.
25. Winder SJ. Structure-function relationships in dystrophin and utrophin. Biochem Soc Trans 1996; 24: 497-501.
26. Winder SJ. The membrane-cytoskeleton interface: The role of dystrophin and utrophin. J Musc Res Cell Motil 1997; 18: 617-629.
27. Gramolini AO, Jasmin BJ. Duchenne muscular dystrophy and the neuromuscular junction: The utrophin link. BioEssays 1997; 19: 747-750.
28. Tinsley JM, Davies KE. Utrophin: A potential replacement for dystrophin? Neuromusc Disord 1993; 3: 537-539.
29. Tinsley JM et al. Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene. Nature 1996; 384: 349-353.
30. Deconinck N et al. Expression of truncated utrophin leads to major functional improvements in dystrophin-deficient muscles of mice. Nature Med 1997; 3: 1216-1221.
31. Rafael JA et al. Skeletal muscle-specific expression of a utrophin transgene rescues utrophin-dystrophin deficient mice. Nat Genet 1998; 19: 79-82.
32. Dennis CL, Tinsley JM, Deconinck AE, Davies KE. Molecular and functional analysis of the utrophin promoter. Nucleic Acid Res 1996; 24: 1646-1652.
33. Kadonaga JT, Carner KR, Masiarz FR, Tjian R. Isolation of cDNA encoding transcription factor Sp1 and functional analysis of the DNA binding domain. Cell 1987; 51: 1079-1090.
34. Christy BC, Lau LF, Nathans D. A gene activated in mouse 3T3 cells by serum growth factors encodes a protein with 'zinc finger' sequences. Proc Natl Acad Sci USA 1988; 85: 7857-7861.
35. Choo Y, Klug A. Toward a code for the interactions of zinc fingers with DNA: Selection of randomized fingers displayed on phage. Proc Natl Acad Sci USA 1994; 91: 11163-11167.
36. Sadowski I. Uses for GAL4 expression in mammalian cells. Genet Eng (NY) 1995; 17: 119-148.
37. Choo Y et al. Promoter-specific activation of gene expression directed by bacteriophage-selected zinc fingers. J Mol Biol 1997; 273: 525-532.
38. Beerli RR, Segal DJ, Dreier B, Barbas III CF. Toward controlling gene expression at will: Specific regulation of the crbB-2/HER-2 promoter by using polydactyl zinc finger proteins constructed from modular building blocks. Proc Natl Acad Sci USA 1998; 95: 14628-14633.
39. Erlod-Ericson M, Pabo CO. Binding studies with mutants of zif268. Contribution of individual side chains to binding affinity and specificity in the zif268 zinc finger-DNA complex. J Biol Chem 1999; 274: 19281-19285.
40. Wolfe SA, Greisman HA, Ramm EI, Pabo CO. Analysis of zinc fingers optimized via phage display: Evaluating the utility of a recognition code. J Mol Biol 1999; 285: 1917-1934.
41. Berg JM. Sp1 and the subfamily of zinc finger proteins with guanine-rich binding sites. Proc Natl Acad Sci USA 1992; 89: 11109-11110.
42. Biesiada E, Hamamori Y, Kedes L, Sartorelli V. Myogenic basic helix-loop-helix proteins and Sp1 interact as components of a multiprotein transcriptional complex required for activity of the human cardiac alpha-actin promoter. Mol Cell Biol 1999; 19: 2577-2584.
43. Wadzinski BE et al. NH2-terminal modification of the phosphatase 2A catalytic subunit allows functional expression in mammalian cells. J Biol Chem 1992; 267: 16883-16888.
44. Smith DB, Johnson KS. Single-step purification of polypeptidcs expressed in Escherichia coli as fusions with glutathione S-transferase. Gene 1988; 67: 31-40.