Transgenic mice expressing an artificial zinc finger regulator targeting an endogenous gene

Methods in Molecular Biology

Volumn 649, Issue , 2010, Pages 183-206

  Passananti, Claudio ^a   Corbi, Nicoletta ^a   Onori, Annalisa ^a   Di Certo, Maria Grazia ^b   Mattei, Elisabetta ^b  

^a Istituto Di Biologia E Patologia Molecolari Rome   (Italy)

^b CNR   (Italy)

Author keywords

artificial transcription factor; dystrophin; gene therapy; muscular dystrophy; synthetic gene; transgenic mice; utrophin; Zinc finger

Indexed keywords

MUS; MUS MUSCULUS;

TRANSCRIPTION FACTOR; UTROPHIN; ZINC FINGER PROTEIN;

ANIMAL; ARTICLE; CHROMATIN IMMUNOPRECIPITATION; DUCHENNE MUSCULAR DYSTROPHY; FEMALE; GENE THERAPY; GENETICS; GENOTYPE; HUMAN; IMMUNOBLOTTING; MALE; METABOLISM; METHODOLOGY; MOUSE; POLYACRYLAMIDE GEL ELECTROPHORESIS; TRANSGENIC MOUSE; WESTERN BLOTTING;

ANIMALS; BLOTTING, WESTERN; CHROMATIN IMMUNOPRECIPITATION; ELECTROPHORESIS, POLYACRYLAMIDE GEL; FEMALE; GENE THERAPY; GENOTYPE; HUMANS; IMMUNOBLOTTING; MALE; MICE; MICE, TRANSGENIC; MUSCULAR DYSTROPHY, DUCHENNE; TRANSCRIPTION FACTORS; UTROPHIN; ZINC FINGERS;

EID: 80054937413     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-60761-753-2_11     Document Type: Article

References (27)

1. Corbi, N., Libri, V., Onori, A., and Passananti, C. (2004) Synthetic zinc finger peptides: Old and novel applications. Biochem Cell Biol. 82, 428-436.
2. Blancafort, P., Segal, D.J., and Barbas, C.F., III. (2004) Designing transcription factor architectures for drug discovery. Mol Pharmacol 66, 1361-1371.
3. Choo, Y. and Klug, A. (1997) Physical basis of a protein-DNA recognition code. Curr Opin Struct Biol. 7, 117-125.
4. 2 zinc finger proteins. Annu Rev Biochem. 70, 313-340.
5. Dreier, B., Segal, D.J., and Barbas, C.F., III. (2000) Insights into the molecular recognition of the 5'-GNN-3' family of DNA sequences by zinc finger domains. J Mol Biol. 303, 489-502.
6. Dreier, B., Beerli, R.R., Segal, D.J., Flippin, J.D., and Barbas, C.F., III. (2001) Development of zinc finger domains for recognition of the 5'-ANN-3' family of DNA sequences and their use in the construction of artificial transcription factors. J Biol Chem. 276, 29466-29478.
7. Dreier, B., Fuller, R.P., Segal, D.J., Lund, C.V., Blancafort, P., Huber, A., Koksch, B., and Barbas, C.F., III. (2005) Development of zinc finger domains for recognition of the 5'-CNN-3' family DNA sequences and their use in the construction of artificial transcription factors. J Biol Chem. 280, 35588-35597.
8. Papworth, M., Kolasinska, P., and Minczuk, M. (2006) Designer zinc-finger proteins and their applications. Gene. 366, 27-38.
9. Beltran, A., Liu, Y., Parikh, S., Temple, B., and Blancafort, P. (2006) Interrogating genomes with combinatorial artificial transcription factor libraries: Asking zinc finger questions. Assay Drug Dev Technol. 4, 317-331.
10. Blancafort, P. and Beltran, A.S. (2008) Rational design, selection and specificity of artificial transcription factors (ATFs): The influence of chromatin in target gene regulation. Comb Chem High Throughput Screen. 11(2), 146-158.
11. Papworth, M., Moore, M., Isalan, M., Minczuk, M., Choo, Y., and Klug, A. (2003) Inhibition of herpes simplex virus 1 gene expression by designer zinc-finger transcription factors. Proc Natl Acad Sci USA. 100, 1621-1626.
12. Reynolds, L., Ullman, C., Moore, M., Isalan, M., West, M.J., Clapham, P., Klug, A., and Choo, Y. (2003) Repression of the HIV-1 5' LTR promoter and inhibition of HIV-1 replication by using engineered zinc-finger transcription factors. Proc Natl Acad Sci USA. 100, 1615-1620.
13. Beltran, A., Parikh, S., Liu, Y., Cuevas, B.D., Johnson, G.L., Futscher, B.W., and Blancafort, P. (2007) Re-activation of a dormant tumor suppressor gene maspin by designed transcription factors. Oncogene. 26(19), 2791-2798.
14. 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) Induction of angiogenesis in a mouse model using engineered transcription factors. Nat Med 8, 1427-1432.
15. Lu, Y., Tian, C., Danialou, G., Gilbert, R., Petrof, B.J., Karpati, G., and Nalbantoglu, J. (2008) Targeting artificial transcription factors to the Utrophin A promoter: Effects on dystrophic pathology and muscle function. J Biol Chem. 283(50), 34720-34727.
16. Corbi, N., Libri, V., Fanciulli, M., Tinsley, J.M., Davies, K.E., and Passananti, C. (2000) The artificial zinc finger coding gene "Jazz" binds the utrophin promoter and activates transcription. Gene Ther. 7, 1076-1083.
17. Onori, A., Desantis, A., Buontempo, S., Di Certo, M.G., Fanciulli, M., Salvatori, L., Passananti, C., and Corbi, N. (2007) The artificial 4-zinc-finger Bagly binds human utrophin promoter A at the endogenous chromosomal site and activates transcription. Biochem Cell Biol. 85(3), 358-365.
18. Mattei, E., Corbi, N., Di Certo, M.G., Strimpakos, G., Severini, C., Onori, A., Desantis, A., Libri, V., Buontempo, S., Floridi, A., Fanciulli, M., Baban, D., Davies, K.E., and Passananti, C. (2007) Utrophin upregulation by an artificial transcription factor in transgenic mice. PLoS ONE. 22(1), e774.
19. Desantis, A., Onori, A., Di Certo, M.G., Mattei, E., Fanciulli, M., Passananti, C., and Corbi, N. (2009) Novel activation domain derived from Che-1 cofactor coupled with the artificial protein Jazz drives utrophin upregulation. Neuromuscular Disorders. 19(2), 158-162.
20. Tinsley, J.M., Potter, A.C., Phelps, S.R., Fisher, R., Trickett, J.I., and Davies, K.E. (1996) Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene. Nature. 384, 349-353.
21. Tinsley, J., Deconinck, N., Fisher, R., Kahn, D., Phelps, S., Gillis, J.M., and Davies, K.E. (1998) Expression of full-length utrophin prevents muscular dystrophy in mdx mice. Nat Med. 4, 1441-1444.
22. Miura, P. and Jasmin, B.J. (2006) Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: How close are we? Trends Mol Med. 3, 122-129.
23. Dennis, C.L., Tinsley, J.M., Deconinck, A.E., and Davies, K.E. (1996) Molecular and functional analysis of the utrophin promoter. Nucleic Acids Res. 24, 1646-1652.
24. Di Certo, M.G., Corbi, N., Strimpakos, G., Onori, A., Luvisetto, S., Severini, C., Guglielmotti, A., Matassa, E.M., Pisani, C., Floridi, A., Benassi, B., Fanciulli, M., Magrelli, A., Mattei, E., and Passananti, C. (2010) The artificial gene Jazz, a transcriptional regulator of utrophin, corrects the dystrophic pathology in mdx mice. Hum Mol Genet. 19, 752-760.
25. Musarò, A., McCullagh, K., Paul, A., Houghton, L., Dobrowolny, G., Molinaro, M., Barton, E.R., Sweeney, H.L., and Rosenthal, N. (2001) Localized Igf-1 transgene expression sustains hypertrophy and regeneration in senescent skeletal muscle. Nat Genet. 27(2), 195-200.
26. Nagy, A., Gertsenstein, M., Vintersten, K., and Behringer, R. (2003) Manipulating the Mouse Embryo: A Laboratory Manual. 3rd ed. Cold Spring Harbor Laboratory Press, New York.
27. Laemmli, U.K. (1970) Cleavege of structural proteins during the assembly of the head of bacteriophage T4. Nature. 227, 680-685.