Transcription activator-like effector proteins induce the expression of the frataxin gene

Human Gene Therapy

Volumn 23, Issue 8, 2012, Pages 883-890

  Tremblay, Jacques P ^a,b   Chapdelaine, Pierre ^a,b   Coulombe, Zoé ^a,b   Rousseau, Joel ^a,b  

^a UNIVERSITÉ LAVAL   (Canada)

^b Centre de Recherche du Centre Hospitalier Universitaire de   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

FRATAXIN; MESSENGER RNA; PROTEIN VP16;

ARTICLE; CYTOMEGALOVIRUS; DNA SEQUENCE; FRIEDREICH ATAXIA; GENE EXPRESSION; GENETIC CODE; HUMAN; HUMAN CELL; METHODOLOGY; NUCLEOTIDE SEQUENCE; PROTEIN TARGETING; REAL TIME POLYMERASE CHAIN REACTION; TRANSCRIPTION INITIATION; TRINUCLEOTIDE REPEAT;

BASE SEQUENCE; CELL LINE; FRIEDREICH ATAXIA; GENE EXPRESSION; GENES, REPORTER; HERPES SIMPLEX VIRUS PROTEIN VMW65; HUMANS; INTRONS; IRON-BINDING PROTEINS; LUMINESCENT PROTEINS; MOLECULAR SEQUENCE DATA; PLASMIDS; PROMOTER REGIONS, GENETIC; RNA, MESSENGER; TRANSCRIPTION FACTORS; TRINUCLEOTIDE REPEAT EXPANSION;

EID: 84865252679     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2012.034     Document Type: Article

References (40)

1. Babady, N.E., Carelle, N., Wells, R.D., et al. (2007). Advancements in the pathophysiology of Friedreich's ataxia and new prospects for treatments. Mol. Genet. Metab. 92, 23-35.
2. Becker, E., and Richardson, D.R. (2001). Frataxin: Its role in iron metabolism and the pathogenesis of Friedreich's ataxia. Int. J. Biochem. Cell Biol. 33, 1-10.
3. Becker-Hapak, M., McAllister, S.S., and Dowdy, S.F. (2001). TAT-mediated protein transduction into mammalian cells. Methods 24, 247-256.
4. Boch, J., and Bonas, U. (2009). Xanthomonas AvrBs3 family-type III effectors: Discovery and function. Annu. Rev. Phytopathol. 48, 419-436.
5. Boch, J., Scholze, H., Schornack, S., et al. (2009). Breaking the code of DNA binding specificity of TAL-type III effectors. Science 326, 1509-1512.
6. Campuzano, V., Montermini, L., Molto, M.D., et al. (1996). Friedreich's ataxia: Autosomal recessive disease caused by an intronic GAA triplet repeat expansion. Science 271, 1423-1427.
7. Campuzano, V., Montermini, L., Lutz, Y., et al. (1997). Frataxin is reduced in Friedreich ataxia patients and is associated with mitochondrial membranes. Hum. Mol. Genet. 6, 1771-1780.
8. Cermak, T., Doyle, E.L., Christian, M., et al. (2011). Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting. Nucleic Acids Res. 39, e82.
9. Christian, M., Cermak, T., Doyle, E.L., et al. (2010). Targeting DNA double-strand breaks with TAL effector nucleases. Genetics 186, 757-761.
10. Cooper, J.M., and Schapira, A.H. (2003). Friedreich's ataxia: Disease mechanisms, antioxidant and coenzyme Q10 therapy. Biofactors 18, 163-171.
11. Furler, S., Paterna, J.C., Weibel, M., and Bueler, H. (2001). Recombinant AAV vectors containing the foot and mouth disease virus 2A sequence confer efficient bicistronic gene expression in cultured cells and rat substantia nigra neurons. Gene Ther. 8, 864-873.
12. Gu, K., Tian, D., Qiu, C., and Yin, Z. (2009). Transcription activator-like type III effector AvrXa27 depends on OsTFIIAc5 for the activation of Xa27 transcription in rice that triggers disease resistance to Xanthomonas oryzae pv. oryzae. Mol. Plant Pathol. 10, 829-835.
13. Harding, A.E. (1981). Friedreich's ataxia: A clinical and genetic study of 90 families with an analysis of early diagnostic criteria and intrafamilial clustering of clinical features. Brain 104, 589-620.
14. Hauck, B., Murphy, S.L., Smith, P.H., et al. (2009). Undetectable transcription of cap in a clinical AAV vector: Implications for preformed capsid in immune responses. Mol. Ther. 17, 144-152.
15. Herzog, R.W. (2007). Immune responses to AAV capsid: Are mice not humans after all? Mol. Ther. 15, 649-650.
16. Li, K., Singh, A., Crooks, D.R., et al. (2010). Expression of human frataxin is regulated by transcription factors SRF and TFAP2. PLoS One 5, e12286.
17. Li, T., Huang, S., Jiang, W.Z., et al. (2010). TAL nucleases (TALNs): Hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain. Nucleic Acids Res. 39, 359-372.
18. Li, T., Huang, S., Zhao, X., et al. (2011). Modularly assembled designer TAL effector nucleases for targeted gene knockout and gene replacement in eukaryotes. Nucleic Acids Res. 39, 6315-6325.
19. Li, Y.R., Zou, H.S., Che, Y.Z., et al. (2011). A novel regulatory role of HrpD6 in regulating hrp-hrc-hpa genes in Xanthomonas oryzae pv. oryzicola. Mol. Plant Microbe Interact. 24, 1086-1101.
20. Luu-The, V., Paquet, N., Calvo, E., and Cumps, J. (2005). Improved real-time RT-PCR method for high-throughput measurements using second derivative calculation and double correction. Biotechniques 38, 287-293.
21. Lynch, D.R., Farmer, J.M., Balcer, L.J., and Wilson, R.B. (2002). Friedreich ataxia: Effects of genetic understanding on clinical evaluation and therapy. Arch. Neurol. 59, 743-747.
22. Morris, M.C., Depollier, J., Mery, J., et al. (2001). A peptide carrier for the delivery of biologically active proteins into mammalian cells. Nat. Biotechnol. 19, 1173-1176.
23. Moscou, M.J., and Bogdanove, A.J. (2009). A simple cipher governs DNA recognition by TAL effectors. Science 326, 1501.
24. Ohshima, S., Shin, J.H., Yuasa, K., et al. (2009). Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle. Mol. Ther. 17, 73-80.
25. Pandolfo, M. (1999). Molecular pathogenesis of Friedreich ataxia. Arch. Neurol. 56, 1201-1208.
26. Pandolfo, M. (2006). Iron and Friedreich ataxia. J. Neural Transm. Suppl. 2006, 143-146.
27. Pandolfo, M. (2012). Friedreich ataxia. Handb. Clin. Neurol. 103, 275-294.
28. Paul, A.V., Mugavero, J., Molla, A., and Wimmer, E. (1998). Internal ribosomal entry site scanning of the poliovirus polyprotein: Implications for proteolytic processing. Virology 250, 241-253.
29. Romer, P., Recht, S., and Lahaye, T. (2009). A single plant resistance gene promoter engineered to recognize multiple TAL effectors from disparate pathogens. Proc. Natl. Acad. Sci. U.S.A. 106, 20526-20531.
30. Romer, P., Recht, S., Strauss, T., et al. (2010). Promoter elements of rice susceptibility genes are bound and activated by specific TAL effectors from the bacterial blight pathogen, Xanthomonas oryzae pv. oryzae. New Phytol. 187, 1048-1057.
31. Rotig, A., de Lonlay, P., Chretien, D., et al. (1997). Aconitase and mitochondrial iron-sulphur protein deficiency in Friedreich ataxia. Nat. Genet. 17, 215-217.
32. Scholze, H., and Boch, J. (2010). TAL effector-DNA specificity. Virulence 1, 428-432.
33. Singh, G., Binstadt, B.A., Black, D.F., et al. (2001). Electro-convulsive therapy and Friedreich's ataxia. J. ECT 17, 53-54.
34. Song, C., and Yang, B. (2010). Mutagenesis of 18 type III effectors reveals virulence function of XopZPXO99 in Xanthomonas oryzae pv. oryzae. Mol. Plant Microbe Interact. 23, 893-902.
35. Tilstra, J., Rehman, K.K., Hennon, T., et al. (2007). Protein transduction: Identification, characterization and optimization. Biochem. Soc. Trans. 35, 811-85.
36. Tinsley, J.M., Potter, A.C., Phelps, S.R., et al. (1996). Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene [see comments]. Nature 384, 349-353.
37. Vives, E., Richard, J.P., Rispal, C., and Lebleu, B. (2003). TAT peptide internalization: Seeking the mechanism of entry. Curr. Protein Pept. Sci. 4, 125-132.
38. Wallis, J., Shaw, J., Wilkes, D., et al. (1989). Prenatal diagnosis of Friedreich ataxia. Am. J. Med. Genet. 34, 458-461.
39. Yu, Y., Streubel, J., Balzergue, S., et al. (2011). Colonization of rice leaf blades by an African strain of Xanthomonas oryzae pv. oryzae depends on a new TAL effector that induces the rice nodulin-3 Os11N3 gene. Mol. Plant Microbe Interact. 24, 1102-1113.
40. Zhang, F., Cong, L., Lodato, S., et al. (2011). Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription. Nat. Biotechnol. 29, 149-154.