A cell-based high-throughput screening assay for posttranscriptional utrophin upregulation

Journal of Biomolecular Screening

Volumn 18, Issue 4, 2013, Pages 400-406

  Moorwood, Catherine ^a   Soni, Neha ^a   Patel, Gopal ^a   Wilton, Steve D ^b   Khurana, Tejvir S ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

Author keywords

assay; high throughput screening; posttranscriptional; translational; utrophin

Indexed keywords

GENOMIC DNA; LUCIFERASE; MICRORNA; MICRORNA 206; UNCLASSIFIED DRUG; UTROPHIN;

3' UNTRANSLATED REGION; 5' UNTRANSLATED REGION; ANIMAL CELL; ARTICLE; CELL ASSAY; CONTROLLED STUDY; DNA ISOLATION; DUCHENNE MUSCULAR DYSTROPHY; GENE EXPRESSION; GENE MUTATION; GENE SEQUENCE; GENE TARGETING; GENETIC TRANSFECTION; HIGH THROUGHPUT SCREENING; MOUSE; MUSCLE CELL; NONHUMAN; POSTTRANSCRIPTIONAL GENE SILENCING; PRIORITY JOURNAL; PROMOTER REGION; RNA ISOLATION; TRANSCRIPTION REGULATION; UPREGULATION;

3' UNTRANSLATED REGIONS; 5' UNTRANSLATED REGIONS; ANIMALS; CELL LINE; HIGH-THROUGHPUT SCREENING ASSAYS; LUCIFERASES; MICE; MICRORNAS; REPRODUCIBILITY OF RESULTS; TRANSCRIPTION, GENETIC; UP-REGULATION; UTROPHIN;

ANIMALIA;

EID: 84875699165     PISSN: 10870571     EISSN: 1552454X     Source Type: Journal    
DOI: 10.1177/1087057112465648     Document Type: Article

References (31)

1. Hoffman E. P., Brown R. H., Kunkel L. M.. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987 ; 51: 919-928
2. Engel A. G., Franzini-Armstrong C. Myology: Basic and Clinical. 3 rd ed.; McGraw-Hill: New York, 2004; p 1960. McGraw-Hill: New York ; 2004 1960 :
3. Emery A. E., Muntoni F. Duchenne Muscular Dystrophy. Oxford University Press: Oxford ; 2003 :
4. Kohler M., Clarenbach C. F., Bahler C., Brack T., Russi E. W., Bloch K. E.. Disability and Survival in Duchenne Muscular Dystrophy. J. Neurol. Neurosurg. Psychiatry. 2009 ; 80: 320-325
5. Khurana T. S., Watkins S. C., Chafey P., Chelly J., Tome F. M., Fardeau M., Kaplan J. C., Kunkel L. M.. Immunolocalization and Developmental Expression of Dystrophin Related Protein in Skeletal Muscle. Neuromuscul. Disord. 1991 ; 1: 185-194
6. Burton E. A., Tinsley J. M., Holzfeind P. J., Rodrigues N. R., Davies K. E.. A Second Promoter Provides an Alternative Target for Therapeutic Up-regulation of Utrophin in Duchenne Muscular Dystrophy. Proc. Natl. Acad. Sci. U. S. A. 1999 ; 96: 14025-14030
7. Weir A. P., Burton E. A., Harrod G., Davies K. E.. A- and B-Utrophin Have Different Expression Patterns and Are Differentially Up-regulated in mdx Muscle. J. Biol. Chem. 2002 ; 277: 45285-45290
8. Love D. R., Hill D. F., Dickson G., Spurr N. K., Byth B. C., Marsden R. F., Walsh F. S., Edwards Y. H., Davies K. E.. An Autosomal Transcript in Skeletal Muscle with Homology to Dystrophin. Nature. 1989 ; 339: 55-58
9. Khurana T. S., Hoffman E. P., Kunkel L. M.. Identification of a Chromosome 6-Encoded Dystrophin-Related Protein. J. Biol. Chem. 1990 ; 265: 16717-16720
10. Tinsley J. M., Blake D. J., Roche A., Fairbrother U., Riss J., Byth B. C., Knight A. E., Kendrick-Jones J., Suthers G. K., Love D. R., et al. Primary Structure of Dystrophin-Related Protein. Nature. 1992 ; 360: 591-593
11. Tinsley J., Deconinck N., Fisher R., Kahn D., Phelps S., Gillis J. M., Davies K.. Expression of Full-Length Utrophin Prevents Muscular Dystrophy in mdx Mice. Nat. Med. 1998 ; 4: 1441-1444
12. Tinsley J. M., Fairclough R. J., Storer R., Wilkes F. J., Potter A. C., Squire S. E., Powell D. S., Cozzoli A., Capogrosso R. F., Lambert A., Wilson F. X., Wren S. P., De Luca A., Davies K. E.. Daily Treatment with SMTC1100, a Novel Small Molecule Utrophin Upregulator, Dramatically Reduces the Dystrophic Symptoms in the mdx Mouse. PloS One. 2011 ; 6: e19189
13. Cerletti M., Negri T., Cozzi F., Colpo R., Andreetta F., Croci D., Davies K. E., Cornelio F., Pozza O., Karpati G., Gilbert R., Mora M.. Dystrophic Phenotype of Canine X-Linked Muscular Dystrophy Is Mitigated by Adenovirus-Mediated Utrophin Gene Transfer. Gene Ther. 2003 ; 10: 750-757
14. Krag T. O., Bogdanovich S., Jensen C. J., Fischer M. D., Hansen-Schwartz J., Javazon E. H., Flake A. W., Edvinsson L., Khurana T. S.. Heregulin Ameliorates the Dystrophic Phenotype in mdx Mice. Proc. Natl. Acad. Sci. U. S. A. 2004 ; 101: 13856-13860
15. Barton E. R., Morris L., Kawana M., Bish L. T., Toursel T.. Systemic Administration of L-Arginine Benefits mdx Skeletal Muscle Function. Muscle Nerve. 2005 ; 32: 751-760
16. Voisin V., Sebrie C., Matecki S., Yu H., Gillet B., Ramonatxo M., Israel M., De la Porte S.. L-Arginine Improves Dystrophic Phenotype in mdx Mice. Neurobiol. Dis. 2005 ; 20: 123-130
17. Lu Y., Tian C., Danialou G., Gilbert R., Petrof B. J., Karpati G., Nalbantoglu J.. Targeting Artificial Transcription Factors to the Utrophin A Promoter: Effects on Dystrophic Pathology and Muscle Function. J. Biol. Chem. 2008 ; 283: 34720-34727
18. Sonnemann K. J., Heun-Johnson H., Turner A. J., Baltgalvis K. A., Lowe D. A., Ervasti J. M.. Functional Substitution by TAT-Utrophin in Dystrophin-Deficient Mice. PLoS Med. 2009 ; 6: e1000083
19. Gramolini A. O., Angus L. M., Schaeffer L., Burton E. A., Tinsley J. M., Davies K. E., Changeux J. P., Jasmin B. J.. Induction of Utrophin Gene Expression by Heregulin in Skeletal Muscle Cells: Role of the N-box Motif and GA Binding Protein. Proc. Natl. Acad. Sci. U. S. A. 1999 ; 96: 3223-3227
20. Khurana T. S., Rosmarin A. G., Shang J., Krag T. O., Das S., Gammeltoft S.. Activation of Utrophin Promoter by Heregulin via the ets-Related Transcription Factor Complex GA-Binding Protein Alpha/Beta. Mol. Biol. Cell. 1999 ; 10: 2075-2086
21. Galvagni F., Capo S., Oliviero S.. Sp1 and Sp3 Physically Interact and Co-operate with GABP for the Activation of the Utrophin Promoter. J. Mol. Biol. 2001 ; 306: 985-996
22. Perkins K. J., Burton E. A., Davies K. E.. The Role of Basal and Myogenic Factors in the Transcriptional Activation of Utrophin Promoter A: Implications for Therapeutic Up-regulation in Duchenne Muscular Dystrophy. Nucleic Acids Res. 2001 ; 29: 4843-4850
23. Rodova M., Brownback K., Werle M. J.. Okadaic Acid Augments Utrophin in Myogenic Cells. Neurosci. Lett. 2004 ; 363: 163-167
24. Miura P., Chakkalakal J. V., Boudreault L., Belanger G., Hebert R. L., Renaud J. M., Jasmin B. J.. Pharmacological Activation of PPARbeta/Delta Stimulates Utrophin A Expression in Skeletal Muscle Fibers and Restores Sarcolemmal Integrity in Mature mdx Mice. Human Mol. Genet. 2009 ; 18: 4640-4649
25. Miura P., Thompson J., Chakkalakal J. V., Holcik M., Jasmin B. J.. The Utrophin A 5′-Untranslated Region Confers Internal Ribosome Entry Site-Mediated Translational Control during Regeneration of Skeletal Muscle Fibers. J. Biol. Chem.. 2005 ; 280: 32997-33005
26. Rosenberg M. I., Georges S. A., Asawachaicharn A., Analau E., Tapscott S. J.. MyoD Unhibits Fstl1 and Utrn Expression by Inducing Transcription of miR-206. J. Cell Biol. 2006 ; 175: 77-85
27. Chakkalakal J. V., Miura P., Belanger G., Michel R. N., Jasmin B. J.. Modulation of Utrophin A mRNA Stability in Fast versus Slow Muscles via an AU-Rich Element and Calcineurin Signaling. Nucleic Acids Res. 2008 ; 36: 826-838
28. Basu U., Lozynska O., Moorwood C., Patel G., Wilton S. D., Khurana T. S.. Translational Regulation of Utrophin by miRNAs. PloS One. 2011 ; 6: e29376
29. Moorwood C., Lozynska O., Suri N., Napper A. D., Diamond S. L., Khurana T. S.. Drug Discovery for Duchenne Muscular Dystrophy via Utrophin Promoter Activation Screening. PloS One. 2011 ; 6: e26169
30. Chancellor D. R., Davies K. E., De Moor O., Dorgan C. R., Johnson P. D., Lambert A. G., Lawrence D., Lecci C., Maillol C., Middleton P. J., Nugent G., Poignant S. D., Potter A. C., Price P. D., Pye R. J., Storer R., Tinsley J. M., van Well R., Vickers R., Vile J., Wilkes F. J., Wilson F. X., Wren S. P., Wynne G. M.. Discovery of 2-arylbenzoxazoles as Upregulators of Utrophin Production for the Treatment of Duchenne Muscular Dystrophy. J. Med. Chem. 2011 ; 54: 3241-3250
31. National Institutes of Health Molecular Libraries Program. MLSMR Compounds. http://mli.nih.gov/mli/compound-repository/mlsmr-compounds/.