The HDAC Inhibitor TSA Ameliorates a Zebrafish Model of Duchenne Muscular Dystrophy

PLoS Currents

Volumn , Issue SEP, 2013, Pages

  Johnson, Nathan M ^a   Farr III, Gist H ^a   Maves, Lisa ^a,b  

^a SEATTLE CHILDREN S RESEARCH INSTITUTE   (United States)

^b UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ACTIN; DYSTROPHIN; MORPHOLINO OLIGONUCLEOTIDE; PHALLOIDIN; TRICHOSTATIN A;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BIREFRINGENCE; CONFOCAL MICROSCOPY; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EMBRYO; IMMUNOCYTOCHEMISTRY; IMMUNOHISTOCHEMISTRY; MUSCLE INJURY; NONHUMAN; PHENOTYPE;

EID: 84884698672     PISSN: None     EISSN: 21573999     Source Type: Journal    
DOI: 10.1371/currents.md.8273cf41db10e2d15dd3ab827cb4b027     Document Type: Article

References (33)

1. Dalkilic I, Kunkel LM. 2003. Muscular dystrophies: genes to pathogenesis. Curr Opin Genet Dev. 13, 231-238.
2. Wallace GQ, McNally EM. 2009. Mechanisms of muscle degeneration, regeneration, and repair in the muscular dystrophies. Annu Rev Physiol. 71, 37-57.
3. Monaco AP, Neve RL, Colletti-Feener C, Bertelson CJ, Kurnit DM, Kunkel LM. 1986. Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene. Nature 323, 646-650.
4. Fairclough RJ, Bareja A, Davies KE. 2011. Progress in therapy for Duchenne muscular dystrophy. Exp. Physiol. 96, 1101-1113.
5. Pichavant C, Aartsma-Rus A, Clemens PR, Davies KE, Dickson G, Takeda S, Wilton SD, Wolff JA, Wooddell CI, Xiao X, Tremblay JP. 2011. Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther. 19, 830-840.
6. Tedesco FS, Cossu G. 2012. Stem cell therapies for muscle disorders. Curr Opin Neurol. 25, 597-603.
7. Consalvi S, Saccone, V, Giordani, L, Minetti, G, Mozzetta, C, Puri PL. 2011 Histone deacetylase inhibitors in the treatment of muscular dystrophies: epigenetic drugs for genetic diseases. Mol Med. 17, 457-465.
8. Abdel-Hamid H, Clemens PR. 2012. Pharmacological therapies for muscular dystrophies. Curr Opin Neurol. 25, 604-608.
9. Zon LI, Peterson RT. 2005. In vivo drug discovery in the zebrafish. Nat Rev Drug Discov. 4, 35-44.
10. Bassett DI, Bryson-Richardson RJ, Daggett DF, Gautier P, Keenan DG, Currie PD. 2003. Dystrophin is required for the formation of stable muscle attachments in the zebrafish embryo. Development 130, 5851-5860.
11. Guyon JR, Mosley AN, Zhou Y, O'Brien K F, Sheng X, Chiang K, Davidson AJ, Volinski JM, Zon LI, Kunkel LM. 2003. The dystrophin associated protein complex in zebrafish. Hum. Mol. Genet. 12, 601-615.
12. Berger J, Berger S, Hall TE, Lieschke GJ, Currie PD. 2010. Dystrophin-deficient zebrafish feature aspects of the Duchenne muscular dystrophy pathology. Neuromuscul Disord. 20, 826-832.
13. Kawahara G, Karpf JA, Myers JA, Alexander MS, Guyon JR, Kunkel LM. 2011. Drug screening in a zebrafish model of Duchenne muscular dystrophy. Proc Natl Acad Sci U S A. 108, 5331-5336.
14. Winder SJ, Lipscomb L, Angela Parkin C, Juusola M. 2011. The proteasomal inhibitor MG132 prevents muscular dystrophy in zebrafish. PLoS Curr. 3:RRN1286.
15. Goody MF, Kelly MW, Reynolds CJ, Khalil A, Crawford BD, Henry CA. 2012. NAD+ biosynthesis ameliorates a zebrafish model of muscular dystrophy. PLoS Biol. 10, e1001409.
16. Dowling JJ, Arbogast S, Hur J, Nelson DD, McEvoy A, Waugh T, Marty I, Lunardi J, Brooks SV, Kuwada JY, Ferreiro A. 2012. Oxidative stress and successful antioxidant treatment in models of RYR1-related myopathy. Brain 135, 1115-1127.
17. Etard C, Behra M, Ertzer R, Fischer N, Jesuthasan S, Blader P, Geisler R, Strahle U. 2005. Mutation in the delta-subunit of the nAChR suppresses the muscle defects caused by lack of Dystrophin. Dev Dyn. 234, 1016-1025.
18. Minetti GC, Colussi C, Adami R, Serra C, Mozzetta C, Parente V, Fortuni S, Straino S, Sampaolesi M, Di Padova M, Illi B, Gallinari P, Steinkühler C, Capogrossi MC, Sartorelli V, Bottinelli R, Gaetano C, Puri PL. 2006. Functional and morphological recovery of dystrophic muscles in mice treated with deacetylase inhibitors. Nat Med. 12, 1147-1150.
19. Colussi C, Mozzetta C, Gurtner A, Illi B, Rosati J, Straino S, Ragone G, Pescatori M, Zaccagnini G, Antonini A, Minetti G, Martelli F, Piaggio G, Gallinari P, Steinkuhler C, Clementi E, Dell'Aversana C, Altucci L, Mai A, Capogrossi MC, Puri PL, Gaetano C. 2008. HDAC2 blockade by nitric oxide and histone deacetylase inhibitors reveals a common target in Duchenne muscular dystrophy treatment. Proc Natl Acad Sci U S A. 105, 19183-19187.
20. Consalvi S, Saccone, V, Giordani, L, Minetti, G, Mozzetta, C, Puri PL. 2011 Histone deacetylase inhibitors in the treatment of muscular dystrophies: epigenetic drugs for genetic diseases. Mol Med. 17, 457-465.
21. Mozzetta C, Consalvi S, Saccone V, Tierney M, Diamantini A, Mitchell KJ, Marazzi G, Borsellino G, Battistini L, Sassoon D, Sacco A, Puri PL. 2013. Fibroadipogenic progenitors mediate the ability of HDAC inhibitors to promote regeneration in dystrophic muscles of young, but not old Mdx mice. EMBO Mol Med. 5, 1-14.
22. Westerfield M. 2007. The Zebrafish Book, 5th Edition; A guide for the laboratory use of zebrafish (Danio rerio).
23. Higashijima S, Okamoto H, Ueno N, Hotta Y, Eguchi G. 1997. High-frequency generation of transgenic zebrafish which reliably express GFP in whole muscles or the whole body by using promoters of zebrafish origin. Dev Biol. 192, 289-299.
24. Granato M, van Eeden FJ, Schach U, Trowe T, Brand M, Furutani-Seiki M, Haffter P, Hammerschmidt M, Heisenberg CP, Jiang YJ, Kane DA, Kelsh RN, Mullins MC, Odenthal J, Nüsslein-Volhard C. 1996. Genes controlling and mediating locomotion behavior of the zebrafish embryo and larva. Development 123, 399-413.
25. Berger J, Berger S, Jacoby AS, Wilton SD, Currie PD. 2011. Evaluation of exon-skipping strategies for Duchenne muscular dystrophy utilizing dystrophin-deficient zebrafish. J. Cell. Mol. Med. 15, 2643-2651.
26. Maves L, Jackman W, Kimmel CB. 2002. FGF3 and FGF8 mediate a rhombomere 4 signaling activity in the zebrafish hindbrain. Development 129, 3825-3837.
27. Bird NC, Windner SE, Devoto SH. 2012. Immunocytochemistry to study myogenesis in zebrafish. Methods Mol. Biol. 798, 153-169.
28. Kawahara G, Guyon JR, Nakamura Y, Kunkel LM. 2010. Zebrafish model for human FKRP muscular dystrophies. Hum Mol Genet. 19, 623-633.
29. Ekker SC, Larson JD. 2001. Morphant technology in model developmental systems. Genesis 30, 89-93.
30. Draper BW, Morcos PA, Kimmel CB. 2001. Inhibition of zebrafish fgf8 pre-mRNA splicing with morpholino oligos: a quantifiable method for gene knockdown. Genesis 30, 154-156.
31. Maves L, Waskiewicz AJ, Paul B, Cao Y, Tyler A, Moens CB, Tapscott SJ. 2007. Pbx homeodomain proteins direct Myod activity to promote fast-muscle differentiation. Development 134, 3371-3382.
32. Felsenfeld AL, Walker C, Westerfield M, Kimmel C, Streisinger G. 1990. Mutations affecting skeletal muscle myofibril structure in the zebrafish. Development 108, 443-459.
33. Berger J, Sztal T, Currie PD. 2012. Quantification of birefringence readily measures the level of muscle damage in zebrafish. Biochem. Biophys. Res. Commun. 423, 785-788.