Pharmacologically targeting the primary defect and downstream pathology in duchenne muscular dystrophy

Current Gene Therapy

Volumn 12, Issue 3, 2012, Pages 206-244

  Fairclough, Rebecca J ^a   Perkins, Kelly J ^a   Davies, Kay E ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

Cardiac; Duchenne; Dystrophy; Fibrosis; Inflammation; Muscular; Pharmacological; Utrophin

Indexed keywords

4 BENZHYDRYLOXY 1 [3 (1H TETRAZOL 5 YL)PROPYL]PIPERIDINE; 5 AMINO 4 IMIDAZOLECARBOXAMIDE RIBOSIDE; [2 METHYL 4 [4 METHYL 2 (4 TRIFLUOROMETHYLPHENYL) 5 THIAZOLYLMETHYLTHIO]PHENOXY]ACETIC ACID; ARGININE; ATALUREN; BETA ADRENERGIC RECEPTOR BLOCKING AGENT; BMN 195; BORTEZOMIB; CATENA; CORTICOSTEROID; CURCUMIN; DEFLAZACORT; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; ETANERCEPT; FLAVOCOXID; GENTAMICIN; GLUCOCORTICOID; HYDROXYUREA; I KAPPA B; IMATINIB; INFLIXIMAB; LAMININ; LIMBREL; MATRIX METALLOPROTEINASE 2 INHIBITOR; MATRIX METALLOPROTEINASE 9 INHIBITOR; MATRIX METALLOPROTEINASE INHIBITOR; MOLSIDOMINE; NABUMETONE; NB 74; NB 84; NEGAMYCIN; PHOSPHODIESTERASE V INHIBITOR; POLOXAMER; PREDNISOLONE; PRT 01; PYRROLIDINE DITHIOCARBAMATE; RTC 13; RTC 14; UNCLASSIFIED DRUG; UNINDEXED DRUG; URSODEOXYCHOLIC ACID; VALPROIC ACID;

CELL BASED GENE THERAPY; DIET SUPPLEMENTATION; DUCHENNE MUSCULAR DYSTROPHY; EXPERIMENTAL MODEL; FIBROSIS; GENE; GENE THERAPY; HUMAN; INFLAMMATION; LIFE EXPECTANCY; NONHUMAN; NONSENSE MUTATION; PATHOLOGY; PATHOPHYSIOLOGY; RENIN ANGIOTENSIN ALDOSTERONE SYSTEM; REVIEW; UPREGULATION;

AMINOGLYCOSIDES; ANTIGENS, CD; CODON, NONSENSE; DYSTROPHIN; GENE EXPRESSION; GENETIC THERAPY; HUMANS; INTEGRIN ALPHA CHAINS; MOLECULAR TARGETED THERAPY; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; SURAMIN; UTROPHIN;

EID: 84862635331     PISSN: 15665232     EISSN: 18755631     Source Type: Journal    
DOI: 10.2174/156652312800840595     Document Type: Review

References (574)

1. rd ed. Oxford: Oxford University Press 2008.
2. Koenig M, Monaco AP, Kunkel LM. The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein. Cell 1988; 53: 219-28.
3. Levine BA, Moir AJ, Patchell VB, Perry SV. The interaction of actin with dystrophin. FEBS Lett 1990; 263: 159-62.
4. Ibraghimov-Beskrovnaya O, Ervasti JM, Leveille CJ, Slaughter CA, Sernett SW, Campbell KP. Primary structure of dystrophinassociated glycoproteins linking dystrophin to the extracellular matrix. Nature 1992; 355: 696-702.
5. Davies KE, Nowak KJ. Molecular mechanisms of muscular dystrophies: old and new players. Nat Rev Mol Cell Biol 2006; 7: 762-73.
6. Jung D, Yang B, Meyer J, Chamberlain JS, Campbell KP. Identification and characterization of the dystrophin anchoring site on beta-dystroglycan. J Biol Chem 1995; 270: 27305-10.
7. Yang B, Jung D, Rafael JA, Chamberlain JS, Campbell KP. Identification of alpha-syntrophin binding to syntrophin triplet, dystrophin, and utrophin. J Biol Chem 1995; 270: 4975-8.
8. Sadoulet-Puccio HM, Rajala M, Kunkel LM. Dystrobrevin and dystrophin: an interaction through coiled-coil motifs. Proc Natl Acad Sci U S A 1997; 94: 12413-8.
9. Oak SA, Zhou YW, Jarrett HW. Skeletal muscle signaling pathway through the dystrophin glycoprotein complex and Rac1. J Biol Chem 2003; 278: 39287-95.
10. Brenman JE, Chao DS, Xia H, Aldape K, Bredt DS. Nitric oxide synthase complexed with dystrophin and absent from skeletal muscle sarcolemma in Duchenne muscular dystrophy. Cell 1995; 82: 743-52.
11. Yang B, Jung D, Motto D, Meyer J, Koretzky G, Campbell KP. SH3 domain-mediated interaction of dystroglycan and Grb2. J Biol Chem 1995; 270: 11711-4.
12. Abramovici H, Hogan AB, Obagi C, Topham MK, Gee SH. Diacylglycerol kinase-zeta localization in skeletal muscle is regulated by phosphorylation and interaction with syntrophins. Mol Biol Cell 2003; 14: 4499-511.
13. Spence HJ, Dhillon AS, James M, Winder SJ. Dystroglycan, a scaffold for the ERK-MAP kinase cascade. EMBO Rep 2004; 5: 484-9.
14. Lapidos KA, Kakkar R, McNally EM. The dystrophin glycoprotein complex: signaling strength and integrity for the sarcolemma. Circ Res 2004; 94: 1023-31.
15. Judge LM, Haraguchiln M, Chamberlain JS. Dissecting the signaling and mechanical functions of the dystrophin-glycoprotein complex. J Cell Sci 2006; 119: 1537-46.
16. Ervasti JM, Campbell KP. Membrane organization of the dystrophin-glycoprotein complex. Cell 1991; 66: 1121-31.
17. Ervasti JM, Ohlendieck K, Kahl SD, Gaver MG, Campbell KP. Deficiency of a glycoprotein component of the dystrophin complex in dystrophic muscle. Nature 1990; 345: 315-9.
18. Zubrzycka-Gaarn EE, Bulman DE, Karpati G, et al. The Duchenne muscular dystrophy gene product is localized in sarcolemma of human skeletal muscle. Nature 1988; 333: 466-9.
19. Lynch GS, Rafael JA, Chamberlain JS, Faulkner JA. Contractioninduced injury to single permeabilized muscle fibers from mdx, transgenic mdx, and control mice. Am J Physiol Cell Physiol 2000; 279: C1290-4.
20. Carnwath JW, Shotton DM. Muscular dystrophy in the mdx mouse: histopathology of the soleus and extensor digitorum longus muscles. J Neurol Sci 1987; 80: 39-54.
21. Tanabe Y, Esaki K, Nomura T. Skeletal muscle pathology in X chromosome-linked muscular dystrophy (mdx) mouse. Acta Neuropathol 1986; 69: 91-5.
22. Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010; 9: 77-93.
23. Wang CH, Bonnemann CG, Rutkowski A, et al. Consensus statement on standard of care for congenital muscular dystrophies. J Child Neurol 2010; 25: 1559-81.
24. Moxley RT, 3rd, Ashwal S, Pandya S, et al. Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology 2005; 64: 13-20.
25. Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev 2008: CD003725.
26. DeSilva S, Drachman DB, Mellits D, Kuncl RW. Prednisone treatment in Duchenne muscular dystrophy. Long-term benefit. Arch Neurol 1987; 44: 818-22.
27. Moxley RT, 3rd, Pandya S. Weekend high-dosage prednisone: a new option for treatment of Duchenne muscular dystrophy. Neurology 2011; 77: 416-7.
28. Escolar DM, Hache LP, Clemens PR, et al. Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology 2011; 77: 444-52.
29. Mazzone E, Vasco G, Sormani MP, et al. Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study. Neurology 2011; 77: 250-6.
30. Drachman DB, Toyka KV, Myer E. Prednisone in Duchenne muscular dystrophy. Lancet 1974; 2: 1409-12.
31. Bulfield G, Siller WG, Wight PA, Moore KJ. X chromosomelinked muscular dystrophy (mdx) in the mouse. Proc Natl Acad Sci U S A 1984; 81: 1189-92.
32. McGeachie JK, Grounds MD, Partridge TA, Morgan JE. Age-related changes in replication of myogenic cells in mdx mice: quantitative autoradiographic studies. J Neurol Sci 1993; 119: 169-79.
33. Spencer MJ, Tidball JG. Calpain translocation during muscle fiber necrosis and regeneration in dystrophin-deficient mice. Exp Cell Res 1996; 226: 264-72.
34. Stuckey DJ, Carr CA, Camelliti P, Tyler DJ, Davies KE, Clarke K. In vivo MRI Characterization of Progressive Cardiac Dysfunction in the mdx Mouse Model of Muscular Dystrophy. PLoS ONE 2012; 7: e28569.
35. Quinlan JG, Hahn HS, Wong BL, Lorenz JN, Wenisch AS, Levin LS. Evolution of the mdx mouse cardiomyopathy: physiological and morphological findings. Neuromuscul Disord 2004; 14: 491-6.
36. Wehling-Henricks M, Jordan MC, Roos KP, Deng B, Tidball JG. Cardiomyopathy in dystrophin-deficient hearts is prevented by expression of a neuronal nitric oxide synthase transgene in the myocardium. Hum Mol Genet 2005; 14: 1921-33.
37. Van Erp C, Irwin NG, Hoey AJ. Long-term administration of pirfenidone improves cardiac function in mdx mice. Muscle Nerve 2006; 34: 327-34.
38. Stedman HH, Sweeney HL, Shrager JB, et al. The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy. Nature 1991; 352: 536-9.
39. Willmann R, Possekel S, Dubach-Powell J, Meier T, Ruegg MA. Mammalian animal models for Duchenne muscular dystrophy. Neuromuscul Disord 2009; 19: 241-9.
40. Deconinck AE, Rafael JA, Skinner JA, et al. Utrophin-dystrophindeficient mice as a model for Duchenne muscular dystrophy. Cell 1997; 90: 717-27.
41. Grady RM, Teng H, Nichol MC, Cunningham JC, Wilkinson RS, Sanes JR. Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy. Cell 1997; 90: 729-38.
42. Zhou L, Rafael-Fortney JA, Huang P, et al. Haploinsufficiency of utrophin gene worsens skeletal muscle inflammation and fibrosis in mdx mice. J Neurol Sci 2008; 264: 106-11.
43. van Putten M, Kumar D, Hulsker M, et al. Comparison of skeletal muscle pathology and motor function of dystrophin and utrophin deficient mouse strains. Neuromuscul Disord 2012.
44. Sharp NJ, Kornegay JN, Van Camp SD, et al. An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy. Genomics 1992; 13: 115-21.
45. Pichavant C, Aartsma-Rus A, Clemens PR, et al. Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther 2011; 19: 830-40.
46. Goyenvalle A, Davies KE. Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy. Skelet Muscle 2011; 1: 8.
47. Goyenvalle A, Seto JT, Davies KE, Chamberlain J. Therapeutic approaches to muscular dystrophy. Hum Mol Genet 2011; 20: R69-78.
48. Fairclough RJ, Bareja A, Davies KE. Progress in therapy for Duchenne muscular dystrophy. Experimental physiology 2011; 96: 1101-13.
49. Khurana TS, Davies KE. Pharmacological strategies for muscular dystrophy. Nat Rev Drug Discov 2003; 2: 379-90.
50. Mendell JR, Campbell K, Rodino-Klapac L, et al. Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med 2010; 363: 1429-37.
51. Malik V, Rodino-Klapac LR, Viollet L, et al. Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy. Ann Neurol 2010; 67: 771-80.
52. Chen YW, Nagaraju K, Bakay M, et al. Early onset of inflammation and later involvement of TGFbeta in Duchenne muscular dystrophy. Neurology 2005; 65: 826-34.
53. Gosselin LE, Williams JE, Deering M, Brazeau D, Koury S, Martinez DA. Localization and early time course of TGF-beta 1 mRNA expression in dystrophic muscle. Muscle Nerve 2004; 30: 645-53.
54. Weiner AM, Weber K. A single UGA codon functions as a natural termination signal in the coliphage q beta coat protein cistron. J Mol Biol 1973; 80: 837-55.
55. Lovett PS, Ambulos NP, Jr., Mulbry W, Noguchi N, Rogers EJ. UGA can be decoded as tryptophan at low efficiency in Bacillus subtilis. J Bacteriol 1991; 173: 1810-2.
56. Keeling KM, Brooks DA, Hopwood JJ, Li P, Thompson JN, Bedwell DM. Gentamicin-mediated suppression of Hurler syndrome stop mutations restores a low level of alpha-Liduronidase activity and reduces lysosomal glycosaminoglycan accumulation. Hum Mol Genet 2001; 10: 291-9.
57. Zingman LV, Park S, Olson TM, Alekseev AE, Terzic A. Aminoglycoside-induced translational read-through in disease: overcoming nonsense mutations by pharmacogenetic therapy. Clin Pharm Ther 2007; 81: 99-103.
58. Mendell JT, Dietz HC. When the message goes awry: diseaseproducing mutations that influence mRNA content and performance. Cell 2001; 107: 411-4.
59. Burke JF, Mogg AE. Suppression of a nonsense mutation in mammalian cells in vivo by the aminoglycoside antibiotics G-418 and paromomycin. Nucleic Acids Res 1985; 13: 6265-72.
60. Barton-Davis ER, Cordier L, Shoturma DI, Leland SE, Sweeney HL. Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice. J Clin Invest 1999; 104: 375-81.
61. Wagner KR, Hamed S, Hadley DW, et al. Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations. Ann Neurol 2001; 49: 706-11.
62. Politano L, Nigro G, Nigro V, et al. Gentamicin administration in Duchenne patients with premature stop codon. Preliminary results. Acta Myol 2003; 22: 15-21.
63. Selimoglu E. Aminoglycoside-induced ototoxicity. Curr Pharm Des 2007; 13: 119-26.
64. Tang HY, Hutcheson E, Neill S, Drummond-Borg M, Speer M, Alford RL. Genetic susceptibility to aminoglycoside ototoxicity: how many are at risk? Genet Med: Am Coll Med Genet 2002; 4: 336-45.
65. Mingeot-Leclercq MP, Tulkens PM. Aminoglycosides: nephrotoxicity. Antimicrob Agents Chemother 1999; 43: 1003-12.
66. Guan MX, Fischel-Ghodsian N, Attardi G. A biochemical basis for the inherited susceptibility to aminoglycoside ototoxicity. Hum Mol Genet 2000; 9: 1787-93.
67. Arakawa M, Shiozuka M, Nakayama Y, et al. Negamycin restores dystrophin expression in skeletal and cardiac muscles of mdx mice. Journal of biochemistry 2003; 134: 751-8.
68. Balaban B, Tok F, Tan AK, Matthews DJ. Botulinum toxin a treatment in children with cerebral palsy: its effects on walking and energy expenditure. Am J Phys Med Rehabil 2012; 91: 53-64.
69. Nudelman I, Glikin D, Smolkin B, Hainrichson M, Belakhov V, Baasov T. Repairing faulty genes by aminoglycosides: development of new derivatives of geneticin (G418) with enhanced suppression of diseases-causing nonsense mutations. Bioorg Med Chem 2010; 18: 3735-46.
70. Carter AP, Clemons WM, Brodersen DE, Morgan-Warren RJ, Wimberly BT, Ramakrishnan V. Functional insights from the structure of the 30S ribosomal subunit and its interactions with antibiotics. Nature 2000; 407: 340-8.
71. Borovinskaya MA, Pai RD, Zhang W, et al. Structural basis for aminoglycoside inhibition of bacterial ribosome recycling. Nature structural & molecular biology 2007; 14: 727-32.
72. Yukihara M, Ito K, Tanoue O, et al. Effective drug delivery system for duchenne muscular dystrophy using hybrid liposomes including gentamicin along with reduced toxicity. Biol Pharm Bull 2011; 34: 712-6.
73. Du L, Damoiseaux R, Nahas S, et al. Nonaminoglycoside compounds induce readthrough of nonsense mutations. J Exp Med 2009; 206: 2285-97.
74. Welch EM, Barton ER, Zhuo J, et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature 2007; 447: 87-91.
75. Childers MK, Okamura CS, Bogan DJ, et al. Eccentric contraction injury in dystrophic canine muscle. Arch Phys Med Rehabil 2002; 83: 1572-8.
76. Dellorusso C, Crawford RW, Chamberlain JS, Brooks SV. Tibialis anterior muscles in mdx mice are highly susceptible to contractioninduced injury. J Muscle Res Cell Motil 2001; 22: 467-75.
77. Brooks SV. Rapid recovery following contraction-induced injury to in situ skeletal muscles in mdx mice. J Muscle Res Cell Motil 1998; 19: 179-87.
78. Hirawat S, Welch EM, Elfring GL, et al. Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following singleand multiple-dose administration to healthy male and female adult volunteers. J Clin Pharmacol 2007; 47: 430-44.
79. Love DR, Hill DF, Dickson G, et al. An autosomal transcript in skeletal muscle with homology to dystrophin. Nature 1989; 339: 55-8.
80. Khurana TS, Hoffman EP, Kunkel LM. Identification of a chromosome 6-encoded dystrophin-related protein. J Biol Chem 1990; 265: 16717-20.
81. Pearce M, Blake DJ, Tinsley JM, et al. The utrophin and dystrophin genes share similarities in genomic structure. Hum Mol Genet 1993; 2: 1765-72.
82. Tinsley JM, Blake DJ, Roche A, et al. Primary Structure of Dystrophin-Related Protein. Nature 1992; 360: 591-3.
83. Blake DJ, Tinsley JM, Davies KE. Utrophin: a structural and functional comparison to dystrophin. Brain Pathol 1996; 6: 37-47.
84. Nguyen TM, Ellis JM, Love DR, et al. Localization of the DMDL gene-encoded dystrophin-related protein using a panel of nineteen monoclonal antibodies: presence at neuromuscular junctions, in the sarcolemma of dystrophic skeletal muscle, in vascular and other smooth muscles, and in proliferating brain cell lines. J Cell Biol 1991; 115: 1695-700.
85. Ishikawa-Sakurai M, Yoshida M, Imamura M, Davies KE, Ozawa E. ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to beta-dystroglycan. Human Molecular Genetics 2004; 13: 693-702.
86. Amann KJ, Guo AW, Ervasti JM. Utrophin lacks the rod domain actin binding activity of dystrophin. J Biol Chem 1999; 274: 35375-80.
87. Amann KJ, Renley BA, Ervasti JM. A cluster of basic repeats in the dystrophin rod domain binds F-actin through an electrostatic interaction. Journal of Biological Chemistry 1998; 273: 28419-23.
88. Rybakova IN, Ervasti JM. Identification of spectrin-like repeats required for high affinity utrophin-actin interaction. J Biol Chem 2005; 280: 23018-23.
89. Rybakova IN, Patel JR, Davies KE, Yurchenco PD, Ervasti JM. Utrophin binds laterally along actin filaments and can couple costameric actin with sarcolemma when overexpressed in dystrophin-deficient muscle. Mol Biol Cell 2002; 13: 1512-21.
90. Clerk A, Morris GE, Dubowitz V, Davies KE, Sewry CA. Dystrophin-related protein, utrophin, in normal and dystrophic human fetal skeletal muscle. Histochem J 1993; 25: 554-61.
91. Tome FM, Matsumura K, Chevallay M, Campbell KP, Fardeau M. Expression of dystrophin-associated glycoproteins during human fetal muscle development: a preliminary immunocytochemical study. Neuromuscul Disord 1994; 4: 343-8.
92. Schofield J, Houzelstein D, Davies K, Buckingham M, Edwards YH. Expression of the dystrophin-related protein (utrophin) gene during mouse embryogenesis. Dev Dyn 1993; 198: 254-64.
93. Mizuno Y, Nonaka I, Hirai S, Ozawa E. Reciprocal expression of dystrophin and utrophin in muscles of Duchenne muscular dystrophy patients, female DMD-carriers and control subjects. J Neurol Sci 1993; 119: 43-52.
94. Pons F, Nicholson LV, Robert A, Voit T, Leger JJ. Dystrophin and dystrophin-related protein (utrophin) distribution in normal and dystrophin-deficient skeletal muscles. Neuromuscul Disord 1993; 3: 507-14.
95. Weir AP, Burton EA, Harrod G, Davies KE. A- and B-utrophin have different expression patterns and are differentially upregulated in mdx muscle. J Biol Chem 2002; 277: 45285-90.
96. Kleopa KA, Drousiotou A, Mavrikiou E, Ormiston A, Kyriakides T. Naturally occurring utrophin correlates with disease severity in Duchenne muscular dystrophy. Hum Mol Genet 2006; 15: 1623-8.
97. Fisher R, Tinsley JM, Phelps SR, et al. Non-toxic ubiquitous overexpression of utrophin in the mdx mouse. Neuromuscul Disord 2001; 11: 713-21.
98. Rafael JA, Tinsley JM, Potter AC, Deconinck AE, Davies KE. Skeletal muscle-specific expression of a utrophin transgene rescues utrophin-dystrophin deficient mice. Nat Genet 1998; 19: 79-82.
99. Tinsley J, Deconinck N, Fisher R, et al. Expression of full-length utrophin prevents muscular dystrophy in mdx mice. Nat Med 1998; 4: 1441-4.
100. Tinsley JM, Potter AC, Phelps SR, Fisher R, Trickett JI, Davies KE. Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene. Nature 1996; 384: 349-53.
101. Gilbert R, Nalbanoglu J, Tinsley JM, Massie B, Davies KE, Karpati G. Efficient utrophin expression following adenovirus gene transfer in dystrophic muscle. Biochem Biophys Res Commun 1998; 242: 244-7.
102. Wakefield PM, Tinsley JM, Wood MJ, Gilbert R, Karpati G, Davies KE. Prevention of the dystrophic phenotype in dystrophin/utrophindeficient muscle following adenovirus-mediated transfer of a utrophin minigene. Gene Ther 2000; 7: 201-4.
103. Tinsley JM, Fairclough RJ, Storer R, et al. Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse. PLoS One 2011; 6: e19189.
104. Cerletti M, Negri T, Cozzi F, et al. Dystrophic phenotype of canine X-linked muscular dystrophy is mitigated by adenovirus-mediated utrophin gene transfer. Gene Ther 2003; 10: 750-7.
105. Squire S, Raymackers JM, Vandebrouck C, et al. Prevention of pathology in mdx mice by expression of utrophin: analysis using an inducible transgenic expression system. Hum Mol Genet 2002; 11: 3333-44.
106. Ahmad A, Brinson M, Hodges BL, Chamberlain JS, Amalfitano A. Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for duchenne muscular dystrophy. Hum Mol Genet 2000; 9: 2507-15.
107. Dennis CL, Tinsley JM, Deconinck AE, Davies KE. Molecular and functional analysis of the utrophin promoter. Nucleic Acids Res 1996; 24: 1646-52.
108. Burton EA, Tinsley JM, Holzfeind PJ, Rodrigues NR, Davies KE. 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: 14025-30.
109. Weir AP, Morgan JE, Davies KE. A-utrophin up-regulation in mdx skeletal muscle is independent of regeneration. Neuromuscul Disord 2004; 14: 19-23.
110. Angus LM, Chakkalakal JV, Mejat A, et al. Calcineurin-NFAT signaling, together with GABP and peroxisome PGC-1{alpha}, drives utrophin gene expression at the neuromuscular junction. Am J Physiol Cell Physiol 2005; 289: C908-17.
111. Chakkalakal JV, Stocksley MA, Harrison MA, et al. Expression of utrophin A mRNA correlates with the oxidative capacity of skeletal muscle fiber types and is regulated by calcineurin/NFAT signaling. Proc Natl Acad Sci U S A 2003; 100: 7791-6.
112. Krag TO, Bogdanovich S, Jensen CJ, et al. Heregulin ameliorates the dystrophic phenotype in mdx mice. Proc Natl Acad Sci U S A 2004; 101: 13856-60.
113. Gramolini AO, Dennis CL, Tinsley JM, et al. Local transcriptional control of utrophin expression at the neuromuscular synapse. J Biol Chem 1997; 272: 8117-20.
114. Gyrd-Hansen M, Krag TO, Rosmarin AG, Khurana TS. Sp1 and the ets-related transcription factor complex GABP alpha/beta functionally cooperate to activate the utrophin promoter. J Neurol Sci 2002; 197: 27-35.
115. Khurana TS, Rosmarin AG, Shang J, Krag TO, 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: 2075-86.
116. Gramolini AO, Angus LM, Schaeffer L, et al. 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: 3223-7.
117. Ljubicic V, Miura P, Burt M, et al. Chronic AMPK activation evokes the slow, oxidative myogenic program and triggers beneficial adaptations in mdx mouse skeletal muscle. Hum Mol Genet 2011; 20: 3478-93.
118. Miura P, Chakkalakal JV, Boudreault L, et al. Pharmacological activation of PPARbeta/delta stimulates utrophin A expression in skeletal muscle fibers and restores sarcolemmal integrity in mature mdx mice. Hum Mol Genet 2009; 18: 4640-9.
119. Chakkalakal JV, Miura P, Belanger G, Michel RN, Jasmin BJ. Modulation of utrophin A mRNA stability in fast versus slow muscles via an AU-rich element and calcineurin signaling. Nucleic Acids Res 2008; 36: 826-38.
120. Gramolini AO, Karpati G, Jasmin BJ. Discordant expression of utrophin and its transcript in human and mouse skeletal muscles. J Neuropathol Exp Neurol 1999; 58: 235-44.
121. Miura P, Andrews M, Holcik M, Jasmin BJ. IRES-mediated translation of utrophin A is enhanced by glucocorticoid treatment in skeletal muscle cells. PLoS One 2008; 3: e2309.
122. Miura P, Coriati A, Belanger G, et al. The utrophin A 5'-UTR drives cap-independent translation exclusively in skeletal muscles of transgenic mice and interacts with eEF1A2. Hum Mol Genet 2010; 19: 1211-20.
123. Basu U, Lozynska O, Moorwood C, Patel G, Wilton SD, Khurana TS. Translational Regulation of Utrophin by miRNAs. PLoS ONE 2011; 6: e29376.
124. Rosenberg MI, Georges SA, Asawachaicharn A, Analau E, Tapscott SJ. MyoD inhibits Fstl1 and Utrn expression by inducing transcription of miR-206. J Cell Biol 2006; 175: 77-85.
125. Cokic VP, Smith RD, Beleslin-Cokic BB, et al. Hydroxyurea induces fetal hemoglobin by the nitric oxide-dependent activation of soluble guanylyl cyclase. J Clin Invest 2003; 111: 231-9.
126. Olivieri NF, Weatherall DJ. The therapeutic reactivation of fetal haemoglobin. Hum Mol Genet 1998; 7: 1655-8.
127. Thomson S, Mahadevan LC, Clayton AL. MAP kinase-mediated signalling to nucleosomes and immediate-early gene induction. Semin Cell Dev Biol 1999; 10: 205-14.
128. Whitehead NP, Pham C, Gervasio OL, Allen DG. N-Acetylcysteine ameliorates skeletal muscle pathophysiology in mdx mice. J Physiol 2008; 586: 2003-14.
129. Chaubourt E, Fossier P, Baux G, Leprince C, Israel M, De La Porte S. Nitric oxide and l-arginine cause an accumulation of utrophin at the sarcolemma: a possible compensation for dystrophin loss in Duchenne muscular dystrophy. Neurobiol Dis 1999; 6: 499-507.
130. Chaubourt E, Voisin V, Fossier P, Baux G, Israel M, de La Porte S. The NO way to increase muscular utrophin expression? C R Acad Sci III 2000; 323: 735-40.
131. Voisin V, Sebrie C, Matecki S, et al. L-arginine improves dystrophic phenotype in mdx mice. Neurobiol Dis 2005; 20: 123-30.
132. Barton ER, Morris L, Kawana M, Bish LT, Toursel T. Systemic administration of L-arginine benefits mdx skeletal muscle function. Muscle Nerve 2005; 32: 751-60.
133. Hnia K, Gayraud J, Hugon G, et al. L-arginine decreases inflammation and modulates the nuclear factor-kappaB/matrix metalloproteinase cascade in mdx muscle fibers. Am J Pathol 2008; 172: 1509-19.
134. Guerron AD, Rawat R, Sali A, et al. Functional and molecular effects of arginine butyrate and prednisone on muscle and heart in the mdx mouse model of Duchenne Muscular Dystrophy. PLoS One 2010; 5: e11220.
135. Wehling-Henricks M, Jordan MC, Gotoh T, Grody WW, Roos KP, Tidball JG. Arginine metabolism by macrophages promotes cardiac and muscle fibrosis in mdx muscular dystrophy. PLoS One 2010; 5: e10763.
136. Marques MJ, Barbin IC, Taniguti AP, Oggian DS, Ferretti R, Santo Neto H. Myocardial fibrosis is unaltered by long-term administration of L-arginine in dystrophin deficient mdx mice: a histomorphometric analysis. Acta Biol Hung 2010; 61: 168-74.
137. Gramolini AO, Belanger G, Thompson JM, Chakkalakal JV, Jasmin BJ. Increased expression of utrophin in a slow vs. a fast muscle involves posttranscriptional events. Am J Physiol Cell Physiol 2001; 281: C1300-9.
138. Consolino CM, Brooks SV. Susceptibility to sarcomere injury induced by single stretches of maximally activated muscles of mdx mice. J Appl Physiol 2004; 96: 633-8.
139. Webster C, Silberstein L, Hays AP, Blau HM. Fast muscle fibers are preferentially affected in Duchenne muscular dystrophy. Cell 1988; 52: 503-13.
140. Moens P, Baatsen PH, Marechal G. Increased susceptibility of EDL muscles from mdx mice to damage induced by contractions with stretch. J Muscle Res Cell Motil 1993; 14: 446-51.
141. Chakkalakal JV, Harrison MA, Carbonetto S, Chin E, Michel RN, Jasmin BJ. Stimulation of calcineurin signaling attenuates the dystrophic pathology in mdx mice. Hum Mol Genet 2004; 13: 379-88.
142. St-Pierre SJ, Chakkalakal JV, Kolodziejczyk SM, Knudson JC, Jasmin BJ, Megeney LA. Glucocorticoid treatment alleviates dystrophic myofiber pathology by activation of the calcineurin/NF- AT pathway. FASEB J 2004; 18: 1937-9.
143. Luquet S, Lopez-Soriano J, Holst D, et al. Peroxisome proliferatoractivated receptor delta controls muscle development and oxidative capability. FASEB J 2003; 17: 2299-301.
144. Wang YX, Zhang CL, Yu RT, et al. Regulation of muscle fiber type and running endurance by PPARdelta. PLoS Biol 2004; 2: e294.
145. Lin J, Wu H, Tarr PT, et al. Transcriptional co-activator PGC-1 alpha drives the formation of slow-twitch muscle fibres. Nature 2002; 418: 797-801.
146. Narkar VA, Downes M, Yu RT, et al. AMPK and PPARdelta agonists are exercise mimetics. Cell 2008; 134: 405-15.
147. Handschin C, Kobayashi YM, Chin S, Seale P, Campbell KP, Spiegelman BM. PGC-1alpha regulates the neuromuscular junction program and ameliorates Duchenne muscular dystrophy. Genes Dev 2007; 21: 770-83.
148. Selsby JT, Morine KJ, Pendrak K, Barton ER, Sweeney HL. Rescue of Dystrophic Skeletal Muscle by PGC-1alpha Involves a Fast to Slow Fiber Type Shift in the mdx Mouse. PLoS ONE 2012; 7: e30063.
149. Leick L, Fentz J, Bienso RS, et al. PGC-1{alpha} is required for AICAR-induced expression of GLUT4 and mitochondrial proteins in mouse skeletal muscle. Am J Physiol Endocrinol Metab 2010; 299: E456-65.
150. Ljubicic V, Khogali S, Renaud JM, Jasmin BJ. Chronic AMPK stimulation attenuates adaptive signaling in dystrophic skeletal muscle. Am J Physiol Cell Physiol 2011; 302: C110-21.
151. Lopez-Soriano J, Chiellini C, Maffei M, Grimaldi PA, Argiles JM. Roles of skeletal muscle and peroxisome proliferator-activated receptors in the development and treatment of obesity. Endocr Rev 2006; 27: 318-29.
152. Gardner OS, Dewar BJ, Graves LM. Activation of mitogenactivated protein kinases by peroxisome proliferator-activated receptor ligands: an example of nongenomic signaling. Mol Pharm 2005; 68: 933-41.
153. Peraza MA, Burdick AD, Marin HE, Gonzalez FJ, Peters JM. The toxicology of ligands for peroxisome proliferator-activated receptors (PPAR). Toxicol Sci 2006; 90: 269-95.
154. Lampen A, Siehler S, Ellerbeck U, Gottlicher M, Nau H. New molecular bioassays for the estimation of the teratogenic potency of valproic acid derivatives in vitro: activation of the peroxisomal proliferator-activated receptor (PPARdelta). Toxicol Appl Pharmacol 1999; 160: 238-49.
155. Pold R, Jensen LS, Jessen N, et al. Long-term AICAR administration and exercise prevents diabetes in ZDF rats. Diabetes 2005; 54: 928-34.
156. Morgan JE, Beauchamp JR, Pagel CN, et al. Myogenic cell lines derived from transgenic mice carrying a thermolabile T antigen: a model system for the derivation of tissue-specific and mutationspecific cell lines. Dev Biol 1994; 162: 486-98.
157. Jat PS, Noble MD, Ataliotis P, et al. Direct derivation of conditionally immortal cell lines from an H-2Kb-tsA58 transgenic mouse. Proc Natl Acad Sci U S A 1991; 88: 5096-100.
158. Chancellor DR, Davies KE, De Moor O, et al. Discovery of 2- arylbenzoxazoles as upregulators of utrophin production for the treatment of Duchenne muscular dystrophy. J Med Chem 2011; 54: 3241-50.
159. De Moor O, Dorgan CR, Johnson PD, et al. Discovery and SAR of 2-arylbenzotriazoles and 2-arylindazoles as potential treatments for Duchenne muscular dystrophy. Bioorg Med Chem Lett 2011; 21: 4828-31.
160. Moorwood C, Lozynska O, Suri N, Napper AD, Diamond SL, Khurana TS. Drug discovery for Duchenne muscular dystrophy via utrophin promoter activation screening. PLoS One 2011; 6: e26169.
161. Corbi N, Libri V, Fanciulli M, Tinsley JM, Davies KE, Passananti C. The artificial zinc finger coding gene 'Jazz' binds the utrophin promoter and activates transcription. Gene Ther 2000; 7: 1076-83.
162. Mattei E, Corbi N, Di Certo MG, et al. Utrophin up-regulation by an artificial transcription factor in transgenic mice. PLoS One 2007; 2: e774.
163. Onori A, Desantis A, Buontempo S, et al. The artificial 4-zinc-finger protein Bagly binds human utrophin promoter A at the endogenous chromosomal site and activates transcription. Biochem Cell Biol 2007; 85: 358-65.
164. Desantis A, Onori A, Di Certo MG, et al. Novel activation domain derived from Che-1 cofactor coupled with the artificial protein Jazz drives utrophin upregulation. Neuromuscul Disord 2009; 19: 158-62.
165. Di Certo MG, Corbi N, Strimpakos G, et al. The artificial gene Jazz, a transcriptional regulator of utrophin, corrects the dystrophic pathology in mdx mice. Hum Mol Genet 2010; 19: 752-60.
166. Schwarze SR, Ho A, Vocero-Akbani A, Dowdy SF. In vivo protein transduction: delivery of a biologically active protein into the mouse. Science 1999; 285: 1569-72.
167. Sonnemann KJ, Heun-Johnson H, Turner AJ, Baltgalvis KA, Lowe DA, Ervasti JM. Functional substitution by TAT-utrophin in dystrophin-deficient mice. PLoS Med 2009; 6: e1000083.
168. Odom GL, Gregorevic P, Allen JM, Finn E, Chamberlain JS. Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophindeficient mice. Mol Ther: J Amn Soc Gene Ther 2008; 16: 1539-45.
169. Call JA, Ervasti JM, Lowe DA. TAT-muUtrophin mitigates the pathophysiology of dystrophin and utrophin double-knockout mice. J Appl Physiol 2011; 111: 200-5.
170. Fadic R, Mezzano V, Alvarez K, Cabrera D, Holmgren J, Brandan E. Increase in decorin and biglycan in Duchenne Muscular Dystrophy: role of fibroblasts as cell source of these proteoglycans in the disease. J Cell Mol Med 2006; 10: 758-69.
171. Zanotti S, Negri T, Cappelletti C, et al. Decorin and biglycan expression is differentially altered in several muscular dystrophies. Brain 2005; 128: 2546-55.
172. Mercado ML, Amenta AR, Hagiwara H, et al. Biglycan regulates the expression and sarcolemmal localization of dystrobrevin, syntrophin, and nNOS. FASEB J 2006; 20: 1724-6.
173. Amenta AR, Yilmaz A, Bogdanovich S, et al. Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice. Proc Natl Acad Sci U S A 2011; 108: 762-7.
174. Hynes RO. Integrins: versatility, modulation, and signaling in cell adhesion. Cell 1992; 69: 11-25.
175. Mayer U, Saher G, Fassler R, et al. Absence of integrin alpha 7 causes a novel form of muscular dystrophy. Nat Genet 1997; 17: 318-23.
176. Rooney JE, Gurpur PB, Burkin DJ. Laminin-111 protein therapy prevents muscle disease in the mdx mouse model for Duchenne muscular dystrophy. Proc Natl Acad Sci U S A 2009; 106: 7991-6.
177. Rooney JE, Gurpur PB, Yablonka-Reuveni Z, Burkin DJ. Laminin- 111 restores regenerative capacity in a mouse model for alpha7 integrin congenital myopathy. Am J Pathol 2009; 174: 256-64.
178. Vignier N, Moghadaszadeh B, Gary F, Beckmann J, Mayer U, Guicheney P. Structure, genetic localization, and identification of the cardiac and skeletal muscle transcripts of the human integrin alpha7 gene (ITGA7). Biochem Biophys Res Commun 1999; 260: 357-64.
179. Hodges BL, Hayashi YK, Nonaka I, Wang W, Arahata K, Kaufman SJ. Altered expression of the alpha7beta1 integrin in human and murine muscular dystrophies. J Cell Sci 1997; 110 (Pt 22): 2873-81.
180. Burkin DJ, Wallace GQ, Nicol KJ, Kaufman DJ, Kaufman SJ. Enhanced expression of the alpha 7 beta 1 integrin reduces muscular dystrophy and restores viability in dystrophic mice. J Cell Biol 2001; 152: 1207-18.
181. Vachon PH, Xu H, Liu L, et al. Integrins (alpha7beta1) in muscle function and survival. Disrupted expression in merosin-deficient congenital muscular dystrophy. J Clin Invest 1997; 100: 1870-81.
182. Guo C, Willem M, Werner A, et al. Absence of alpha 7 integrin in dystrophin-deficient mice causes a myopathy similar to Duchenne muscular dystrophy. Hum Mol Genet 2006; 15: 989-98.
183. Liu J, Burkin DJ, Kaufman SJ. Increasing alpha 7 beta 1-integrin promotes muscle cell proliferation, adhesion, and resistance to apoptosis without changing gene expression. Am J Physiol Cell Physiol 2008; 294: C627-40.
184. Liu J, Milner DJ, Boppart MD, Ross RS, Kaufman SJ. beta1D chain increases alpha7beta1 integrin and laminin and protects against sarcolemmal damage in mdx mice. Hum Mol Genet 2011.
185. Doe JA, Wuebbles RD, Allred ET, Rooney JE, Elorza M, Burkin DJ. Transgenic overexpression of the alpha7 integrin reduces muscle pathology and improves viability in the dy(W) mouse model of merosin-deficient congenital muscular dystrophy type 1A. J Cell Sci 2011; 124: 2287-97.
186. Milner DJ, Kaufman SJ. Alpha7beta1 integrin does not alleviate disease in a mouse model of limb girdle muscular dystrophy type 2F. Am J Pathol 2007; 170: 609-19.
187. Gawlik KI, Oliveira BM, Durbeej M. Transgenic expression of Laminin alpha1 chain does not prevent muscle disease in the mdx mouse model for Duchenne muscular dystrophy. Am J Pathol 2011; 178: 1728-37.
188. Gurpur PB, Liu J, Burkin DJ, Kaufman SJ. Valproic acid activates the PI3K/Akt/mTOR pathway in muscle and ameliorates pathology in a mouse model of Duchenne muscular dystrophy. Am J Pathol 2009; 174: 999-1008.
189. Sztajnkrycer MD. Valproic acid toxicity: overview and management. J Toxicol Clin Toxicol 2002; 40: 789-801.
190. Ballas SK, Files B, Luchtman-Jones L, et al. Safety of purified poloxamer 188 in sickle cell disease: phase I study of a non-ionic surfactant in the management of acute chest syndrome. Hemoglobin 2004; 28: 85-102.
191. Yasuda S, Townsend D, Michele DE, Favre EG, Day SM, Metzger JM. Dystrophic heart failure blocked by membrane sealant poloxamer. Nature 2005; 436: 1025-9.
192. Spurney CF, Guerron AD, Yu Q, et al. Membrane sealant Poloxamer P188 protects against isoproterenol induced cardiomyopathy in dystrophin deficient mice. BMC Cardiovas Disord 2011; 11: 20.
193. Townsend D, Turner I, Yasuda S, et al. Chronic administration of membrane sealant prevents severe cardiac injury and ventricular dilatation in dystrophic dogs. J Clin Invest 2010; 120: 1140-50.
194. Ng R, Metzger JM, Claflin DR, Faulkner JA. Poloxamer 188 reduces the contraction-induced force decline in lumbrical muscles from mdx mice. Am J Physiol Cell Physiol 2008; 295: C146-50.
195. Quinlan JG, Wong BL, Niemeier RT, McCullough AS, Levin L, Emanuele M. Poloxamer 188 failed to prevent exercise-induced membrane breakdown in mdx skeletal muscle fibers. Neuromuscul Disord 2006; 16: 855-64.
196. Ozawa E. Our trails and trials in the subsarcolemmal cytoskeleton network and muscular dystrophy researches in the dystrophin era. Proc Jpn Acad Ser B Phys Biol Sci 2010; 86: 798-821.
197. Wehling M, Spencer MJ, Tidball JG. A nitric oxide synthase transgene ameliorates muscular dystrophy in mdx mice. J Cell Biol 2001; 155: 123-31.
198. Hodgetts S, Radley H, Davies M, Grounds MD. Reduced necrosis of dystrophic muscle by depletion of host neutrophils, or blocking TNFalpha function with Etanercept in mdx mice. Neuromuscul Disord 2006; 16: 591-602.
199. Spencer MJ, Montecino-Rodriguez E, Dorshkind K, Tidball JG. Helper (CD4(+)) and cytotoxic (CD8(+)) T cells promote the pathology of dystrophin-deficient muscle. Clin Immunol 2001; 98: 235-43.
200. Wehling-Henricks M, Lee JJ, Tidball JG. Prednisolone decreases cellular adhesion molecules required for inflammatory cell infiltration in dystrophin-deficient skeletal muscle. Neuromuscul Disord 2004; 14: 483-90.
201. Hayden MS, Ghosh S. Signaling to NF-kappaB. Genes Dev 2004; 18: 2195-224.
202. Porreca E, Guglielmi MD, Uncini A, et al. Haemostatic abnormalities, cardiac involvement and serum tumor necrosis factor levels in X-linked dystrophic patients. Thromb Haemost 1999; 81: 543-6.
203. Lundberg I, Brengman JM, Engel AG. Analysis of cytokine expression in muscle in inflammatory myopathies, Duchenne dystrophy, and non-weak controls. J Neuroimmunol 1995; 63: 9-16.
204. Watanabe N. Clinical significance of measurement of circulating tumor necrosis factor alpha]. Rinsho Byori 2001; 49: 829-33.
205. Kherif S, Lafuma C, Dehaupas M, et al. Expression of matrix metalloproteinases 2 and 9 in regenerating skeletal muscle: a study in experimentally injured and mdx muscles. Dev Biol 1999; 205: 158-70.
206. Bonizzi G, Karin M. The two NF-kappaB activation pathways and their role in innate and adaptive immunity. Trends Immunol 2004; 25: 280-8.
207. Peters RT, Maniatis T. A new family of IKK-related kinases may function as I kappa B kinase kinases. Biochim Biophys Acta 2001; 1471: M57-62.
208. Karin M, Ben-Neriah Y. Phosphorylation meets ubiquitination: the control of NF-[kappa]B activity. Ann Rev Immunol 2000; 18: 621-63.
209. Roman-Blas JA, Jimenez SA. NF-kappaB as a potential therapeutic target in osteoarthritis and rheumatoid arthritis. Osteoarthritis and cartilage/OARS, Osteoarthr Res Soc 2006; 14: 839-48.
210. Solt LA, May MJ. The IkappaB kinase complex: master regulator of NF-kappaB signaling. Immunologic Research 2008; 42: 3-18.
211. Kumar A, Takada Y, Boriek AM, Aggarwal BB. Nuclear factor-kappaB: its role in health and disease. J Mol Med (Berl) 2004; 82: 434-48.
212. Barnes PJ, Karin M. Nuclear factor-kappaB: a pivotal transcription factor in chronic inflammatory diseases. N Engl J Med 1997; 336: 1066-71.
213. Monici MC, Aguennouz M, Mazzeo A, Messina C, Vita G. Activation of nuclear factor-kappaB in inflammatory myopathies and Duchenne muscular dystrophy. Neurology 2003; 60: 993-7.
214. Kumar A, Boriek AM. Mechanical stress activates the nuclear factor-kappaB pathway in skeletal muscle fibers: a possible role in Duchenne muscular dystrophy. FASEB J 2003; 17: 386-96.
215. Messina S, Altavilla D, Aguennouz M, et al. Lipid peroxidation inhibition blunts nuclear factor-kappaB activation, reduces skeletal muscle degeneration, and enhances muscle function in mdx mice. Am J Pathol 2006; 168: 918-26.
216. Messina S, Bitto A, Aguennouz M, et al. Nuclear factor kappa-B blockade reduces skeletal muscle degeneration and enhances muscle function in mdx mice. Exp Neurol 2006; 198: 234-41.
217. Acharyya S, Villalta SA, Bakkar N, et al. Interplay of IKK/NF- kappaB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophy. J Clin Invest 2007; 117: 889-901.
218. Disatnik MH, Dhawan J, Yu Y, et al. Evidence of oxidative stress in mdx mouse muscle: studies of the pre-necrotic state. J Neurol Sci 1998; 161: 77-84.
219. De Bosscher K, Schmitz ML, Vanden Berghe W, Plaisance S, Fiers W, Haegeman G. Glucocorticoid-mediated repression of nuclear factor-kappaB-dependent transcription involves direct interference with transactivation. Proc Natl Acad Sci U S A 1997; 94: 13504-9.
220. De Bosscher K, Vanden Berghe W, Vermeulen L, Plaisance S, Boone E, Haegeman G. Glucocorticoids repress NF-kappaB-driven genes by disturbing the interaction of p65 with the basal transcription machinery, irrespective of coactivator levels in the cell. Proc Natl Acad Sci U S A 2000; 97: 3919-24.
221. Carlson CG, Samadi A, Siegel A. Chronic treatment with agents that stabilize cytosolic IkappaB-alpha enhances survival and improves resting membrane potential in mdx muscle fibers subjected to chronic passive stretch. Neurobiol Dis 2005; 20: 719-30.
222. Cuzzocrea S, Chatterjee PK, Mazzon E, et al. Pyrrolidine dithiocarbamate attenuates the development of acute and chronic inflammation. Br J Pharmacol 2002; 135: 496-510.
223. Miura T, Ouchida R, Yoshikawa N, et al. Functional modulation of the glucocorticoid receptor and suppression of NF-kappaB- dependent transcription by ursodeoxycholic acid. J Biol Chem 2001; 276: 47371-8.
224. De Bosscher K, Vanden Berghe W, Haegeman G. The interplay between the glucocorticoid receptor and nuclear factor-kappaB or activator protein-1: molecular mechanisms for gene repression. Endocr Rev 2003; 24: 488-522.
225. Siegel AL, Bledsoe C, Lavin J, et al. Treatment with inhibitors of the NF-kappaB pathway improves whole body tension development in the mdx mouse. Neuromuscul Disord 2009; 19: 131-9.
226. Miles MT, Cottey E, Cottey A, Stefanski C, Carlson CG. Reduced resting potentials in dystrophic (mdx) muscle fibers are secondary to NF-kappaB-dependent negative modulation of ouabain sensitive Na+-K+ pump activity. J Neurol Sci 2011; 303: 53-60.
227. Pan Y, Chen C, Shen Y, et al. Curcumin alleviates dystrophic muscle pathology in mdx mice. Mol Cells 2008; 25: 531-7.
228. Messina S, Bitto A, Aguennouz M, et al. Flavocoxid counteracts muscle necrosis and improves functional properties in mdx mice: a comparison study with methylprednisolone. Exp Neurol 2009; 220: 349-58.
229. Tang Y, Reay DP, Salay MN, et al. Inhibition of the IKK/NF- kappaB pathway by AAV gene transfer improves muscle regeneration in older mdx mice. Gene Ther 2010; 17: 1476-83.
230. Gregorevic P, Allen JM, Minami E, et al. rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 2006; 12: 787-9.
231. Yue Y, Liu M, Duan D. C-terminal-truncated microdystrophin recruits dystrobrevin and syntrophin to the dystrophin-associated glycoprotein complex and reduces muscular dystrophy in symptomatic utrophin/dystrophin double-knockout mice. Mol Ther 2006; 14: 79-87.
232. Wang B, Li J, Fu FH, Xiao X. Systemic human minidystrophin gene transfer improves functions and life span of dystrophin and dystrophin/utrophin-deficient mice. J Orthop Res 2009; 27: 421-6.
233. May MJ, D'Acquisto F, Madge LA, Glockner J, Pober JS, Ghosh S. Selective inhibition of NF-kappaB activation by a peptide that blocks the interaction of NEMO with the IkappaB kinase complex. Science 2000; 289: 1550-4.
234. Tilstra J, Rehman KK, Hennon T, Plevy SE, Clemens P, Robbins PD. Protein transduction: identification, characterization and optimization. Biochem Soc Trans 2007; 35: 811-5.
235. Delfin DA, Xu Y, Peterson JM, Guttridge DC, Rafael-Fortney JA, Janssen PM. Improvement of cardiac contractile function by peptide- based inhibition of NF-kappaB in the utrophin/dystrophin-deficient murine model of muscular dystrophy. J Transl Med 2011; 9: 68.
236. Li H, Mittal A, Makonchuk DY, Bhatnagar S, Kumar A. Matrix metalloproteinase-9 inhibition ameliorates pathogenesis and improves skeletal muscle regeneration in muscular dystrophy. Hum Mol Genet 2009; 18: 2584-98.
237. Fisher JF, Mobashery S. Recent advances in MMP inhibitor design. Cancer Metast Rev 2006; 25: 115-36.
238. Peterson JM, Kline W, Canan BD, et al. Peptide-based inhibition of NF-kappaB rescues diaphragm muscle contractile dysfunction in a murine model of Duchenne muscular dystrophy. Mol Med 2011; 17: 508-15.
239. Reay DP, Yang M, Watchko JF, et al. Systemic delivery of NEMO binding domain/IKKgamma inhibitory peptide to young mdx mice improves dystrophic skeletal muscle histopathology. Neurobiol Dis 2011; 43: 598-608.
240. Zahid M, Phillips BE, Albers SM, Giannoukakis N, Watkins SC, Robbins PD. Identification of a cardiac specific protein transduction domain by in vivo biopanning using a M13 phage peptide display library in mice. PLoS ONE 2010; 5: e12252.
241. Lawrence T, Fong C. The resolution of inflammation: antiinflammatory roles for NF-kappaB. Int J Biochem Cell Biol 2010; 42: 519-23.
242. Gordon JR, Burd PR, Galli SJ. Mast cells as a source of multifunctional cytokines. Immunol Today 1990; 11: 458-64.
243. Collins RA, Grounds MD. The role of tumor necrosis factor-alpha (TNF-alpha) in skeletal muscle regeneration. Studies in TNF- alpha(-/-) and TNF-alpha(-/-)/LT-alpha(-/-) mice. J Histochem Cytochem 2001; 49: 989-1001.
244. Tidball JG. Inflammatory processes in muscle injury and repair. Am J Physiol Regul Integr Comp Physiol 2005; 288: R345-53.
245. Pizza FX, Peterson JM, Baas JH, Koh TJ. Neutrophils contribute to muscle injury and impair its resolution after lengthening contractions in mice. J Physiol 2005; 562: 899-913.
246. Lapointe BM, Frenette J, Cote CH. Lengthening contractioninduced inflammation is linked to secondary damage but devoid of neutrophil invasion. J Appl Physiol 2002; 92: 1995-2004.
247. Pizza FX, McLoughlin TJ, McGregor SJ, Calomeni EP, Gunning WT. Neutrophils injure cultured skeletal myotubes. Am J Physiol Cell Physiol 2001; 281: C335-41.
248. Saito K, Kobayashi D, Komatsu M, et al. A sensitive assay of tumor necrosis factor alpha in sera from Duchenne muscular dystrophy patients. Clin Chem 2000; 46: 1703-4.
249. Huang P, Zhao XS, Fields M, Ransohoff RM, Zhou L. Imatinib attenuates skeletal muscle dystrophy in mdx mice. FASEB J 2009; 23: 2539-48.
250. Kageyama Y, Takahashi M, Torikai E, et al. Treatment with anti- TNF-alpha antibody infliximab reduces serum IL-15 levels in patients with rheumatoid arthritis. Clin Rheumatol 2007; 26: 505-9.
251. Grounds MD, Torrisi J. Anti-TNFalpha (Remicade) therapy protects dystrophic skeletal muscle from necrosis. FASEB J 2004; 18: 676-82.
252. Pierno S, Nico B, Burdi R, et al. Role of tumour necrosis factor alpha, but not of cyclo-oxygenase-2-derived eicosanoids, on functional and morphological indices of dystrophic progression in mdx mice: a pharmacological approach. Neuropathol Appl Neurobiol 2007; 33: 344-59.
253. Gosselin LE, Martinez DA. Impact of TNF-alpha blockade on TGF-beta1 and type I collagen mRNA expression in dystrophic muscle. Muscle Nerve 2004; 30: 244-6.
254. Scallon B, Cai A, Radewonuk J, Naso M. Addition of an extra immunoglobulin domain to two anti-rodent TNF monoclonal antibodies substantially increased their potency. Mol Immunol 2004; 41: 73-80.
255. Piers AT, Lavin T, Radley-Crabb HG, Bakker AJ, Grounds MD, Pinniger GJ. Blockade of TNF in vivo using cV1q antibody reduces contractile dysfunction of skeletal muscle in response to eccentric exercise in dystrophic mdx and normal mice. Neuromuscul Disord 2011; 21: 132-41.
256. Radley HG, Davies MJ, Grounds MD. Reduced muscle necrosis and long-term benefits in dystrophic mdx mice after cV1q (blockade of TNF) treatment. Neuromuscul Disord 2008; 18: 227-38.
257. Chen YW, Zhao P, Borup R, Hoffman EP. Expression profiling in the muscular dystrophies: identification of novel aspects of molecular pathophysiology. J Cell Biol 2000; 151: 1321-36.
258. Haslett JN, Sanoudou D, Kho AT, et al. Gene expression comparison of biopsies from Duchenne muscular dystrophy (DMD) and normal skeletal muscle. Proc Natl Acad Sci U S A 2002; 99: 15000-5.
259. Pescatori M, Broccolini A, Minetti C, et al. Gene expression profiling in the early phases of DMD: a constant molecular signature characterizes DMD muscle from early postnatal life throughout disease progression. FASEB J 2007; 21: 1210-26.
260. Sterrenburg E, van der Wees CG, White SJ, et al. Gene expression profiling highlights defective myogenesis in DMD patients and a possible role for bone morphogenetic protein 4. Neurobiol Dis 2006; 23: 228-36.
261. Radley HG, Grounds MD. Cromolyn administration (to block mast cell degranulation) reduces necrosis of dystrophic muscle in mdx mice. Neurobiol Dis 2006; 23: 387-97.
262. Marques MJ, Ventura Machado R, Minatel E, Santo Neto H. Disodium cromoglycate protects dystrophin-deficient muscle fibers from leakiness. Muscle Nerve 2008; 37: 61-7.
263. Spurney CF, Cha HJ, Sali A, et al. Evaluation of skeletal and cardiac muscle function after chronic administration of thymosin beta-4 in the dystrophin deficient mouse. PLoS ONE 2010; 5: e8976.
264. Urade Y, Ujihara M, Horiguchi Y, et al. Mast cells contain spleen-type prostaglandin D synthetase. J Biol Chem 1990; 265: 371-5.
265. Okinaga T, Mohri I, Fujimura H, et al. Induction of hematopoietic prostaglandin D synthase in hyalinated necrotic muscle fibers: its implication in grouped necrosis. Acta Neuropathol 2002; 104: 377-84.
266. Newton R, Kuitert LM, Slater DM, Adcock IM, Barnes PJ. Cytokine induction of cytosolic phospholipase A2 and cyclooxygenase-2 mRNA is suppressed by glucocorticoids in human epithelial cells. Life Sci 1997; 60: 67-78.
267. Mohri I, Aritake K, Taniguchi H, et al. Inhibition of prostaglandin D synthase suppresses muscular necrosis. Am J Pathol 2009; 174: 1735-44.
268. Kamauchi S, Urade Y. Hematopoietic prostaglandin d synthase inhibitors for the treatment of duchenne muscular dystrophy]. Brain Nerve 2011; 63: 1261-9.
269. Leask A, Abraham DJ. TGF-beta signaling and the fibrotic response. FASEB J 2004; 18: 816-27.
270. Hartel JV, Granchelli JA, Hudecki MS, Pollina CM, Gosselin LE. Impact of prednisone on TGF-beta1 and collagen in diaphragm muscle from mdx mice. Muscle Nerve 2001; 24: 428-32.
271. Andreetta F, Bernasconi P, Baggi F, et al. Immunomodulation of TGF-beta 1 in mdx mouse inhibits connective tissue proliferation in diaphragm but increases inflammatory response: implications for antifibrotic therapy. J Neuroimmunol 2006; 175: 77-86.
272. Zhou L, Porter JD, Cheng G, et al. Temporal and spatial mRNA expression patterns of TGF-beta1, 2, 3 and TbetaRI, II, III in skeletal muscles of mdx mice. Neuromuscul Disord 2006; 16: 32-8.
273. Taniguti AP, Pertille A, Matsumura CY, Santo Neto H, Marques MJ. Prevention of muscle fibrosis and myonecrosis in mdx mice by suramin, a TGF-beta1 blocker. Muscle Nerve 2011; 43: 82-7.
274. Bernasconi P, Torchiana E, Confalonieri P, et al. Expression of transforming growth factor-beta 1 in dystrophic patient muscles correlates with fibrosis. Pathogenetic role of a fibrogenic cytokine. J Clin Invest 1995; 96: 1137-44.
275. Santibanez JF, Quintanilla M, Bernabeu C. TGF-beta/TGF-beta receptor system and its role in physiological and pathological conditions. Clin Sci (Lond) 2011; 121: 233-51.
276. Border WA, Noble NA. Transforming growth factor beta in tissue fibrosis. N Engl J Med 1994; 331: 1286-92.
277. Joyce ME, Roberts AB, Sporn MB, Bolander ME. Transforming growth factor-beta and the initiation of chondrogenesis and osteogenesis in the rat femur. J Cell Biol 1990; 110: 2195-207.
278. Wahl SM, Hunt DA, Wakefield LM, et al. Transforming growth factor type beta induces monocyte chemotaxis and growth factor production. Proc Natl Acad Sci U S A 1987; 84: 5788-92.
279. Postlethwaite AE, Keski-Oja J, Moses HL, Kang AH. Stimulation of the chemotactic migration of human fibroblasts by transforming growth factor beta. J Exp Med 1987; 165: 251-6.
280. Carlson ME, Hsu M, Conboy IM. Imbalance between pSmad3 and Notch induces CDK inhibitors in old muscle stem cells. Nature 2008; 454: 528-32.
281. Zhu S, Goldschmidt-Clermont PJ, Dong C. Transforming growth factor-beta-induced inhibition of myogenesis is mediated through Smad pathway and is modulated by microtubule dynamic stability. Circ Res 2004; 94: 617-25.
282. Allen RE, Boxhorn LK. Regulation of skeletal muscle satellite cell proliferation and differentiation by transforming growth factor beta, insulin-like growth factor I, and fibroblast growth factor. J Cell Physiol 1989; 138: 311-5.
283. Cohn RD, van Erp C, Habashi JP, et al. Angiotensin II type 1 receptor blockade attenuates TGF-beta-induced failure of muscle regeneration in multiple myopathic states. Nat Med 2007; 13: 204-10.
284. Jiang BH, Aoki M, Zheng JZ, Li J, Vogt PK. Myogenic signaling of phosphatidylinositol 3-kinase requires the serine-threonine kinase Akt/protein kinase B. Proc Natl Acad Sci U S A 1999; 96: 2077-81.
285. Rommel C, Bodine SC, Clarke BA, et al. Mediation of IGF-1- induced skeletal myotube hypertrophy by PI(3)K/Akt/mTOR and PI(3)K/Akt/GSK3 pathways. Nat Cell Biol 2001; 3: 1009-13.
286. Halevy O, Cantley LC. Differential regulation of the phosphoinositide 3-kinase and MAP kinase pathways by hepatocyte growth factor vs. insulin-like growth factor-I in myogenic cells. Exp Cell Res 2004; 297: 224-34.
287. Jones NC, Fedorov YV, Rosenthal RS, Olwin BB. ERK1/2 is required for myoblast proliferation but is dispensable for muscle gene expression and cell fusion. J Cell Physiol 2001; 186: 104-15.
288. Li J, Johnson SE. ERK2 is required for efficient terminal differentiation of skeletal myoblasts. Biochem Biophys Res Commun 2006; 345: 1425-33.
289. Gredinger E, Gerber AN, Tamir Y, Tapscott SJ, Bengal E. Mitogen-activated protein kinase pathway is involved in the differentiation of muscle cells. J Biol Chem 1998; 273: 10436-44.
290. Itoh S, Ten Dijke P. Negative regulation of TGF-beta receptor/Smad signal transduction. Current opinion in cell biology 2007; 19: 176-84.
291. Yamamoto T, Noble NA, Miller DE, Border WA. Sustained expression of TGF-beta 1 underlies development of progressive kidney fibrosis. Kidney Int 1994; 45: 916-27.
292. Fakhfakh R, Lamarre Y, Skuk D, Tremblay JP. Losartan enhances the success of myoblast transplantation. Cell Transplant 2011.
293. Cheson BD, Levine AM, Mildvan D, et al. Suramin therapy in AIDS and related disorders. Report of the US Suramin Working Group. JAMA 1987; 258: 1347-51.
294. Reed E, Cooper MR, LaRocca RV, Bostick-Bruton F, Myers CE. Suramin in advanced platinum-resistant ovarian cancer. Eur J Cancer 1992; 28A: 864-6.
295. Mietz H, Chevez-Barrios P, Feldman RM, Lieberman MW. Suramin inhibits wound healing following filtering procedures for glaucoma. The British Journal of Ophthalmology 1998; 82: 816-20.
296. Sahu D, Saroha A, Roy S, Das S, Srivastava PS, Das HR. Suramin ameliorates collagen induced arthritis. Inter Immunopharm 2011.
297. Coffey RJ, Jr., Leof EB, Shipley GD, Moses HL. Suramin inhibition of growth factor receptor binding and mitogenicity in AKR-2B cells. J Cell Physiol 1987; 132: 143-8.
298. Liu N, Zhuang S. Tissue protective and anti-fibrotic actions of suramin: new uses of an old drug. Curr Clin Pharmacol 2011; 6: 137-42.
299. McGeary RP, Bennett AJ, Tran QB, Cosgrove KL, Ross BP. Suramin: clinical uses and structure-activity relationships. Mini Rev Med Chem 2008; 8: 1384-94.
300. Chan YS, Li Y, Foster W, et al. Antifibrotic effects of suramin in injured skeletal muscle after laceration. J Appl Physiol 2003; 95: 771-80.
301. Pines M, Nagler A. Halofuginone: a novel antifibrotic therapy. Gen Pharmacol 1998; 30: 445-50.
302. Pines M, Snyder D, Yarkoni S, Nagler A. Halofuginone to treat fibrosis in chronic graft-versus-host disease and scleroderma. Biol Blood Marrow Transpl 2003; 9: 417-25.
303. Roffe S, Hagai Y, Pines M, Halevy O. Halofuginone inhibits Smad3 phosphorylation via the PI3K/Akt and MAPK/ERK pathways in muscle cells: effect on myotube fusion. Exp Cell Res 2010; 316: 1061-9.
304. Turgeman T, Hagai Y, Huebner K, et al. Prevention of muscle fibrosis and improvement in muscle performance in the mdx mouse by halofuginone. Neuromuscul Disord 2008; 18: 857-68.
305. Huebner KD, Jassal DS, Halevy O, Pines M, Anderson JE. Functional resolution of fibrosis in mdx mouse dystrophic heart and skeletal muscle by halofuginone. Am J Physiol Heart Circ Physiol 2008; 294: H1550-61.
306. de Jonge MJ, Dumez H, Verweij J, et al. Phase I and pharmacokinetic study of halofuginone, an oral quinazolinone derivative in patients with advanced solid tumours. Eur J Cancer 2006; 42: 1768-74.
307. Wang S, Wilkes MC, Leof EB, Hirschberg R. Imatinib mesylate blocks a non-Smad TGF-beta pathway and reduces renal fibrogenesis in vivo. FASEB J 2005; 19: 1-11.
308. Vuorinen K, Gao F, Oury TD, Kinnula VL, Myllarniemi M. Imatinib mesylate inhibits fibrogenesis in asbestos-induced interstitial pneumonia. Exp Lung Res 2007; 33: 357-73.
309. Leipner C, Grun K, Muller A, et al. Imatinib mesylate attenuates fibrosis in coxsackievirus b3-induced chronic myocarditis. Cardiovasc Res 2008; 79: 118-26.
310. Yoshiji H, Noguchi R, Kuriyama S, et al. Imatinib mesylate (STI- 571) attenuates liver fibrosis development in rats. American journal of physiology Gastrointest Liver Physiol 2005; 288: G907-13.
311. Daniels CE, Wilkes MC, Edens M, et al. Imatinib mesylate inhibits the profibrogenic activity of TGF-beta and prevents bleomycin-mediated lung fibrosis. J Clin Invest 2004; 114: 1308-16.
312. Druker BJ, Guilhot F, O'Brien SG, et al. Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia. N Engl J Med 2006; 355: 2408-17.
313. Guilhot F. Indications for imatinib mesylate therapy and clinical management. Oncologist 2004; 9: 271-81.
314. Bizario JC, Cerri DG, Rodrigues LC, et al. Imatinib mesylate ameliorates the dystrophic phenotype in exercised mdx mice. J Neuroimmunol 2009; 212: 93-101.
315. Lee KY, Ho SC, Lin HC, et al. Neutrophil-derived elastase induces TGF-beta1 secretion in human airway smooth muscle via NFkappaB pathway. Am J Respir Cell Mol Biol 2006; 35: 407-14.
316. Maffia PC, Zittermann SE, Scimone ML, et al. Neutrophil elastase converts human immature dendritic cells into transforming growth factor-beta1-secreting cells and reduces allostimulatory ability. Am J Pathol 2007; 171: 928-37.
317. Zhao XY, Zhao LY, Zheng QS, et al. Chymase induces profibrotic response via transforming growth factor-beta 1/Smad activation in rat cardiac fibroblasts. Mol Cell Biochem 2008; 310: 159-66.
318. Arbogast S, Smith J, Matuszczak Y, et al. Bowman-Birk inhibitor concentrate prevents atrophy, weakness, and oxidative stress in soleus muscle of hindlimb-unloaded mice. J Appl Physiol 2007; 102: 956-64.
319. Morris CA, Morris LD, Kennedy AR, Sweeney HL. Attenuation of skeletal muscle atrophy via protease inhibition. J Appl Physiol 2005; 99: 1719-27.
320. Morris CA, Selsby JT, Morris LD, Pendrak K, Sweeney HL. Bowman-Birk inhibitor attenuates dystrophic pathology in mdx mice. J Appl Physiol 2010; 109: 1492-9.
321. Billings PC, St Clair WH, Maki PA, Kennedy AR. Distribution of the Bowman Birk protease inhibitor in mice following oral administration. Cancer Lett 1992; 62: 191-7.
322. Oreffo VI, Billings PC, Kennedy AR, Witschi H. Acute effects of the Bowman-Birk protease inhibitor in mice. Toxicology 1991; 69: 165-76.
323. Lichtenstein GR, Deren JJ, Katz S, Lewis JD, Kennedy AR, Ware JH. Bowman-Birk inhibitor concentrate: a novel therapeutic agent for patients with active ulcerative colitis. Dig Dis Sci 2008; 53: 175-80.
324. Schaefer CJ, Ruhrmund DW, Pan L, Seiwert SD, Kossen K. Antifibrotic activities of pirfenidone in animal models. Eur Respirat Rev 2011; 20: 85-97.
325. Azuma A, Taguchi Y, Ogura T, et al. Exploratory analysis of a phase III trial of pirfenidone identifies a subpopulation of patients with idiopathic pulmonary fibrosis as benefiting from treatment. Respirat Res 2011; 12: 143.
326. Shimizu T, Fukagawa M, Kuroda T, et al. Pirfenidone prevents collagen accumulation in the remnant kidney in rats with partial nephrectomy. Kidney Inter Suppl 1997; 63: S239-43.
327. Nakazato H, Oku H, Yamane S, Tsuruta Y, Suzuki R. A novel antifibrotic agent pirfenidone suppresses tumor necrosis factor-alpha at the translational level. Eur J Pharmacol 2002; 446: 177-85.
328. Gosselin LE, Williams JE, Personius K, Farkas GA. A comparison of factors associated with collagen metabolism in different skeletal muscles from dystrophic (mdx) mice: impact of pirfenidone. Muscle Nerve 2007; 35: 208-16.
329. Oku H, Nakazato H, Horikawa T, Tsuruta Y, Suzuki R. Pirfenidone suppresses tumor necrosis factor-alpha, enhances interleukin-10 and protects mice from endotoxic shock. Eur J Pharmacol 2002; 446: 167-76.
330. Miric G, Dallemagne C, Endre Z, Margolin S, Taylor SM, Brown L. Reversal of cardiac and renal fibrosis by pirfenidone and spironolactone in streptozotocin-diabetic rats. Br J Pharmacol 2001; 133: 687-94.
331. Khairallah M, Khairallah R, Young ME, Dyck JR, Petrof BJ, Des Rosiers C. Metabolic and signaling alterations in dystrophindeficient hearts precede overt cardiomyopathy. J Mol Cell Cardiol 2007; 43: 119-29.
332. Miyazono K, Kamiya Y, Morikawa M. Bone morphogenetic protein receptors and signal transduction. J Biochem 2010; 147: 35-51.
333. Wang H, Noulet F, Edom-Vovard F, Tozer S, Le Grand F, Duprez D. Bmp signaling at the tips of skeletal muscles regulates the number of fetal muscle progenitors and satellite cells during development. Dev Cell 2010; 18: 643-54.
334. Clever JL, Sakai Y, Wang RA, Schneider DB. Inefficient skeletal muscle repair in inhibitor of differentiation knockout mice suggests a crucial role for BMP signaling during adult muscle regeneration. Am J Physiol Cell Physiol 2010; 298: C1087-99.
335. Frank NY, Kho AT, Schatton T, et al. Regulation of myogenic progenitor proliferation in human fetal skeletal muscle by BMP4 and its antagonist Gremlin. J Cell Biol 2006; 175: 99-110.
336. Fukada S, Uezumi A, Ikemoto M, et al. Molecular signature of quiescent satellite cells in adult skeletal muscle. Stem Cells 2007; 25: 2448-59.
337. Ono Y, Calhabeu F, Morgan JE, Katagiri T, Amthor H, Zammit PS. BMP signalling permits population expansion by preventing premature myogenic differentiation in muscle satellite cells. Cell Death Differ 2011; 18: 222-34.
338. Linker C, Lesbros C, Stark MR, Marcelle C. Intrinsic signals regulate the initial steps of myogenesis in vertebrates. Development 2003; 130: 4797-807.
339. Reshef R, Maroto M, Lassar AB. Regulation of dorsal somitic cell fates: BMPs and Noggin control the timing and pattern of myogenic regulator expression. Genes Dev 1998; 12: 290-303.
340. Tylzanowski P, Mebis L, Luyten FP. The Noggin null mouse phenotype is strain dependent and haploinsufficiency leads to skeletal defects. Dev Dyn 2006; 235: 1599-607.
341. Friedrichs M, Wirsdoerfer F, Flohe SB, Schneider S, Wuelling M, Vortkamp A. BMP signaling balances proliferation and differentiation of muscle satellite cell descendants. BMC Cell Biol 2011; 12: 26.
342. Turk R, Sterrenburg E, de Meijer EJ, van Ommen GJ, den Dunnen JT, t Hoen PA. Muscle regeneration in dystrophin-deficient mdx mice studied by gene expression profiling. BMC Genomics 2005; 6: 98.
343. Shi S, Hoogaars WM, de Gorter DJ, et al. BMP antagonists enhance myogenic differentiation and ameliorate the dystrophic phenotype in a DMD mouse model. Neurobiol Dis 2011; 41: 353-60.
344. Cuny GD, Yu PB, Laha JK, et al. Structure-activity relationship study of bone morphogenetic protein (BMP) signaling inhibitors. Bioorg Med Chem Lett 2008; 18: 4388-92.
345. Yu PB, Deng DY, Lai CS, et al. BMP type I receptor inhibition reduces heterotopic [corrected] ossification. Nat Med 2008; 14: 1363-9.
346. Yu PB, Hong CC, Sachidanandan C, et al. Dorsomorphin inhibits BMP signals required for embryogenesis and iron metabolism. Nat Chem Biol 2008; 4: 33-41.
347. Niesler CU, Myburgh KH, Moore F. The changing AMPK expression profile in differentiating mouse skeletal muscle myoblast cells helps confer increasing resistance to apoptosis. Exp Physiol 2007; 92: 207-17.
348. Hao J, Ho JN, Lewis JA, et al. In vivo structure-activity relationship study of dorsomorphin analogues identifies selective VEGF and BMP inhibitors. ACS Chem Biol 2010; 5: 245-53.
349. Fayssoil A, Nardi O, Orlikowski D, Annane D. Cardiomyopathy in Duchenne muscular dystrophy: pathogenesis and therapeutics. Heart Failure Rev 2010; 15: 103-7.
350. Toussaint M, Steens M, Wasteels G, Soudon P. Diurnal ventilation via mouthpiece: survival in end-stage Duchenne patients. Eur Respir J 2006; 28: 549-55.
351. Spurney CF. Cardiomyopathy of Duchenne muscular dystrophy: current understanding and future directions. Muscle Nerve 2011; 44: 8-19.
352. Finsterer J, Stollberger C. Cardiac involvement in Becker muscular dystrophy. Can J Cardiol 2008; 24: 786-92.
353. Alter J, Lou F, Rabinowitz A, et al. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 2006; 12: 175-7.
354. Lu QL, Rabinowitz A, Chen YC, et al. Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci U S A 2005; 102: 198-203.
355. Grounds MD, Radley HG, Lynch GS, Nagaraju K, De Luca A. Towards developing standard operating procedures for pre-clinical testing in the mdx mouse model of Duchenne muscular dystrophy. Neurobiol Dis 2008; 31: 1-19.
356. De Luca A, Nico B, Liantonio A, et al. A multidisciplinary evaluation of the effectiveness of cyclosporine a in dystrophic mdx mice. Am J Pathol 2005; 166: 477-89.
357. Cox GF, Kunkel LM. Dystrophies and heart disease. Current Opinion in Cardiology 1997; 12: 329-43.
358. Williams IA, Allen DG. Intracellular calcium handling in ventricular myocytes from mdx mice. Am J Physiol Heart Circ Physiol 2007; 292: H846-55.
359. Constantin B, Sebille S, Cognard C. New insights in the regulation of calcium transfers by muscle dystrophin-based cytoskeleton: implications in DMD. J Muscle Res Cell Motil 2006; 27: 375-86.
360. Feng J, Schaus BJ, Fallavollita JA, Lee TC, Canty JM, Jr. Preload induces troponin I degradation independently of myocardial ischemia. Circulation 2001; 103: 2035-7.
361. Gao WD, Atar D, Liu Y, Perez NG, Murphy AM, Marban E. Role of troponin I proteolysis in the pathogenesis of stunned myocardium. Circ Res 1997; 80: 393-9.
362. Ameen V, Robson LG. Experimental models of duchenne muscular dystrophy: relationship with cardiovascular disease. Open Cardiovas Med J 2010; 4: 265-77.
363. Khan R, Sheppard R. Fibrosis in heart disease: understanding the role of transforming growth factor-beta in cardiomyopathy, valvular disease and arrhythmia. Immunology 2006; 118: 10-24.
364. Fischer JW, Stoll M, Hahn AW, Unger T. Differential regulation of thrombospondin-1 and fibronectin by angiotensin II receptor subtypes in cultured endothelial cells. Cardiovasc Res 2001; 51: 784-91.
365. Chua CC, Hamdy RC, Chua BH. Regulation of thrombospondin-1 production by angiotensin II in rat heart endothelial cells. Biochim Biophys Acta 1997; 1357: 209-14.
366. Scott-Burden T, Resink TJ, Hahn AW, Buhler FR. Induction of thrombospondin expression in vascular smooth muscle cells by angiotensin II. J Cardiovas Pharmacol 1990; 16 Suppl 7: S17-20.
367. Hunt SA, Abraham WT, Chin MH, et al. ACC/AHA 2005 Guideline Update for the Diagnosis and Management of Chronic Heart Failure in the Adult: a report of the American College of Cardiology/American Heart Association Task Force on Practice Guidelines (Writing Committee to Update the 2001 Guidelines for the Evaluation and Management of Heart Failure): developed in collaboration with the American College of Chest Physicians and the International Society for Heart and Lung Transplantation: endorsed by the Heart Rhythm Society. Circulation 2005; 112: e154-235.
368. Kaspar RW, Allen HD, Montanaro F. Current understanding and management of dilated cardiomyopathy in Duchenne and Becker muscular dystrophy. J Am Acad Nurse Practit 2009; 21: 241-9.
369. Ramaciotti C, Heistein LC, Coursey M, et al. Left ventricular function and response to enalapril in patients with duchenne muscular dystrophy during the second decade of life. Am J Cardiol 2006; 98: 825-7.
370. Jefferies JL, Eidem BW, Belmont JW, et al. Genetic predictors and remodeling of dilated cardiomyopathy in muscular dystrophy. Circulation 2005; 112: 2799-804.
371. Ogata H, Ishikawa Y, Minami R. Beneficial effects of beta-blockers and angiotensin-converting enzyme inhibitors in Duchenne muscular dystrophy. J Cardiol 2009; 53: 72-8.
372. Kajimoto H, Ishigaki K, Okumura K, et al. Beta-blocker therapy for cardiac dysfunction in patients with muscular dystrophy. Cir J 2006; 70: 991-4.
373. Bauer R, Straub V, Blain A, Bushby K, MacGowan GA. Contrasting effects of steroids and angiotensin-converting-enzyme inhibitors in a mouse model of dystrophin-deficient cardiomyopathy. Eur J Heart Fail 2009; 11: 463-71.
374. English KM, Gibbs JL. Cardiac monitoring and treatment for children and adolescents with neuromuscular disorders. Dev Med Child Neurol 2006; 48: 231-5.
375. Bushby K, Muntoni F, Bourke JP. 107th ENMC international workshop: the management of cardiac involvement in muscular dystrophy and myotonic dystrophy. 7th-9th June 2002, Naarden, the Netherlands. Neuromuscul Disord 2003; 13: 166-72.
376. Hor KN, Wansapura J, Markham LW, et al. Circumferential strain analysis identifies strata of cardiomyopathy in Duchenne muscular dystrophy: a cardiac magnetic resonance tagging study. J Am Coll Cardiol 2009; 53: 1204-10.
377. van Bockel EA, Lind JS, Zijlstra JG, et al. Cardiac assessment of patients with late stage Duchenne muscular dystrophy. Netherlands heart journal: monthly journal of the Netherlands Society of Cardiology and the Netherlands Heart Foundation 2009; 17: 232-7.
378. Finsterer J, Stollberger C. Heart Hum Dystrophinopathies. Cardiol 2003; 99: 1-19.
379. Duboc D, Meune C, Pierre B, et al. Perindopril preventive treatment on mortality in Duchenne muscular dystrophy: 10 years' follow-up. Am Heart J 2007; 154: 596-602.
380. Duboc D, Meune C, Lerebours G, Devaux JY, Vaksmann G, Becane HM. Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy. J Am Coll Cardiol 2005; 45: 855-7.
381. Spurney CF, Sali A, Guerron AD, et al. Losartan decreases cardiac muscle fibrosis and improves cardiac function in dystrophindeficient mdx mice. Journal of Cardiovascular Pharmacology and Therapeutics 2011; 16: 87-95.
382. Bish LT, Yarchoan M, Sleeper MM, et al. Chronic losartan administration reduces mortality and preserves cardiac but not skeletal muscle function in dystrophic mice. PLoS ONE 2011; 6: e20856.
383. Nelson CA, Hunter RB, Quigley LA, et al. Inhibiting TGF-beta activity improves respiratory function in mdx mice. Am J Pathol 2011; 178: 2611-21.
384. Niebroj-Dobosz I, Fidzianska A, Hausmanowa-Petrusewicz I. Controversies about the function of dystrophin in muscle. Folia Neuropathol 2001; 39: 253-8.
385. Johnston AP, Baker J, De Lisio M, Parise G. Skeletal muscle myoblasts possess a stretch-responsive local angiotensin signalling system. Journal of the renin-angiotensin-aldosterone system: JRAAS 2011; 12: 75-84.
386. Sun G, Haginoya K, Dai H, et al. Intramuscular renin-angiotensin system is activated in human muscular dystrophy. J Neurol Sci 2009; 280: 40-8.
387. Johnston AP, Baker J, Bellamy LM, McKay BR, De Lisio M, Parise G. Regulation of muscle satellite cell activation and chemotaxis by angiotensin II. PLoS ONE 2010; 5: e15212.
388. Cozzoli A, Nico B, Sblendorio VT, et al. Enalapril treatment discloses an early role of angiotensin II in inflammation- and oxidative stress-related muscle damage in dystrophic mdx mice. Pharmacol Res 2011; 64: 482-92.
389. Wei Y, Sowers JR, Clark SE, Li W, Ferrario CM, Stump CS. Angiotensin II-induced skeletal muscle insulin resistance mediated by NF-kappaB activation via NADPH oxidase. Am J Physiol Endocrinol Metab 2008; 294: E345-51.
390. Rando TA, Disatnik MH, Yu Y, Franco A. Muscle cells from mdx mice have an increased susceptibility to oxidative stress. Neuromuscul Disord 1998; 8: 14-21.
391. Rafael-Fortney JA, Chimanji NS, Schill KE, et al. Early treatment with lisinopril and spironolactone preserves cardiac and skeletal muscle in Duchenne muscular dystrophy mice. Circulation 2011; 124: 582-8.
392. Spencer MJ, Tidball JG. Do immune cells promote the pathology of dystrophin-deficient myopathies? Neuromuscul Disord 2001; 11: 556-64.
393. Tidball JG, Wehling-Henricks M. The role of free radicals in the pathophysiology of muscular dystrophy. J Appl Physiol 2007; 102: 1677-86.
394. Gissel H. The role of Ca2+ in muscle cell damage. Ann N Y Acad Sci 2005; 1066: 166-80.
395. Halliwell B, Gutteridge JMC. Free radicals in biology and medicine. 4th ed. Oxford: Oxford University Press; 2007.
396. Ragusa RJ, Chow CK, Porter JD. Oxidative stress as a potential pathogenic mechanism in an animal model of Duchenne muscular dystrophy. Neuromuscul Disord 1997; 7: 379-86.
397. Haycock JW, Mac Neil S, Mantle D. Differential protein oxidation in Duchenne and Becker muscular dystrophy. Neuroreport 1998; 9: 2201-7.
398. El-Shafey AF, Armstrong AE, Terrill JR, Grounds MD, Arthur PG. Screening for increased protein thiol oxidation in oxidatively stressed muscle tissue. Free Radical Research 2011; 45: 991-9.
399. Suzuki YJ, Ford GD. Redox regulation of signal transduction in cardiac and smooth muscle. J Mol Cell Cardiol 1999; 31: 345-53.
400. Arthur PG, Grounds MD, Shavlakadze T. Oxidative stress as a therapeutic target during muscle wasting: considering the complex interactions. Curr Opin Clin Nutr Metab Care 2008; 11: 408-16.
401. Yeung EW, Whitehead NP, Suchyna TM, Gottlieb PA, Sachs F, Allen DG. Effects of stretch-activated channel blockers on [Ca2+]i and muscle damage in the mdx mouse. J Physiol 2005; 562: 367-80.
402. Robert V, Massimino ML, Tosello V, et al. Alteration in calcium handling at the subcellular level in mdx myotubes. J Biol Chem 2001; 276: 4647-51.
403. Nethery D, Callahan LA, Stofan D, Mattera R, DiMarco A, Supinski G. PLA(2) dependence of diaphragm mitochondrial formation of reactive oxygen species. J Appl Physiol 2000; 89: 72-80.
404. Martins AS, Shkryl VM, Nowycky MC, Shirokova N. Reactive oxygen species contribute to Ca2+ signals produced by osmotic stress in mouse skeletal muscle fibres. J Physiol 2008; 586: 197-210.
405. Howl JD, Publicover SJ. Permeabilisation of the sarcolemma in mouse diaphragm exposed to Bay K 8644 in vitro: time course, dependence on Ca2+ and effects of enzyme inhibitors. Acta Neuropathol 1990; 79: 438-43.
406. Canton M, Skyschally A, Menabo R, et al. Oxidative modification of tropomyosin and myocardial dysfunction following coronary microembolization. Eur Heart J 2006; 27: 875-81.
407. Canton M, Neverova I, Menabo R, Van Eyk J, Di Lisa F. Evidence of myofibrillar protein oxidation induced by postischemic reperfusion in isolated rat hearts. Am J Physiol Heart Circ Physiol 2004; 286: H870-7.
408. Ilsley JL, Sudol M, Winder SJ. The WW domain: linking cell signalling to the membrane cytoskeleton. Cell Signal 2002; 14: 183-9.
409. Sotgia F, Bonuccelli G, Bedford M, et al. Localization of phospho-beta-dystroglycan (pY892) to an intracellular vesicular compartment in cultured cells and skeletal muscle fibers in vivo. Biochemistry 2003; 42: 7110-23.
410. Hnia K, Hugon G, Rivier F, Masmoudi A, Mercier J, Mornet D. Modulation of p38 mitogen-activated protein kinase cascade and metalloproteinase activity in diaphragm muscle in response to free radical scavenger administration in dystrophin-deficient mdx mice. Am J Pathol 2007; 170: 633-43.
411. Powers SK, Kavazis AN, McClung JM. Oxidative stress and disuse muscle atrophy. J Appl Physiol 2007; 102: 2389-97.
412. Kadowaki M, Kanazawa T. Amino acids as regulators of proteolysis. J Nutr 2003; 133: 2052S-6S.
413. Takahashi M, Nishihira J, Shimpo M, et al. Macrophage migration inhibitory factor as a redox-sensitive cytokine in cardiac myocytes. Cardiovasc Res 2001; 52: 438-45.
414. Nguyen HX, Tidball JG. Interactions between neutrophils and macrophages promote macrophage killing of rat muscle cells in vitro. J Physiol 2003; 547: 125-32.
415. Khairallah M, Khairallah RJ, Young ME, et al. Sildenafil and cardiomyocyte-specific cGMP signaling prevent cardiomyopathic changes associated with dystrophin deficiency. Proc Natl Acad Sci U S A 2008; 105: 7028-33.
416. Renjini R, Gayathri N, Nalini A, Srinivas Bharath MM. Oxidative Damage in Muscular Dystrophy Correlates with the Severity of the Pathology: Role of Glutathione Metabolism. Neurochemical Research 2012; 37: 885-98.
417. Fenichel GM, Brooke MH, Griggs RC, et al. Clinical investigation in Duchenne muscular dystrophy: penicillamine and vitamin E. Muscle Nerve 1988; 11: 1164-8.
418. Gamstorp I, Gustavson KH, Hellstrom O, Nordgren B. A trial of selenium and vitamin E in boys with muscular dystrophy. J Child Neurol 1986; 1: 211-4.
419. Backman E, Nylander E, Johansson I, Henriksson KG, Tagesson C. Selenium and vitamin E treatment of Duchenne muscular dystrophy: no effect on muscle function. Acta neurologica Scandinavica 1988; 78: 429-35.
420. Buetler TM, Renard M, Offord EA, Schneider H, Ruegg UT. Green tea extract decreases muscle necrosis in mdx mice and protects against reactive oxygen species. Am J Clin Nutr 2002; 75: 749-53.
421. Dorchies OM, Wagner S, Vuadens O, et al. Green tea extract and its major polyphenol (-)-epigallocatechin gallate improve muscle function in a mouse model for Duchenne muscular dystrophy. Am J Physiol Cell Physiol 2006; 290: C616-25.
422. Nakae Y, Hirasaka K, Goto J, et al. Subcutaneous injection, from birth, of epigallocatechin-3-gallate, a component of green tea, limits the onset of muscular dystrophy in mdx mice: a quantitative histological, immunohistochemical and electrophysiological study. Histochem Cell Biol 2008; 129: 489-501.
423. Call JA, Voelker KA, Wolff AV, et al. Endurance capacity in maturing mdx mice is markedly enhanced by combined voluntary wheel running and green tea extract. J Appl Physiol 2008; 105: 923-32.
424. Evans NP, Call JA, Bassaganya-Riera J, Robertson JL, Grange RW. Green tea extract decreases muscle pathology and NF-kappaB immunostaining in regenerating muscle fibers of mdx mice. Clin Nutr 2010; 29: 391-8.
425. Liu HS, Chen YH, Hung PF, Kao YH. Inhibitory effect of green tea (-)-epigallocatechin gallate on resistin gene expression in 3T3-L1 adipocytes depends on the ERK pathway. American Journal of Physiology Endocrinology and Metabolism 2006; 290: E273-81.
426. Sah JF, Balasubramanian S, Eckert RL, Rorke EA. Epigallocatechin-3-gallate inhibits epidermal growth factor receptor signaling pathway. Evidence for direct inhibition of ERK1/2 and AKT kinases. J Biol Chem 2004; 279: 12755-62.
427. Zhang Q, Kelly AP, Wang L, et al. Green tea extract and (-)- epigallocatechin-3-gallate inhibit mast cell-stimulated type I collagen expression in keloid fibroblasts via blocking PI-3K/AkT signaling pathways. J Invest Dermatol 2006; 126: 2607-13.
428. Chen PC, Wheeler DS, Malhotra V, Odoms K, Denenberg AG, Wong HR. A green tea-derived polyphenol, epigallocatechin-3- gallate, inhibits IkappaB kinase activation and IL-8 gene expression in respiratory epithelium. Inflammation 2002; 26: 233-41.
429. Acuna-Castroviejo D, Escames G, Rodriguez MI, Lopez LC. Melatonin role in the mitochondrial function. Front Biosci 2007; 12: 947-63.
430. Hibaoui Y, Roulet E, Ruegg UT. Melatonin prevents oxidative stress-mediated mitochondrial permeability transition and death in skeletal muscle cells. Journal of Pineal Research 2009; 47: 238-52.
431. Yeung HM, Hung MW, Fung ML. Melatonin ameliorates calcium homeostasis in myocardial and ischemia-reperfusion injury in chronically hypoxic rats. J Pineal Res 2008; 45: 373-82.
432. Hibaoui Y, Reutenauer-Patte J, Patthey-Vuadens O, Ruegg UT, Dorchies OM. Melatonin improves muscle function of the dystrophic mdx5Cv mouse, a model for Duchenne muscular dystrophy. J Pineal Res 2011; 51: 163-71.
433. Chahbouni M, Escames G, Venegas C, et al. Melatonin treatment normalizes plasma pro-inflammatory cytokines and nitrosative/ oxidative stress in patients suffering from Duchenne muscular dystrophy. J Pineal Res 2010; 48: 282-9.
434. Chahbouni M, Escames G, Lopez LC, et al. Melatonin treatment counteracts the hyperoxidative status in erythrocytes of patients suffering from Duchenne muscular dystrophy. Clin Biochem 2011; 44: 853-8.
435. Hankard R, Mauras N, Hammond D, Haymond M, Darmaun D. Is glutamine a 'conditionally essential' amino acid in Duchenne muscular dystrophy? Clin Nutr 1999; 18: 365-9.
436. Mok E, Constantin B, Favreau F, et al. l-Glutamine administration reduces oxidized glutathione and MAP kinase signaling in dystrophic muscle of mdx mice. Pediatr Res 2008; 63: 268-73.
437. Hankard RG, Hammond D, Haymond MW, Darmaun D. Oral glutamine slows down whole body protein breakdown in Duchenne muscular dystrophy. Pediatr Res 1998; 43: 222-6.
438. Mok E, Eleouet-Da Violante C, Daubrosse C, et al. Oral glutamine and amino acid supplementation inhibit whole-body protein degradation in children with Duchenne muscular dystrophy. Am J Clin Nutr 2006; 83: 823-8.
439. Mok E, Letellier G, Cuisset JM, et al. Lack of functional benefit with glutamine versus placebo in Duchenne muscular dystrophy: a randomized crossover trial. PLoS ONE 2009; 4: e5448.
440. Payne ET, Yasuda N, Bourgeois JM, et al. Nutritional therapy improves function and complements corticosteroid intervention in mdx mice. Muscle Nerve 2006; 33: 66-77.
441. Granchelli JA, Pollina C, Hudecki MS. Pre-clinical screening of drugs using the mdx mouse. Neuromuscul Disord 2000; 10: 235-9.
442. Qureshi MM, McClure WC, Arevalo NL, et al. The Dietary Supplement Protandim Decreases Plasma Osteopontin and Improves Markers of Oxidative Stress in Muscular Dystrophy mdx Mice. Journal of Dietary Supplements 2010; 7: 159-78.
443. Davoodi J, Markert CD, Voelker KA, Hutson SM, Grange RW. Nutrition strategies to improve physical capabilities in duchenne muscular dystrophy. Phys Med Rehabil Clin N Am 2012; 23: 187-99.
444. Evangeliou A, Vasilaki K, Karagianni P, Nikolaidis N. Clinical applications of creatine supplementation on paediatrics. Current Pharmaceutical Biotechnology 2009; 10: 683-90.
445. Escolar DM, Buyse G, Henricson E, et al. CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy. Ann Neurol 2005; 58: 151-5.
446. Tarnopolsky MA, Mahoney DJ, Vajsar J, et al. Creatine monohydrate enhances strength and body composition in Duchenne muscular dystrophy. Neurology 2004; 62: 1771-7.
447. Aruoma OI, Halliwell B, Hoey BM, Butler J. The antioxidant action of N-acetylcysteine: its reaction with hydrogen peroxide, hydroxyl radical, superoxide, and hypochlorous acid. Free Radical Biology & Medicine 1989; 6: 593-7.
448. Medved I, Brown MJ, Bjorksten AR, et al. N-acetylcysteine enhances muscle cysteine and glutathione availability and attenuates fatigue during prolonged exercise in endurance-trained individuals. J Appl Physiol 2004; 97: 1477-85.
449. Terrill JR, Radley-Crabb HG, Grounds MD, Arthur PG. N- Acetylcysteine treatment of dystrophic mdx mice results in protein thiol modifications and inhibition of exercise induced myofibre necrosis. Neuromuscul Disord 2011.
450. Williams IA, Allen DG. The role of reactive oxygen species in the hearts of dystrophin-deficient mdx mice. Am J Physiol Heart Circ Physiol 2007; 293: H1969-77.
451. Farid M, Reid MB, Li YP, Gerken E, Durham WJ. Effects of dietary curcumin or N-acetylcysteine on NF-kappaB activity and contractile performance in ambulatory and unloaded murine soleus. Nutr Metab (Lond) 2005; 2: 20.
452. Smith RA, Murphy MP. Mitochondria-targeted antioxidants as therapies. Discov Med 2011; 11: 106-14.
453. Gane EJ, Weilert F, Orr DW, et al. The mitochondria-targeted anti-oxidant mitoquinone decreases liver damage in a phase II study of hepatitis C patients. Liver International: Official Journal of the International Association for the Study of the Liver 2010; 30: 1019-26.
454. Menazza S, Blaauw B, Tiepolo T, et al. Oxidative stress by monoamine oxidases is causally involved in myofiber damage in muscular dystrophy. Hum Mol Genet 2010; 19: 4207-15.
455. Okamoto T, Kubota N, Takahata K, Takahashi T, Goshima K, Kishi T. Protective effect of coenzyme Q10 on cultured skeletal muscle cell injury induced by continuous electric field stimulation. Biochem Biophys Res Commun 1995; 216: 1006-12.
456. Lonnrot K, Holm P, Lagerstedt A, Huhtala H, Alho H. The effects of lifelong ubiquinone Q10 supplementation on the Q9 and Q10 tissue concentrations and life span of male rats and mice. Biochem Mol Biol Int 1998; 44: 727-37.
457. Papucci L, Schiavone N, Witort E, et al. Coenzyme q10 prevents apoptosis by inhibiting mitochondrial depolarization independently of its free radical scavenging property. J Biol Chem 2003; 278: 28220-8.
458. Spurney CF, Rocha CT, Henricson E, et al. CINRG pilot trial of coenzyme Q10 in steroid-treated Duchenne muscular dystrophy. Muscle Nerve 2011; 44: 174-8.
459. Zs-Nagy I. Chemistry, toxicology, pharmacology and pharmacokinetics of idebenone: a review. Arch Gerontol Geriatr 1990; 11: 177-86.
460. Meier T, Perlman SL, Rummey C, Coppard NJ, Lynch DR. Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study. J Neurol 2011.
461. Buyse GM, Van der Mieren G, Erb M, et al. Long-term blinded placebo-controlled study of SNT-MC17/idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance. Eur Heart J 2009; 30: 116-24.
462. Buyse GM, Goemans N, van den Hauwe M, et al. Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trial. Neuromuscul Disord 2011; 21: 396-405.
463. Kishi T, Watanabe T, Folkers K. Bioenergetics in clinical medicine XV. Inhibition of coenzyme Q10-enzymes by clinically used adrenergic blockers of beta-receptors. Research Communications in Chemical Pathology and Pharmacology 1977; 17: 157-64.
464. Bredt DS, Snyder SH. Isolation of nitric oxide synthetase, a calmodulin-requiring enzyme. Proc Natl Acad Sci U S A 1990; 87: 682-5.
465. Cho HJ, Xie QW, Calaycay J, et al. Calmodulin is a subunit of nitric oxide synthase from macrophages. J Exp Med 1992; 176: 599-604.
466. Stamler JS, Meissner G. Physiology of nitric oxide in skeletal muscle. Physiol Rev 2001; 81: 209-37.
467. Lau KS, Grange RW, Chang WJ, Kamm KE, Sarelius I, Stull JT. Skeletal muscle contractions stimulate cGMP formation and attenuate vascular smooth muscle myosin phosphorylation via nitric oxide. FEBS Lett 1998; 431: 71-4.
468. Kobzik L, Reid MB, Bredt DS, Stamler JS. Nitric oxide in skeletal muscle. Nature 1994; 372: 546-8.
469. Wang T, Xie Z, Lu B. Nitric oxide mediates activity-dependent synaptic suppression at developing neuromuscular synapses. Nature 1995; 374: 262-6.
470. Wakayama Y, Inoue M, Murahashi M, et al. Ultrastructural localization of alpha 1-syntrophin and neuronal nitric oxide synthase in normal skeletal myofiber, and their relation to each other and to dystrophin. Acta Neuropathol 1997; 94: 455-64.
471. Chang WJ, Iannaccone ST, Lau KS, et al. Neuronal nitric oxide synthase and dystrophin-deficient muscular dystrophy. Proc Natl Acad Sci U S A 1996; 93: 9142-7.
472. Bia BL, Cassidy PJ, Young ME, et al. Decreased myocardial nNOS, increased iNOS and abnormal ECGs in mouse models of Duchenne muscular dystrophy. J Mol Cell Cardiol 1999; 31: 1857-62.
473. Wehling-Henricks M, Tidball JG. Neuronal nitric oxide synthase-rescue of dystrophin/utrophin double knockout mice does not require nNOS localization to the cell membrane. PLoS ONE 2011; 6: e25071.
474. Asai A, Sahani N, Kaneki M, Ouchi Y, Martyn JA, Yasuhara SE. Primary role of functional ischemia, quantitative evidence for the two-hit mechanism, and phosphodiesterase-5 inhibitor therapy in mouse muscular dystrophy. PLoS ONE 2007; 2: e806.
475. Garcia-Mata C, Gay R, Sokolovski S, Hills A, Lamattina L, Blatt MR. Nitric oxide regulates K+ and Cl- channels in guard cells through a subset of abscisic acid-evoked signaling pathways. Proc Natl Acad Sci U S A 2003; 100: 11116-21.
476. Corbin JD, Francis SH. Cyclic GMP phosphodiesterase-5: target of sildenafil. J Biol Chem 1999; 274: 13729-32.
477. Boolell M, Allen MJ, Ballard SA, et al. Sildenafil: an orally active type 5 cyclic GMP-specific phosphodiesterase inhibitor for the treatment of penile erectile dysfunction. International Journal of Impotence Research 1996; 8: 47-52.
478. Ravipati G, McClung JA, Aronow WS, Peterson SJ, Frishman WH. Type 5 phosphodiesterase inhibitors in the treatment of erectile dysfunction and cardiovascular disease. Cardiol Rev 2007; 15: 76-86.
479. Singh TP. Clinical use of sildenafil in pulmonary artery hypertension. Expert Rev Respir Med 2010; 4: 13-9.
480. Gomez-Moreno S, Lage E, Hernandez A, et al. Use of oral sildenafil in patients with irreversible pulmonary hypertension not eligible for heart transplantation. Transplant Proc 2005; 37: 1550-1.
481. Takimoto E, Champion HC, Li M, et al. Chronic inhibition of cyclic GMP phosphodiesterase 5A prevents and reverses cardiac hypertrophy. Nat Med 2005; 11: 214-22.
482. Adamo CM, Dai DF, Percival JM, et al. Sildenafil reverses cardiac dysfunction in the mdx mouse model of Duchenne muscular dystrophy. Proc Natl Acad Sci U S A 2010; 107: 19079-83.
483. Ascah A, Khairallah M, Daussin F, et al. Stress-induced opening of the permeability transition pore in the dystrophin-deficient heart is attenuated by acute treatment with sildenafil. Am J Physiol Heart Circ Physiol 2011; 300: H144-53.
484. Sander M, Chavoshan B, Harris SA, et al. Functional muscle ischemia in neuronal nitric oxide synthase-deficient skeletal muscle of children with Duchenne muscular dystrophy. Proc Natl Acad Sci U S A 2000; 97: 13818-23.
485. Mendell JR, Engel WK, Derrer EC. Duchenne muscular dystrophy: functional ischemia reproduces its characteristic lesions. Science 1971; 172: 1143-5.
486. Grange RW, Isotani E, Lau KS, Kamm KE, Huang PL, Stull JT. Nitric oxide contributes to vascular smooth muscle relaxation in contracting fast-twitch muscles. Physiol Genomics 2001; 5: 35-44.
487. Lau KS, Grange RW, Isotani E, et al. nNOS and eNOS modulate cGMP formation and vascular response in contracting fast-twitch skeletal muscle. Physiol Genomics 2000; 2: 21-7.
488. Thomas GD, Sander M, Lau KS, Huang PL, Stull JT, Victor RG. Impaired metabolic modulation of alpha-adrenergic vasoconstriction in dystrophin-deficient skeletal muscle. Proc Natl Acad Sci U S A 1998; 95: 15090-5.
489. Burbach JA. Ultrastructure of cardiocyte degeneration and myocardial calcification in the dystrophic hamster. The American Journal of Anatomy 1987; 179: 291-307.
490. Kobayashi Y, Suzuki H, Iinuma K, Tada K, Yamamoto TY. Endothelial alterations of skeletal muscle capillaries in childhood myopathies. The Tohoku journal of experimental medicine 1983; 140: 381-9.
491. Miike T, Sugino S, Ohtani Y, Taku K, Yoshioka K. Vascular endothelial cell injury and platelet embolism in Duchenne muscular dystrophy at the preclinical stage. J Neurol Sci 1987; 82: 67-80.
492. Musch BC, Papapetropoulos TA, McQueen DA, Hudgson P, Weightman D. A comparison of the structure of small blood vessels in normal, denervated and dystrophic human muscle. J Neurol Sci 1975; 26: 221-34.
493. Hunter EG, Elbrink J. Increased contractility in vascular smooth muscle of dystrophic hamsters. Canadian Journal of Physiology and Pharmacology 1983; 61: 182-5.
494. Miyatake M, Miike T, Zhao J, Yoshioka K, Uchino M, Usuku G. Possible systemic smooth muscle layer dysfunction due to a deficiency of dystrophin in Duchenne muscular dystrophy. J Neurol Sci 1989; 93: 11-7.
495. Bredt DS. Endogenous nitric oxide synthesis: biological functions and pathophysiology. Free Radical Research 1999; 31: 577-96.
496. Burdi R, Rolland JF, Fraysse B, et al. Multiple pathological events in exercised dystrophic mdx mice are targeted by pentoxifylline: outcome of a large array of in vivo and ex vivo tests. J Appl Physiol 2009; 106: 1311-24.
497. Gosselin LE, Williams JE. Pentoxifylline fails to attenuate fibrosis in dystrophic (mdx) diaphragm muscle. Muscle Nerve 2006; 33: 820-3.
498. Zimmerman A, Clemens PR, Tesi-Rocha C, et al. Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy. Muscle Nerve 2011; 44: 170-3.
499. Kaliman P, Canicio J, Testar X, Palacin M, Zorzano A. Insulin-like growth factor-II, phosphatidylinositol 3-kinase, nuclear factor- kappaB and inducible nitric-oxide synthase define a common myogenic signaling pathway. J Biol Chem 1999; 274: 17437-44.
500. Pisconti A, Brunelli S, Di Padova M, et al. Follistatin induction by nitric oxide through cyclic GMP: a tightly regulated signaling pathway that controls myoblast fusion. J Cell Biol 2006; 172: 233-44.
501. Tatsumi R, Hattori A, Ikeuchi Y, Anderson JE, Allen RE. Release of hepatocyte growth factor from mechanically stretched skeletal muscle satellite cells and role of pH and nitric oxide. Mol Biol Cell 2002; 13: 2909-18.
502. Anderson JE. A role for nitric oxide in muscle repair: nitric oxide-mediated activation of muscle satellite cells. Mol Biol Cell 2000; 11: 1859-74.
503. Wallace JL, Reuter B, Cicala C, McKnight W, Grisham MB, Cirino G. Novel nonsteroidal anti-inflammatory drug derivatives with markedly reduced ulcerogenic properties in the rat. Gastroenterology 1994; 107: 173-9.
504. Scatena R. Nitroflurbiprofen (NicOx). Curr Opin Investig Drugs 2004; 5: 551-6.
505. Brunelli S, Sciorati C, D'Antona G, et al. Nitric oxide release combined with nonsteroidal antiinflammatory activity prevents muscular dystrophy pathology and enhances stem cell therapy. Proc Natl Acad Sci U S A 2007; 104: 264-9.
506. Sciorati C, Galvez BG, Brunelli S, et al. Ex vivo treatment with nitric oxide increases mesoangioblast therapeutic efficacy in muscular dystrophy. J Cell Sci 2006; 119: 5114-23.
507. Deponti D, Francois S, Baesso S, et al. Necdin mediates skeletal muscle regeneration by promoting myoblast survival and differentiation. J Cell Biol 2007; 179: 305-19.
508. Mizunoya W, Upadhaya R, Burczynski FJ, Wang G, Anderson JE. Nitric oxide donors improve prednisone effects on muscular dystrophy in the mdx mouse diaphragm. Am J Physiol Cell Physiol 2011; 300: C1065-77.
509. Wang G, Burczynski FJ, Hasinoff BB, Zhang K, Lu Q, Anderson JE. Development of a nitric oxide-releasing analogue of the muscle relaxant guaifenesin for skeletal muscle satellite cell myogenesis. Molecular Pharmaceutics 2009; 6: 895-904.
510. Sawada H, Tsuji S, Kusumoto S, Doi Y, Matsushita H. Preclinical increase in activity of muscle microsomal trypsin-like protease in murine muscular dystrophy, C57BL/10-mdx. FEBS Lett 1986; 199: 193-7.
511. Spencer MJ, Tidball JG. Calpain concentration is elevated although net calcium-dependent proteolysis is suppressed in dystrophin-deficient muscle. Exp Cell Res 1992; 203: 107-14.
512. Turner PR, Schultz R, Ganguly B, Steinhardt RA. Proteolysis results in altered leak channel kinetics and elevated free calcium in mdx muscle. The Journal of membrane biology 1993; 133: 243-51.
513. Spencer MJ, Croall DE, Tidball JG. Calpains are activated in necrotic fibers from mdx dystrophic mice. J Biol Chem 1995; 270: 10909-14.
514. Kumamoto T, Fujimoto S, Ito T, Horinouchi H, Ueyama H, Tsuda T. Proteasome expression in the skeletal muscles of patients with muscular dystrophy. Acta Neuropathol 2000; 100: 595-602.
515. Bodine SC, Latres E, Baumhueter S, et al. Identification of ubiquitin ligases required for skeletal muscle atrophy. Science 2001; 294: 1704-8.
516. Spencer MJ, Mellgren RL. Overexpression of a calpastatin transgene in mdx muscle reduces dystrophic pathology. Hum Mol Genet 2002; 11: 2645-55.
517. Badalamente MA, Stracher A. Delay of muscle degeneration and necrosis in mdx mice by calpain inhibition. Muscle Nerve 2000; 23: 106-11.
518. Sawada H, Nagahiro K, Kikukawa Y, et al. Therapeutic effect of camostat mesilate on Duchenne muscular dystrophy in mdx mice. Biol Pharm Bull 2003; 26: 1025-7.
519. Lescop C, Herzner H, Siendt H, et al. Novel cell-penetrating alpha- keto-amide calpain inhibitors as potential treatment for muscular dystrophy. Bioorg Med Chem Lett 2005; 15: 5176-81.
520. Burdi R, Didonna MP, Pignol B, et al. First evaluation of the potential effectiveness in muscular dystrophy of a novel chimeric compound, BN 82270, acting as calpain-inhibitor and anti-oxidant. Neuromuscul Disord 2006; 16: 237-48.
521. Briguet A, Erb M, Courdier-Fruh I, et al. Effect of calpain and proteasome inhibition on Ca2+-dependent proteolysis and muscle histopathology in the mdx mouse. FASEB J 2008; 22: 4190-200.
522. Selsby J, Pendrak K, Zadel M, et al. Leupeptin-based inhibitors do not improve the mdx phenotype. Am J Physiol Regul Integr Comp Physiol 2010; 299: R1192-201.
523. Childers MK, Bogan JR, Bogan DJ, et al. Chronic administration of a leupeptin-derived calpain inhibitor fails to ameliorate severe muscle pathology in a canine model of duchenne muscular dystrophy. Frontiers in Pharmacology 2011; 2: 89.
524. Bonuccelli G, Sotgia F, Schubert W, et al. Proteasome inhibitor (MG-132) treatment of mdx mice rescues the expression and membrane localization of dystrophin and dystrophin-associated proteins. Am J Pathol 2003; 163: 1663-75.
525. Assereto S, Stringara S, Sotgia F, et al. Pharmacological rescue of the dystrophin-glycoprotein complex in Duchenne and Becker skeletal muscle explants by proteasome inhibitor treatment. Am J Physiol Cell Physiol 2006; 290: C577-82.
526. Kane RC, Bross PF, Farrell AT, Pazdur R. Velcade: U.S. FDA approval for the treatment of multiple myeloma progressing on prior therapy. The Oncologist 2003; 8: 508-13.
527. Bonuccelli G, Sotgia F, Capozza F, Gazzerro E, Minetti C, Lisanti MP. Localized treatment with a novel FDA-approved proteasome inhibitor blocks the degradation of dystrophin and dystrophin-associated proteins in mdx mice. Cell Cycle 2007; 6: 1242-8.
528. Gazzerro E, Assereto S, Bonetto A, et al. Therapeutic potential of proteasome inhibition in Duchenne and Becker muscular dystrophies. Am J Pathol 2010; 176: 1863-77.
529. San Miguel J, Blade J, Boccadoro M, et al. A practical update on the use of bortezomib in the management of multiple myeloma. The Oncologist 2006; 11: 51-61.
530. Zamzami N, Kroemer G. The mitochondrion in apoptosis: how Pandora's box opens. Nat Rev Mol Cell Biol 2001; 2: 67-71.
531. Halestrap AP, Davidson AM. Inhibition of Ca2(+)-induced large-amplitude swelling of liver and heart mitochondria by cyclosporin is probably caused by the inhibitor binding to mitochondrial-matrix peptidyl-prolyl cis-trans isomerase and preventing it interacting with the adenine nucleotide translocase. Biochem J 1990; 268: 153-60.
532. McGuinness O, Yafei N, Costi A, Crompton M. The presence of two classes of high-affinity cyclosporin A binding sites in mitochondria. Evidence that the minor component is involved in the opening of an inner-membrane Ca(2+)-dependent pore. European journal of biochemistry/FEBS 1990; 194: 671-9.
533. Reutenauer J, Dorchies OM, Patthey-Vuadens O, Vuagniaux G, Ruegg UT. Investigation of Debio 025, a cyclophilin inhibitor, in the dystrophic mdx mouse, a model for Duchenne muscular dystrophy. Br J Pharmacol 2008; 155: 574-84.
534. Weller B, Massa R, Karpati G, Carpenter S. Glucocorticoids and immunosuppressants do not change the prevalence of necrosis and regeneration in mdx skeletal muscles. Muscle Nerve 1991; 14: 771-4.
535. Sharma KR, Mynhier MA, Miller RG. Cyclosporine increases muscular force generation in Duchenne muscular dystrophy. Neurology 1993; 43: 527-32.
536. Kirschner J, Schessl J, Schara U, et al. Treatment of Duchenne muscular dystrophy with ciclosporin A: a randomised, double-blind, placebo-controlled multicentre trial. Lancet Neurol 2010; 9: 1053-9.
537. Stupka N, Gregorevic P, Plant DR, Lynch GS. The calcineurin signal transduction pathway is essential for successful muscle regeneration in mdx dystrophic mice. Acta Neuropathol 2004; 107: 299-310.
538. Friday BB, Horsley V, Pavlath GK. Calcineurin activity is required for the initiation of skeletal muscle differentiation. J Cell Biol 2000; 149: 657-66.
539. Hansson MJ, Mattiasson G, Mansson R, et al. The nonimmunosuppressive cyclosporin analogs NIM811 and UNIL025 display nanomolar potencies on permeability transition in brain-derived mitochondria. Journal of Bioenergetics and Biomembranes 2004; 36: 407-13.
540. Millay DP, Sargent MA, Osinska H, et al. Genetic and pharmacologic inhibition of mitochondrial-dependent necrosis attenuates muscular dystrophy. Nat Med 2008; 14: 442-7.
541. Wissing ER, Millay DP, Vuagniaux G, Molkentin JD. Debio-025 is more effective than prednisone in reducing muscular pathology in mdx mice. Neuromuscul Disord 2010; 20: 753-60.
542. Tiepolo T, Angelin A, Palma E, et al. The cyclophilin inhibitor Debio 025 normalizes mitochondrial function, muscle apoptosis and ultrastructural defects in Col6a1-/-myopathic mice. Br J Pharmacol 2009; 157: 1045-52.
543. Whitehead NP, Streamer M, Lusambili LI, Sachs F, Allen DG. Streptomycin reduces stretch-induced membrane permeability in muscles from mdx mice. Neuromuscul Disord 2006; 16: 845-54.
544. Matsumura CY, Taniguti AP, Pertille A, Santo Neto H, Marques MJ. Stretch-activated calcium channel protein TRPC1 is correlated with the different degrees of the dystrophic phenotype in mdx mice. American Journal of Physiology Cell Physiology 2011; 301: C1344-50.
545. Ward ML, Williams IA, Chu Y, Cooper PJ, Ju YK, Allen DG. Stretch-activated channels in the heart: contributions to length-dependence and to cardiomyopathy. Progress in Biophysics and Molecular Biology 2008; 97: 232-49.
546. Yeung EW, Allen DG. Stretch-activated channels in stretchinduced muscle damage: role in muscular dystrophy. Clin Exp Pharmacol Physiol 2004; 31: 551-6.
547. Rolland JF, De Luca A, Burdi R, Andreetta F, Confalonieri P, Conte Camerino D. Overactivity of exercise-sensitive cation channels and their impaired modulation by IGF-1 in mdx native muscle fibers: beneficial effect of pentoxifylline. Neurobiol Dis 2006; 24: 466-74.
548. Suchyna TM, Sachs F. Mechanosensitive channel properties and membrane mechanics in mouse dystrophic myotubes. The Journal of Physiology 2007; 581: 369-87.
549. Jorgensen LH, Blain A, Greally E, et al. Long-term blocking of calcium channels in mdx mice results in differential effects on heart and skeletal muscle. Am J Pathol 2011; 178: 273-83.
550. Gehrig SM, van der Poel C, Sayer TA, et al. Hsp72 preserves muscle function and slows progression of severe muscular dystrophy. Nature 2012.
551. Nicolas-Metral V, Raddatz E, Kucera P, Ruegg UT. Mdx myotubes have normal excitability but show reduced contraction-relaxation dynamics. Journal of Muscle Research and Cell Motility 2001; 22: 69-75.
552. Tutdibi O, Brinkmeier H, Rudel R, Fohr KJ. Increased calcium entry into dystrophin-deficient muscle fibres of mdx and ADR-mdx mice is reduced by ion channel blockers. The Journal of Physiology 1999; 515 (Pt 3): 859-68.
553. Goonasekera SA, Lam CK, Millay DP, et al. Mitigation of muscular dystrophy in mice by SERCA overexpression in skeletal muscle. The Journal of Clinical Investigation 2011; 121: 1044-52.
554. Morine KJ, Sleeper MM, Barton ER, Sweeney HL. Overexpression of SERCA1a in the mdx diaphragm reduces susceptibility to contraction-induced damage. Hum Gene Ther 2010; 21: 1735-9.
555. Literati-Nagy B, Kulcsar E, Literati-Nagy Z, et al. Improvement of insulin sensitivity by a novel drug, BGP-15, in insulin-resistant patients: a proof of concept randomized double-blind clinical trial. Hormone and metabolic research = Hormonund Stoffwechselforschung = Hormones et metabolisme 2009; 41: 374-80.
556. Tupling AR, Gramolini AO, Duhamel TA, et al. HSP70 binds to the fast-twitch skeletal muscle sarco(endo)plasmic reticulum Ca2+ -ATPase (SERCA1a) and prevents thermal inactivation. The Journal of Biological Chemistry 2004; 279: 52382-9.
557. Bornman L, Polla BS, Lotz BP, Gericke GS. Expression of heatshock/ stress proteins in Duchenne muscular dystrophy. Muscle & Nerve 1995; 18: 23-31.
558. Aartsma-Rus A. The risks of therapeutic misconception and individual patient (n=1) trials in rare diseases such as Duchenne dystrophy. Neuromuscul Disord 2011; 21: 13-5.
559. Catlin N, Bettelheim K, Henderson I. Individual patient (n=1) trials in Duchenne dystrophy. Neuromuscul Disord 2011; 21: 525-6; author reply 7-8.
560. Balaban B, Matthews DJ, Clayton GH, Carry T. Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: long-term effect. Am J Phys Med Rehabil 2005; 84: 843-50.
561. Biggar WD, Harris VA, Eliasoph L, Alman B. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord 2006; 16: 249-55.
562. King WM, Ruttencutter R, Nagaraja HN, et al. Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy. Neurology 2007; 68: 1607-13.
563. Houde S, Filiatrault M, Fournier A, et al. Deflazacort use in Duchenne muscular dystrophy: an 8-year follow-up. Pediatr Neurol 2008; 38: 200-6.
564. Archer JD, Vargas CC, Anderson JE. Persistent and improved functional gain in mdx dystrophic mice after treatment with Larginine and deflazacort. FASEB J 2006; 20: 738-40.
565. Segalat L, Grisoni K, Archer J, Vargas C, Bertrand A, Anderson JE. CAPON expression in skeletal muscle is regulated by position, repair, NOS activity, and dystrophy. Exp Cell Res 2005; 302: 170-9.
566. Grounds MD, Davies M, Torrisi J, Shavlakadze T, White J, Hodgetts S. Silencing TNFalpha activity by using Remicade or Enbrel blocks inflammation in whole muscle grafts: an in vivo bioassay to assess the efficacy of anti-cytokine drugs in mice. Cell Tissue Res 2005; 320: 509-15.
567. Ishikawa Y, Bach JR, Minami R. A management trial for Duchenne cardiomyopathy. Am J Phys Med Rehabil 1995; 74: 345-50.
568. Matsumura T, Tamura T, Kuru S, Kikuchi Y, Kawai M. Carvedilol can prevent cardiac events in Duchenne muscular dystrophy. Intern Med 2010; 49: 1357-63.
569. Shaddy RE, Tani LY, Gidding SS, et al. Beta-blocker treatment of dilated cardiomyopathy with congestive heart failure in children: a multi-institutional experience. The Journal of Heart and Lung Transplantation: The Official Publication of the International Society for Heart Transplantation 1999; 18: 269-74.
570. Saito T, Matsumura T, Miyai I, Nozaki S, Shinno S. [Carvedilol effectiveness for left ventricular-insufficient patients with Duchenne muscular dystrophy]. Rinsho Shinkeigaku 2001; 41: 691-4.
571. Folkers K, Simonsen R. Two successful double-blind trials with coenzyme Q10 (vitamin Q10) on muscular dystrophies and neurogenic atrophies. Biochim Biophys Acta 1995; 1271: 281-6.
572. Vandenburgh H, Shansky J, Benesch-Lee F, et al. Automated drug screening with contractile muscle tissue engineered from dystrophic myoblasts. FASEB J 2009; 23: 3325-34.
573. Yeung EW, Head SI, Allen DG. Gadolinium reduces short-term stretch-induced muscle damage in isolated mdx mouse muscle fibres. J Physiol 2003; 552: 449-58.
574. Kumar A, Khandelwal N, Malya R, Reid MB, Boriek AM. Loss of dystrophin causes aberrant mechanotransduction in skeletal muscle fibers. FASEB J 2004; 18: 102-13.