Targeting fibrosis in duchenne muscular dystrophy

Journal of Neuropathology and Experimental Neurology

Volumn 69, Issue 8, 2010, Pages 771-776

  Zhou, Lan ^a   Lu, Haiyan ^a  

^a LERNER RESEARCH INSTITUTE   (United States)

Author keywords

Antifibrotic therapy; Duchenne muscular dystrophy; Muscle fibrosis.

Indexed keywords

ANTIFIBROTIC AGENT; DECORIN; FIBRINOGEN; HALOFUGINONE; IMATINIB; LOSARTAN; MYOSTATIN; TRANSFORMING GROWTH FACTOR BETA ANTIBODY;

CELL THERAPY; DRUG EFFECT; DRUG INHIBITION; DUCHENNE MUSCULAR DYSTROPHY; EOSINOPHIL; FIBROGENESIS; FIBROSIS; GENE THERAPY; HEART FAILURE; HUMAN; LIMB WEAKNESS; LUNG FIBROSIS; MUSCLE BIOPSY; MUSCLE FUNCTION; MUSCLE REGENERATION; MYOFIBROSIS; MYOSITIS; PHENOTYPE; PREMATURITY; PRIORITY JOURNAL; REVIEW; SCAR FORMATION; SKIN FIBROSIS; STEM CELL TRANSPLANTATION; T LYMPHOCYTE; UNSPECIFIED SIDE EFFECT; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

ANIMALS; DYSTROPHIN; FIBROSIS; GENE THERAPY; HUMANS; MALE; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; TISSUE THERAPY;

EID: 77955472624     PISSN: 00223069     EISSN: None     Source Type: Journal    
DOI: 10.1097/NEN.0b013e3181e9a34b     Document Type: Review

References (63)

1. Wynn TA. Common and unique mechanisms regulate fibrosis in various fibroproliferative diseases. J Clin Invest 2007;117:524-529
2. Wynn TA. Cellular and molecular mechanisms of fibrosis. J Pathol 2008; 214:199-210
3. Emery AE. Population frequencies of inherited neuromuscular diseasesV a world survey. Neuromuscul Disord 1991;1:19-29
4. Emery AEH. Duchenne Muscular Dystrophy. Oxford, UK: Oxford University Press, 1993
5. Desguerre I, Mayer M, Leturcq F, et al. Endomysial fibrosis in Duchenne muscular dystrophy: A marker of poor outcome associated with macrophage alternative activation. J Neuropathol Exp Neurol 2009;68:762-773
6. Border WA, Noble NA, Yamamoto T, et al. Natural inhibitor of transforming growth factor-beta protects against scarring in experimental kidney disease. Nature 1992;360:361-364
7. Clouthier DE, Comerford SA, Hammer RE. Hepatic fibrosis, glomerulosclerosis, and a lipodystrophy-like syndrome in PEPCKYTGF-beta1 transgenic mice. J Clin Invest 1997;100:2697-2713
8. Sato M, Muragaki Y, Saika S, et al. Targeted disruption of TGFbeta1/Smad3 signaling protects against renal tubulointerstitial fibrosis induced by unilateral ureteral obstruction. J Clin Invest 2003;112: 1486-1494
9. Sime PJ, Xing Z, Graham FL, et al. Adenovector-mediated gene transfer of active transforming growth factor-beta1 induces prolonged severe fibrosis in rat lung. J Clin Invest 1997;100:768-776
10. Heydemann A, Ceco E, Lim JE, et al. Latent TGF-betaYbinding protein 4 modifies muscular dystrophy in mice. J Clin Invest 2009;119:3703-3712
11. Shi Y, Massague J. Mechanisms of TGF-beta signaling from cell membrane to the nucleus. Cell 2003;113:685-700
12. Runyan CE, Schnaper HW, Poncelet AC. Smad3 and PKCdelta mediate TGF-beta1Yinduced collagen I expression in human mesangial cells. Am J Physiol Renal Physiol 2003;285:F413-22
13. Border WA, Noble NA. Transforming growth factor beta in tissue fibrosis. N Engl J Med 1994;331:1286-1292
14. 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;96:1137-1144
15. 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;16:32-38
16. Reed CC, Iozzo RV. The role of decorin in collagen fibrillogenesis and skin homeostasis. Glycoconj J 2002;19:249-255
17. Gosselin LE, Williams JE, Deering M, et al. Localization and early time course of TGF-beta1 mRNA expression in dystrophic muscle. Muscle Nerve 2004;30:645-653
18. Li Y, Foster W, Deasy BM, et al. Transforming growth factor-beta1 induces the differentiation of myogenic cells into fibrotic cells in injured skeletal muscle: A key event in muscle fibrogenesis. Am J Pathol 2004; 164:1007-1019
19. 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
20. Rosenkranz S. TGF-beta1 and angiotensin networking in cardiac remodeling. Cardiovasc Res 2004;63:423-432
21. Cohn RD, van Erp C, Habashi JP, et al. Angiotensin II type 1 receptor blockade attenuates TGF-betaYinduced failure of muscle regeneration in multiple myopathic states. Nat Med 2007;13:204-210
22. Granot I, Halevy O, Hurwitz S, et al. Halofuginone: An inhibitor of collagen type I synthesis. Biochim Biophys Acta 1993;1156:107-112
23. Halevy O, Nagler A, Levi-Schaffer F, et al. Inhibition of collagen type I synthesis by skin fibroblasts of graft versus host disease and scleroderma patients: Effect of halofuginone. Biochem Pharmacol 1996;52:1057-1063
24. McGaha TL, Phelps RG, Spiera H, et al. Halofuginone, an inhibitor of type-I collagen synthesis and skin sclerosis, blocks transforminggrowth-factor- betaYmediated Smad3 activation in fibroblasts. J Invest Dermatol 2002;118:461-470
25. 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;18:857-868
26. Huebner KD, Jassal DS, Halevy O, et al. Functional resolution of fibrosis in mdx mouse dystrophic heart and skeletal muscle by halofuginone. Am J Physiol Heart Circ Physiol 2008;294:H1550-61
27. Granot I, Bartov I, Plavnik I, et al. Increased skin tearing in broilers and reduced collagen synthesis in skin in vivo and in vitro in response to the coccidiostat halofuginone. Poult Sci 1991;70:1559-1563
28. Vidal B, Serrano AL, Tjwa M, et al. Fibrinogen drives dystrophic muscle fibrosis via a TGFbeta/alternative macrophage activation pathway. Genes Dev 2008;22:1747-1752
29. Druker BJ. Imatinib as a paradigm of targeted therapies. Adv Cancer Res 2004;91:1-30
30. Abdollahi A, Li M, Ping G, et al. Inhibition of platelet-derived growth factor signaling attenuates pulmonary fibrosis. J Exp Med 2005;201: 925-935
31. 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;114:1308-1316
32. Yoshiji H, Noguchi R, Kuriyama S, et al. Imatinib mesylate (STI-571) attenuates liver fibrosis development in rats. Am J Physiol Gastrointest Liver Physiol 2005;288:G907-13
33. Wang S, Wilkes MC, Leof EB, et al. Imatinib mesylate blocks a non-Smad TGF-beta pathway and reduces renal fibrogenesis in vivo. Faseb J 2005;19:1-11
34. Distler JH, Jungel A, Huber LC, et al. Imatinib mesylate reduces production of extracellular matrix and prevents development of experimental dermal fibrosis. Arthritis Rheum 2007;56:311-322
35. Tidball JG, Spencer MJ, St Pierre BA. PDGF-receptor concentration is elevated in regenerative muscle fibers in dystrophin-deficient muscle. Exp Cell Res 1992;203:141-149
36. Zhao Y, Haginoya K, Sun G, et al. Platelet-derived growth factor and its receptors are related to the progression of human muscular dystrophy: An immunohistochemical study. J Pathol 2003;201:149-159
37. 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;264:106-111
38. Huang P, Zhao XS, Fields M, et al. Imatinib attenuates skeletal muscle dystrophy in mdx mice. FASEB J 2009;23:2539-2548
39. Bizario JC, Cerri DG, Rodrigues LC, et al. Imatinib mesylate ameliorates the dystrophic phenotype in exercised mdx mice. J Neuroimmunol 2009; 212:93-101
40. Spencer MJ, Montecino-Rodriguez E, Dorshkind K, et al. Helper (CD4(+)) and cytotoxic (CD8(+)) T cells promote the pathology of dystrophin-deficient muscle. Clin Immunol 2001;98:235-243
41. Wehling M, Spencer MJ, Tidball JG. A nitric oxide synthase transgene ameliorates muscular dystrophy in mdx mice. J Cell Biol 2001;155: 123-131
42. Hodgetts S, Radley H, Davies M, et al. 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
43. Cai B, Spencer MJ, Nakamura G, et al. Eosinophilia of dystrophindeficient muscle is promoted by perforin-mediated cytotoxicity by T cell effectors. Am J Pathol 2000;156:1789-1796
44. Gorospe JR, Tharp M, Demitsu T, et al. Dystrophin-deficient myofibers are vulnerable to mast cell granuleYinduced necrosis. Neuromuscul Disord 1994;4:325-333
45. Wynn TA. Fibrotic disease and the T(H)1/T(H)2 paradigm. Nat Rev Immunol 2004;4:583-594
46. Farini A, Meregalli M, Belicchi M, et al. T and B lymphocyte depletion has a marked effect on the fibrosis of dystrophic skeletal muscles in the scid/mdx mouse. J Pathol 2007;213:229-238
47. Morrison J, Lu QL, Pastoret C, et al. T-cellYdependent fibrosis in the mdx dystrophic mouse. Lab Invest 2000;80:881-891
48. Morrison J, Palmer DB, Cobbold S, et al. Effects of T-lymphocyte depletion on muscle fibrosis in the mdx mouse. Am J Pathol 2005;166: 1701-1710
49. Gordon S. Alternative activation of macrophages. Nat Rev Immunol 2003;3:23-35
50. Martinez FO, Helming L, Gordon S. Alternative activation of macrophages: An immunologic functional perspective. Annu Rev Immunol 2009;27:451-483
51. Pesce JT, Ramalingam TR, Mentink-Kane MM, et al. Arginase-1-expressing macrophages suppress TH2 cytokine-driven inflammation and fibrosis. PLoS Pathog 2009;5:e1000371
52. 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;172:1509-1519
53. Villalta SA, Nguyen HX, Deng B, et al. Shifts in macrophage phenotypes and macrophage competition for arginine metabolism affect the severity of muscle pathology in muscular dystrophy. Hum Mol Genet 2009;18: 482-496
54. Wehling-Henricks M, Sokolow S, Lee JJ, et al. Major basic protein-1 promotes fibrosis of dystrophic muscle and attenuates the cellular immune response in muscular dystrophy. Hum Mol Genet 2008;17: 2280-2292
55. Vetrone SA, Montecino-Rodriguez E, Kudryashova E, et al. Osteopontin promotes fibrosis in dystrophic mouse muscle by modulating immune cell subsets and intramuscular TGF-beta. J Clin Invest 2009;119:1583-1594
56. Barton ER, Morris L, Musaro A, et al. Muscle-specific expression of insulin-like growth factor I counters muscle decline in mdx mice. J Cell Biol 2002;157:137-148
57. Wagner KR, McPherron AC, Winik N, et al. Loss of myostatin attenuates severity of muscular dystrophy in mdx mice. Ann Neurol 2002;52: 832-836
58. Li ZB, Kollias HD, Wagner KR. Myostatin directly regulates skeletal muscle fibrosis. J Biol Chem 2008;283:19371-19378
59. Cordier L, Hack AA, Scott MO, et al. Rescue of skeletal muscles of gamma-sarcoglycanYdeficient mice with adeno-associated virus
60. Gargioli C, Coletta M, De Grandis F, et al. PlGF-MMP-9Yexpressing cells restore microcirculation and efficacy of cell therapy in aged dystrophic muscle. Nat Med 2008;14:973-978
61. Dupont-Versteegden EE, McCarter RJ. Differential expression of muscular dystrophy in diaphragm versus hindlimb muscles of mdx mice. Muscle Nerve 1992;15:1105-1110
62. Stedman HH, Sweeney HL, Shrager JB, et al. The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy. Nature 1991;352:536-539
63. Wagner KR, Fleckenstein JL, Amato AA, et al. A phase I/II trial of MYO-029 in adult subjects with muscular dystrophy. Ann Neurol 2008; 63:561-71