inbred mdx mice

Summary

Summary: A strain of mice arising from a spontaneous MUTATION (mdx) in inbred C57BL mice. This mutation is X chromosome-linked and produces viable homozygous animals that lack the muscle protein DYSTROPHIN, have high serum levels of muscle ENZYMES, and possess histological lesions similar to human MUSCULAR DYSTROPHY. The histological features, linkage, and map position of mdx make these mice a worthy animal model of DUCHENNE MUSCULAR DYSTROPHY.

Top Publications

  1. Barton E, Morris L, Kawana M, Bish L, Toursel T. Systemic administration of L-arginine benefits mdx skeletal muscle function. Muscle Nerve. 2005;32:751-60 pubmed
    ..Together, these results show that L-arginine treatment can be beneficial to mdx muscle function, perhaps through a combination of enhanced calcium handling and increased utrophin, thereby decreasing muscle degeneration...
  2. Van Erp C, Irwin N, Hoey A. Long-term administration of pirfenidone improves cardiac function in mdx mice. Muscle Nerve. 2006;34:327-34 pubmed
    ..These results show that the TGF-beta antagonist, pirfenidone, can improve cardiac function in mdx mice, potentially providing a new avenue for developing cardiac therapies for patients with Duchenne muscular dystrophy...
  3. Jearawiriyapaisarn N, Moulton H, Buckley B, Roberts J, Sazani P, Fucharoen S, et al. Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. Mol Ther. 2008;16:1624-9 pubmed publisher
    ..This is the first report of oligonucleotide-mediated exon skipping and dystrophin protein induction in the heart of treated animals. ..
  4. Percival J, Gregorevic P, Odom G, Banks G, Chamberlain J, Froehner S. rAAV6-microdystrophin rescues aberrant Golgi complex organization in mdx skeletal muscles. Traffic. 2007;8:1424-39 pubmed
    ..In summary, GC distribution abnormalities are a novel component of mdx skeletal muscle pathology rescued by microdystrophin expression. ..
  5. Matsumura C, Pertille A, Albuquerque T, Santo Neto H, Marques M. Diltiazem and verapamil protect dystrophin-deficient muscle fibers of MDX mice from degeneration: a potential role in calcium buffering and sarcolemmal stability. Muscle Nerve. 2009;39:167-76 pubmed publisher
    ..Possible interactions of these drugs with the sarcoplasmic reticulum and sarcolemma may also contribute to the improvement of the dystrophic phenotype. ..
  6. Nunes V, Cavaçana N, Canovas M, Strauss B, Zatz M. Stem cells from umbilical cord blood differentiate into myotubes and express dystrophin in vitro only after exposure to in vivo muscle environment. Biol Cell. 2007;99:185-96 pubmed
    ..Nevertheless, we showed that the HUCB-derived stem cells were capable of acquiring a muscle phenotype after exposure to an in vivo muscle environment, which was required to activate the differentiation programme. ..
  7. Bani C, Lagrota Candido J, Pinheiro D, Leite P, Salimena M, Henriques Pons A, et al. Pattern of metalloprotease activity and myofiber regeneration in skeletal muscles of mdx mice. Muscle Nerve. 2008;37:583-92 pubmed publisher
  8. Minetti G, Colussi C, Adami R, Serra C, Mozzetta C, Parente V, et al. Functional and morphological recovery of dystrophic muscles in mice treated with deacetylase inhibitors. Nat Med. 2006;12:1147-50 pubmed
    ..These results provide a rationale for using deacetylase inhibitors in the pharmacological therapy of muscular dystrophies...
  9. Williams I, Allen D. The role of reactive oxygen species in the hearts of dystrophin-deficient mdx mice. Am J Physiol Heart Circ Physiol. 2007;293:H1969-77 pubmed
    ..Therapies designed to reduce oxidative damage might be beneficial to DMD patients with heart failure. ..

More Information

Publications71

  1. Wang Z, Allen J, Riddell S, Gregorevic P, Storb R, Tapscott S, et al. Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy. Hum Gene Ther. 2007;18:18-26 pubmed
    ..Our data indicate that AAV2 and AAV6 capsid proteins can elicit primary cellular immune responses when injected into the skeletal muscle of random-bred dogs, and suggest the possibility of cellular immunity to AAV vectors in humans. ..
  2. Whitehead N, Streamer M, Lusambili L, Sachs F, Allen D. Streptomycin reduces stretch-induced membrane permeability in muscles from mdx mice. Neuromuscul Disord. 2006;16:845-54 pubmed
    ..The results suggest that Ca(2+) entry through SACs activates Ca(2+) -dependent pathways, which are the main cause of the increased membrane permeability in mdx muscle. ..
  3. Puttini S, Lekka M, Dorchies O, Saugy D, Incitti T, Ruegg U, et al. Gene-mediated restoration of normal myofiber elasticity in dystrophic muscles. Mol Ther. 2009;17:19-25 pubmed publisher
    ..AFM may thus provide a quantification of the functional benefit of gene therapies from live tissues coupled to single-cell resolution...
  4. Messina S, Bitto A, Aguennouz M, Minutoli L, Monici M, Altavilla D, et al. Nuclear factor kappa-B blockade reduces skeletal muscle degeneration and enhances muscle function in Mdx mice. Exp Neurol. 2006;198:234-41 pubmed
    ..Most importantly, these new findings may have clinical implications for the pharmacological treatment of patients with DMD...
  5. 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 pubmed
    ..It also brought the response to eccentric contraction-induced injury to the normal range. In summary, our results suggest that the DeltaR4/DeltaC microgene holds great promise in preventing muscular dystrophy...
  6. Chakkalakal J, Michel S, Chin E, Michel R, Jasmin B. Targeted inhibition of Ca2+ /calmodulin signaling exacerbates the dystrophic phenotype in mdx mouse muscle. Hum Mol Genet. 2006;15:1423-35 pubmed
    ..Finally, our results further support the concept that strategies aimed at promoting the slow oxidative myofiber program in muscle may be effective in altering the relentless progression of DMD...
  7. Malerba A, Thorogood F, Dickson G, Graham I. Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice. Hum Gene Ther. 2009;20:955-65 pubmed publisher
    ..These results clearly demonstrate the key role of the optimization of dosing regimen for the systemic administration of PMO in patients, and support the clinical feasibility of this approach with naked PMO...
  8. Hodgetts S, Radley H, Davies M, Grounds M. 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 pubmed
    ..Etanercept is a highly specific anti-inflammatory drug, widely used clinically, and potential application to muscular dystrophies is suggested by this reduced breakdown of mdx skeletal muscle...
  9. Sirsi S, Williams J, Lutz G. Poly(ethylene imine)-poly(ethylene glycol) copolymers facilitate efficient delivery of antisense oligonucleotides to nuclei of mature muscle cells of mdx mice. Hum Gene Ther. 2005;16:1307-17 pubmed
    ..Dose-response analysis indicated saturation of endocytotic uptake of the polyplex. Overall, we conclude that PEG-PEI copolymers represent high-capacity, nontoxic carriers for efficient delivery of AO to nuclei of mature myofibers...
  10. Hnia K, Gayraud J, Hugon G, Ramonatxo M, de la Porte S, Matecki S, 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 pubmed publisher
  11. Heemskerk H, De Winter C, De Kimpe S, van Kuik Romeijn P, Heuvelmans N, Platenburg G, et al. In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping. J Gene Med. 2009;11:257-66 pubmed publisher
    ..In vivo studies are mainly performed using 2'-O-methyl phosphorothioate (2OMePS) or morpholino (PMO) AONs. These compounds were never directly compared...
  12. Radley H, De Luca A, Lynch G, Grounds M. Duchenne muscular dystrophy: focus on pharmaceutical and nutritional interventions. Int J Biochem Cell Biol. 2007;39:469-77 pubmed
  13. Banks G, Chamberlain J. The value of mammalian models for duchenne muscular dystrophy in developing therapeutic strategies. Curr Top Dev Biol. 2008;84:431-53 pubmed publisher
    ..Although each mammalian model has its limitations, together they have been essential for the development of several treatment strategies for DMD that target dystrophin replacement, disease progression, and muscle regeneration...
  14. Keeling R, Golumbek P, Streif E, Connolly A. Weekly oral prednisolone improves survival and strength in male mdx mice. Muscle Nerve. 2007;35:43-8 pubmed
    ..As corticosteroids remain the most validated long-term treatment of DMD, this work may allow for better prediction of synergistic treatments likely to translate to effective improvement for boys with this progressive muscular dystrophy...
  15. Peter A, Crosbie R. Hypertrophic response of Duchenne and limb-girdle muscular dystrophies is associated with activation of Akt pathway. Exp Cell Res. 2006;312:2580-91 pubmed
    ..Current investigations are focused on introducing constitutively active and dominant-negative Akt into prenecrotic mdx mice to determine how early modification of Akt activity influences disease pathogenesis...
  16. Yasuda S, Townsend D, Michele D, Favre E, Day S, Metzger J. Dystrophic heart failure blocked by membrane sealant poloxamer. Nature. 2005;436:1025-9 pubmed
  17. Darabi R, Gehlbach K, Bachoo R, Kamath S, Osawa M, Kamm K, et al. Functional skeletal muscle regeneration from differentiating embryonic stem cells. Nat Med. 2008;14:134-43 pubmed publisher
    ..These data demonstrate the therapeutic potential of ES cells in muscular dystrophy...
  18. Baban D, Davies K. Microarray analysis of mdx mice expressing high levels of utrophin: therapeutic implications for dystrophin deficiency. Neuromuscul Disord. 2008;18:239-47 pubmed publisher
    ..This study confirms that a strategy to up-regulate utrophin is likely to be beneficial in dystrophin deficiency...
  19. Colussi C, Gaetano C, Capogrossi M. AAV-dependent targeting of myostatin function: follistatin strikes back at muscular dystrophy. Gene Ther. 2008;15:1075-6 pubmed publisher
  20. Voisin V, Sebrie C, Matecki S, Yu H, Gillet B, Ramonatxo M, et al. L-arginine improves dystrophic phenotype in mdx mice. Neurobiol Dis. 2005;20:123-30 pubmed
    ..Our results suggest that pharmacological activators of the NO pathway may constitute a realistic treatment for Duchenne and Becker muscular dystrophies...
  21. Messina S, Altavilla D, Aguennouz M, Seminara P, Minutoli L, Monici 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 pubmed
    ..Most importantly, these new findings may have clinical implications for the pharmacological treatment of patients with DMD...
  22. Spurney C, Knoblach S, Pistilli E, Nagaraju K, Martin G, Hoffman E. Dystrophin-deficient cardiomyopathy in mouse: expression of Nox4 and Lox are associated with fibrosis and altered functional parameters in the heart. Neuromuscul Disord. 2008;18:371-81 pubmed publisher
    ..This is the first report of an altered profibrotic gene expression profile in cardiac tissue of dystrophic mice showing echocardiographic evidence of cardiomyopathy...
  23. Friedrich O, von Wegner F, Chamberlain J, Fink R, Rohrbach P. L-type Ca2+ channel function is linked to dystrophin expression in mammalian muscle. PLoS ONE. 2008;3:e1762 pubmed publisher
    ..g. impaired L-type Ca2+ currents. In regenerating mdx muscle, 'revertant' fibres restore dystrophin expression. Their functionality involving DHPR-Ca2+-channels is elusive...
  24. Vidal B, Serrano A, Tjwa M, Suelves M, Ardite E, De Mori R, et al. Fibrinogen drives dystrophic muscle fibrosis via a TGFbeta/alternative macrophage activation pathway. Genes Dev. 2008;22:1747-52 pubmed publisher
    ..Fibrinogen, by engaging its alphavbeta3 receptor on fibroblasts, also directly promotes collagen synthesis. These data unveil a profibrotic role of fibrinogen deposition in muscle dystrophy...
  25. Odom G, Gregorevic P, Allen J, Finn E, Chamberlain J. Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice. Mol Ther. 2008;16:1539-45 pubmed publisher
    ..This approach may hold promise as a treatment option for DMD because it avoids the potential immune responses that are associated with the delivery of exogenous dystrophin...
  26. Zhang W, Ten Hove M, Schneider J, Stuckey D, Sebag Montefiore L, Bia B, et al. Abnormal cardiac morphology, function and energy metabolism in the dystrophic mdx mouse: an MRI and MRS study. J Mol Cell Cardiol. 2008;45:754-60 pubmed publisher
    ..We conclude that the absence of dystrophin in adult mdx mouse heart, as in the heart of human patient, is associated with right ventricular dilatation, left ventricular diastolic dysfunction and abnormal energy metabolism...
  27. Chan J, Waddington S, O Donoghue K, Kurata H, Guillot P, Gotherstrom C, et al. Widespread distribution and muscle differentiation of human fetal mesenchymal stem cells after intrauterine transplantation in dystrophic mdx mouse. Stem Cells. 2007;25:875-84 pubmed
    ..Although the low-level of chimerism achieved is not curative for DMD, this approach may be useful in other severe mesenchymal or enzyme deficiency syndromes, where low-level protein expression may ameliorate disease pathology...
  28. Qiao C, Li J, Jiang J, Zhu X, Wang B, Li J, et al. Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice. Hum Gene Ther. 2008;19:241-54 pubmed publisher
    ..These results clearly demonstrate the efficacy of AAV8-mediated myostatin propeptide gene delivery in a rodent model of DMD, and warrant further investigation in large animal models and eventually in human patients...
  29. Montarras D, Morgan J, Collins C, Relaix F, Zaffran S, Cumano A, et al. Direct isolation of satellite cells for skeletal muscle regeneration. Science. 2005;309:2064-7 pubmed
    ..Such cells, grafted into muscles of mdx nu/nu mice, contributed both to fiber repair and to the muscle satellite cell compartment. Expansion of these cells in culture before engraftment reduced their regenerative capacity...
  30. Cui C, Uyama T, Miyado K, Terai M, Kyo S, Kiyono T, et al. Menstrual blood-derived cells confer human dystrophin expression in the murine model of Duchenne muscular dystrophy via cell fusion and myogenic transdifferentiation. Mol Biol Cell. 2007;18:1586-94 pubmed
    ..These results demonstrate that the endometrial progenitor cells and menstrual blood-derived cells can transfer dystrophin into dystrophied myocytes through cell fusion and transdifferentiation in vitro and in vivo...
  31. Personius K, Sawyer R. Terminal Schwann cell structure is altered in diaphragm of mdx mice. Muscle Nerve. 2005;32:656-63 pubmed
  32. Lansman J, Franco Obregón A. Mechanosensitive ion channels in skeletal muscle: a link in the membrane pathology of muscular dystrophy. Clin Exp Pharmacol Physiol. 2006;33:649-56 pubmed
  33. Ito K, Kimura S, Ozasa S, Matsukura M, Ikezawa M, Yoshioka K, et al. Smooth muscle-specific dystrophin expression improves aberrant vasoregulation in mdx mice. Hum Mol Genet. 2006;15:2266-75 pubmed
    ..In addition, the elevated serum creatine kinase levels observed in mdx mice were significantly reduced in SMTg/mdx mice. This is the first report of a functional role of dystrophin in vascular smooth muscle...
  34. Woods C, Novo D, DiFranco M, Capote J, Vergara J. Propagation in the transverse tubular system and voltage dependence of calcium release in normal and mdx mouse muscle fibres. J Physiol. 2005;568:867-80 pubmed
    ..Taken together, our data suggest that the intrinsic ability of the sarcoplasmic reticulum to release Ca2+ may be altered in the mdx mouse...
  35. Williams I, Allen D. Intracellular calcium handling in ventricular myocytes from mdx mice. Am J Physiol Heart Circ Physiol. 2007;292:H846-55 pubmed
    ..Early in the disease process and before the onset of clinical symptoms increased, SAC activity may underlie the abnormal Ca(2+) handling in young mdx mice...
  36. Spurney C, Gordish Dressman H, Guerron A, Sali A, Pandey G, Rawat R, et al. Preclinical drug trials in the mdx mouse: assessment of reliable and sensitive outcome measures. Muscle Nerve. 2009;39:591-602 pubmed publisher
    ..These data provide an experimental basis upon which standardization of preclinical drug testing can be developed. Muscle Nerve, 2008...
  37. Adams M, Tesch Y, Percival J, Albrecht D, Conhaim J, Anderson K, et al. Differential targeting of nNOS and AQP4 to dystrophin-deficient sarcolemma by membrane-directed alpha-dystrobrevin. J Cell Sci. 2008;121:48-54 pubmed
    ..Thus, although nNOS and AQP4 both require interaction with the PDZ domain of alpha-syntrophin for sarcolemmal association, their localization is regulated differentially...
  38. Ferlini A, Sabatelli P, Fabris M, Bassi E, Falzarano S, Vattemi G, et al. Dystrophin restoration in skeletal, heart and skin arrector pili smooth muscle of mdx mice by ZM2 NP-AON complexes. Gene Ther. 2010;17:432-8 pubmed publisher
  39. Li H, Mittal A, Makonchuk D, 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 pubmed publisher
    ..Collectively, our study suggests that the increased production of MMP-9 exacerbates dystrophinopathy and MMP-9 represents as one of the most promising therapeutic targets for the prevention of disease progression in DMD...
  40. Wehling Henricks M, Sokolow S, Lee J, Myung K, Villalta S, Tidball J. Major basic protein-1 promotes fibrosis of dystrophic muscle and attenuates the cellular immune response in muscular dystrophy. Hum Mol Genet. 2008;17:2280-92 pubmed publisher
  41. Boittin F, Petermann O, Hirn C, Mittaud P, Dorchies O, Roulet E, et al. Ca2+-independent phospholipase A2 enhances store-operated Ca2+ entry in dystrophic skeletal muscle fibers. J Cell Sci. 2006;119:3733-42 pubmed
    ..The Ca2+-independent phospholipase A2 pathway therefore appears as an attractive target to reduce excessive Ca2+ influx and subsequent degeneration occurring in dystrophic fibers...
  42. Yin H, Lu Q, Wood M. Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice. Mol Ther. 2008;16:38-45 pubmed
    ..Our results demonstrate that PNAs have a higher efficiency of exon skipping than 2'O methyl phosphorothioate AOs do, and have a potential use in AO chemistry for antisense therapy of DMD...
  43. Williams J, Sirsi S, Latta D, Lutz G. Induction of dystrophin expression by exon skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG-PEI copolymers. Mol Ther. 2006;14:88-96 pubmed
    ..We conclude that low Mw PEI2000(PEG550) copolymers function as high-capacity, nontoxic AO carriers suitable for in vivo transfection of skeletal muscle and are promising compounds for potential use in molecular therapy of DMD...
  44. Han R, Grounds M, Bakker A. Measurement of sub-membrane [Ca2+] in adult myofibers and cytosolic [Ca2+] in myotubes from normal and mdx mice using the Ca2+ indicator FFP-18. Cell Calcium. 2006;40:299-307 pubmed
    ..This study suggests that the sub-sarcolemmal Ca(2+) homeostasis is well maintained in isolated adult mdx myofibers and also further supports the hypothesis that cytosolic Ca(2+) handling is compromised in mdx myotubes...
  45. Fletcher S, Honeyman K, Fall A, Harding P, Johnsen R, Wilton S. Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide. J Gene Med. 2006;8:207-16 pubmed
    ..The antisense oligonucleotide chemistry most widely used to alter pre-mRNA processing is 2'-O-methyl-modified bases on a phosphorothioate backbone...
  46. Rooney J, Gurpur P, Burkin D. 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 pubmed publisher
    ..These findings demonstrate that laminin-111 is a highly potent therapeutic agent for the mdx mouse model of DMD and represents a paradigm for the systemic delivery of extracellular matrix proteins as therapies for genetic diseases...
  47. Wolff A, Niday A, Voelker K, Call J, Evans N, Granata K, et al. Passive mechanical properties of maturing extensor digitorum longus are not affected by lack of dystrophin. Muscle Nerve. 2006;34:304-12 pubmed
    ..Determining this threshold may have important clinical implications for treatments of muscular dystrophy involving physical activity...
  48. Lowe D, Williams B, Thomas D, Grange R. Molecular and cellular contractile dysfunction of dystrophic muscle from young mice. Muscle Nerve. 2006;34:92-100 pubmed
    ..Elucidating the molecular mechanisms underlying muscle weakness at the onset of disease is important for designing treatment strategies...
  49. Suchyna T, Sachs F. Mechanosensitive channel properties and membrane mechanics in mouse dystrophic myotubes. J Physiol. 2007;581:369-87 pubmed
    ..Spontaneous Ca2+ transients in mdx mouse cells are sensitive to depolarization and are inhibited by the specific MSC inhibitor GsMTx4, in both the D and L forms...
  50. Fletcher S, Honeyman K, Fall A, Harding P, Johnsen R, Steinhaus J, et al. Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse. Mol Ther. 2007;15:1587-92 pubmed
    ..We demonstrate dystrophin expression and near-normal muscle architecture in all muscles examined, except for cardiac muscle. The CPP greatly enhanced uptake of the PMO, resulting in widespread dystrophin expression...
  51. Barton E. Impact of sarcoglycan complex on mechanical signal transduction in murine skeletal muscle. Am J Physiol Cell Physiol. 2006;290:C411-9 pubmed
    ..This study provides evidence that the SGs are involved in the transduction of mechanical information in skeletal muscle, potentially unique from the entire DGC...
  52. Archer J, Vargas C, Anderson J. Persistent and improved functional gain in mdx dystrophic mice after treatment with L-arginine and deflazacort. FASEB J. 2006;20:738-40 pubmed
    ..Results suggest a potential new treatment option for improving the quality of life for boys with DMD...
  53. Welch E, Barton E, Zhuo J, Tomizawa Y, Friesen W, Trifillis P, et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature. 2007;447:87-91 pubmed
  54. Buyse G, Van der Mieren G, Erb M, D hooge J, Herijgers P, Verbeken E, 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 pubmed publisher
    ..We have performed such a trial of SNT-MC17/idebenone in the mdx mouse model of DMD, based on the drug's potential to improve mitochondrial respiratory chain function and reduce oxidative stress...
  55. Cohn R, Liang H, Shetty R, Abraham T, Wagner K. Myostatin does not regulate cardiac hypertrophy or fibrosis. Neuromuscul Disord. 2007;17:290-6 pubmed
    ..2% vs. 12%). The physiological role of myostatin in cardiac muscle appears significantly different than that in skeletal muscle as it does not induce cardiac hypertrophy and does not modulate cardiac fibrosis in mdx mice...
  56. Denti M, Rosa A, D Antona G, Sthandier O, De Angelis F, Nicoletti C, et al. Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model. Proc Natl Acad Sci U S A. 2006;103:3758-63 pubmed
    ..This approach provides solid bases for a systemic use of AAV-mediated antisense-U1 small nuclear RNA expression for the therapeutic treatment of Duchenne muscular dystrophy...
  57. Haenggi T, Fritschy J. Role of dystrophin and utrophin for assembly and function of the dystrophin glycoprotein complex in non-muscle tissue. Cell Mol Life Sci. 2006;63:1614-31 pubmed
    ..Here, we focus on recent studies of the DGC in brain, blood-brain barrier and choroid plexus, retina, and kidney and discuss the role of dystrophin isoforms and utrophin for assembly of the complex in these tissues...
  58. Yablonka Reuveni Z, Anderson J. Satellite cells from dystrophic (mdx) mice display accelerated differentiation in primary cultures and in isolated myofibers. Dev Dyn. 2006;235:203-12 pubmed
    ..Furthermore, the study demonstrated that FDB myofibers are an excellent model of the in vivo state of muscle, as they accurately represented the dystrophic phenotype...
  59. Turk R, Sterrenburg E, de Meijer E, van Ommen G, den Dunnen J, t Hoen P. Muscle regeneration in dystrophin-deficient mdx mice studied by gene expression profiling. BMC Genomics. 2005;6:98 pubmed
    ..To characterize the molecular processes associated with regeneration, we compared gene expression levels in hindlimb muscle tissue of mdx and control mice at 9 timepoints, ranging from 1-20 weeks of age...
  60. Alter J, Lou F, Rabinowitz A, Yin H, Rosenfeld J, Wilton S, et al. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med. 2006;12:175-7 pubmed
    ..Although the level of dystrophin expression achieved varies considerably between muscles, antisense therapy may provide a realistic hope for the treatment of a majority of individuals with DMD...
  61. Hirn C, Shapovalov G, Petermann O, Roulet E, Ruegg U. Nav1.4 deregulation in dystrophic skeletal muscle leads to Na+ overload and enhanced cell death. J Gen Physiol. 2008;132:199-208 pubmed publisher
    ..4 gating properties and increased Na(+) concentrations are strongly correlated with increased cell death in mdx fibers and that both cell death and Na(+) overload can be reversed by 3 nM tetrodotoxin, a specific Na(v)1.4 blocker...
  62. Yokota T, Lu Q, Morgan J, Davies K, Fisher R, Takeda S, et al. Expansion of revertant fibers in dystrophic mdx muscles reflects activity of muscle precursor cells and serves as an index of muscle regeneration. J Cell Sci. 2006;119:2679-87 pubmed
    ..This expansion of revertant clusters depicts the cumulative history of regeneration, thus providing a useful index for functional evaluation of therapies that counteract muscle degeneration...