• 1
    Burns R (1786) To a mouse, on turning her up in her nest with the plough. In Poems, Chiefly in the Scottish Dialect, pp. 138140. John Wilson, Kilmarnock, UK.
  • 2
    Philip M, Benatar M, Fisher M & Savitz SI (2009) Methodological quality of animal studies of neuroprotective agents currently in phase II/III acute ischemic stroke trials. Stroke 40, 577581.
  • 3
    Benatar M (2007) Lost in translation: treatment trials in the SOD1 mouse and in human ALS. Neurobiol Dis 26, 113.
  • 4
    Landis SC, Amara SG, Asadullah K, Austin CP, Blumenstein R, Bradley EW, Crystal RG, Darnell RB, Ferrante RJ, Fillit H, et al. (2012) A call for transparent reporting to optimize the predictive value of preclinical research. Nature 490, 187191.
  • 5
    Prinz F, Schlange T & Asadullah K (2011) Believe it or not: how much can we rely on published data on potential drug targets? Nat Rev Drug Discov 10, 712.
  • 6
    Scott S, Kranz JE, Cole J, Lincecum JM, Thompson K, Kelly N, Bostrom A, Theodoss J, Al–Nakhala BM, Vieira FG, et al. (2008) Design, power, and interpretation of studies in the standard murine model of ALS. Amyotroph Lateral Scler 9, 415.
  • 7
    Bulfield G, Siller WG, Wight PA & Moore KJ (1984) X chromosome-linked muscular dystrophy (mdx) in the mouse. Proc Natl Acad Sci USA 81, 11891192.
  • 8
    Haldane JBS (1928) On being the right size. In Possible Worlds and Other Essays. Harper and Brothers, New York.
  • 9
    Thompson DW (1945) On Growth and Form. Cambridge University Press, New York.
  • 10
    Harrison BC, Allen DL & Leinwand LA (2011) IIb or not IIb? Regulation of myosin heavy chain gene expression in mice and men. Skelet Muscle 1, 5.
  • 11
    Rayavarapu S, Coley W, Cakir E, Jahnke V, Takeda S, Aoki Y, Gordish-Dressman H, Jaiswal JK, Hoffman EP, Brown KJ, et al. (2013) Identification of disease specific pathways using in vivo SILAC proteomics in dystrophin-deficient mdx mouse. Mol Cell Proteomics, doi:10.1074/mcp.M112.023127.
  • 12
    Liu JX, Hoglund AS, Karlsson P, Lindblad J, Qaisar R, Aare S, Bengtsson E & Larsson L (2009) Myonuclear domain size and myosin isoform expression in muscle fibres from mammals representing a 100,000-fold difference in body size. Exp Physiol 94, 117129.
  • 13
    Carlson BM (1973) The regeneration of skeletal muscle: a review. Am J Anat 137, 119150.
  • 14
    Lepper C, Partridge TA & Fan CM (2011) An absolute requirement for Pax7-positive satellite cells in acute injury-induced skeletal muscle regeneration. Development 138, 36393646.
  • 15
    Sambasivan R, Yao R, Kissenpfennig A, Van Wittenberghe L, Paldi A, Gayraud-Morel B, Guenou H, Malissen B, Tajbakhsh S & Galy A (2011) Pax7-expressing satellite cells are indispensable for adult skeletal muscle regeneration. Development 138, 36473656.
  • 16
    Murphy MM, Lawson JA, Mathew SJ, Hutcheson DA & Kardon G (2011) Satellite cells, connective tissue fibroblasts and their interactions are crucial for muscle regeneration. Development 138, 36253637.
  • 17
    Morgan JE, Hoffman EP & Partridge TA (1990) Normal myogenic cells from newborn mice restore normal histology to degenerating muscles of the mdx mouse. J Cell Biol 111, 24372449.
  • 18
    Skuk D, Goulet M, Roy B & Tremblay JP (2002) Efficacy of myoblast transplantation in nonhuman primates following simple intramuscular cell injections: toward defining strategies applicable to humans. Exp Neurol 175, 112126.
  • 19
    Skuk D & Tremblay JP (2003) Myoblast transplantation: the current status of a potential therapeutic tool for myopathies. J Muscle Res Cell Motil 24, 285300.
  • 20
    Alter J, Lou F, Rabinowitz A, Yin H, Rosenfeld J, Wilton SD, Partridge TA & Lu QL (2006) Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 12, 175177.
  • 21
    Yokota T, Lu QL, Partridge T, Kobayashi M, Nakamura A, Takeda S & Hoffman E (2009) Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs. Ann Neurol 65, 66766676.
  • 22
    Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, et al. (2011) Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet 378, 595605.
  • 23
    Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, Holling T, Janson AA, Platenburg GJ, Sipkens JA, et al. (2011) Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med 364, 15131522.
  • 24
    Odom GL, Gregorevic P, Allen JM, Finn E & Chamberlain JS (2008) Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice. Mol Ther 16, 15391545.
  • 25
    Lai Y, Thomas GD, Yue Y, Yang HT, Li D, Long C, Judge L, Bostick B, Chamberlain JS, Terjung RL, et al. (2009) Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy. J Clin Invest 119, 624635.
  • 26
    Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L & Danos O (2004) Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 306, 17961799.
  • 27
    Goyenvalle A, Babbs A, van Ommen GJ, Garcia L & Davies KE (2009) Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: promising tool for DMD therapy. Mol Ther 17, 12341240.
  • 28
    Denti MA, Rosa A, D'Antona G, Sthandier O, De Angelis FG, Nicoletti C, Allocca M, Pansarasa O, Parente V, Musaro A, et al. (2006) Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice. Hum Gene Ther 17, 565574.
  • 29
    Dellavalle A, Sampaolesi M, Tonlorenzi R, Tagliafico E, Sacchetti B, Perani L, Innocenzi A, Galvez BG, Messina G, Morosetti R, et al. (2007) Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells. Nat Cell Biol 9, 255267.
  • 30
    Sampaolesi M, Blot S, D'Antona G, Granger N, Tonlorenzi R, Innocenzi A, Mognol P, Thibaud JL, Galvez BG, Barthelemy I, et al. (2006) Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs. Nature 444, 574579.
  • 31
    Negroni E, Riederer I, Chaouch S, Belicchi M, Razini P, Di Santo J, Torrente Y, Butler-Browne GS & Mouly V (2009) In vivo myogenic potential of human CD133+ muscle-derived stem cells: a quantitative study. Mol Ther 17, 17711778.
  • 32
    Torrente Y, Belicchi M, Marchesi C, Dantona G, Cogiamanian F, Pisati F, Gavina M, Giordano R, Tonlorenzi R, Fagiolari G, et al. (2007) Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients. Cell Transplant 16, 563577.
  • 33
    Vieira NM, Brandalise V, Zucconi E, Secco M, Strauss BE & Zatz M (2009) Isolation, characterization, and differentiation potential of canine adipose-derived stem cells. Cell Transplant 19, 279289.
  • 34
    Rouger K, Larcher T, Dubreil L, Deschamps JY, Le Guiner C, Jouvion G, Delorme B, Lieubeau B, Carlus M, Fornasari B, et al. (2011) Systemic delivery of allogenic muscle stem cells induces long-term muscle repair and clinical efficacy in Duchenne muscular dystrophy dogs. Am J Pathol 179, 25012518.
  • 35
    Standring S, Collins P, Crossman AR, Gatzoulis MA, Healy JC, Johnson D, Mahedevan V, Newell RLM & Wigley C (2008) Cells tissues and systems – functional anatomy of the musculoskeletal system. In Gray's Anatomy, pp. 81126. Churchill Livingstone, Edinburgh, UK.
  • 36
    Pastoret C & Sebille A (1995) mdx mice show progressive weakness and muscle deterioration with age. J Neurol Sci 129, 97105.
  • 37
    Cheek DB, Graystone JE & Read MS (1970) Cellular growth, nutrition and development. Pediatrics 45, 315334.
  • 38
    White RB, Bierinx AS, Gnocchi VF & Zammit PS (2010) Dynamics of muscle fibre growth during postnatal mouse development. BMC Dev Biol 10, 21.
  • 39
    Lepper C, Conway SJ & Fan CM (2009) Adult satellite cells and embryonic muscle progenitors have distinct genetic requirements. Nature 460, 627631.
  • 40
    Moss FP & Leblond CP (1970) Nature of dividing nuclei in skeletal muscle of growing rats. J Cell Biol 44, 459462.
  • 41
    Moss FP & Leblond CP (1971) Satellite cells as the source of nuclei in muscles of growing rats. Anat Rec 170, 421435.
  • 42
    Cheek DB (1985) The control of cell mass and replication. The DNA unit – a personal 20-year study. Early Hum Dev 12, 211239.
  • 43
    Renault V, Piron-Hamelin G, Forestier C, DiDonna S, Decary S, Hentati F, Saillant G, Butler-Browne GS & Mouly V (2000) Skeletal muscle regeneration and the mitotic clock. Exp Gerontol 35, 711719.
  • 44
    Cheek DB (1977) Human growth – methods of approach. Aust Paediatr J 13, 7787.
  • 45
    McGeachie JK & Grounds MD (1987) Initiation and duration of muscle precursor replication after mild and severe injury to skeletal muscle. Cell Tissue Res 248, 125130.
  • 46
    Grounds MD & Shavlakadze T (2011) Growing muscle has different sarcolemmal properties from adult muscle: a proposal with scientific and clinical implications. Reasons to reassess skeletal muscle molecular dynamics, cellular responses and suitability of experimental models of muscle disorders. BioEssays 33, 458468.
  • 47
    Porter JD, Khanna S, Kaminski HJ, Rao JS, Merriam AP, Richmonds CR, Leahy P, Li J, Guo W & Andrade FH (2002) A chronic inflammatory response dominates the skeletal muscle molecular signature in dystrophin-deficient mdx mice. Hum Mol Genet 11, 263272.
  • 48
    Hirata A, Masuda S, Tamura T, Kai K, Ojima K, Fukase A, Motoyoshi K, Kamakura K, Miyagoe-Suzuki Y & Takeda S (2003) Expression profiling of cytokines and related genes in regenerating skeletal muscle after cardiotoxin injection: a role for osteopontin. Am J Pathol 163, 203215.
  • 49
    Vetrone SA, Montecino-Rodriguez E, Kudryashova E, Kramerova I, Hoffman EP, Liu SD, Miceli MC & Spencer MJ (2009) Osteopontin promotes fibrosis in dystrophic mouse muscle by modulating immune cell subsets and intramuscular TGF–β. J Clin Invest 119, 15831594.
  • 50
    Uaesoontrachoon K, Yoo HJ, Tudor EM, Pike RN, Mackie EJ & Pagel CN (2008) Osteopontin and skeletal muscle myoblasts: association with muscle regeneration and regulation of myoblast function in vitro. Int J Biochem Cell Biol 40, 23032314.
  • 51
    Hoffman EP, Gordish-Dressman H, McLane VD, Devaney JM, Thompson PD, Visich P, Gordon PM, Pescatello LS, Zoeller RF, Moyna NM, et al. (2013) Alterations in osteopontin modify muscle size in females in both humans and mice. Med Sci Sports Exerc, in press.
  • 52
    Shinohara ML, Kim HJ, Kim JH, Garcia VA & Cantor H (2008) Alternative translation of osteopontin generates intracellular and secreted isoforms that mediate distinct biological activities in dendritic cells. Proc Natl Acad Sci USA 105, 72357239.
  • 53
    Chae S, Jun HO, Lee EG, Yang SJ, Lee DC, Jung JK, Park KC, Yeom YI & Kim KW (2009) Osteopontin splice variants differentially modulate the migratory activity of hepatocellular carcinoma cell lines. Int J Oncol 35, 14091416.
  • 54
    Tang X, Li J, Yu B, Su L, Yu Y, Yan M, Liu B & Zhu Z (2013) Osteopontin splice variants differentially exert clinicopathological features and biological functions in gastric cancer. Int J Biol Sci 9, 5566.
  • 55
    Grau JB, Poggio P, Sainger R, Vernick WJ, Seefried WF, Branchetti E, Field BC, Bavaria JE, Acker MA & Ferrari G (2012) Analysis of osteopontin levels for the identification of asymptomatic patients with calcific aortic valve disease. Ann Thorac Surg 93, 7986.
  • 56
    Weidle UH, Maisel D, Klostermann S, Weiss EH & Schmitt M (2011) Differential splicing generates new transmembrane receptor and extracellular matrix-related targets for antibody-based therapy of cancer. Cancer Genomics Proteomics 8, 211226.
  • 57
    Cooper BJ, Winand NJ, Stedman H, Valentine BA, Hoffman EP, Kunkel LM, Scott MO, Fischbeck KH, Kornegay JN, Avery RJ, et al. (1988) The homologue of the Duchenne locus is defective in X–linked muscular dystrophy of dogs. Nature 334, 154156.
  • 58
    Sharp NJ, Kornegay JN, Van Camp SD, Herbstreith MH, Secore SL, Kettle S, Hung WY, Constantinou CD, Dykstra MJ, Roses AD, et al. (1992) An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy. Genomics 13, 115121.
  • 59
    Kornegay JN, Bogan JR, Bogan DJ, Childers MK & Grange RW (2011) Golden retriever muscular dystrophy (GRMD): developing and maintaining a colony and physiological functional measurements. Methods Mol Biol 709, 105123.
  • 60
    Miyazato LG, Moraes JR, Beretta DC & Kornegay JN (2011) Muscular dystrophy in dogs: does the crossing of breeds influence disease phenotype? Vet Pathol 48, 655662.
  • 61
    Yokota T, Nakamura A, Nagata T, Saito T, Kobayashi M, Aoki Y, Echigoya Y, Partridge T, Hoffman EP & Takeda S (2012) Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs. Nucleic Acid Ther 22, 306315.
  • 62
    Partridge TA, Morgan JE, Coulton GR, Hoffman EP & Kunkel LM (1989) Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts. Nature 337, 176179.
  • 63
    Morrison J, Partridge T & Bou-Gharios G (2005) Nude mutation influences limb skeletal muscle development. Matrix Biol 23, 535542.
  • 64
    Deconinck AE, Rafael JA, Skinner JA, Brown SC, Potter AC, Metzinger L, Watt DJ, Dickson JG, Tinsley JM & Davies KE (1997) Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy. Cell 90, 717727.
  • 65
    Ward CL & Davies KE (1998) Genetic modelling of muscular dystrophies. Neuropathol Appl Neurobiol 24, 96100.
  • 66
    Wakefield PM, Tinsley JM, Wood MJ, Gilbert R, Karpati G & Davies KE (2000) Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene. Gene Ther 7, 201204.
  • 67
    Decary S, Hamida CB, Mouly V, Barbet JP, Hentati F & Butler-Browne GS (2000) Shorter telomeres in dystrophic muscle consistent with extensive regeneration in young children. Neuromuscul Disord 10, 113120.
  • 68
    Decary S, Mouly V, Hamida CB, Sautet A, Barbet JP & Butler-Browne GS (1997) Replicative potential and telomere length in human skeletal muscle: implications for satellite cell-mediated gene therapy. Hum Gene Ther 8, 14291438.
  • 69
    Decary S, Mouly V & Butler-Browne GS (1996) Telomere length as a tool to monitor satellite cell amplification for cell-mediated gene therapy. Hum Gene Ther 7, 13471350.
  • 70
    Sacco A, Mourkioti F, Tran R, Choi J, Llewellyn M, Kraft P, Shkreli M, Delp S, Pomerantz JH, Artandi SE, et al. (2010) Short telomeres and stem cell exhaustion model Duchenne muscular dystrophy in mdx/mTR mice. Cell 143, 10591071.
  • 71
    Megeney LA, Kablar B, Perry RL, Ying C, May L & Rudnicki MA (1999) Severe cardiomyopathy in mice lacking dystrophin and MyoD. Proc Natl Acad Sci USA 96, 220225.
  • 72
    Aoki Y, Nakamura A, Yokota T, Saito T, Okazawa H, Nagata T & Takeda S (2010) In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse. Mol Ther 18, 19952005.
  • 73
    Aoki Y, Yokota T, Nagata T, Nakamura A, Tanihata J, Saito T, Duguez SM, Nagaraju K, Hoffman EP, Partridge T, et al. (2012) Bodywide skipping of exons 45–55 in dystrophic mdx52 mice by systemic antisense delivery. Proc Natl Acad Sci USA 109, 1376313768.
  • 74
    Willmann R, De Luca A, Benatar M, Grounds M, Dubach J, Raymackers JM & Nagaraju K (2012) Enhancing translation: guidelines for standard pre-clinical experiments in mdx mice. Neuromuscul Disord 22, 4349.