Animal models of muscular dystrophy.

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Altered Tnnt3 characterizes selective weakness of fast fibers in mice overexpressing FSHD region gene 1 (FRG1). Am J Physiol Regul Integr Comp Physiol (2013) 0.82

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Removing the immune response from muscular dystrophy research. Mol Ther (2013) 0.75

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A gene related to Caenorhabditis elegans spermatogenesis factor fer-1 is mutated in limb-girdle muscular dystrophy type 2B. Nat Genet (1998) 3.76

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Mutations in the laminin alpha 2-chain gene (LAMA2) cause merosin-deficient congenital muscular dystrophy. Nat Genet (1995) 3.05

Dysferlin interacts with annexins A1 and A2 and mediates sarcolemmal wound-healing. J Biol Chem (2003) 3.00

Increased protein degradation results from elevated free calcium levels found in muscle from mdx mice. Nature (1988) 2.97

The DUX4 gene at the FSHD1A locus encodes a pro-apoptotic protein. Neuromuscul Disord (2007) 2.85

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Disruption of muscle architecture and myocardial degeneration in mice lacking desmin. J Cell Biol (1996) 2.44

Short telomeres and stem cell exhaustion model Duchenne muscular dystrophy in mdx/mTR mice. Cell (2010) 2.43

Facioscapulohumeral muscular dystrophy in mice overexpressing FRG1. Nature (2005) 2.42

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Collagen VI deficiency induces early onset myopathy in the mouse: an animal model for Bethlem myopathy. Hum Mol Genet (1998) 2.30

A common nonsense mutation results in alpha-actinin-3 deficiency in the general population. Nat Genet (1999) 2.27

Dystrophin-deficient mdx mice display a reduced life span and are susceptible to spontaneous rhabdomyosarcoma. FASEB J (2007) 2.22

Autosomal recessive limb-girdle muscular dystrophy, LGMD2F, is caused by a mutation in the delta-sarcoglycan gene. Nat Genet (1996) 2.22

Murine muscular dystrophy caused by a mutation in the laminin alpha 2 (Lama2) gene. Nat Genet (1994) 2.19

Involvement of TRPC in the abnormal calcium influx observed in dystrophic (mdx) mouse skeletal muscle fibers. J Cell Biol (2002) 2.14

Muscle development in mdx mutant mice. Muscle Nerve (1985) 2.12

Mice transgenic for the human myotonic dystrophy region with expanded CTG repeats display muscular and brain abnormalities. Hum Mol Genet (2001) 2.08

Desmin myopathy, a skeletal myopathy with cardiomyopathy caused by mutations in the desmin gene. N Engl J Med (2000) 2.04

Gamma-sarcoglycan deficiency leads to muscle membrane defects and apoptosis independent of dystrophin. J Cell Biol (1998) 1.98

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Mouse model of desmin-related cardiomyopathy. Circulation (2001) 1.84

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Ullrich scleroatonic muscular dystrophy is caused by recessive mutations in collagen type VI. Proc Natl Acad Sci U S A (2001) 1.69

Differential expression of dystrophin isoforms in strains of mdx mice with different mutations. Hum Mol Genet (1996) 1.69

Skeletal muscle-specific expression of a utrophin transgene rescues utrophin-dystrophin deficient mice. Nat Genet (1998) 1.68

Laminin alpha2 chain-null mutant mice by targeted disruption of the Lama2 gene: a new model of merosin (laminin 2)-deficient congenital muscular dystrophy. FEBS Lett (1997) 1.67

Disruption of the beta-sarcoglycan gene reveals pathogenetic complexity of limb-girdle muscular dystrophy type 2E. Mol Cell (2000) 1.66

Desmin cytoskeleton linked to muscle mitochondrial distribution and respiratory function. J Cell Biol (2000) 1.64

Recovery of induced mutations for X chromosome-linked muscular dystrophy in mice. Proc Natl Acad Sci U S A (1989) 1.64

Tragedy in a heartbeat: malfunctioning desmin causes skeletal and cardiac muscle disease. J Clin Invest (2009) 1.63

Identification of the Syrian hamster cardiomyopathy gene. Hum Mol Genet (1997) 1.63

An Actn3 knockout mouse provides mechanistic insights into the association between alpha-actinin-3 deficiency and human athletic performance. Hum Mol Genet (2008) 1.62

The value of mammalian models for duchenne muscular dystrophy in developing therapeutic strategies. Curr Top Dev Biol (2008) 1.62

Merosin-deficient congenital muscular dystrophy. Partial genetic correction in two mouse models. J Clin Invest (1998) 1.60

Both hypertrophic and dilated cardiomyopathies are caused by mutation of the same gene, delta-sarcoglycan, in hamster: an animal model of disrupted dystrophin-associated glycoprotein complex. Proc Natl Acad Sci U S A (1997) 1.59

Calcium influx is sufficient to induce muscular dystrophy through a TRPC-dependent mechanism. Proc Natl Acad Sci U S A (2009) 1.58

ACTN3 genotype is associated with increases in muscle strength in response to resistance training in women. J Appl Physiol (1985) (2005) 1.57

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Mutations in the FKRP gene can cause muscle-eye-brain disease and Walker-Warburg syndrome. J Med Genet (2004) 1.56

Mitochondrial DNA and ACTN3 genotypes in Finnish elite endurance and sprint athletes. Eur J Hum Genet (2005) 1.54

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Null mutation of calpain 3 (p94) in mice causes abnormal sarcomere formation in vivo and in vitro. Hum Mol Genet (2004) 1.48

Type VI collagen mutations in Bethlem myopathy, an autosomal dominant myopathy with contractures. Nat Genet (1996) 1.47

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Elevated subsarcolemmal Ca2+ in mdx mouse skeletal muscle fibers detected with Ca2+-activated K+ channels. Proc Natl Acad Sci U S A (2000) 1.46

Defective muscle basement membrane and lack of M-laminin in the dystrophic dy/dy mouse. Proc Natl Acad Sci U S A (1994) 1.46

Structural and functional features of the alpha 3 chain indicate a bridging role for chicken collagen VI in connective tissues. Biochemistry (1990) 1.45

Null mutation in the desmin gene gives rise to a cardiomyopathy. J Mol Cell Cardiol (1997) 1.44

CUGBP1 overexpression in mouse skeletal muscle reproduces features of myotonic dystrophy type 1. Hum Mol Genet (2010) 1.44

New mdx mutation disrupts expression of muscle and nonmuscle isoforms of dystrophin. Nat Genet (1993) 1.42

Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med (2004) 5.59

Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells. Nat Cell Biol (2007) 5.50

Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat Med (2002) 5.07

rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med (2006) 3.13

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 (2009) 2.89

Sarcolemma-localized nNOS is required to maintain activity after mild exercise. Nature (2008) 2.87

Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol Ther (2004) 2.84

Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression. Mol Ther (2007) 2.76

Syndecan-4-expressing muscle progenitor cells in the SP engraft as satellite cells during muscle regeneration. Cell Stem Cell (2009) 2.57

Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart. Circulation (2003) 2.22

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors. Nat Biotechnol (2005) 2.16

Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Mol Ther (2005) 2.08

Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure. Mol Ther (2007) 1.85

Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy. Hum Gene Ther (2007) 1.70

Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle. Mol Ther (2007) 1.65

The value of mammalian models for duchenne muscular dystrophy in developing therapeutic strategies. Curr Top Dev Biol (2008) 1.62

Dystrophin deficiency in Drosophila reduces lifespan and causes a dilated cardiomyopathy phenotype. Aging Cell (2008) 1.52

Dissecting the signaling and mechanical functions of the dystrophin-glycoprotein complex. J Cell Sci (2006) 1.51

Emergent dilated cardiomyopathy caused by targeted repair of dystrophic skeletal muscle. Mol Ther (2008) 1.47

Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin. Proc Natl Acad Sci U S A (2002) 1.47

Recombinant adeno-associated virus transduction and integration. Mol Ther (2008) 1.44

Functional deficits in nNOSmu-deficient skeletal muscle: myopathy in nNOS knockout mice. PLoS One (2008) 1.42

Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation. J Clin Invest (2002) 1.41

Plectin 1f scaffolding at the sarcolemma of dystrophic (mdx) muscle fibers through multiple interactions with beta-dystroglycan. J Cell Biol (2007) 1.39

Prevention of muscle aging by myofiber-associated satellite cell transplantation. Sci Transl Med (2010) 1.36

Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice. Mol Ther (2008) 1.34

Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice. Mol Ther (2008) 1.29

Emerging strategies for cell and gene therapy of the muscular dystrophies. Expert Rev Mol Med (2009) 1.29

Viral-mediated gene therapy for the muscular dystrophies: successes, limitations and recent advances. Biochim Biophys Acta (2006) 1.25

Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin. Neuromuscul Disord (2002) 1.24

Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6. Mol Ther (2010) 1.21

Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy. Hum Mol Genet (2008) 1.19

An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells. Nat Commun (2013) 1.17

Gene therapy strategies for Duchenne muscular dystrophy utilizing recombinant adeno-associated virus vectors. Mol Ther (2005) 1.15

Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin. J Cell Sci (2010) 1.14

Gene and cell-mediated therapies for muscular dystrophy. Muscle Nerve (2013) 1.14

Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies. Mol Ther (2012) 1.13

Evaluation of vascular delivery methodologies to enhance rAAV6-mediated gene transfer to canine striated musculature. Mol Ther (2009) 1.13

rAAV6-microdystrophin rescues aberrant Golgi complex organization in mdx skeletal muscles. Traffic (2007) 1.13

Immune responses to AAV in canine muscle monitored by cellular assays and noninvasive imaging. Mol Ther (2009) 1.11

A highly functional mini-dystrophin/GFP fusion gene for cell and gene therapy studies of Duchenne muscular dystrophy. Hum Mol Genet (2006) 1.11

Phenotypic improvement of dystrophic muscles by rAAV/microdystrophin vectors is augmented by Igf1 codelivery. Mol Ther (2005) 1.10

Expression of Dp260 in muscle tethers the actin cytoskeleton to the dystrophin-glycoprotein complex and partially prevents dystrophy. Hum Mol Genet (2002) 1.10

Follistatin-mediated skeletal muscle hypertrophy is regulated by Smad3 and mTOR independently of myostatin. J Cell Biol (2012) 1.10

Inhibitory control over Ca(2+) sparks via mechanosensitive channels is disrupted in dystrophin deficient muscle but restored by mini-dystrophin expression. PLoS One (2008) 1.08

The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins. PLoS Genet (2010) 1.08

Sustained human factor VIII expression in hemophilia A mice following systemic delivery of a gutless adenoviral vector. Mol Ther (2002) 1.07

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Gene therapy in large animal models of muscular dystrophy. ILAR J (2009) 1.05

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Proteomic analysis of mdx skeletal muscle: Great reduction of adenylate kinase 1 expression and enzymatic activity. Proteomics (2003) 1.00

Microtubule binding distinguishes dystrophin from utrophin. Proc Natl Acad Sci U S A (2014) 0.98

Onset of experimental severe cardiac fibrosis is mediated by overexpression of Angiotensin-converting enzyme 2. Hypertension (2009) 0.97

Truncated dystrophins can influence neuromuscular synapse structure. Mol Cell Neurosci (2009) 0.97

AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy. Mol Ther (2011) 0.97

Lentiviral transduction of microglial cells. Glia (2005) 0.96

Therapy for neuromuscular disorders. Curr Opin Genet Dev (2009) 0.96

Circular permutation of Bacillus circulans xylanase: a kinetic and structural study. Biochemistry (2010) 0.95

Spectrin-like repeats from dystrophin and alpha-actinin-2 are not functionally interchangeable. Hum Mol Genet (2002) 0.94

L-type Ca2+ channel function is linked to dystrophin expression in mammalian muscle. PLoS One (2008) 0.92

Molecular and cellular adaptations to chronic myotendinous strain injury in mdx mice expressing a truncated dystrophin. Hum Mol Genet (2008) 0.91

Fluorophore-labeled myosin-specific antibodies simplify muscle-fiber phenotyping. Muscle Nerve (2008) 0.90

Gutted adenoviral vector growth using E1/E2b/E3-deleted helper viruses. J Gene Med (2002) 0.90

Dystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expression. Mol Ther (2009) 0.90

Sequencing protocols to genotype mdx, mdx(4cv), and mdx(5cv) mice. Muscle Nerve (2010) 0.89

Immune Responses to rAAV6: The Influence of Canine Parvovirus Vaccination and Neonatal Administration of Viral Vector. Front Microbiol (2011) 0.88

Generation and growth of gutted adenoviral vectors. Methods Enzymol (2002) 0.88

Increased sphingosine-1-phosphate improves muscle regeneration in acutely injured mdx mice. Skelet Muscle (2013) 0.88

Characterization of aquaporin-4 in muscle and muscular dystrophy. FASEB J (2002) 0.85

Diaphragm muscle strip preparation for evaluation of gene therapies in mdx mice. Clin Exp Pharmacol Physiol (2008) 0.85

Electromyographic studies in mdx and wild-type C57 mice. Muscle Nerve (2006) 0.85

Age-dependent dystrophin loss and genetic reconstitution establish a molecular link between dystrophin and heart performance during aging. Mol Ther (2011) 0.85

Expression of the dystrophin isoform Dp116 preserves functional muscle mass and extends lifespan without preventing dystrophy in severely dystrophic mice. Hum Mol Genet (2011) 0.84

Donor origin of multipotent adult progenitor cells in radiation chimeras. Blood (2005) 0.84

Probing electrostatic interactions along the reaction pathway of a glycoside hydrolase: histidine characterization by NMR spectroscopy. Biochemistry (2007) 0.84

Mechanism of Thermoanaerobacterium saccharolyticum beta-xylosidase: kinetic studies. Biochemistry (2002) 0.84

Differential expression of the skeletal muscle proteome in mdx mice at different ages. Electrophoresis (2004) 0.83

Biodistribution and safety profile of recombinant adeno-associated virus serotype 6 vectors following intravenous delivery. J Virol (2008) 0.83

BPAG1 isoform-b: complex distribution pattern in striated and heart muscle and association with plectin and alpha-actinin. Exp Cell Res (2009) 0.83

Viral vectors for gene transfer to striated muscle. Curr Opin Mol Ther (2004) 0.83

ACE inhibitor bulks up muscle. Nat Med (2007) 0.82

A case for reverse protonation: identification of Glu160 as an acid/base catalyst in Thermoanaerobacterium saccharolyticum beta-xylosidase and detailed kinetic analysis of a site-directed mutant. Biochemistry (2002) 0.82

Enhanced effect of microdystrophin gene transfection by HSV-VP22 mediated intercellular protein transport. BMC Neurosci (2007) 0.82

Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors. Curr Gene Ther (2012) 0.81

Viral vector-mediated gene therapies. Curr Opin Neurol (2015) 0.81

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Herpes simplex virus VP22 enhances adenovirus-mediated microdystrophin gene transfer to skeletal muscles in dystrophin-deficient (mdx) mice. Hum Gene Ther (2007) 0.80

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