Published in Mol Ther on December 19, 2005
Systemic Trans-splicing adeno-associated viral delivery efficiently transduces the heart of adult mdx mouse, a model for duchenne muscular dystrophy. Hum Gene Ther (2009) 1.53
AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis. Hum Mol Genet (2008) 1.44
Cardiac expression of a mini-dystrophin that normalizes skeletal muscle force only partially restores heart function in aged Mdx mice. Mol Ther (2008) 1.36
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs. Mol Ther (2010) 1.35
Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies. Hum Gene Ther (2009) 1.26
Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector-mediated gene therapy. Blood (2008) 1.24
Gene therapy in large animal models of muscular dystrophy. ILAR J (2009) 1.05
Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice. J Gene Med (2009) 1.04
Missense mutations in dystrophin that trigger muscular dystrophy decrease protein stability and lead to cross-beta aggregates. Proc Natl Acad Sci U S A (2010) 1.01
A simplified immune suppression scheme leads to persistent micro-dystrophin expression in Duchenne muscular dystrophy dogs. Hum Gene Ther (2011) 1.00
Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy. Mol Ther (2009) 1.00
Dystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expression. Mol Ther (2009) 0.90
Dystrophin deficiency compromises force production of the extensor carpi ulnaris muscle in the canine model of Duchenne muscular dystrophy. PLoS One (2012) 0.90
Engraftment of ES-Derived Myogenic Progenitors in a Severe Mouse Model of Muscular Dystrophy. J Stem Cell Res Ther (2012) 0.89
Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy. Mol Ther (2012) 0.88
A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform. Mol Ther Methods Clin Dev (2016) 0.81
Mechanical and non-mechanical functions of Dystrophin can prevent cardiac abnormalities in Drosophila. Exp Gerontol (2013) 0.78
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy. Nat Commun (2017) 0.77
Advances in gene therapy for muscular dystrophies. F1000Res (2016) 0.75
X-Linked Dilated Cardiomyopathy: A Cardiospecific Phenotype of Dystrophinopathy. Pharmaceuticals (Basel) (2015) 0.75
Silencing of Her2, CCNB1 and PKC Genes by siRNA Results in Prolonged Retardation of Neuroblastoma Cell Division. Acta Naturae (2011) 0.75
Intracardiac injection of AAV9-NPC1 significantly ameliorates Purkinje cell death and behavioral abnormalities in mouse Niemann-Pick type C disease. J Lipid Res (2017) 0.75
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
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
TGF-beta regulates miR-206 and miR-29 to control myogenic differentiation through regulation of HDAC4. J Biol Chem (2011) 2.06
Suppression of microRNA-29 expression by TGF-β1 promotes collagen expression and renal fibrosis. J Am Soc Nephrol (2011) 2.06
Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure. Mol Ther (2007) 1.85
Therapeutic inhibition of the miR-34 family attenuates pathological cardiac remodeling and improves heart function. Proc Natl Acad Sci U S A (2012) 1.72
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
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
Beta 2-agonist administration reverses muscle wasting and improves muscle function in aged rats. J Physiol (2003) 1.25
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
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
Evaluation of vascular delivery methodologies to enhance rAAV6-mediated gene transfer to canine striated musculature. Mol Ther (2009) 1.13
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
Elevated expression of activins promotes muscle wasting and cachexia. FASEB J (2013) 1.13
rAAV6-microdystrophin rescues aberrant Golgi complex organization in mdx skeletal muscles. Traffic (2007) 1.13
The bone morphogenetic protein axis is a positive regulator of skeletal muscle mass. J Cell Biol (2013) 1.12
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
Interleukin-15 administration improves diaphragm muscle pathology and function in dystrophic mdx mice. Am J Pathol (2005) 1.10
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
ACTN3 genotype influences muscle performance through the regulation of calcineurin signaling. J Clin Invest (2013) 1.08
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
Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials. Front Microbiol (2011) 1.07
Beta 2-agonist fenoterol has greater effects on contractile function of rat skeletal muscles than clenbuterol. Am J Physiol Regul Integr Comp Physiol (2002) 1.06
Gene therapy in large animal models of muscular dystrophy. ILAR J (2009) 1.05
Functional capacity of dystrophins carrying deletions in the N-terminal actin-binding domain. Hum Mol Genet (2007) 1.04
The calcineurin signal transduction pathway is essential for successful muscle regeneration in mdx dystrophic mice. Acta Neuropathol (2004) 1.03
Generation of a specific activin antagonist by modification of the activin A propeptide. Endocrinology (2011) 1.00
Gene therapy for muscular dystrophy - a review of promising progress. Expert Opin Biol Ther (2003) 1.00
Proteomic analysis of mdx skeletal muscle: Great reduction of adenylate kinase 1 expression and enzymatic activity. Proteomics (2003) 1.00
miR-206 represses hypertrophy of myogenic cells but not muscle fibers via inhibition of HDAC4. PLoS One (2013) 0.99
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
Lentiviral transduction of microglial cells. Glia (2005) 0.96
Therapy for neuromuscular disorders. Curr Opin Genet Dev (2009) 0.96
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
Beta2-adrenoceptor agonist fenoterol enhances functional repair of regenerating rat skeletal muscle after injury. J Appl Physiol (1985) (2003) 0.91
Dystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expression. Mol Ther (2009) 0.90
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
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
Phosphoinositide 3-kinase p110α is a master regulator of exercise-induced cardioprotection and PI3K gene therapy rescues cardiac dysfunction. Circ Heart Fail (2012) 0.87
Characterization of aquaporin-4 in muscle and muscular dystrophy. FASEB J (2002) 0.85
Electromyographic studies in mdx and wild-type C57 mice. Muscle Nerve (2006) 0.85
Diaphragm muscle strip preparation for evaluation of gene therapies in mdx mice. Clin Exp Pharmacol Physiol (2008) 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
Biodistribution and safety profile of recombinant adeno-associated virus serotype 6 vectors following intravenous delivery. J Virol (2008) 0.83
Differential expression of the skeletal muscle proteome in mdx mice at different ages. Electrophoresis (2004) 0.83