Published in Biochem Biophys Res Commun on May 17, 2002
Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med (2004) 5.59
Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci U S A (2004) 3.34
Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Mol Ther (2005) 2.08
Treatment of human disease by adeno-associated viral gene transfer. Hum Genet (2006) 1.84
Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice. Mol Ther (2009) 1.76
Transgenic expression of {alpha}7{beta}1 integrin maintains muscle integrity, increases regenerative capacity, promotes hypertrophy, and reduces cardiomyopathy in dystrophic mice. Am J Pathol (2005) 1.37
Therapeutic approaches to muscular dystrophy. Hum Mol Genet (2011) 1.30
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
Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle. Mol Ther (2008) 1.21
Gene and cell-mediated therapies for muscular dystrophy. Muscle Nerve (2013) 1.14
Cloning of cDNA encoding a regeneration-associated muscle protease whose expression is attenuated in cell lines derived from Duchenne muscular dystrophy patients. Am J Pathol (2004) 0.89
Approaching a new age in Duchenne muscular dystrophy treatment. Neurotherapeutics (2008) 0.88
Mutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice. PLoS One (2013) 0.82
Enhanced effect of microdystrophin gene transfection by HSV-VP22 mediated intercellular protein transport. BMC Neurosci (2007) 0.82
Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy. Pharmaceuticals (Basel) (2013) 0.82
Current Translational Research and Murine Models For Duchenne Muscular Dystrophy. J Neuromuscul Dis (2016) 0.80
Dystrophin-deficient large animal models: translational research and exon skipping. Am J Transl Res (2015) 0.80
DMD Mutations in 576 Dystrophinopathy Families: A Step Forward in Genotype-Phenotype Correlations. PLoS One (2015) 0.79
Metabogenic and Nutriceutical Approaches to Address Energy Dysregulation and Skeletal Muscle Wasting in Duchenne Muscular Dystrophy. Nutrients (2015) 0.77
Phosphorylation within the cysteine-rich region of dystrophin enhances its association with β-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting. Hum Mol Genet (2014) 0.77
In vitro stability of therapeutically relevant, internally truncated dystrophins. Skelet Muscle (2015) 0.75
Progress toward Gene Therapy for Duchenne Muscular Dystrophy. Mol Ther (2017) 0.75
Functional correction of dystrophin actin binding domain mutations by genome editing. JCI Insight (2017) 0.75
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol (2009) 5.35
Negative feedback regulation ensures the one receptor-one olfactory neuron rule in mouse. Science (2003) 5.05
Mesenchymal progenitors distinct from satellite cells contribute to ectopic fat cell formation in skeletal muscle. Nat Cell Biol (2010) 4.57
Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci U S A (2004) 3.34
Transgenic expression of Helicobacter pylori CagA induces gastrointestinal and hematopoietic neoplasms in mouse. Proc Natl Acad Sci U S A (2008) 3.17
A neuronal identity code for the odorant receptor-specific and activity-dependent axon sorting. Cell (2006) 3.15
Odorant receptor-derived cAMP signals direct axonal targeting. Science (2006) 3.03
Pre-target axon sorting establishes the neural map topography. Science (2009) 2.42
Molecular signature of quiescent satellite cells in adult skeletal muscle. Stem Cells (2007) 2.23
Myeloproliferative stem cell disorders by deregulated Rap1 activation in SPA-1-deficient mice. Cancer Cell (2003) 2.11
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther (2011) 2.06
Cardiac side population cells have a potential to migrate and differentiate into cardiomyocytes in vitro and in vivo. J Cell Biol (2007) 2.02
Adenovirus vector vaccination induces expansion of memory CD4 T cells with a mucosal homing phenotype that are readily susceptible to HIV-1. Proc Natl Acad Sci U S A (2009) 1.95
Fibrosis and adipogenesis originate from a common mesenchymal progenitor in skeletal muscle. J Cell Sci (2011) 1.94
Characterization of the pufferfish Otx2 cis-regulators reveals evolutionarily conserved genetic mechanisms for vertebrate head specification. Development (2003) 1.90
Purification and cell-surface marker characterization of quiescent satellite cells from murine skeletal muscle by a novel monoclonal antibody. Exp Cell Res (2004) 1.84
MicroRNA-206 is highly expressed in newly formed muscle fibers: implications regarding potential for muscle regeneration and maturation in muscular dystrophy. Cell Struct Funct (2008) 1.76
NO production results in suspension-induced muscle atrophy through dislocation of neuronal NOS. J Clin Invest (2007) 1.70
Transgenic expression of a myostatin inhibitor derived from follistatin increases skeletal muscle mass and ameliorates dystrophic pathology in mdx mice. FASEB J (2007) 1.65
Coordinate control of axon defasciculation and myelination by laminin-2 and -8. J Cell Biol (2005) 1.56
Cutting Edge: Ectopic expression of CD40 ligand on B cells induces lupus-like autoimmune disease. J Immunol (2002) 1.55
Identification of muscle-specific microRNAs in serum of muscular dystrophy animal models: promising novel blood-based markers for muscular dystrophy. PLoS One (2011) 1.52
Muscle regeneration by reconstitution with bone marrow or fetal liver cells from green fluorescent protein-gene transgenic mice. J Cell Sci (2002) 1.51
Expression profiling of cytokines and related genes in regenerating skeletal muscle after cardiotoxin injection: a role for osteopontin. Am J Pathol (2003) 1.49
Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice. Mol Ther (2010) 1.48
Dilated cardiomyopathy caused by aberrant endoplasmic reticulum quality control in mutant KDEL receptor transgenic mice. Mol Cell Biol (2004) 1.48
Cre-loxP-controlled periodic Aurora-A overexpression induces mitotic abnormalities and hyperplasia in mammary glands of mouse models. Oncogene (2004) 1.47
Intracellular cell-autonomous association of Notch and its ligands: a novel mechanism of Notch signal modification. Dev Biol (2002) 1.47
Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy. Nature (2011) 1.41
Prognostic impact of left ventricular noncompaction in patients with Duchenne/Becker muscular dystrophy--prospective multicenter cohort study. Int J Cardiol (2013) 1.40
The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy. Mol Ther (2010) 1.38
Identification of Cis-regulatory elements in the mouse Pax9/Nkx2-9 genomic region: implication for evolutionary conserved synteny. Genetics (2003) 1.37
Major clinical and histopathological characteristics of canine X-linked muscular dystrophy in Japan, CXMDJ. Acta Myol (2005) 1.36
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) 1.34
Deficiency of Cbl-b gene enhances infiltration and activation of macrophages in adipose tissue and causes peripheral insulin resistance in mice. Diabetes (2007) 1.33
Functional heterogeneity of side population cells in skeletal muscle. Biochem Biophys Res Commun (2006) 1.33
In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse. Mol Ther (2010) 1.33
Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery. Proc Natl Acad Sci U S A (2012) 1.32
Activation of calcium signaling through Trpv1 by nNOS and peroxynitrite as a key trigger of skeletal muscle hypertrophy. Nat Med (2012) 1.31
Follow-up of three patients with a large in-frame deletion of exons 45-55 in the Duchenne muscular dystrophy (DMD) gene. J Clin Neurosci (2008) 1.30
Laminin alpha1 chain reduces muscular dystrophy in laminin alpha2 chain deficient mice. Hum Mol Genet (2004) 1.29
Scalable purification of adeno-associated virus serotype 1 (AAV1) and AAV8 vectors, using dual ion-exchange adsorptive membranes. Hum Gene Ther (2009) 1.29
Suppression of macrophage functions impairs skeletal muscle regeneration with severe fibrosis. Exp Cell Res (2008) 1.29
Canine X-linked muscular dystrophy in Japan (CXMDJ). Exp Anim (2003) 1.27
Ubiquitin ligase Cbl-b is a negative regulator for insulin-like growth factor 1 signaling during muscle atrophy caused by unloading. Mol Cell Biol (2009) 1.27
Codon and mRNA sequence optimization of microdystrophin transgenes improves expression and physiological outcome in dystrophic mdx mice following AAV2/8 gene transfer. Mol Ther (2008) 1.24
Poly(A) binding protein nuclear 1 levels affect alternative polyadenylation. Nucleic Acids Res (2012) 1.23
AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype. Mol Ther (2004) 1.21
Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle. Mol Ther (2008) 1.21
Progression of dystrophic features and activation of mitogen-activated protein kinases and calcineurin by physical exercise, in hearts of mdx mice. FEBS Lett (2002) 1.20
Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice. Hum Gene Ther (2009) 1.19
Sequential arrival and graded secretion of Sema3F by olfactory neuron axons specify map topography at the bulb. Cell (2010) 1.18
Skeletal muscle gene expression in space-flown rats. FASEB J (2004) 1.18
Negamycin restores dystrophin expression in skeletal and cardiac muscles of mdx mice. J Biochem (2003) 1.17
Intracellular localization of dysferlin and its association with the dihydropyridine receptor. Acta Myol (2005) 1.17
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther (2013) 1.16
Hesr1 and Hesr3 are essential to generate undifferentiated quiescent satellite cells and to maintain satellite cell numbers. Development (2011) 1.16
Anti-inflammatory activity of PYNOD and its mechanism in humans and mice. J Immunol (2010) 1.15
Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through. Am J Pathol (2011) 1.15
Generation of transplantable, functional satellite-like cells from mouse embryonic stem cells. FASEB J (2009) 1.15
Genetic background affects properties of satellite cells and mdx phenotypes. Am J Pathol (2010) 1.14
Alpha1-syntrophin modulates turnover of ABCA1. J Biol Chem (2004) 1.13
Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice. Hum Mol Genet (2002) 1.13
A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground. Arch Neurol (2009) 1.11
Muscle CD31(-) CD45(-) side population cells promote muscle regeneration by stimulating proliferation and migration of myoblasts. Am J Pathol (2008) 1.10
Residual laminin-binding activity and enhanced dystroglycan glycosylation by LARGE in novel model mice to dystroglycanopathy. Hum Mol Genet (2008) 1.10
Molecular and phenotypic characterization of a mouse model of oculopharyngeal muscular dystrophy reveals severe muscular atrophy restricted to fast glycolytic fibres. Hum Mol Genet (2010) 1.10
Molecular, cellular and physiological investigation of myostatin propeptide-mediated muscle growth in adult mice. Neuromuscul Disord (2009) 1.09
Alpha1-syntrophin-deficient skeletal muscle exhibits hypertrophy and aberrant formation of neuromuscular junctions during regeneration. J Cell Biol (2002) 1.09