Published in J Gene Med on September 01, 2011
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Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy. Dis Model Mech (2015) 1.30
Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy. Mol Ther (2013) 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
Mapping a neutralizing epitope onto the capsid of adeno-associated virus serotype 8. J Virol (2012) 1.05
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Systemic gene transfer reveals distinctive muscle transduction profile of tyrosine mutant AAV-1, -6, and -9 in neonatal dogs. Mol Ther Methods Clin Dev (2014) 0.93
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Duchenne muscular dystrophy gene therapy in the canine model. Hum Gene Ther Clin Dev (2015) 0.86
Intra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune tolerance. Mol Ther (2015) 0.85
A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence. Mol Ther Methods Clin Dev (2015) 0.83
Abnormal splicing switch of DMD's penultimate exon compromises muscle fibre maintenance in myotonic dystrophy. Nat Commun (2015) 0.81
Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors. Curr Gene Ther (2012) 0.81
The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer. Curr Gene Ther (2015) 0.80
Systemic delivery of adeno-associated viral vectors. Curr Opin Virol (2016) 0.78
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy. Nat Commun (2017) 0.77
Conserved regions of the DMD 3' UTR regulate translation and mRNA abundance in cultured myotubes. Neuromuscul Disord (2014) 0.76
Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy. Expert Opin Orphan Drugs (2015) 0.75
Gene transfer of arginine kinase to skeletal muscle using adeno-associated virus. Gene Ther (2014) 0.75
Progress toward Gene Therapy for Duchenne Muscular Dystrophy. Mol Ther (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
Mesenchymal progenitors distinct from satellite cells contribute to ectopic fat cell formation in skeletal muscle. Nat Cell Biol (2010) 4.57
BSPAR Standards of Care for children and young people with juvenile idiopathic arthritis. Rheumatology (Oxford) (2010) 2.68
Regulation of osteoclast apoptosis by ubiquitylation of proapoptotic BH3-only Bcl-2 family member Bim. EMBO J (2003) 2.30
Molecular signature of quiescent satellite cells in adult skeletal muscle. Stem Cells (2007) 2.23
Multiple roles of Rev3, the catalytic subunit of polzeta in maintaining genome stability in vertebrates. EMBO J (2003) 2.12
RAD18 and RAD54 cooperatively contribute to maintenance of genomic stability in vertebrate cells. EMBO J (2002) 2.06
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
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
Vertebrate POLQ and POLbeta cooperate in base excision repair of oxidative DNA damage. Mol Cell (2006) 1.79
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
A randomized trial of parenteral methotrexate comparing an intermediate dose with a higher dose in children with juvenile idiopathic arthritis who failed to respond to standard doses of methotrexate. Arthritis Rheum (2004) 1.71
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
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
Intracellular cell-autonomous association of Notch and its ligands: a novel mechanism of Notch signal modification. Dev Biol (2002) 1.47
rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse. Biochem Biophys Res Commun (2006) 1.45
Neuroprotective effects of glial cell line-derived neurotrophic factor mediated by an adeno-associated virus vector in a transgenic animal model of amyotrophic lateral sclerosis. J Neurosci (2002) 1.45
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
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
Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene. Biochem Biophys Res Commun (2002) 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
Cell and gene therapy using mesenchymal stem cells (MSCs). J Autoimmun (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
Development of new inbred transgenic strains of rats with LacZ or GFP. Biochem Biophys Res Commun (2005) 1.24
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
β-catenin as a potential key target for tumor suppression. Int J Cancer (2011) 1.23
A new noninvasive measurement system for wave intensity: evaluation of carotid arterial wave intensity and reproducibility. Heart Vessels (2002) 1.22
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
Interleukin-10 expression mediated by an adeno-associated virus vector prevents monocrotaline-induced pulmonary arterial hypertension in rats. Circ Res (2007) 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
Sexual reproduction is the default mode in apomictic Hieracium subgenus Pilosella, in which two dominant loci function to enable apomixis. Plant J (2011) 1.16
Hesr1 and Hesr3 are essential to generate undifferentiated quiescent satellite cells and to maintain satellite cell numbers. Development (2011) 1.16
Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through. Am J Pathol (2011) 1.15
Clinical usefulness of carotid arterial wave intensity in assessing left ventricular systolic and early diastolic performance. Heart Vessels (2003) 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
Retroviral vector-producing mesenchymal stem cells for targeted suicide cancer gene therapy. J Gene Med (2009) 1.10
Current teaching of paediatric musculoskeletal medicine within UK medical schools--a need for change. Rheumatology (Oxford) (2009) 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
Anionic polymer, poly(methyl vinyl ether-maleic anhydride)-coated beads-based capture of human influenza A and B virus. Bioorg Med Chem (2008) 1.09
Sport-specific characteristics of trunk muscles in collegiate wrestlers and judokas. J Strength Cond Res (2008) 1.09
Alpha1-syntrophin-deficient skeletal muscle exhibits hypertrophy and aberrant formation of neuromuscular junctions during regeneration. J Cell Biol (2002) 1.09
Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases. Nucleic Acids Res (2012) 1.09
Smooth muscle-specific dystrophin expression improves aberrant vasoregulation in mdx mice. Hum Mol Genet (2006) 1.09
Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in. Hum Gene Ther (2013) 1.08
Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy). Orphanet J Rare Dis (2013) 1.08
Specific and efficient transduction of Cochlear inner hair cells with recombinant adeno-associated virus type 3 vector. Mol Ther (2005) 1.07
A lack of self-consciousness in autism. Am J Psychiatry (2002) 1.06
Comparison of selection pressures on the HA gene of pandemic (2009) and seasonal human and swine influenza A H1 subtype viruses. Virology (2010) 1.05
High-speed, sub-15 nm feature size thermochemical nanolithography. Nano Lett (2007) 1.05
Langerin negative dendritic cells promote potent CD8+ T-cell priming by skin delivery of live adenovirus vaccine microneedle arrays. Proc Natl Acad Sci U S A (2013) 1.05
Identification of disease specific pathways using in vivo SILAC proteomics in dystrophin deficient mdx mouse. Mol Cell Proteomics (2013) 1.05
Clinical usefulness of wave intensity analysis. Med Biol Eng Comput (2008) 1.05
Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice. Hum Gene Ther (2013) 1.04
Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment. Mol Ther (2010) 1.03
Interleukin-10-mediated inhibition of angiogenesis and tumor growth in mice bearing VEGF-producing ovarian cancer. Cancer Res (2003) 1.02
Viral-mediated temporally controlled dopamine production in a rat model of Parkinson disease. Mol Ther (2005) 1.02