Published in Hum Gene Ther on July 01, 2008
Overexpression of Galgt2 reduces dystrophic pathology in the skeletal muscles of alpha sarcoglycan-deficient mice. Am J Pathol (2009) 0.92
Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy. Pharmaceuticals (Basel) (2013) 0.82
Systemic delivery of adeno-associated viral vectors. Curr Opin Virol (2016) 0.78
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med (2006) 13.51
Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther (2006) 4.64
Mesenchymal progenitors distinct from satellite cells contribute to ectopic fat cell formation in skeletal muscle. Nat Cell Biol (2010) 4.57
Inhibition of hepatitis B virus in mice by RNA interference. Nat Biotechnol (2003) 3.15
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood (2003) 2.74
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
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
Characterization of genome integrity for oversized recombinant AAV vector. Mol Ther (2009) 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
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
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
Intraganglionic AAV6 results in efficient and long-term gene transfer to peripheral sensory nervous system in adult rats. PLoS One (2013) 1.51
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
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
Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype. J Virol (2006) 1.36
The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice. J Virol (2007) 1.35
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
The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver. Mol Ther (2008) 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
Fukutin is required for maintenance of muscle integrity, cortical histiogenesis and normal eye development. Hum Mol Genet (2003) 1.27
Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle. Hum Gene Ther (2010) 1.26
Development of new inbred transgenic strains of rats with LacZ or GFP. Biochem Biophys Res Commun (2005) 1.24
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
An intronic LINE-1 element insertion in the dystrophin gene aborts dystrophin expression and results in Duchenne-like muscular dystrophy in the corgi breed. Lab Invest (2010) 1.20
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
ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to beta-dystroglycan. Hum Mol Genet (2004) 1.12
A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground. Arch Neurol (2009) 1.11
Discovery of β-1,4-D-mannosyl-N-acetyl-D-glucosamine phosphorylase involved in the metabolism of N-glycans. J Biol Chem (2013) 1.11
Muscle CD31(-) CD45(-) side population cells promote muscle regeneration by stimulating proliferation and migration of myoblasts. Am J Pathol (2008) 1.10
Mutation analysis of the IL36RN gene in 14 Japanese patients with generalized pustular psoriasis. Hum Mutat (2012) 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
Alpha1-syntrophin-deficient skeletal muscle exhibits hypertrophy and aberrant formation of neuromuscular junctions during regeneration. J Cell Biol (2002) 1.09