Published in Mol Ther on February 01, 2007
Interleukin-10 prevents diet-induced insulin resistance by attenuating macrophage and cytokine response in skeletal muscle. Diabetes (2009) 2.00
DUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53-dependent myopathy in vivo. Ann Neurol (2010) 1.89
Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer. Mol Ther (2011) 1.86
Genetic enhancement of stem cell engraftment, survival, and efficacy. Circ Res (2008) 1.42
Golgi and sarcolemmal neuronal NOS differentially regulate contraction-induced fatigue and vasoconstriction in exercising mouse skeletal muscle. J Clin Invest (2010) 1.37
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Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice. Am J Physiol Cell Physiol (2008) 1.16
Enhancing muscle membrane repair by gene delivery of MG53 ameliorates muscular dystrophy and heart failure in δ-Sarcoglycan-deficient hamsters. Mol Ther (2012) 1.13
Bioengineered human myobundles mimic clinical responses of skeletal muscle to drugs. Elife (2015) 1.10
Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer. PLoS One (2012) 1.10
Gene doping: the hype and the reality. Br J Pharmacol (2008) 1.10
Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy. Mol Ther (2008) 1.09
Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery. Mol Ther (2013) 1.06
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Hypoxia induces re-entry of committed cells into pluripotency. Stem Cells (2013) 1.01
Targeted genomic integration of a selectable floxed dual fluorescence reporter in human embryonic stem cells. PLoS One (2012) 1.00
Myocyte-mediated arginase expression controls hyperargininemia but not hyperammonemia in arginase-deficient mice. Mol Ther (2014) 0.97
The potential of adeno-associated viral vectors for gene delivery to muscle tissue. Expert Opin Drug Deliv (2014) 0.94
Adeno-associated virus serotype 9 efficiently targets ischemic skeletal muscle following systemic delivery. Gene Ther (2013) 0.93
AAV-mediated overexpression of human α7 integrin leads to histological and functional improvement in dystrophic mice. Mol Ther (2013) 0.92
Immunomodulatory gene therapy in lysosomal storage disorders. Curr Gene Ther (2009) 0.92
KLF3 regulates muscle-specific gene expression and synergizes with serum response factor on KLF binding sites. Mol Cell Biol (2010) 0.90
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Approaching a new age in Duchenne muscular dystrophy treatment. Neurotherapeutics (2008) 0.88
Desmin-regulated lentiviral vectors for skeletal muscle gene transfer. Mol Ther (2009) 0.87
Human embryonic stem cell-derived cardiomyocytes migrate in response to gradients of fibronectin and Wnt5a. Stem Cells Dev (2013) 0.87
Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy. Nat Commun (2017) 0.86
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Transcription factor rational design improves directed differentiation of human mesenchymal stem cells into skeletal myocytes. Mol Ther (2011) 0.85
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Gene therapy to treat cardiac arrhythmias. Nat Rev Cardiol (2015) 0.83
Polymers for improving the in vivo transduction efficiency of AAV2 vectors. PLoS One (2010) 0.83
Adeno-associated viral vector-mediated transgene expression is independent of DNA methylation in primate liver and skeletal muscle. PLoS One (2011) 0.82
Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease. Gene Ther (2010) 0.81
Multi-parametric MRI at 14T for muscular dystrophy mice treated with AAV vector-mediated gene therapy. PLoS One (2015) 0.81
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Cardiac gene therapy: are we there yet? Gene Ther (2016) 0.77
Transduction of skeletal muscles with common reporter genes can promote muscle fiber degeneration and inflammation. PLoS One (2012) 0.77
Using AAV vectors expressing the β2-adrenoceptor or associated Gα proteins to modulate skeletal muscle mass and muscle fibre size. Sci Rep (2016) 0.77
Partial rescue of growth failure in growth hormone (GH)-deficient mice by a single injection of a double-stranded adeno-associated viral vector expressing the GH gene driven by a muscle-specific regulatory cassette. Hum Gene Ther (2009) 0.76
Systemic RNAi delivery to the muscles of ROSA26 mice reduces lacZ expression. PLoS One (2014) 0.76
Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice. Mol Ther (2017) 0.75
Deletion of Pofut1 in Mouse Skeletal Myofibers Induces Muscle Aging-Related Phenotypes in cis and in trans. Mol Cell Biol (2017) 0.75
Efficacy of Gene Therapy Is Dependent on Disease Progression in Dystrophic Mice with Mutations in the FKRP Gene. Mol Ther Methods Clin Dev (2017) 0.75
Recombinant adeno-associated viral (rAAV) vectors mediate efficient gene transduction in cultured neonatal and adult microglia. J Neurochem (2015) 0.75
Empower multiplex cell and tissue-specific CRISPR-mediated gene manipulation with self-cleaving ribozymes and tRNA. Nucleic Acids Res (2016) 0.75
Mitochondrial Maturation in Human Pluripotent Stem Cell Derived Cardiomyocytes. Stem Cells Int (2017) 0.75
A compact unc45b-promoter drives muscle-specific expression in zebrafish and mouse. Genesis (2016) 0.75
The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery. Mol Ther Nucleic Acids (2012) 0.75
MicroRNA-regulated viral vectors for gene therapy. World J Exp Med (2016) 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
RNA interference in adult mice. Nature (2002) 5.33
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
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood (2003) 2.74
Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol (2002) 2.54
Foamy virus vector integration sites in normal human cells. Proc Natl Acad Sci U S A (2006) 2.52
Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo. Nat Biotechnol (2002) 2.31
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
Interleukin-10 prevents diet-induced insulin resistance by attenuating macrophage and cytokine response in skeletal muscle. Diabetes (2009) 2.00
Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure. Mol Ther (2007) 1.85
Lamin A/C and emerin are critical for skeletal muscle satellite cell differentiation. Genes Dev (2006) 1.73
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
Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nat Biotechnol (2002) 1.69
The value of mammalian models for duchenne muscular dystrophy in developing therapeutic strategies. Curr Top Dev Biol (2008) 1.62
Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors. Nat Med (2007) 1.58
A novel leucine zipper targets AKAP15 and cyclic AMP-dependent protein kinase to the C terminus of the skeletal muscle Ca2+ channel and modulates its function. J Biol Chem (2001) 1.57
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
Determinants of hepatitis C translational initiation in vitro, in cultured cells and mice. Mol Ther (2002) 1.51
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
Emergent dilated cardiomyopathy caused by targeted repair of dystrophic skeletal muscle. Mol Ther (2008) 1.47
Activation of dopamine neurons is critical for aversive conditioning and prevention of generalized anxiety. Nat Neurosci (2011) 1.46
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
Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo. Mol Ther (2003) 1.42
Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation. J Clin Invest (2002) 1.41
In vitro generation of differentiated cardiac myofibers on micropatterned laminin surfaces. J Biomed Mater Res (2002) 1.38
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
In vivo antiviral efficacy of prenylation inhibitors against hepatitis delta virus. J Clin Invest (2003) 1.33
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
Spatially organized layers of cardiomyocytes on biodegradable polyurethane films for myocardial repair. J Biomed Mater Res A (2003) 1.19
Quantitative proteomic identification of six4 as the trex-binding factor in the muscle creatine kinase enhancer. Mol Cell Biol (2004) 1.17
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
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
rAAV6-microdystrophin rescues aberrant Golgi complex organization in mdx skeletal muscles. Traffic (2007) 1.13
Elevated expression of activins promotes muscle wasting and cachexia. FASEB J (2013) 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