Published in Mol Ther on December 06, 2011
Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy. Dis Model Mech (2015) 1.30
Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping. Mol Ther (2012) 1.18
Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients. Mol Ther (2014) 1.17
Targeting RNA splicing for disease therapy. Wiley Interdiscip Rev RNA (2013) 1.17
Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus. Hum Mol Genet (2015) 1.16
Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy. Hum Genet (2016) 1.04
AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy. Mol Ther (2013) 1.03
Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice. EMBO Mol Med (2013) 1.01
Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6. J Virol (2012) 0.99
The potential of adeno-associated viral vectors for gene delivery to muscle tissue. Expert Opin Drug Deliv (2014) 0.94
Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches. Nucleic Acids Res (2013) 0.91
Progress and prospects of gene therapy clinical trials for the muscular dystrophies. Hum Mol Genet (2015) 0.89
An emerging adeno-associated viral vector pipeline for cardiac gene therapy. Hum Gene Ther (2013) 0.88
Pharmacologic management of Duchenne muscular dystrophy: target identification and preclinical trials. ILAR J (2014) 0.88
Duchenne muscular dystrophy gene therapy in the canine model. Hum Gene Ther Clin Dev (2015) 0.86
MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy. Gene Ther (2012) 0.86
Dystrophin-deficient large animal models: translational research and exon skipping. Am J Transl Res (2015) 0.80
Tadalafil Treatment Delays the Onset of Cardiomyopathy in Dystrophin-Deficient Hearts. J Am Heart Assoc (2016) 0.77
Cardiac AAV9 Gene Delivery Strategies in Adult Canines: Assessment by Long-term Serial SPECT Imaging of Sodium Iodide Symporter Expression. Mol Ther (2015) 0.77
Viral Vector-Mediated Antisense Therapy for Genetic Diseases. Genes (Basel) (2017) 0.75
Progress toward Gene Therapy for Duchenne Muscular Dystrophy. Mol Ther (2017) 0.75
The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery. Mol Ther Nucleic Acids (2012) 0.75
100-fold but not 50-fold dystrophin overexpression aggravates electrocardiographic defects in the mdx model of Duchenne muscular dystrophy. Mol Ther Methods Clin Dev (2016) 0.75
Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy. Expert Opin Orphan Drugs (2015) 0.75
Functional correction of dystrophin actin binding domain mutations by genome editing. JCI Insight (2017) 0.75
Canine and Feline Models of Human Genetic Diseases and Their Contributions to Advancing Clinical Therapies . Yale J Biol Med (2017) 0.75
Nanotherapy for Duchenne muscular dystrophy. Wiley Interdiscip Rev Nanomed Nanobiotechnol (2017) 0.75
Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med (2007) 7.29
The muscular dystrophies. Lancet (2002) 6.41
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
Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat Med (2002) 5.07
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat Med (1999) 4.61
Cardiac motion tracking using CINE harmonic phase (HARP) magnetic resonance imaging. Magn Reson Med (1999) 4.15
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat Med (2003) 4.03
Comparison of magnetic resonance feature tracking for strain calculation with harmonic phase imaging analysis. JACC Cardiovasc Imaging (2010) 3.50
Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci U S A (2004) 3.34
Imaging heart motion using harmonic phase MRI. IEEE Trans Med Imaging (2000) 3.15
Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs. Ann Neurol (2009) 3.01
Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule. Muscle Nerve (2006) 2.98
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science (2004) 2.71
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. Mol Ther (2008) 2.69
An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy. Genomics (1992) 2.29
Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum Mol Genet (2003) 2.17
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Ther (2006) 2.00
Canine X-linked muscular dystrophy. An animal model of Duchenne muscular dystrophy: clinical studies. J Neurol Sci (1988) 1.98
Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers. Cardiovasc Res (2009) 1.82
A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells. Mol Ther (2009) 1.80
Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice. Mol Ther (2009) 1.76
Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells. Hum Mol Genet (2001) 1.76
Circumferential strain analysis identifies strata of cardiomyopathy in Duchenne muscular dystrophy: a cardiac magnetic resonance tagging study. J Am Coll Cardiol (2009) 1.74
Tissue Doppler imaging detects early asymptomatic myocardial abnormalities in a dog model of Duchenne's cardiomyopathy. Eur Heart J (2004) 1.65
Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs. Mol Ther (2010) 1.56
Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model. Proc Natl Acad Sci U S A (2006) 1.55
Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino. Gene Ther (2009) 1.52
Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy. Biochim Biophys Acta (2010) 1.47
Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery. Mol Ther (2009) 1.35
Occult cardiac contractile dysfunction in dystrophin-deficient children revealed by cardiac magnetic resonance strain imaging. Circulation (2005) 1.28
Producing recombinant adeno-associated virus in foster cells: overcoming production limitations using a baculovirus-insect cell expression strategy. Hum Gene Ther (2009) 1.24
Percutaneous transendocardial delivery of self-complementary adeno-associated virus 6 achieves global cardiac gene transfer in canines. Mol Ther (2008) 1.18
Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice. Hum Gene Ther (2008) 1.17
Early manifestation of alteration in cardiac function in dystrophin deficient mdx mouse using 3D CMR tagging. J Cardiovasc Magn Reson (2009) 1.13
Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping. Mol Ther (2010) 1.09
Gene therapy in large animal models of muscular dystrophy. ILAR J (2009) 1.05
Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy. Mol Ther (2009) 1.00
A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping. PLoS One (2010) 0.98
Duchenne's cardiomyopathy in a canine model: electrocardiographic and echocardiographic studies. J Am Coll Cardiol (1991) 0.93
Gene therapy for muscle disease. Exp Cell Res (2010) 0.90
An explanation for the constitutive exon 9 cassette splicing of the DMD gene. Hum Mol Genet (1994) 0.87
Tissue Doppler assessment of diastolic and systolic alterations of radial and longitudinal left ventricular motions in Golden Retrievers during the preclinical phase of cardiomyopathy associated with muscular dystrophy. Am J Vet Res (2004) 0.87
Biocompatibility of cardiovascular gene delivery catheters with adenovirus vectors: an important determinant of the efficiency of cardiovascular gene transfer. Mol Ther (2000) 0.82
Large-scale production of recombinant adeno-associated viral vectors. Methods Mol Biol (2008) 0.81
Cardiac magnetic resonance imaging in normal dogs and two dogs with heart base tumor. Vet Radiol Ultrasound (2010) 0.81
Non-invasive evaluation of the cardiac function in golden retriever dogs by radionuclide angiography. Neuromuscul Disord (1994) 0.81
The potential of exon skipping for treatment for Duchenne muscular dystrophy. J Child Neurol (2010) 0.81
Percutaneous transendocardial delivery of self-complementary adeno-associated virus 6 in the canine. Methods Mol Biol (2011) 0.79
Percutaneous transendocardial VEGF gene therapy: MRI guided delivery and characterization of 3D myocardial strain. Int J Cardiol (2009) 0.77
Matrix elasticity directs stem cell lineage specification. Cell (2006) 43.00
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A (2002) 11.11
Myotubes differentiate optimally on substrates with tissue-like stiffness: pathological implications for soft or stiff microenvironments. J Cell Biol (2004) 7.38
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A (2008) 7.16
Clades of Adeno-associated viruses are widely disseminated in human tissues. J Virol (2004) 7.12
PTC124 targets genetic disorders caused by nonsense mutations. Nature (2007) 6.67
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab (2003) 5.27
RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc Natl Acad Sci U S A (2005) 4.79
RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nat Med (2004) 4.63
Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis (2009) 4.27
Insect cells as a factory to produce adeno-associated virus type 2 vectors. Hum Gene Ther (2002) 3.92
New recombinant serotypes of AAV vectors. Curr Gene Ther (2005) 3.87
The mechanism of myosin VI translocation and its load-induced anchoring. Cell (2004) 3.85
Mesenchymal stem cell injection after myocardial infarction improves myocardial compliance. Am J Physiol Heart Circ Physiol (2006) 3.78
A structural state of the myosin V motor without bound nucleotide. Nature (2003) 3.63
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci U S A (2009) 3.58
Weight change and health outcomes at 3 years after bariatric surgery among individuals with severe obesity. JAMA (2013) 3.57
Postoperative atrial fibrillation and mortality after coronary artery bypass surgery. J Am Coll Cardiol (2004) 3.49
Adeno-associated viruses undergo substantial evolution in primates during natural infections. Proc Natl Acad Sci U S A (2003) 3.31
Three myosin V structures delineate essential features of chemo-mechanical transduction. EMBO J (2004) 3.21
Role of the lever arm in the processive stepping of myosin V. Proc Natl Acad Sci U S A (2002) 2.86
A simian replication-defective adenoviral recombinant vaccine to HIV-1 gag. J Immunol (2003) 2.85
Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med (2006) 2.78
Muscle-specific expression of insulin-like growth factor I counters muscle decline in mdx mice. J Cell Biol (2002) 2.76
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results. Hum Gene Ther (2011) 2.75
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science (2004) 2.71
Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. Hum Gene Ther (2007) 2.64
Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum Gene Ther (2010) 2.51
The structure of the myosin VI motor reveals the mechanism of directionality reversal. Nature (2005) 2.50
A model of myosin V processivity. J Biol Chem (2004) 2.49
Genetic and pharmacologic inhibition of mitochondrial-dependent necrosis attenuates muscular dystrophy. Nat Med (2008) 2.49
Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. Mol Ther (2005) 2.48
Myostatin activation in patients with advanced heart failure and after mechanical unloading. Eur J Heart Fail (2010) 2.40
A force-dependent state controls the coordination of processive myosin V. Proc Natl Acad Sci U S A (2005) 2.39
Natural killer T cell ligand alpha-galactosylceramide enhances protective immunity induced by malaria vaccines. J Exp Med (2002) 2.38
Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery. Nat Med (2002) 2.37
Novel, chimpanzee serotype 68-based adenoviral vaccine carrier for induction of antibodies to a transgene product. J Virol (2002) 2.31
Myosin VI steps via a hand-over-hand mechanism with its lever arm undergoing fluctuations when attached to actin. J Biol Chem (2004) 2.31
Effects of experimental weight perturbation on skeletal muscle work efficiency in human subjects. Am J Physiol Regul Integr Comp Physiol (2003) 2.28
Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat. Hum Gene Ther (2008) 2.27
Early quadriceps strength loss after total knee arthroplasty. The contributions of muscle atrophy and failure of voluntary muscle activation. J Bone Joint Surg Am (2005) 2.26
A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs. Mol Ther (2008) 2.22
Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype. Blood (2003) 2.21
Human cone photoreceptor dependence on RPE65 isomerase. Proc Natl Acad Sci U S A (2007) 2.21
Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing. Hum Gene Ther (2010) 2.19
Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice. Cell Stem Cell (2007) 2.17
Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver. Mol Ther (2006) 2.15
Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood (2005) 2.12
The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther (2009) 2.08
Ambulatory continuous femoral nerve blocks decrease time to discharge readiness after tricompartment total knee arthroplasty: a randomized, triple-masked, placebo-controlled study. Anesthesiology (2008) 2.07
Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. Mol Ther (2008) 2.06
Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. J Virol (2007) 2.05
An inherently bifunctional subset of Foxp3+ T helper cells is controlled by the transcription factor eos. Immunity (2013) 1.98
A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency. Hum Gene Ther (2002) 1.96
Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets. Hum Gene Ther (2007) 1.95
Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy. Hum Mutat (2007) 1.89
Toll-like receptor 4 mediates innate immune responses to Haemophilus influenzae infection in mouse lung. J Immunol (2002) 1.88
Misregulated alternative splicing of BIN1 is associated with T tubule alterations and muscle weakness in myotonic dystrophy. Nat Med (2011) 1.87
Isolation and characterization of adenoviruses persistently shed from the gastrointestinal tract of non-human primates. PLoS Pathog (2009) 1.85
Systematic evaluation of AAV vectors for liver directed gene transfer in murine models. Mol Ther (2009) 1.85
HIV-1 suppression and durable control by combining single broadly neutralizing antibodies and antiretroviral drugs in humanized mice. Proc Natl Acad Sci U S A (2013) 1.85
Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. Blood (2004) 1.83
Neovasculogenic therapy to augment perfusion and preserve viability in ischemic cardiomyopathy. Ann Thorac Surg (2006) 1.82
Novel AAV serotypes for improved ocular gene transfer. J Gene Med (2008) 1.80
Full-length myosin VI dimerizes and moves processively along actin filaments upon monomer clustering. Mol Cell (2006) 1.80
Genome-wide DNA methylation analysis reveals novel epigenetic changes in chronic lymphocytic leukemia. Epigenetics (2012) 1.80
A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells. Mol Ther (2009) 1.80
A flexible domain is essential for the large step size and processivity of myosin VI. Mol Cell (2005) 1.78
Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered. Proc Natl Acad Sci U S A (2006) 1.78
Induction of CD8+ T cells to an HIV-1 antigen through a prime boost regimen with heterologous E1-deleted adenoviral vaccine carriers. J Immunol (2003) 1.75
Structural unity among viral origin binding proteins: crystal structure of the nuclease domain of adeno-associated virus Rep. Mol Cell (2002) 1.74
Multiple mechanisms linked to platelet activation result in lysophosphatidic acid and sphingosine 1-phosphate generation in blood. J Biol Chem (2002) 1.72
Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors. Hum Gene Ther (2011) 1.71
Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector. Mol Ther (2006) 1.71
Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses. Hum Gene Ther (2009) 1.71
Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model. Invest Ophthalmol Vis Sci (2011) 1.69
Total elbow arthroplasty as an outpatient procedure using a continuous infraclavicular nerve block at home: a prospective case report. Reg Anesth Pain Med (2006) 1.67
Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors. Mol Ther (2006) 1.64
Chimpanzee adenovirus vaccine protects against Zaire Ebola virus. Virology (2005) 1.63
Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins. J Clin Invest (2002) 1.63
Prognostic significance of delayed-enhancement magnetic resonance imaging: survival of 857 patients with and without left ventricular dysfunction. Circulation (2009) 1.61
In vivo somatic cell gene transfer of an engineered Noggin mutein prevents BMP4-induced heterotopic ossification. J Bone Joint Surg Am (2003) 1.59
Preoperative statin therapy is not associated with a decrease in the incidence of postoperative atrial fibrillation in patients undergoing cardiac surgery. Am Heart J (2007) 1.58
The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice. J Clin Invest (2011) 1.58
Adeno-associated virus antibody profiles in newborns, children, and adolescents. Clin Vaccine Immunol (2011) 1.57
beta-Defensin 1 contributes to pulmonary innate immunity in mice. Infect Immun (2002) 1.57
Polymerase chain reaction (PCR) amplification of parvoviral DNA from the brains of dogs and cats with cerebellar hypoplasia. J Vet Intern Med (2003) 1.57
The structural basis for the large powerstroke of myosin VI. Cell (2007) 1.56