Published in Genet Vaccines Ther on October 15, 2008
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol (2010) 2.02
Therapeutic approaches to muscular dystrophy. Hum Mol Genet (2011) 1.30
Systemic myostatin inhibition via liver-targeted gene transfer in normal and dystrophic mice. PLoS One (2010) 1.10
AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy. Mol Ther (2013) 1.03
B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study. Mol Ther Methods Clin Dev (2014) 0.88
Long-term systemic myostatin inhibition via liver-targeted gene transfer in golden retriever muscular dystrophy. Hum Gene Ther (2011) 0.85
Adeno-associated virus-mediated gene therapy for metabolic myopathy. Hum Gene Ther (2013) 0.80
Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA. Mol Ther Methods Clin Dev (2015) 0.76
A chemical method for fast and sensitive detection of DNA synthesis in vivo. Proc Natl Acad Sci U S A (2008) 10.09
Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci U S A (1996) 4.76
Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ Res (2006) 3.63
Genetic fate of recombinant adeno-associated virus vector genomes in muscle. J Virol (2003) 2.07
Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. Blood (2004) 1.78
Limb-girdle muscular dystrophy in the United States. J Neuropathol Exp Neurol (2006) 1.59
Primary adhalinopathy (alpha-sarcoglycanopathy): clinical, pathologic, and genetic correlation in 20 patients with autosomal recessive muscular dystrophy. Neurology (1997) 1.21
Long-term skeletal muscle protection after gene transfer in a mouse model of LGMD-2D. Mol Ther (2007) 1.13
Phenotypic correction of alpha-sarcoglycan deficiency by intra-arterial injection of a muscle-specific serotype 1 rAAV vector. Mol Ther (2007) 1.13
Temperature dependency of force loss and Ca(2+) homeostasis in mouse EDL muscle after eccentric contractions. Am J Physiol Regul Integr Comp Physiol (2002) 1.04
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther (2008) 8.83
Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine heart. Circulation (2002) 7.97
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
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods (2002) 5.30
Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci U S A (2008) 3.84
Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularization. Nat Med (2002) 3.81
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Arch Ophthalmol (2011) 3.79
Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol (2009) 3.69
Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ Res (2006) 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
Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum Gene Ther (2009) 3.07
Pompe disease diagnosis and management guideline. Genet Med (2006) 3.03
Vision 1 year after gene therapy for Leber's congenital amaurosis. N Engl J Med (2009) 2.57
Early postresuscitation hypotension is associated with increased mortality following pediatric cardiac arrest. Crit Care Med (2014) 2.26
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol (2010) 2.02
Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression. Virology (2008) 1.96
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. Hum Gene Ther (2006) 1.83
Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease). Curr Mol Med (2002) 1.82
Neural deficits contribute to respiratory insufficiency in Pompe disease. Proc Natl Acad Sci U S A (2009) 1.67
Cardiac and clinical phenotype in Barth syndrome. Pediatrics (2006) 1.61
Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum Gene Ther (2004) 1.54
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther (2013) 1.54
LateTIME: a phase-II, randomized, double-blinded, placebo-controlled, pilot trial evaluating the safety and effect of administration of bone marrow mononuclear cells 2 to 3 weeks after acute myocardial infarction. Tex Heart Inst J (2010) 1.52
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther (2010) 1.51
Pompe disease gene therapy. Hum Mol Genet (2011) 1.39
Establishing intensivist-driven ultrasound at the PICU bedside--it's about time*. Pediatr Crit Care Med (2014) 1.38
Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors. Mol Ther (2007) 1.31
Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system. Mol Ther (2005) 1.30
Improved method of recombinant AAV2 delivery for systemic targeted gene therapy. Mol Ther (2002) 1.29
Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies. Hum Gene Ther (2009) 1.26
Skeletal muscles of ambulant children with Duchenne muscular dystrophy: validation of multicenter study of evaluation with MR imaging and MR spectroscopy. Radiology (2013) 1.22
Gene therapy for aromatic L-amino acid decarboxylase deficiency. Sci Transl Med (2012) 1.22
Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes. Hum Gene Ther (2013) 1.19
The Barth Syndrome Registry: distinguishing disease characteristics and growth data from a longitudinal study. Am J Med Genet A (2012) 1.18
Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II. Hum Gene Ther (2005) 1.18
Structural insight into the unique properties of adeno-associated virus serotype 9. J Virol (2012) 1.18
Research priorities in hypertrophic cardiomyopathy: report of a Working Group of the National Heart, Lung, and Blood Institute. Circulation (2010) 1.18
Enhancing rAAV vector expression in the lung. J Gene Med (2005) 1.17
Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease. Mol Ther (2010) 1.17
Tolerance induction to cytoplasmic beta-galactosidase by hepatic AAV gene transfer: implications for antigen presentation and immunotoxicity. PLoS One (2009) 1.17
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther (2013) 1.16
Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse. Invest Ophthalmol Vis Sci (2011) 1.15
Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy. PLoS One (2012) 1.15
Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites. Hum Gene Ther (2007) 1.14
Long-term skeletal muscle protection after gene transfer in a mouse model of LGMD-2D. Mol Ther (2007) 1.13
Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors. Mol Ther (2002) 1.11
Hypoglossal neuropathology and respiratory activity in pompe mice. Front Physiol (2011) 1.10
The respiratory neuromuscular system in Pompe disease. Respir Physiol Neurobiol (2013) 1.09
B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease. J Pediatr (2013) 1.09
Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy. Sci Transl Med (2012) 1.06
Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease. Development (2004) 1.04
Glycosylation-independent lysosomal targeting of acid α-glucosidase enhances muscle glycogen clearance in pompe mice. J Biol Chem (2012) 1.04
AAV vectors for cardiac gene transfer: experimental tools and clinical opportunities. Mol Ther (2011) 1.04
Gene therapy approaches for lysosomal storage disease: next-generation treatment. Hum Gene Ther (2012) 1.03
Characterization of a transgenic short hairpin RNA-induced murine model of Tafazzin deficiency. Hum Gene Ther (2011) 1.03
Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease. Mol Ther (2013) 0.99
Impaired cardiac reserve and severely diminished skeletal muscle O₂ utilization mediate exercise intolerance in Barth syndrome. Am J Physiol Heart Circ Physiol (2011) 0.99
Noninvasive monitoring and tracking of muscle stem cell transplants. Transplantation (2004) 0.99
Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice. Hum Gene Ther (2003) 0.98
Sustained hepatic and renal glucose-6-phosphatase expression corrects glycogen storage disease type Ia in mice. Hum Mol Genet (2002) 0.97
A new method for recombinant adeno-associated virus vector delivery to murine diaphragm. Mol Ther (2004) 0.96
Production, purification and preliminary X-ray crystallographic studies of adeno-associated virus serotype 9. Acta Crystallogr Sect F Struct Biol Cryst Commun (2009) 0.96
Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B. Front Microbiol (2011) 0.96
AAV-mediated knockdown of phospholamban leads to improved contractility and calcium handling in cardiomyocytes. J Gene Med (2008) 0.96
Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia. Hum Gene Ther (2010) 0.95
Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9. Hum Gene Ther Methods (2012) 0.95
Non-invasive analysis of myoblast transplants in rodent cardiac muscle. Int J Cardiovasc Imaging (2004) 0.93
Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice. Mol Ther (2011) 0.91
Gene delivery to intestinal epithelial cells in vitro and in vivo with recombinant adeno-associated virus types 1, 2 and 5. Dig Dis Sci (2007) 0.91
Virus-based gene delivery systems. Clin Pharmacokinet (2002) 0.87
Mapping the T helper cell response to acid α-glucosidase in Pompe mice. Mol Genet Metab (2012) 0.86
Biochemical correction of short-chain acyl-coenzyme A dehydrogenase deficiency after portal vein injection of rAAV8-SCAD. Hum Gene Ther (2008) 0.86
The effect of DNA-dependent protein kinase on adeno-associated virus replication. PLoS One (2010) 0.86
Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase. Hum Gene Ther Clin Dev (2013) 0.84
Substrate metabolism during basal and hyperinsulinemic conditions in adolescents and young-adults with Barth syndrome. J Inherit Metab Dis (2012) 0.83
Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees. Hum Gene Ther (2008) 0.83
Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors. Mol Ther (2005) 0.82
Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with Pompe disease. Hum Gene Ther Clin Dev (2014) 0.82
MRI/MRS evaluation of a female carrier of Duchenne muscular dystrophy. Neuromuscul Disord (2012) 0.81
Reprogramming adipose tissue-derived mesenchymal stem cells into pluripotent stem cells by a mutant adeno-associated viral vector. Hum Gene Ther Methods (2013) 0.81
PRX-08066, a novel 5-hydroxytryptamine receptor 2B antagonist, reduces monocrotaline-induced pulmonary arterial hypertension and right ventricular hypertrophy in rats. J Pharmacol Exp Ther (2010) 0.80
A degradable, bioactive, gelatinized alginate hydrogel to improve stem cell/growth factor delivery and facilitate healing after myocardial infarction. Med Hypotheses (2012) 0.80
Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy. Mol Ther (2012) 0.80
Adeno-associated virus-mediated gene therapy for metabolic myopathy. Hum Gene Ther (2013) 0.80
Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis. Am J Physiol Gastrointest Liver Physiol (2011) 0.80
Analysis of regional congenital cardiac surgical outcomes in Florida using the Society of Thoracic Surgeons Congenital Heart Surgery Database. Cardiol Young (2009) 0.79
Recombinant adeno-associated virus-mediated gene delivery of long chain acyl coenzyme A dehydrogenase (LCAD) into LCAD-deficient mice. J Gene Med (2008) 0.79