Published in Ann Neurol on September 01, 2009
Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med (2010) 4.28
AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells. Blood (2009) 3.65
Follistatin gene delivery enhances muscle growth and strength in nonhuman primates. Sci Transl Med (2009) 2.21
Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood (2013) 2.09
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. Mol Ther (2011) 2.04
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol (2010) 2.02
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. Blood (2010) 1.50
Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery. Mol Ther (2009) 1.35
Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs. Mol Ther (2010) 1.35
AAV's anatomy: roadmap for optimizing vectors for translational success. Curr Gene Ther (2010) 1.27
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy. Mol Ther (2014) 1.22
Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle. Mol Ther (2009) 1.21
Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPL(S447X)) in a phase II clinical trial of lipoprotein lipase deficiency gene therapy. Hum Gene Ther (2014) 1.19
Gene therapy for muscular dystrophy: lessons learned and path forward. Neurosci Lett (2012) 1.16
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
Immune responses to AAV in canine muscle monitored by cellular assays and noninvasive imaging. Mol Ther (2009) 1.11
CpG-depleted adeno-associated virus vectors evade immune detection. J Clin Invest (2013) 1.10
Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer. PLoS One (2012) 1.10
Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials. Front Microbiol (2011) 1.07
Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery. Mol Ther (2013) 1.06
Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy. Mol Ther (2011) 1.04
Sustained normalization of neurological disease after intracranial gene therapy in a feline model. Sci Transl Med (2014) 1.03
RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy. Mol Ther (2012) 1.00
Progress in gene therapy of dystrophic heart disease. Gene Ther (2012) 0.96
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models. Ann Clin Transl Neurol (2015) 0.95
Evading the immune response upon in vivo gene therapy with viral vectors. Curr Opin Mol Ther (2009) 0.94
The potential of adeno-associated viral vectors for gene delivery to muscle tissue. Expert Opin Drug Deliv (2014) 0.94
Recombinant adeno-associated viral vector production and purification. Methods Mol Biol (2012) 0.92
Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates. Hum Gene Ther Clin Dev (2014) 0.91
Gene and cell therapy based treatment strategies for inflammatory bowel diseases. World J Gastrointest Pathophysiol (2011) 0.91
Gene therapy for muscular dystrophy: moving the field forward. Pediatr Neurol (2014) 0.91
Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions. Front Immunol (2014) 0.91
δ-Sarcoglycan-deficient muscular dystrophy: from discovery to therapeutic approaches. Skelet Muscle (2011) 0.90
Single tyrosine mutation in AAV8 and AAV9 capsids is insufficient to enhance gene delivery to skeletal muscle and heart. Hum Gene Ther Methods (2012) 0.89
Progress and prospects of gene therapy clinical trials for the muscular dystrophies. Hum Mol Genet (2015) 0.89
B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study. Mol Ther Methods Clin Dev (2014) 0.88
Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy. Diabetes (2013) 0.88
A versatile adeno-associated virus vector producer cell line method for scalable vector production of different serotypes. Hum Gene Ther (2011) 0.86
Recent developments in the treatment of Duchenne muscular dystrophy and spinal muscular atrophy. Ther Adv Neurol Disord (2013) 0.86
Adeno-associated virus serotype 1 (AAV1)- and AAV5-antibody complex structures reveal evolutionary commonalities in parvovirus antigenic reactivity. J Virol (2014) 0.85
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes. Mol Ther (2017) 0.85
Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase. Hum Gene Ther Clin Dev (2013) 0.84
Gene therapy for muscular dystrophy reaches human clinical trial. Ann Neurol (2009) 0.83
Muscle Gene Therapy for Hemophilia. J Genet Syndr Gene Ther (2012) 0.82
AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes. Mol Ther Methods Clin Dev (2015) 0.81
Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors. Curr Gene Ther (2012) 0.81
Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy. J Neuromuscul Dis (2015) 0.81
Hoechst increases adeno-associated virus-mediated transgene expression in airway epithelia by inducing the cytomegalovirus promoter. J Gene Med (2012) 0.80
Effects of irradiating adult mdx mice before full-length dystrophin cDNA transfer on host anti-dystrophin immunity. Gene Ther (2010) 0.80
Adeno-associated virus-mediated gene therapy for metabolic myopathy. Hum Gene Ther (2013) 0.80
The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer. Curr Gene Ther (2015) 0.80
Dystrophin Gene Replacement and Gene Repair Therapy for Duchenne Muscular Dystrophy in 2016: An Interview. Hum Gene Ther Clin Dev (2016) 0.79
RNAi-based gene therapy for dominant Limb Girdle Muscular Dystrophies. Curr Gene Ther (2012) 0.79
A dystrophic muscle broadens the contribution and activation of immune cells reacting to rAAV gene transfer. Gene Ther (2014) 0.79
Delivery of recombinant adeno-associated virus vectors to rat diaphragm muscle via direct intramuscular injection. Hum Gene Ther Methods (2013) 0.78
Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine. Am J Phys Med Rehabil (2014) 0.78
High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline. Hum Gene Ther (2015) 0.78
RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice. Mol Ther Nucleic Acids (2014) 0.78
A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB. Hum Gene Ther Clin Dev (2015) 0.78
Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning. Hum Gene Ther Clin Dev (2015) 0.78
Could gene therapy be the future for muscular dystrophy? Therapy (2010) 0.77
The emerging role of viral vectors as vehicles for DMD gene editing. Genome Med (2016) 0.76
Novel approaches in the treatment of myositis and myopathies. Ther Adv Musculoskelet Dis (2012) 0.76
Gene therapy in monogenic congenital myopathies. Methods (2015) 0.76
Regulatory and Exhausted T Cell Responses to AAV Capsid. Hum Gene Ther (2017) 0.76
Gene therapy for muscular dystrophies: progress and challenges. J Clin Neurol (2010) 0.76
Improving the course of muscular dystrophy? Acta Myol (2012) 0.75
Transfer of Therapeutic Genes into Fetal Rhesus Monkeys using Recombinant Adeno-Associated Type I Viral Vectors. Hum Gene Ther Clin Dev (2016) 0.75
Generation and characterization of anti-Adeno-associated virus serotype 8 (AAV8) and anti-AAV9 monoclonal antibodies. J Virol Methods (2016) 0.75
Assaying the Stability and Inactivation of AAV Serotype 1 Vectors. Hum Gene Ther Methods (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
Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy. Expert Opin Orphan Drugs (2015) 0.75
Influence of immune responses in gene/stem cell therapies for muscular dystrophies. Biomed Res Int (2014) 0.75
The Classification, Natural History and Treatment of the Limb Girdle Muscular Dystrophies. J Neuromuscul Dis (2015) 0.75
Trendelenburg-Like Gait, Instability and Altered Step Patterns in a Mouse Model for Limb Girdle Muscular Dystrophy 2i. PLoS One (2016) 0.75
Reply to Letter to the Editor. Mol Ther (2017) 0.75
Multiple newly identified loci associated with prostate cancer susceptibility. Nat Genet (2008) 15.43
Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model. Cell (2009) 11.10
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
Adaptive immune responses in acute and chronic hepatitis C virus infection. Nature (2005) 5.50
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods (2002) 5.30
HCV persistence and immune evasion in the absence of memory T cell help. Science (2003) 5.23
Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol (2013) 4.57
Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med (2010) 4.28
Memory CD8+ T cells are required for protection from persistent hepatitis C virus infection. J Exp Med (2003) 4.20
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
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
Vector-mediated gene transfer engenders long-lived neutralizing activity and protection against SIV infection in monkeys. Nat Med (2009) 3.25
Essential metabolic, anti-inflammatory, and anti-tumorigenic functions of miR-122 in liver. J Clin Invest (2012) 3.14
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
Genetic epidemiology and public health: hope, hype, and future prospects. Lancet (2005) 3.00
Interplay of IKK/NF-kappaB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophy. J Clin Invest (2007) 2.99
Dystrophin glycoprotein complex dysfunction: a regulatory link between muscular dystrophy and cancer cachexia. Cancer Cell (2005) 2.73
A 2-year randomised placebo-controlled trial of doxycycline for lymphangioleiomyomatosis. Eur Respir J (2013) 2.72
Vision 1 year after gene therapy for Leber's congenital amaurosis. N Engl J Med (2009) 2.57
Once versus three-times daily regimens of tobramycin treatment for pulmonary exacerbations of cystic fibrosis--the TOPIC study: a randomised controlled trial. Lancet (2005) 2.42
Clinical practice. Painful sensory neuropathy. N Engl J Med (2003) 2.40
Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors. Proc Natl Acad Sci U S A (2008) 2.31
Early postresuscitation hypotension is associated with increased mortality following pediatric cardiac arrest. Crit Care Med (2014) 2.26
A randomized trial of nicotine-replacement therapy patches in pregnancy. N Engl J Med (2012) 2.22
Follistatin gene delivery enhances muscle growth and strength in nonhuman primates. Sci Transl Med (2009) 2.21
Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders. Mol Ther (2011) 2.20
The Image Gently campaign: working together to change practice. AJR Am J Roentgenol (2008) 2.16
Detection of diarrheagenic Escherichia coli by use of melting-curve analysis and real-time multiplex PCR. J Clin Microbiol (2008) 2.11
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The 'Image Gently' campaign: increasing CT radiation dose awareness through a national education and awareness program. Pediatr Radiol (2008) 2.03
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Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol (2010) 2.02
Survey of acute renal failure in patients with cystic fibrosis in the UK. Thorax (2007) 1.93
Primary Ewing's sarcoma of cranial bones: analysis of ten patients. Acta Neurochir (Wien) (2011) 1.92
A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy. J Transl Med (2007) 1.86
Physiological properties of hypothalamic MCH neurons identified with selective expression of reporter gene after recombinant virus infection. Neuron (2004) 1.83
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
Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy. Ann Neurol (2010) 1.81
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Factors influencing place of delivery for women in Kenya: an analysis of the Kenya demographic and health survey, 2008/2009. BMC Pregnancy Childbirth (2013) 1.78
Evaluation of the removal of point-of-sale tobacco displays in Ireland. Tob Control (2010) 1.76
Effects of changes in hemoglobin level on quality of life and cognitive function in inflammatory bowel disease patients. Inflamm Bowel Dis (2006) 1.73
Characterization of adeno-associated virus genomes isolated from human tissues. J Virol (2005) 1.72
Mutational escape from CD8+ T cell immunity: HCV evolution, from chimpanzees to man. J Exp Med (2005) 1.69
Neural deficits contribute to respiratory insufficiency in Pompe disease. Proc Natl Acad Sci U S A (2009) 1.67
Loss of immune escape mutations during persistent HCV infection in pregnancy enhances replication of vertically transmitted viruses. Nat Med (2013) 1.65
Prescribing of smoking cessation medication in England since the introduction of varenicline. Addiction (2011) 1.65
Methylenetetrahydrofolate reductase (MTHFR) genetic polymorphisms and psychiatric disorders: a HuGE review. Am J Epidemiol (2006) 1.63
Cardiac and clinical phenotype in Barth syndrome. Pediatrics (2006) 1.61
The freeze on mass media campaigns in England: a natural experiment of the impact of tobacco control campaigns on quitting behaviour. Addiction (2014) 1.60
Prevalence of maternal smoking and environmental tobacco smoke exposure during pregnancy and impact on birth weight: retrospective study using Millennium Cohort. BMC Public Health (2007) 1.59
Barriers and motivators to gaining access to smoking cessation services amongst deprived smokers--a qualitative study. BMC Health Serv Res (2006) 1.57
Pooled analysis of alcohol dehydrogenase genotypes and head and neck cancer: a HuGE review. Am J Epidemiol (2004) 1.56
Novel infectious cDNA clones of hepatitis C virus genotype 3a (strain S52) and 4a (strain ED43): genetic analyses and in vivo pathogenesis studies. J Virol (2010) 1.55
Mutation of FIG4 causes a rapidly progressive, asymmetric neuronal degeneration. Brain (2008) 1.55
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