Published in Hum Gene Ther on March 01, 2007
Gene therapy using adeno-associated virus vectors. Clin Microbiol Rev (2008) 3.30
Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood (2013) 2.09
The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther (2009) 2.08
Progress and prospects: immune responses to viral vectors. Gene Ther (2009) 2.05
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells. Blood (2013) 1.98
Systematic evaluation of AAV vectors for liver directed gene transfer in murine models. Mol Ther (2009) 1.85
The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice. J Clin Invest (2009) 1.73
Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors. Hum Gene Ther (2011) 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
Adeno-associated virus antibody profiles in newborns, children, and adolescents. Clin Vaccine Immunol (2011) 1.57
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J Clin Invest (2009) 1.54
Cellular immune response to cryptic epitopes during therapeutic gene transfer. Proc Natl Acad Sci U S A (2009) 1.53
Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo. J Virol (2007) 1.52
Improved induction of immune tolerance to factor IX by hepatic AAV-8 gene transfer. Hum Gene Ther (2009) 1.36
Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector-mediated gene therapy. Blood (2008) 1.24
Myocardial gene delivery using molecular cardiac surgery with recombinant adeno-associated virus vectors in vivo. Gene Ther (2011) 1.21
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver. Gene Ther (2010) 1.21
Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates. Mol Ther (2011) 1.18
Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses. Mol Ther (2008) 1.11
Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs. Hum Gene Ther (2011) 1.05
Transient transfection methods for clinical adeno-associated viral vector production. Hum Gene Ther (2009) 1.05
Animal models of hemophilia. Prog Mol Biol Transl Sci (2012) 1.04
Gene therapy for haemophilia. Br J Haematol (2008) 1.03
Adeno-associated virus vectors serotype 2 induce prolonged proliferation of capsid-specific CD8+ T cells in mice. Mol Ther (2010) 1.02
Direct, help-independent priming of CD8+ T cells by adeno-associated virus-transduced hepatocytes. Hepatology (2010) 1.02
A preclinical animal model to assess the effect of pre-existing immunity on AAV-mediated gene transfer. Mol Ther (2009) 1.00
In vivo reduction or blockade of interleukin-1β in primary osteoarthritis influences expression of mediators implicated in pathogenesis. Osteoarthritis Cartilage (2012) 0.97
Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo. J Virol (2009) 0.97
Adeno-associated virus serotype 2 induces cell-mediated immune responses directed against multiple epitopes of the capsid protein VP1. J Gen Virol (2009) 0.94
Evading the immune response upon in vivo gene therapy with viral vectors. Curr Opin Mol Ther (2009) 0.94
Adjunctive β2-agonists reverse neuromuscular involvement in murine Pompe disease. FASEB J (2012) 0.91
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Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors. J Thromb Haemost (2011) 0.90
Cytotoxic T lymphocyte responses to transgene product, not adeno-associated viral capsid protein, limit transgene expression in mice. Hum Gene Ther (2009) 0.90
Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia. J Genet Syndr Gene Ther (2012) 0.90
Duchenne muscular dystrophy gene therapy in the canine model. Hum Gene Ther Clin Dev (2015) 0.86
Gene therapy for hemophilia: the clot thickens. Hum Gene Ther (2014) 0.84
Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes. Mol Ther (2013) 0.84
Biodistribution and safety profile of recombinant adeno-associated virus serotype 6 vectors following intravenous delivery. J Virol (2008) 0.83
AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes. Mol Ther Methods Clin Dev (2015) 0.81
Kinetics of adeno-associated virus serotype 2 (AAV2) and AAV8 capsid antigen presentation in vivo are identical. Hum Gene Ther (2013) 0.81
Immunological ignorance allows long-term gene expression after perinatal recombinant adeno-associated virus-mediated gene transfer to murine airways. Hum Gene Ther (2014) 0.81
IL12-mediated liver inflammation reduces the formation of AAV transcriptionally active forms but has no effect over preexisting AAV transgene expression. PLoS One (2013) 0.80
Measuring immune responses to recombinant AAV gene transfer. Methods Mol Biol (2011) 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
Enhanced T cell function in a mouse model of human glycosylation. J Immunol (2013) 0.79
AAV Gene Therapy for Liver Disease. Hum Gene Ther (2016) 0.79
Systemic delivery of adeno-associated viral vectors. Curr Opin Virol (2016) 0.78
Elimination of contaminating cap genes in AAV vector virions reduces immune responses and improves transgene expression in a canine gene therapy model. Gene Ther (2014) 0.78
The liver as a target organ for gene therapy: state of the art, challenges, and future perspectives. Pharmaceuticals (Basel) (2012) 0.77
Helper-Dependent Adenoviral Vectors. J Genet Syndr Gene Ther (2011) 0.75
Adenoviral Vectors for Hemophilia Gene Therapy. J Genet Syndr Gene Ther (2013) 0.75
An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8(+) T Cells. Mol Ther Methods Clin Dev (2017) 0.75
Plasmacytoid and conventional dendritic cells cooperate in cross-priming AAV capsid-specific CD8(+) T cells. Blood (2017) 0.75
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A (2002) 11.11
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
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab (2003) 5.27
Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis (2009) 4.27
New recombinant serotypes of AAV vectors. Curr Gene Ther (2005) 3.87
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
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
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
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
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results. Hum Gene Ther (2011) 2.75
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
Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. Mol Ther (2005) 2.48
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
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
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
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
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
Toll-like receptor 4 mediates innate immune responses to Haemophilus influenzae infection in mouse lung. J Immunol (2002) 1.88
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
Genome-wide DNA methylation analysis reveals novel epigenetic changes in chronic lymphocytic leukemia. Epigenetics (2012) 1.80
Novel AAV serotypes for improved ocular gene transfer. J Gene Med (2008) 1.80
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
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
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
Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins. J Clin Invest (2002) 1.63
Chimpanzee adenovirus vaccine protects against Zaire Ebola virus. Virology (2005) 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
High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes. J Virol (2006) 1.55
The complex and evolving story of T cell activation to AAV vector-encoded transgene products. Mol Ther (2010) 1.55
AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice. Mol Ther (2004) 1.55
Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey. Sci Transl Med (2011) 1.54
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther (2013) 1.54
Race/ethnicity and the incidence of new-onset atrial fibrillation after isolated coronary artery bypass surgery. Heart Rhythm (2010) 1.51
Human airway epithelial cells sense Pseudomonas aeruginosa infection via recognition of flagellin by Toll-like receptor 5. Infect Immun (2005) 1.51
Lessons learned from the clinical development and market authorization of Glybera. Hum Gene Ther Clin Dev (2013) 1.51
Effect of blood collection technique in mice on clinical pathology parameters. Hum Gene Ther (2002) 1.48
Preoperative statin therapy decreases risk of postoperative renal insufficiency. Cardiovasc Ther (2010) 1.47
Trigger events: enviroclimatic coupling of Ebola hemorrhagic fever outbreaks. Am J Trop Med Hyg (2004) 1.45
Complete prevention of atherosclerosis in apoE-deficient mice by hepatic human apoE gene transfer with adeno-associated virus serotypes 7 and 8. Arterioscler Thromb Vasc Biol (2006) 1.45
Arrhythmogenicity of weight-loss supplements marketed on the Internet. Heart Rhythm (2009) 1.43
Physiological modulation of CFTR activity by AMP-activated protein kinase in polarized T84 cells. Am J Physiol Cell Physiol (2003) 1.42
Macaque model for severe acute respiratory syndrome. J Virol (2004) 1.41
Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. Mol Ther (2008) 1.41
Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver. Hepatology (2009) 1.41
Vaccines based on novel adeno-associated virus vectors elicit aberrant CD8+ T-cell responses in mice. J Virol (2007) 1.41
Host immune responses to chronic adenovirus infections in human and nonhuman primates. J Virol (2008) 1.41
Usefulness of multislice spiral computed tomography coronary angiography in patients with acute chest pain in the emergency department. J Cardiovasc Comput Tomogr (2007) 1.40
Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV envelope proteins. Mol Ther (2002) 1.39
Chimpanzee adenovirus CV-68 adapted as a gene delivery vector interacts with the coxsackievirus and adenovirus receptor. J Gen Virol (2002) 1.38
Resolution of primary severe acute respiratory syndrome-associated coronavirus infection requires Stat1. J Virol (2004) 1.34
PEGylation of E1-deleted adenovirus vectors allows significant gene expression on readministration to liver. Hum Gene Ther (2002) 1.32
Gene transfer into skeletal muscle using novel AAV serotypes. J Gene Med (2005) 1.31
Erythropoietin gene therapy leads to autoimmune anemia in macaques. Blood (2003) 1.30