Published in Mol Ther on May 03, 2006
Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet J Rare Dis (2012) 2.46
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood (2007) 2.05
AAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice. Mol Ther (2009) 1.22
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice. Nat Biotechnol (2016) 1.10
Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction. Hum Gene Ther (2010) 1.07
A preclinical animal model to assess the effect of pre-existing immunity on AAV-mediated gene transfer. Mol Ther (2009) 1.00
Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II. Mol Ther (2006) 0.99
Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome. Mol Genet Metab (2011) 0.96
Sustained correction of OTC deficiency in spf( ash) mice using optimized self-complementary AAV2/8 vectors. Gene Ther (2011) 0.95
Sexually dimorphic patterns of episomal rAAV genome persistence in the adult mouse liver and correlation with hepatocellular proliferation. Mol Ther (2009) 0.93
Induction and prevention of severe hyperammonemia in the spfash mouse model of ornithine transcarbamylase deficiency using shRNA and rAAV-mediated gene delivery. Mol Ther (2011) 0.93
Phenotypic correction of ornithine transcarbamylase deficiency using low dose helper-dependent adenoviral vectors. J Gene Med (2008) 0.86
Perinatal gene transfer to the liver. Curr Pharm Des (2011) 0.84
Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction. PLoS One (2012) 0.83
AAV-mediated liver-specific MPV17 expression restores mtDNA levels and prevents diet-induced liver failure. Mol Ther (2013) 0.83
Minimal ureagenesis is necessary for survival in the murine model of hyperargininemia treated by AAV-based gene therapy. Gene Ther (2014) 0.82
Acute metabolic decompensation due to influenza in a mouse model of ornithine transcarbamylase deficiency. Dis Model Mech (2013) 0.81
Biochemical correction of very long-chain acyl-CoA dehydrogenase deficiency following adeno-associated virus gene therapy. Mol Ther (2009) 0.79
Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1. Gene Ther (2013) 0.78
Vector-related tumorigenesis not found in ornithine transcarbamylase-deficient mice. Mol Ther (2006) 0.77
Gene therapy for inborn errors of liver metabolism: progress towards clinical applications. Ital J Pediatr (2008) 0.76
Ultrasound‑targeted microbubble destruction enhances gene transduction of adeno-associated virus in a less-permissive cell type, NIH/3T3. Mol Med Rep (2013) 0.76
The approaches for manipulating mitochondrial proteome. Environ Mol Mutagen (2010) 0.76
A new mouse model of mild ornithine transcarbamylase deficiency (spf-j) displays cerebral amino acid perturbations at baseline and upon systemic immune activation. PLoS One (2015) 0.75
AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice. Mol Genet Metab (2017) 0.75
Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders. J Inherit Metab Dis (2012) 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
Aberrant luminal progenitors as the candidate target population for basal tumor development in BRCA1 mutation carriers. Nat Med (2009) 10.88
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
Control of mammary stem cell function by steroid hormone signalling. Nature (2010) 5.38
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab (2003) 5.27
Long-distance growth and connectivity of neural stem cells after severe spinal cord injury. Cell (2012) 5.15
Fbxw7/Cdc4 is a p53-dependent, haploinsufficient tumour suppressor gene. Nature (2004) 4.35
Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis (2009) 4.27
Regulated recruitment of HP1 to a euchromatic gene induces mitotically heritable, epigenetic gene silencing: a mammalian cell culture model of gene variegation. Genes Dev (2003) 3.95
New recombinant serotypes of AAV vectors. Curr Gene Ther (2005) 3.87
FBXW7 targets mTOR for degradation and cooperates with PTEN in tumor suppression. Science (2008) 3.78
Genomics, intellectual disability, and autism. N Engl J Med (2012) 3.74
Giant magnetoresistance in organic spin-valves. Nature (2004) 3.72
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
Transcriptome analyses of mouse and human mammary cell subpopulations reveal multiple conserved genes and pathways. Breast Cancer Res (2010) 3.40
Adeno-associated viruses undergo substantial evolution in primates during natural infections. Proc Natl Acad Sci U S A (2003) 3.31
The Research Assessment Exercise is bad for UK medicine. Lancet (2005) 3.06
Dominant-negative DISC1 transgenic mice display schizophrenia-associated phenotypes detected by measures translatable to humans. Proc Natl Acad Sci U S A (2007) 3.02
Structural insights into the mechanism of abscisic acid signaling by PYL proteins. Nat Struct Mol Biol (2009) 2.94
Cross-sectional multicenter study of patients with urea cycle disorders in the United States. Mol Genet Metab (2008) 2.90
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
Chronic wasting disease of elk: transmissibility to humans examined by transgenic mouse models. J Neurosci (2005) 2.46
High and low hemoglobin glycation phenotypes in type 1 diabetes: a challenge for interpretation of glycemic control. J Diabetes Complications (2002) 2.42
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
Isolation and culture of epithelial progenitors and mesenchymal stem cells from human endometrium. Biol Reprod (2009) 2.34
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
Clinical response and pharmacokinetics from a phase 1 study of an active dosing schedule of flavopiridol in relapsed chronic lymphocytic leukemia. Blood (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
Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver. Mol Ther (2006) 2.15
Multiple recognition assay reveals prostasomes as promising plasma biomarkers for prostate cancer. Proc Natl Acad Sci U S A (2011) 2.14
Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood (2005) 2.12
Prohibitin in squamous cell carcinoma of the lung: its expression and possible clinical significance. Hum Pathol (2012) 2.09
The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther (2009) 2.08
Establishing a consortium for the study of rare diseases: The Urea Cycle Disorders Consortium. Mol Genet Metab (2010) 2.07
Chronic daily headache in children and adolescents presenting to tertiary headache clinics. Headache (2002) 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
Genome-wide association study identifies eight new risk loci for polycystic ovary syndrome. Nat Genet (2012) 2.02
Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy. Muscle Nerve (2007) 1.99
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
Reassessment of the roles of integrase and the central DNA flap in human immunodeficiency virus type 1 nuclear import. J Virol (2002) 1.91
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
Metabolic and neuropsychological phenotype in women heterozygous for ornithine transcarbamylase deficiency. Ann Neurol (2004) 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
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
Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model. Invest Ophthalmol Vis Sci (2011) 1.69
miRCancer: a microRNA-cancer association database constructed by text mining on literature. Bioinformatics (2013) 1.69
Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins. J Clin Invest (2002) 1.63