Published in Curr Opin Biotechnol on October 01, 2002
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
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
Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum Gene Ther (2010) 2.51
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther (2010) 1.51
Transient transfection methods for clinical adeno-associated viral vector production. Hum Gene Ther (2009) 1.05
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material. Hum Gene Ther (2014) 0.91
Efficient AAV1-AAV2 hybrid vector for gene therapy of hemophilia. Hum Gene Ther (2006) 0.88
Immunotherapy: rAAV2 expressing interleukin-15 inhibits HeLa cell tumor growth in mice. J Biomed Sci (2009) 0.87
Cell cycle regulation to repair the infarcted myocardium. Heart Fail Rev (2003) 0.84
Immobilized cobalt affinity chromatography provides a novel, efficient method for herpes simplex virus type 1 gene vector purification. J Virol (2004) 0.83
Long-term expression of rAAV2-hIL15 enhances immunoglobulin production and lymphokine-activated killer cell-mediated human glioblastoma cell death. Mol Clin Oncol (2013) 0.79
Gene delivery with viral vectors for cerebrovascular diseases. Front Biosci (Elite Ed) (2013) 0.78
Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials. Hum Gene Ther Methods (2015) 0.78
Silencing of Her2, CCNB1 and PKC Genes by siRNA Results in Prolonged Retardation of Neuroblastoma Cell Division. Acta Naturae (2011) 0.75
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 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
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
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. Mol Ther (2006) 2.13
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
Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Hum Gene Ther (2006) 1.81
alpha-1 antitrypsin inhibits caspase-3 activity, preventing lung endothelial cell apoptosis. Am J Pathol (2006) 1.81
Long-term, efficient inhibition of microRNA function in mice using rAAV vectors. Nat Methods (2012) 1.67
Controlling neuropathic pain by adeno-associated virus driven production of the anti-inflammatory cytokine, interleukin-10. Mol Pain (2005) 1.66
Repeated intrathecal injections of plasmid DNA encoding interleukin-10 produce prolonged reversal of neuropathic pain. Pain (2006) 1.66
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther (2010) 1.51
DNA-dependent PK inhibits adeno-associated virus DNA integration. Proc Natl Acad Sci U S A (2004) 1.45
Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neurons. Hum Gene Ther (2008) 1.42
A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Hum Gene Ther (2002) 1.40
Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects. Mol Ther (2002) 1.34
Systemic overexpression of IL-10 induces CD4+CD25+ cell populations in vivo and ameliorates type 1 diabetes in nonobese diabetic mice in a dose-dependent fashion. J Immunol (2003) 1.32
MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression. Mol Ther (2010) 1.30
Improved method of recombinant AAV2 delivery for systemic targeted gene therapy. Mol Ther (2002) 1.29
In vivo complementation of complex I by the yeast Ndi1 enzyme. Possible application for treatment of Parkinson disease. J Biol Chem (2006) 1.25
Interleukin 10 attenuates neointimal proliferation and inflammation in aortic allografts by a heme oxygenase-dependent pathway. Proc Natl Acad Sci U S A (2005) 1.22
IL-10 regulation of lupus in the NZM2410 murine model. Lab Invest (2006) 1.21
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. J Clin Invest (2013) 1.20
A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice. Mol Ther (2013) 1.19
Enhancing rAAV vector expression in the lung. J Gene Med (2005) 1.17
Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors. Hum Gene Ther (2005) 1.17
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther (2013) 1.16
Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector. Hum Gene Ther (2004) 1.16
Recurrent pneumonia in children: a case report and approach to diagnosis. Clin Pediatr (Phila) (2006) 1.12
Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method. Hum Gene Ther (2010) 1.08
Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector. Mol Ther (2005) 1.07
Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles. Mol Ther (2012) 1.07
Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector. J Gene Med (2006) 1.04
Cystic fibrosis transmembrane conductance regulator deficiency exacerbates islet cell dysfunction after beta-cell injury. Diabetes (2006) 1.03
In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA. Lab Invest (2007) 1.03
Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responses. Am J Respir Cell Mol Biol (2010) 1.02
Deposition and expression of aerosolized rAAV vectors in the lungs of Rhesus macaques. Mol Ther (2002) 1.00
A single-subunit NADH-quinone oxidoreductase renders resistance to mammalian nerve cells against complex I inhibition. Mol Ther (2002) 0.99
Gene therapy for cystic fibrosis. Clin Rev Allergy Immunol (2008) 0.99
Histone deacetylase inhibitor (HDACi) suberoylanilide hydroxamic acid (SAHA)-mediated correction of α1-antitrypsin deficiency. J Biol Chem (2012) 0.98
Ex vivo transduced liver progenitor cells as a platform for gene therapy in mice. Hepatology (2004) 0.98
Recombinant adeno-associated virus vectors for gene therapy. Expert Opin Biol Ther (2004) 0.97
Effects of CFTR, interleukin-10, and Pseudomonas aeruginosa on gene expression profiles in a CF bronchial epithelial cell Line. Mol Ther (2004) 0.97
Defective acid sphingomyelinase pathway with Pseudomonas aeruginosa infection in cystic fibrosis. Am J Respir Cell Mol Biol (2009) 0.97
Functional expression of the single subunit NADH dehydrogenase in mitochondria in vivo: a potential therapy for complex I deficiencies. Hum Gene Ther (2004) 0.95
Recombinant AAV serotype and capsid mutant comparison for pulmonary gene transfer of alpha-1-antitrypsin using invasive and noninvasive delivery. Mol Ther (2008) 0.95
Gene delivery in renal tubular epithelial cells using recombinant adeno-associated viral vectors. J Am Soc Nephrol (2003) 0.94
Recombinant adeno-associated virus-mediated global anterograde delivery of glial cell line-derived neurotrophic factor to the spinal cord: comparison of rubrospinal and corticospinal tracts in the rat. Hum Gene Ther (2008) 0.93
The pyruvate dehydrogenase complex as a target for gene therapy. Curr Gene Ther (2003) 0.91
Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaques. Mol Ther (2003) 0.90
Systemic correction of a fatty acid oxidation defect by intramuscular injection of a recombinant adeno-associated virus vector. Hum Gene Ther (2006) 0.90
The role of gene and cell therapy in the era of health care reform. Hum Gene Ther (2011) 0.89
Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction. Hum Gene Ther (2013) 0.88
Localized gene expression following administration of adeno-associated viral vectors via pancreatic ducts. Mol Ther (2005) 0.88
Towards a rAAV-based gene therapy for ADA-SCID: from ADA deficiency to current and future treatment strategies. Pharmacogenomics (2008) 0.87
Partial correction of the CFTR-dependent ABPA mouse model with recombinant adeno-associated virus gene transfer of truncated CFTR gene. J Gene Med (2008) 0.87
Virus-based gene delivery systems. Clin Pharmacokinet (2002) 0.87
Biochemical correction of short-chain acyl-coenzyme A dehydrogenase deficiency after portal vein injection of rAAV8-SCAD. Hum Gene Ther (2008) 0.86
Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndrome. Hum Gene Ther (2012) 0.86
Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vector. Mol Ther (2002) 0.85
Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus. Curr Protoc Microbiol (2013) 0.85
Cellular fusion for gene delivery to SCA1 affected Purkinje neurons. Mol Cell Neurosci (2011) 0.85
No immune responses by the expression of the yeast Ndi1 protein in rats. PLoS One (2011) 0.85
Enhanced IgE allergic response to Aspergillus fumigatus in CFTR-/- mice. Lab Invest (2006) 0.84
Methylphenidate does not improve cognitive function in healthy sleep-deprived young adults. J Investig Med (2004) 0.84
Murine model for cystic fibrosis bone disease demonstrates osteopenia and sex-related differences in bone formation. Pediatr Res (2009) 0.83
Effect of cigarette smoke exposure and structural modifications on the α-1 Antitrypsin interaction with caspases. Mol Med (2012) 0.83
Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors. Mol Ther (2005) 0.82
Gene therapy for pyruvate dehydrogenase E1alpha deficiency using recombinant adeno-associated virus 2 (rAAV2) vectors. Mol Ther (2002) 0.81
Induction of group IVC phospholipase A2 in allergic asthma: transcriptional regulation by TNFα in bronchoepithelial cells. Biochem J (2012) 0.81
The murine alpha(1)-proteinase inhibitor gene family: polymorphism, chromosomal location, and structure. Genomics (2002) 0.81
Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy. Mol Ther (2012) 0.80
Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17e. Mol Ther (2009) 0.80
CFTR mutations impart elevated immune reactivity in a murine model of cystic fibrosis related diabetes. Cytokine (2008) 0.80
The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency. Pharmacogenomics (2007) 0.80
Glucose-responsive expression of the human insulin promoter in HepG2 human hepatoma cells. Ann N Y Acad Sci (2003) 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
Endocrine parameters of cystic fibrosis: back to basics. J Cell Biochem (2009) 0.78
Gene-based therapy for alpha-1 antitrypsin deficiency. COPD (2013) 0.78
Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials. Hum Gene Ther Methods (2015) 0.78
Autoimmunity in a genetic disease—a cautionary tale. N Engl J Med (2010) 0.77
N-glycosylation augmentation of the cystic fibrosis epithelium improves Pseudomonas aeruginosa clearance. Am J Respir Cell Mol Biol (2010) 0.77
Current status of gene therapy for α-1 antitrypsin deficiency. Expert Opin Biol Ther (2014) 0.77
Gene transfer in the lung using recombinant adeno-associated virus. Curr Protoc Microbiol (2012) 0.76
Effect of allergy and inflammation on eicosanoid gene expression in CFTR deficiency. J Cyst Fibros (2012) 0.75
Special Note from the Editor-in-Chief. Hum Gene Ther (2016) 0.75
Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiency. Hum Gene Ther (2011) 0.75
It is Time for Zero Tolerance for Sexual Harassment in Academic Medicine. Acad Med (2017) 0.75
Developing Core Competencies for the Prevention and Management of Prescription Drug Misuse: A Medical Education Collaboration in Massachusetts. Acad Med (2016) 0.75
A Gene Therapy Scientist's Life Well-Lived. Hum Gene Ther (2016) 0.75
Gene Drives: Biological Shield or Ecological Menace? Hum Gene Ther (2016) 0.75
The Target's the Thing. Hum Gene Ther (2016) 0.75
Why Human Gene Therapy Scientists Should Care About Model Organisms. Hum Gene Ther (2016) 0.75
Gene Therapy for Alcoholism and Other Substance Use Disorders. Hum Gene Ther (2017) 0.75
In vivo expression of human ATP:cob(I)alamin adenosyltransferase (ATR) using recombinant adeno-associated virus (rAAV) serotypes 2 and 8. J Gene Med (2007) 0.75