Published in Clin Pediatr (Phila) on January 23, 2006
Endobronchial tumor in children: Unusual finding in recurrent pneumonia, report of three cases. World J Clin Pediatr (2015) 0.76
Case 1: Recurrent pneumonia in a 24-month-old boy. Paediatr Child Health (2013) 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
Laparoscopic cholecystectomy for biliary dyskinesia in children: frequency increasing. J Pediatr Surg (2013) 2.00
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
Production of clinical-grade recombinant adeno-associated virus vectors. Curr Opin Biotechnol (2002) 1.26
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
beta-Catenin is expressed aberrantly in tumors expressing shadow cells. Pilomatricoma, craniopharyngioma, and calcifying odontogenic cyst. Am J Clin Pathol (2003) 1.13
Lung transplantation for pulmonary fibrosis in dyskeratosis congenita: Case Report and systematic literature review. BMC Blood Disord (2011) 1.12
Not so fast--don't deprive children with cystic fibrosis of the option for lung transplantation. Am J Respir Crit Care Med (2006) 1.10
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
FAK inhibition decreases cell invasion, migration and metastasis in MYCN amplified neuroblastoma. Clin Exp Metastasis (2012) 1.05
Ketone bodies inhibit the viability of human neuroblastoma cells. J Pediatr Surg (2009) 1.05
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
Isolated cranial nerve III palsy as the presenting manifestation of HIV-related large B-cell lymphoma: clinical, radiological and postmortem observations: report of a case and review of the literature. Surv Ophthalmol (2005) 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
Official American Thoracic Society technical standards: flexible airway endoscopy in children. Am J Respir Crit Care Med (2015) 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
Laparoscopic surgery in children with congenital heart disease. J Pediatr Surg (2012) 0.96
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
Twenty years of progress in congenital diaphragmatic hernia at the University of Florida. Am Surg (2003) 0.95
The risk, prevention, and outcome of cytomegalovirus after pediatric lung transplantation. Transplantation (2009) 0.94
Gene delivery in renal tubular epithelial cells using recombinant adeno-associated viral vectors. J Am Soc Nephrol (2003) 0.94
Extracorporeal membrane oxygenation in pediatric lung transplantation. J Thorac Cardiovasc Surg (2010) 0.93
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
Targeting pediatric cancer stem cells with oncolytic virotherapy. Pediatr Res (2012) 0.92
The pyruvate dehydrogenase complex as a target for gene therapy. Curr Gene Ther (2003) 0.91
Mesenteric lipoblastoma: a rare location in children. J Pediatr Surg (2008) 0.90
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
Routine utilization of single-incision pediatric endosurgery (SIPES): a 5-year institutional experience. J Laparoendosc Adv Surg Tech A (2015) 0.89
The role of gene and cell therapy in the era of health care reform. Hum Gene Ther (2011) 0.89
Feasibility of single-incision pediatric endosurgery for treatment of appendicitis in 415 children. J Laparoendosc Adv Surg Tech A (2012) 0.88
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
Pre-transplant mechanical ventilation increases short-term morbidity and mortality in pediatric patients with cystic fibrosis. J Heart Lung Transplant (2007) 0.88
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
Equestrian injuries in children. J Pediatr Surg (2009) 0.87
Towards a rAAV-based gene therapy for ADA-SCID: from ADA deficiency to current and future treatment strategies. Pharmacogenomics (2008) 0.87
Virus-based gene delivery systems. Clin Pharmacokinet (2002) 0.87
Preclinical evaluation of engineered oncolytic herpes simplex virus for the treatment of neuroblastoma. PLoS One (2013) 0.87
Toxic serum trough concentrations after administration of nebulized tobramycin. Pharmacotherapy (2003) 0.87
Increased mortality after pulmonary fungal infection within the first year after pediatric lung transplantation. J Heart Lung Transplant (2008) 0.87
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
Biochemical correction of short-chain acyl-coenzyme A dehydrogenase deficiency after portal vein injection of rAAV8-SCAD. Hum Gene Ther (2008) 0.86
Cellular fusion for gene delivery to SCA1 affected Purkinje neurons. Mol Cell Neurosci (2011) 0.85
Rabbit study of an in situ forming hydrogel vitreous substitute. Invest Ophthalmol Vis Sci (2009) 0.85
Inhibition of FAK and VEGFR-3 binding decreases tumorigenicity in neuroblastoma. Mol Carcinog (2013) 0.85
No immune responses by the expression of the yeast Ndi1 protein in rats. PLoS One (2011) 0.85
Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus. Curr Protoc Microbiol (2013) 0.85
Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vector. Mol Ther (2002) 0.85
All-terrain vehicle safety in Florida: is legislation really the answer? Am Surg (2010) 0.85
Enhanced IgE allergic response to Aspergillus fumigatus in CFTR-/- mice. Lab Invest (2006) 0.84
Analysis of gene expression patterns in human postburn hypertrophic scars. J Burn Care Rehabil (2003) 0.84
Methylphenidate does not improve cognitive function in healthy sleep-deprived young adults. J Investig Med (2004) 0.84
Exhaust system burn injuries in children. J Pediatr Surg (2005) 0.83
The use of Seprafilm in pediatric surgical patients. Am Surg (2007) 0.83
FAK Inhibition Decreases Hepatoblastoma Survival Both In Vitro and In Vivo. Transl Oncol (2013) 0.83
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
Cytomegalovirus immunoglobulin decreases the risk of cytomegalovirus infection but not disease after pediatric lung transplantation. J Heart Lung Transplant (2009) 0.82
FAK inhibition abrogates the malignant phenotype in aggressive pediatric renal tumors. Mol Cancer Res (2014) 0.82
Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors. Mol Ther (2005) 0.82
Lung transplantation for childhood diffuse lung disease. Pediatr Pulmonol (2012) 0.81
Angiodysplasia (vascular malformations) of the colon presenting as an acute abdomen. J Pediatr Surg (2012) 0.81