Published in Hum Gene Ther on November 01, 2004
New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy. Hum Genet (2006) 1.65
Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations. J Neurosurg Pediatr (2010) 1.57
Suppression of Huntington's disease pathology in Drosophila by human single-chain Fv antibodies. Proc Natl Acad Sci U S A (2005) 1.52
Role of dichloroacetate in the treatment of genetic mitochondrial diseases. Adv Drug Deliv Rev (2008) 1.33
Do all of the neurologic diseases in patients with DNA repair gene mutations result from the accumulation of DNA damage? DNA Repair (Amst) (2008) 1.25
The state of the art of adeno-associated virus-based vectors in gene therapy. Virol J (2007) 1.16
Lysosomal storage diseases--the horizon expands. Nat Rev Neurol (2013) 1.15
Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis. Exp Neurol (2008) 1.13
Proteolytic mapping of the adeno-associated virus capsid. Mol Ther (2006) 1.03
Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis. Hum Gene Ther Methods (2012) 1.01
Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder. Mol Ther (2011) 0.98
Magnetic resonance imaging-guided delivery of adeno-associated virus type 2 to the primate brain for the treatment of lysosomal storage disorders. Hum Gene Ther (2010) 0.97
Disrupting function of FK506-binding protein 1b/12.6 induces the Ca²+-dysregulation aging phenotype in hippocampal neurons. J Neurosci (2011) 0.95
A reversal learning task detects cognitive deficits in a Dachshund model of late-infantile neuronal ceroid lipofuscinosis. Genes Brain Behav (2011) 0.91
Experience, knowledge, and opinions about childhood genetic testing in Batten disease. Mol Genet Metab (2013) 0.87
Quantitative evaluation of monocyte transmigration into the brain following chemical opening of the blood-brain barrier in mice. Brain Res (2006) 0.86
Efficient gene delivery and selective transduction of astrocytes in the mammalian brain using viral vectors. Front Cell Neurosci (2013) 0.86
Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A. Mol Ther (2012) 0.84
Gene therapy for the neurological manifestations in lysosomal storage disorders. J Lipid Res (2014) 0.83
Imaging gene delivery in a mouse model of congenital neuronal ceroid lipofuscinosis. Gene Ther (2011) 0.81
Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy. Mol Ther (2013) 0.79
When ethics constrains clinical research: trial design of control arms in "greater than minimal risk" pediatric trials. Hum Gene Ther (2011) 0.79
Using Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3-Associated Retinal Degeneration. Hum Gene Ther (2016) 0.77
Assessment of disease severity in late infantile neuronal ceroid lipofuscinosis using multiparametric MR imaging. AJNR Am J Neuroradiol (2012) 0.77
Human serum albumin-based nanoparticle-mediated in vitro gene delivery. PLoS One (2014) 0.76
Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene. Hum Gene Ther (2014) 0.76
Vision loss in juvenile neuronal ceroid lipofuscinosis (CLN3 disease). Ann N Y Acad Sci (2016) 0.75
Moving forward toward a cure for Parkinson's: neuropathology of the nigrostriatal pathway determines the location of growth factor delivery. Mol Ther (2011) 0.75
Consideration of gene therapy for paediatric neurotransmitter diseases. J Inherit Metab Dis (2009) 0.75
Recruitment of stem and progenitor cells from the bone marrow niche requires MMP-9 mediated release of kit-ligand. Cell (2002) 9.36
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet (2007) 7.01
Earlier development of the accumbens relative to orbitofrontal cortex might underlie risk-taking behavior in adolescents. J Neurosci (2006) 5.85
Electrical stimulation of the anterior nucleus of thalamus for treatment of refractory epilepsy. Epilepsia (2010) 5.64
Cytokine-mediated deployment of SDF-1 induces revascularization through recruitment of CXCR4+ hemangiocytes. Nat Med (2006) 5.22
Chemokine-mediated interaction of hematopoietic progenitors with the bone marrow vascular niche is required for thrombopoiesis. Nat Med (2003) 4.69
Placental growth factor reconstitutes hematopoiesis by recruiting VEGFR1(+) stem cells from bone-marrow microenvironment. Nat Med (2002) 4.00
European citizens' use of E-health services: a study of seven countries. BMC Public Health (2007) 3.38
Risk-taking and the adolescent brain: who is at risk? Dev Sci (2007) 2.83
Pay for performance in critical care: an executive summary of the position paper by the Society of Critical Care Medicine. Crit Care Med (2009) 2.78
Endothelial-derived angiocrine signals induce and sustain regenerative lung alveolarization. Cell (2011) 2.42
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum Gene Ther (2008) 2.42
The subthalamic nucleus in the context of movement disorders. Brain (2003) 2.10
Low-dose acetazolamide reverses periventricular white matter hyperintensities in iNPH. Neurology (2014) 2.08
Population genetic structure of the people of Qatar. Am J Hum Genet (2010) 1.91
Impact of computerized prescriber order entry on the incidence of adverse drug events in pediatric inpatients. Pediatrics (2007) 1.79
Neurotrophins promote revascularization by local recruitment of TrkB+ endothelial cells and systemic mobilization of hematopoietic progenitors. J Clin Invest (2005) 1.74
Transcriptome analysis of the response of Pseudomonas aeruginosa to hydrogen peroxide. J Bacteriol (2004) 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
Generation of a functional and durable vascular niche by the adenoviral E4ORF1 gene. Proc Natl Acad Sci U S A (2008) 1.69
Possible axonal regrowth in late recovery from the minimally conscious state. J Clin Invest (2006) 1.64
Rapid/sustained anti-anthrax passive immunity mediated by co-administration of Ad/AAV. Mol Ther (2007) 1.57
Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations. J Neurosurg Pediatr (2010) 1.57
Down-regulation of the notch pathway in human airway epithelium in association with smoking and chronic obstructive pulmonary disease. Am J Respir Crit Care Med (2008) 1.57
Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice. Hum Gene Ther (2012) 1.55
Incidence and impact of adverse drug events in pediatric inpatients. Arch Pediatr Adolesc Med (2003) 1.52
Pseudomonas aeruginosa SoxR does not conform to the archetypal paradigm for SoxR-dependent regulation of the bacterial oxidative stress adaptive response. Infect Immun (2005) 1.48
Current medication practice and tracheal intubation safety outcomes from a prospective multicenter observational cohort study. Pediatr Crit Care Med (2015) 1.44
Gene expression profiling of human alveolar macrophages of phenotypically normal smokers and nonsmokers reveals a previously unrecognized subset of genes modulated by cigarette smoking. J Mol Med (Berl) (2006) 1.44
Adenovirus-mediated transfer of a minigene expressing multiple isoforms of VEGF is more effective at inducing angiogenesis than comparable vectors expressing individual VEGF cDNAs. Mol Ther (2004) 1.39
Transcriptome analysis of the Pseudomonas aeruginosa response to iron. Arch Microbiol (2003) 1.36
Protection against P. aeruginosa with an adenovirus vector containing an OprF epitope in the capsid. J Clin Invest (2005) 1.35
Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis. J Neurosci (2006) 1.33
Adenovirus E4 gene promotes selective endothelial cell survival and angiogenesis via activation of the vascular endothelial-cadherin/Akt signaling pathway. J Biol Chem (2003) 1.27
Neurotransplantation of fetal porcine cells in patients with basal ganglia infarcts: a preliminary safety and feasibility study. Cerebrovasc Dis (2005) 1.24
Coordinate control of expression of Nrf2-modulated genes in the human small airway epithelium is highly responsive to cigarette smoking. Mol Med (2009) 1.24
Osteogenic and Ewing sarcomas: estimation of necrotic fraction during induction chemotherapy with dynamic contrast-enhanced MR imaging. Radiology (2003) 1.22
Results of preoperative embolization for metastatic spinal neoplasms. J Neurosurg (2003) 1.19
Cocaine analog coupled to disrupted adenovirus: a vaccine strategy to evoke high-titer immunity against addictive drugs. Mol Ther (2011) 1.18
Novel influenza A(H1N1) in a pediatric health care facility in New York City during the first wave of the 2009 pandemic. Arch Pediatr Adolesc Med (2010) 1.18
Impaired sphingolipid synthesis in the respiratory tract induces airway hyperreactivity. Sci Transl Med (2013) 1.16
Safety and maximum tolerated dose of superselective intraarterial cerebral infusion of bevacizumab after osmotic blood-brain barrier disruption for recurrent malignant glioma. Clinical article. J Neurosurg (2010) 1.15
Epitopes expressed in different adenovirus capsid proteins induce different levels of epitope-specific immunity. J Virol (2006) 1.14
Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis. Exp Neurol (2008) 1.13
Induction of protective immunity to anthrax lethal toxin with a nonhuman primate adenovirus-based vaccine in the presence of preexisting anti-human adenovirus immunity. Infect Immun (2005) 1.13
Down-regulation of the canonical Wnt β-catenin pathway in the airway epithelium of healthy smokers and smokers with COPD. PLoS One (2011) 1.13
Replacing adenoviral vector HVR1 with a malaria B cell epitope improves immunogenicity and circumvents preexisting immunity to adenovirus in mice. J Clin Invest (2010) 1.12
Mortality rates in pediatric septic shock with and without multiple organ system failure. Pediatr Crit Care Med (2003) 1.11
Modification to the capsid of the adenovirus vector that enhances dendritic cell infection and transgene-specific cellular immune responses. J Virol (2004) 1.11
Mortality and functional morbidity after use of PALS/APLS by community physicians. Pediatrics (2009) 1.10
Quality control in microarray assessment of gene expression in human airway epithelium. BMC Genomics (2009) 1.10
Perfusion abnormalities in subchondral bone associated with marrow edema, osteoarthritis, and avascular necrosis. Ann N Y Acad Sci (2007) 1.09
Cigarette smoking induces small airway epithelial epigenetic changes with corresponding modulation of gene expression. Hum Mol Genet (2013) 1.08
Sensitivity of the nucleus accumbens to violations in expectation of reward. Neuroimage (2006) 1.07
Green fluorescent protein selectively induces HSP70-mediated up-regulation of COX-2 expression in endothelial cells. Blood (2003) 1.07
Novel cocaine vaccine linked to a disrupted adenovirus gene transfer vector blocks cocaine psychostimulant and reinforcing effects. Neuropsychopharmacology (2011) 1.07
Monoallelic up-regulation of the imprinted H19 gene in airway epithelium of phenotypically normal cigarette smokers. Cancer Res (2003) 1.07
Safety of direct administration of AAV2(CU)hCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates. Hum Gene Ther (2005) 1.07
Biologic phenotyping of the human small airway epithelial response to cigarette smoking. PLoS One (2011) 1.06
Proinflammatory phenotype and increased caveolin-1 in alveolar macrophages with silenced CFTR mRNA. PLoS One (2010) 1.05
Protective immunity to pseudomonas aeruginosa induced with a capsid-modified adenovirus expressing P. aeruginosa OprF. J Virol (2007) 1.04
Functional MRI of the zebra finch brain during song stimulation suggests a lateralized response topography. Proc Natl Acad Sci U S A (2007) 1.04
Protective immunity against respiratory tract challenge with Yersinia pestis in mice immunized with an adenovirus-based vaccine vector expressing V antigen. J Infect Dis (2006) 1.04
Protective immunity evoked against anthrax lethal toxin after a single intramuscular administration of an adenovirus-based vaccine encoding humanized protective antigen. Hum Gene Ther (2003) 1.04
Variability in small airway epithelial gene expression among normal smokers. Chest (2008) 1.02
Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders. Hum Gene Ther (2005) 1.02
Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis. Hum Gene Ther Methods (2012) 1.01
The cost benefit of electronic patient referrals in Denmark: summary report. Stud Health Technol Inform (2004) 1.00
Proceedings of the diffuse intrinsic pontine glioma (DIPG) Toronto Think Tank: advancing basic and translational research and cooperation in DIPG. J Neurooncol (2011) 1.00
Phenotypic features associated with mutations in steroidogenic acute regulatory protein. J Clin Endocrinol Metab (2005) 1.00
Transfer of the AQP1 cDNA for the correction of radiation-induced salivary hypofunction. Biochim Biophys Acta (2005) 0.99
Frameless stereotactic placement of depth electrodes in epilepsy surgery. J Neurosurg (2005) 0.98
Bilateral intracranial electrodes for lateralizing intractable epilepsy: efficacy, risk, and outcome. Neurosurgery (2010) 0.98
Genetic delivery of an anti-RSV antibody to protect against pulmonary infection with RSV. Virology (2008) 0.98
Smoking-induced upregulation of AKR1B10 expression in the airway epithelium of healthy individuals. Chest (2010) 0.98
Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing. Hum Gene Ther (2009) 0.97
Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway. Hum Mol Genet (2013) 0.96