selected publications
- Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47. EMBO molecular medicine. 2024 Academic Article GET IT
- Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-modified AAV Vector. Human gene therapy. 2024 Academic Article GET IT
- Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy. Molecular therapy : the journal of the American Society of Gene Therapy. 2023 Academic Article GET IT
- Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors. Molecular therapy. Methods & clinical development. 2023 Academic Article GET IT
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Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2.
Science translational medicine.
2020
Academic Article
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Times cited: 46 -
Correction: Type 2 Diabetes Risk Allele Loci in the Qatari Population.
2016
GET IT
Times cited: 1 -
Type 2 Diabetes Risk Allele Loci in the Qatari Population.
PloS one.
2016
Academic Article
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Times cited: 23 -
Genetic modification of neurons to express bevacizumab for local anti-angiogenesis treatment of glioblastoma.
Cancer gene therapy.
2014
Academic Article
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Times cited: 18 -
Smoking accelerates aging of the small airway epithelium.
Respiratory research.
2014
Academic Article
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Times cited: 62 -
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.
Human gene therapy.
2014
Academic Article
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Times cited: 34 -
Smoking dysregulates the human airway basal cell transcriptome at COPD risk locus 19q13.2.
PloS one.
2014
Academic Article
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Times cited: 60 -
HEFT: eQTL analysis of many thousands of expressed genes while simultaneously controlling for hidden factors.
Bioinformatics (Oxford, England).
2013
Academic Article
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Times cited: 17 -
Intrapleural administration of an AAVrh.10 vector coding for human α1-antitrypsin for the treatment of α1-antitrypsin deficiency.
Human gene therapy. Clinical development.
2013
Academic Article
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Times cited: 39 -
Exome sequencing identifies potential risk variants for Mendelian disorders at high prevalence in Qatar.
Human mutation.
2013
Academic Article
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Times cited: 43 -
Prevalence of the apolipoprotein E Arg145Cys dyslipidemia at-risk polymorphism in African-derived populations.
The American journal of cardiology.
2013
Academic Article
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Times cited: 14 -
Airway basal cells of healthy smokers express an embryonic stem cell signature relevant to lung cancer.
Stem cells (Dayton, Ohio).
2013
Academic Article
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Times cited: 28 -
Spectrum of ocular manifestations in CLN2-associated batten (Jansky-Bielschowsky) disease correlate with advancing age and deteriorating neurological function.
PloS one.
2013
Academic Article
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Times cited: 37 -
RGD capsid modification enhances mucosal protective immunity of a non-human primate adenovirus vector expressing Pseudomonas aeruginosa OprF.
Clinical and experimental immunology.
2013
Academic Article
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Times cited: 18 -
Cigarette smoking induces small airway epithelial epigenetic changes with corresponding modulation of gene expression.
Human molecular genetics.
2013
Academic Article
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Times cited: 90 -
RFX3 modulation of FOXJ1 regulation of cilia genes in the human airway epithelium.
Respiratory research.
2013
Academic Article
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Times cited: 65 -
Safety of direct cardiac administration of AdVEGF-All6A+, a replication-deficient adenovirus vector cDNA/genomic hybrid expressing all three major isoforms of human vascular endothelial growth factor, to the ischemic myocardium of rats.
Human gene therapy. Clinical development.
2013
Academic Article
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Times cited: 9 -
In vivo cardiac cellular reprogramming efficacy is enhanced by angiogenic preconditioning of the infarcted myocardium with vascular endothelial growth factor.
Journal of the American Heart Association.
2012
Academic Article
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Times cited: 116 -
Exome sequencing of only seven Qataris identifies potentially deleterious variants in the Qatari population.
PloS one.
2012
Academic Article
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Times cited: 16 -
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.
Human gene therapy methods.
2012
Academic Article
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Times cited: 84 -
Assessment of disease severity in late infantile neuronal ceroid lipofuscinosis using multiparametric MR imaging.
AJNR. American journal of neuroradiology.
2012
Academic Article
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Times cited: 17 -
Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.
Human gene therapy.
2012
Academic Article
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Times cited: 73 -
AAV-directed persistent expression of a gene encoding anti-nicotine antibody for smoking cessation.
Science translational medicine.
2012
Academic Article
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Times cited: 47 -
Inferring genome-wide patterns of admixture in Qataris using fifty-five ancestral populations.
BMC genetics.
2012
Academic Article
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Times cited: 49 -
Empirical Bayes conditional independence graphs for regulatory network recovery.
Bioinformatics (Oxford, England).
2012
Academic Article
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Times cited: 5 -
AAVrh.10-mediated expression of an anti-cocaine antibody mediates persistent passive immunization that suppresses cocaine-induced behavior.
Human gene therapy.
2012
Academic Article
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Times cited: 38 -
Airway basal cell vascular endothelial growth factor-mediated cross-talk regulates endothelial cell-dependent growth support of human airway basal cells.
Cellular and molecular life sciences : CMLS.
2012
Academic Article
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Times cited: 22 -
RNA-Seq quantification of the human small airway epithelium transcriptome.
BMC genomics.
2012
Academic Article
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Times cited: 96 -
Persistent suppression of ocular neovascularization with intravitreal administration of AAVrh.10 coding for bevacizumab.
Human gene therapy.
2011
Academic Article
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Times cited: 20 -
Biologic phenotyping of the human small airway epithelial response to cigarette smoking.
PloS one.
2011
Academic Article
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Times cited: 63 -
The human airway epithelial basal cell transcriptome.
PloS one.
2011
Academic Article
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Times cited: 154 -
Down-regulation of the canonical Wnt β-catenin pathway in the airway epithelium of healthy smokers and smokers with COPD.
PloS one.
2011
Academic Article
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Times cited: 89 -
Glutathione S-transferase copy number variation alters lung gene expression.
The European respiratory journal.
2011
Academic Article
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Times cited: 18 -
Modulation of cystatin A expression in human airway epithelium related to genotype, smoking, COPD, and lung cancer.
Cancer research.
2011
Academic Article
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Times cited: 35 -
Cocaine analog coupled to disrupted adenovirus: a vaccine strategy to evoke high-titer immunity against addictive drugs.
Molecular therapy : the journal of the American Society of Gene Therapy.
2011
Academic Article
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Times cited: 56 -
Smoking-mediated up-regulation of GAD67 expression in the human airway epithelium.
Respiratory research.
2010
Academic Article
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Times cited: 21 -
Smoking-induced upregulation of AKR1B10 expression in the airway epithelium of healthy individuals.
Chest.
2010
Academic Article
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Times cited: 47 -
Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations.
Journal of neurosurgery. Pediatrics.
2010
Academic Article
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Times cited: 59 -
Protective immunity against a lethal respiratory Yersinia pestis challenge induced by V antigen or the F1 capsular antigen incorporated into adenovirus capsid.
Human gene therapy.
2010
Academic Article
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Times cited: 31 -
Population genetic structure of the people of Qatar.
American journal of human genetics.
2010
Academic Article
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Times cited: 92 -
Persistent expression of biologically active anti-HER2 antibody by AAVrh.10-mediated gene transfer.
Cancer gene therapy.
2010
Academic Article
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Times cited: 22 -
Disparate oxidant gene expression of airway epithelium compared to alveolar macrophages in smokers.
Respiratory research.
2009
Academic Article
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Times cited: 19 -
Identification of an exonic splicing silencer in exon 6A of the human VEGF gene.
BMC molecular biology.
2009
Academic Article
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Times cited: 7 -
Quality control in microarray assessment of gene expression in human airway epithelium.
BMC genomics.
2009
Academic Article
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Times cited: 73 -
Coordinate control of expression of Nrf2-modulated genes in the human small airway epithelium is highly responsive to cigarette smoking.
Molecular medicine (Cambridge, Mass.).
2009
Academic Article
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Times cited: 81 -
Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing.
Human gene therapy.
2009
Academic Article
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Times cited: 54 -
Emphysema mediated by lung overexpression of ADAM10.
Clinical and translational science.
2009
Academic Article
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Times cited: 9 -
Adenovirus-mediated delivery of an anti-V antigen monoclonal antibody protects mice against a lethal Yersinia pestis challenge.
Infection and immunity.
2009
Academic Article
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Times cited: 30 -
Down-regulation of the notch pathway in human airway epithelium in association with smoking and chronic obstructive pulmonary disease.
American journal of respiratory and critical care medicine.
2008
Academic Article
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Times cited: 158 -
Generation of a functional and durable vascular niche by the adenoviral E4ORF1 gene.
Proceedings of the National Academy of Sciences of the United States of America.
2008
Academic Article
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Times cited: 118 -
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.
Human gene therapy.
2008
Academic Article
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Times cited: 295 -
Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis.
Experimental neurology.
2008
Academic Article
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Times cited: 60 -
Variability in small airway epithelial gene expression among normal smokers.
Chest.
2008
Academic Article
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Times cited: 46 -
Genetic delivery of the murine equivalent of bevacizumab (avastin), an anti-vascular endothelial growth factor monoclonal antibody, to suppress growth of human tumors in immunodeficient mice.
Human gene therapy.
2008
Academic Article
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Times cited: 13 -
Rapid/sustained anti-anthrax passive immunity mediated by co-administration of Ad/AAV.
Molecular therapy : the journal of the American Society of Gene Therapy.
2007
Academic Article
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Times cited: 36 -
Ablation of tumor-derived stem cells transplanted to the central nervous system by genetic modification of embryonic stem cells with a suicide gene.
Human gene therapy.
2007
Academic Article
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Times cited: 38 -
Protective immunity to pseudomonas aeruginosa induced with a capsid-modified adenovirus expressing P. aeruginosa OprF.
Journal of virology.
2007
Academic Article
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Times cited: 54 -
Neurological deterioration in late infantile neuronal ceroid lipofuscinosis.
Neurology.
2007
Academic Article
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Times cited: 69 -
Assessing disease severity in late infantile neuronal ceroid lipofuscinosis using quantitative MR diffusion-weighted imaging.
AJNR. American journal of neuroradiology.
2007
Academic Article
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Times cited: 25 -
Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.
Molecular therapy : the journal of the American Society of Gene Therapy.
2006
Academic Article
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Times cited: 125 -
Protective immunity against respiratory tract challenge with Yersinia pestis in mice immunized with an adenovirus-based vaccine vector expressing V antigen.
The Journal of infectious diseases.
2006
Academic Article
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Times cited: 43 -
Epitopes expressed in different adenovirus capsid proteins induce different levels of epitope-specific immunity.
Journal of virology.
2006
Academic Article
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Times cited: 62 -
Cytokine-mediated deployment of SDF-1 induces revascularization through recruitment of CXCR4+ hemangiocytes.
Nature medicine.
2006
Academic Article
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Times cited: 577 -
Gene expression profiling of human alveolar macrophages of phenotypically normal smokers and nonsmokers reveals a previously unrecognized subset of genes modulated by cigarette smoking.
Journal of molecular medicine (Berlin, Germany).
2006
Academic Article
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Times cited: 78 -
Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.
The Journal of neuroscience : the official journal of the Society for Neuroscience.
2006
Academic Article
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Times cited: 117 -
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.
Human gene therapy.
2005
Academic Article
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Times cited: 39 -
High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses.
Molecular therapy : the journal of the American Society of Gene Therapy.
2005
Academic Article
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Times cited: 98 -
AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL.
Gene therapy.
2005
Academic Article
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Times cited: 58 -
Alteration of splicing signals in a genomic/cDNA hybrid VEGF gene to modify the ratio of expressed VEGF isoforms enhances safety of angiogenic gene therapy.
Molecular therapy : the journal of the American Society of Gene Therapy.
2005
Academic Article
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Times cited: 25 -
Induction of protective immunity to anthrax lethal toxin with a nonhuman primate adenovirus-based vaccine in the presence of preexisting anti-human adenovirus immunity.
Infection and immunity.
2005
Academic Article
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Times cited: 52 -
Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders.
Human gene therapy.
2005
Review
GET IT
Times cited: 27 -
Thrombopoietin gene transfer-mediated enhancement of angiogenic responses to acute ischemia.
Circulation research.
2005
Academic Article
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Times cited: 23 -
Adenovirus vector E4 gene regulates connexin 40 and 43 expression in endothelial cells via PKA and PI3K signal pathways.
Circulation research.
2005
Academic Article
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Times cited: 21 -
Protection against P. aeruginosa with an adenovirus vector containing an OprF epitope in the capsid.
The Journal of clinical investigation.
2005
Academic Article
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Times cited: 96 -
Neurotrophins promote revascularization by local recruitment of TrkB+ endothelial cells and systemic mobilization of hematopoietic progenitors.
The Journal of clinical investigation.
2005
Academic Article
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Times cited: 217 -
Passive immunotherapy for anthrax toxin mediated by an adenovirus expressing an anti-protective antigen single-chain antibody.
Molecular therapy : the journal of the American Society of Gene Therapy.
2005
Academic Article
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Times cited: 26 -
Intrapleural administration of a serotype 5 adeno-associated virus coding for alpha1-antitrypsin mediates persistent, high lung and serum levels of alpha1-antitrypsin.
Molecular therapy : the journal of the American Society of Gene Therapy.
2004
Academic Article
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Times cited: 39 -
Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis.
Human gene therapy.
2004
Academic Article
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Times cited: 100 -
Coexpression of herpesviral thymidine kinase reporter gene and VEGF gene for noninvasive monitoring of therapeutic gene transfer: an in vitro evaluation.
Journal of nuclear medicine : official publication, Society of Nuclear Medicine.
2004
Academic Article
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Times cited: 18 -
Modification to the capsid of the adenovirus vector that enhances dendritic cell infection and transgene-specific cellular immune responses.
Journal of virology.
2004
Academic Article
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Times cited: 76 -
Adenovirus-mediated transfer of a minigene expressing multiple isoforms of VEGF is more effective at inducing angiogenesis than comparable vectors expressing individual VEGF cDNAs.
Molecular therapy : the journal of the American Society of Gene Therapy.
2004
Academic Article
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Times cited: 46 -
Chemokine-mediated interaction of hematopoietic progenitors with the bone marrow vascular niche is required for thrombopoiesis.
Nature medicine.
2003
Academic Article
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Times cited: 598 -
Adenovirus E4 gene promotes selective endothelial cell survival and angiogenesis via activation of the vascular endothelial-cadherin/Akt signaling pathway.
The Journal of biological chemistry.
2003
Academic Article
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Times cited: 38 -
In vivo trans-splicing of 5' and 3' segments of pre-mRNA directed by corresponding DNA sequences delivered by gene transfer.
Molecular therapy : the journal of the American Society of Gene Therapy.
2003
Academic Article
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Times cited: 27 -
Protective immunity evoked against anthrax lethal toxin after a single intramuscular administration of an adenovirus-based vaccine encoding humanized protective antigen.
Human gene therapy.
2003
Academic Article
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Times cited: 58 -
Sampling-dependent up-regulation of gene expression in sequential samples of human airway epithelial cells.
Molecular medicine (Cambridge, Mass.).
2003
Academic Article
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Times cited: 9 -
Green fluorescent protein selectively induces HSP70-mediated up-regulation of COX-2 expression in endothelial cells.
Blood.
2003
Academic Article
GET IT
Times cited: 46 -
Variability of antioxidant-related gene expression in the airway epithelium of cigarette smokers.
American journal of respiratory cell and molecular biology.
2003
Academic Article
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Times cited: 174 -
Monoallelic up-regulation of the imprinted H19 gene in airway epithelium of phenotypically normal cigarette smokers.
Cancer research.
2003
Academic Article
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Times cited: 51 -
Placental growth factor reconstitutes hematopoiesis by recruiting VEGFR1(+) stem cells from bone-marrow microenvironment.
Nature medicine.
2002
Academic Article
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Times cited: 566 -
Gene therapy with adenovirus-mediated myocardial transfer of vascular endothelial growth factor 121 improves cardiac performance in a pacing model of congestive heart failure.
The Journal of thoracic and cardiovascular surgery.
2002
Academic Article
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Times cited: 37 -
Recruitment of stem and progenitor cells from the bone marrow niche requires MMP-9 mediated release of kit-ligand.
Cell.
2002
Academic Article
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Times cited: 1549 - Adenovirus vector-mediated transfer of the vascular endothelial growth factor cDNA to healing abdominal fascia enhances vascularity and bursting strength in mice with normal and impaired wound healing. Surgery. 2002 Academic Article GET IT
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Intratumoral administration of low doses of an adenovirus vector encoding tumor necrosis factor alpha together with naive dendritic cells elicits significant suppression of tumor growth without toxicity.
Human gene therapy.
2001
Academic Article
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Times cited: 37 -
Feasibility of gene therapy for late neuronal ceroid lipofuscinosis.
Archives of neurology.
2001
Information Resource
GET IT
Times cited: 24 -
Impaired recruitment of bone-marrow-derived endothelial and hematopoietic precursor cells blocks tumor angiogenesis and growth.
Nature medicine.
2001
Academic Article
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Times cited: 1638 -
Cross-strain protection against clinical and laboratory strains of Pseudomonas aeruginosa mediated by dendritic cells genetically modified to express CD40 ligand and pulsed with specific strains of Pseudomonas aeruginosa.
Human gene therapy.
2001
Academic Article
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Times cited: 11 -
Plasma elevation of stromal cell-derived factor-1 induces mobilization of mature and immature hematopoietic progenitor and stem cells.
Blood.
2001
Academic Article
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Times cited: 451 -
Infection of endothelium with E1(-)E4(+), but not E1(-)E4(-), adenovirus gene transfer vectors enhances leukocyte adhesion and migration by modulation of ICAM-1, VCAM-1, CD34, and chemokine expression.
Circulation research.
2001
Academic Article
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Times cited: 27 -
Vascular endothelial growth factor and angiopoietin-1 stimulate postnatal hematopoiesis by recruitment of vasculogenic and hematopoietic stem cells.
The Journal of experimental medicine.
2001
Academic Article
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Times cited: 621 -
Host responses and persistence of vector genome following intrabronchial administration of an E1(-)E3(-) adenovirus gene transfer vector to normal individuals.
Molecular therapy : the journal of the American Society of Gene Therapy.
2001
Academic Article
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Times cited: 27 -
Percutaneous endocardial transfer and expression of genes to the myocardium utilizing fluoroscopic guidance.
Catheterization and cardiovascular interventions : official journal of the Society for Cardiac Angiography & Interventions.
2001
Academic Article
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Times cited: 26 -
Use of quantitative TaqMan real-time PCR to track the time-dependent distribution of gene transfer vectors in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy.
2000
Academic Article
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Times cited: 46 -
Adenoviral vector-mediated gene transfer to primitive human hematopoietic progenitor cells: assessment of transduction and toxicity in long-term culture.
Blood.
2000
Academic Article
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Times cited: 24 -
Free cholesterol enhances adenoviral vector gene transfer and expression in CAR-deficient cells.
Molecular therapy : the journal of the American Society of Gene Therapy.
2000
Academic Article
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Times cited: 35 -
Granulocyte-macrophage colony-stimulating factor gene transfer to dendritic cells or epidermal cells augments their antigen-presenting function including induction of anti-tumor immunity.
The Journal of investigative dermatology.
1999
Academic Article
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Times cited: 22 -
Induction of endogenous genes following infection of human endothelial cells with an E1(-) E4(+) adenovirus gene transfer vector.
Journal of virology.
1999
Academic Article
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Times cited: 28 -
Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus.
The Journal of clinical investigation.
1999
Academic Article
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Times cited: 208 -
Angiogenesis gene therapy: phase I assessment of direct intramyocardial administration of an adenovirus vector expressing VEGF121 cDNA to individuals with clinically significant severe coronary artery disease.
Circulation.
1999
Academic Article
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Times cited: 590 -
Variability of human systemic humoral immune responses to adenovirus gene transfer vectors administered to different organs.
Journal of virology.
1999
Academic Article
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Times cited: 135 -
Fiber swap between adenovirus subgroups B and C alters intracellular trafficking of adenovirus gene transfer vectors.
Journal of virology.
1999
Academic Article
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Times cited: 135 -
Safety of direct myocardial administration of an adenovirus vector encoding vascular endothelial growth factor 121.
Human gene therapy.
1999
Academic Article
GET IT
Times cited: 47 -
Modification of the genetic program of human alveolar macrophages by adenovirus vectors in vitro is feasible but inefficient, limited in part by the low level of expression of the coxsackie/adenovirus receptor.
American journal of respiratory cell and molecular biology.
1999
Academic Article
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Times cited: 71 -
CAR-dependent and CAR-independent pathways of adenovirus vector-mediated gene transfer and expression in human fibroblasts.
The Journal of clinical investigation.
1999
Academic Article
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Times cited: 194 -
Selective expansion of alveolar macrophages in vivo by adenovirus-mediated transfer of the murine granulocyte-macrophage colony-stimulating factor cDNA.
Blood.
1999
Academic Article
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Times cited: 37 -
Reversal of CPT-11 resistance of lung cancer cells by adenovirus-mediated gene transfer of the human carboxylesterase cDNA.
Cancer research.
1998
Academic Article
GET IT
Times cited: 37 -
High-efficiency gene transfer into ex vivo expanded human hematopoietic progenitors and precursor cells by adenovirus vectors.
Blood.
1998
Academic Article
GET IT
Times cited: 73 -
In vivo human carboxylesterase cDNA gene transfer to activate the prodrug CPT-11 for local treatment of solid tumors.
The Journal of clinical investigation.
1998
Academic Article
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Times cited: 74 -
Regional suppression of tumor growth by in vivo transfer of a cDNA encoding a secreted form of the extracellular domain of the flt-1 vascular endothelial growth factor receptor.
Human gene therapy.
1998
Academic Article
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Times cited: 172 -
Regional 'pro-drug' gene therapy: intravenous administration of an adenoviral vector expressing the E. coli cytosine deaminase gene and systemic administration of 5-fluorocytosine suppresses growth of hepatic metastasis of colon carcinoma.
Gene therapy.
1998
Academic Article
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Times cited: 76 -
Fluorescent virions: dynamic tracking of the pathway of adenoviral gene transfer vectors in living cells.
Human gene therapy.
1998
Academic Article
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Times cited: 205 -
Biologic bypass with the use of adenovirus-mediated gene transfer of the complementary deoxyribonucleic acid for vascular endothelial growth factor 121 improves myocardial perfusion and function in the ischemic porcine heart.
The Journal of thoracic and cardiovascular surgery.
1998
Academic Article
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Times cited: 294 -
Circumvention of anti-adenovirus neutralizing immunity by administration of an adenoviral vector of an alternate serotype.
Human gene therapy.
1997
Academic Article
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Times cited: 171