Published in Nucleic Acids Res on November 15, 2003
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Amplification of repeat-containing transcribed sequences (ARTS): a transcriptome fingerprinting strategy to detect functionally relevant microsatellite mutations in cancer. Nucleic Acids Res (2003) 0.78
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol (2007) 9.23
Tie2 identifies a hematopoietic lineage of proangiogenic monocytes required for tumor vessel formation and a mesenchymal population of pericyte progenitors. Cancer Cell (2005) 7.90
An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol (2011) 6.44
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science (2013) 5.64
Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol (2006) 5.58
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol (2007) 5.36
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science (2013) 5.34
Endothelial progenitor cell derived microvesicles activate an angiogenic program in endothelial cells by a horizontal transfer of mRNA. Blood (2007) 4.73
Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells. Nat Med (2003) 4.02
Targeting the ANG2/TIE2 axis inhibits tumor growth and metastasis by impairing angiogenesis and disabling rebounds of proangiogenic myeloid cells. Cancer Cell (2011) 4.02
FcRgamma activation regulates inflammation-associated squamous carcinogenesis. Cancer Cell (2010) 3.80
Stable knockdown of microRNA in vivo by lentiviral vectors. Nat Methods (2008) 3.55
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The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J Clin Invest (2009) 3.34
Dopamine-modified alpha-synuclein blocks chaperone-mediated autophagy. J Clin Invest (2008) 3.23
Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat Med (2006) 3.22
Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications. Nat Rev Genet (2009) 3.09
Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters. Nat Biotechnol (2004) 2.78
Identification of proangiogenic TIE2-expressing monocytes (TEMs) in human peripheral blood and cancer. Blood (2007) 2.74
Forebrain ependymal cells are Notch-dependent and generate neuroblasts and astrocytes after stroke. Nat Neurosci (2009) 2.74
Cleavage of the plasma membrane Na+/Ca2+ exchanger in excitotoxicity. Cell (2005) 2.63
Targeting the tumor and its microenvironment by a dual-function decoy Met receptor. Cancer Cell (2004) 2.61
Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nat Methods (2011) 2.30
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood (2007) 2.28
Secretion of the adipocyte-specific secretory protein adiponectin critically depends on thiol-mediated protein retention. Mol Cell Biol (2007) 2.27
Visualization of dynamics of single endogenous mRNA labeled in live mouse. Science (2014) 2.20
Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med (2009) 2.19
Hepcidin as a therapeutic tool to limit iron overload and improve anemia in β-thalassemic mice. J Clin Invest (2010) 2.18
Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy. Blood (2009) 1.88
Tie2-expressing monocytes: regulation of tumor angiogenesis and therapeutic implications. Trends Immunol (2007) 1.84
Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci Transl Med (2010) 1.84
Generation of HIV-1 derived lentiviral vectors. Methods Enzymol (2002) 1.81
Tumor-targeted interferon-alpha delivery by Tie2-expressing monocytes inhibits tumor growth and metastasis. Cancer Cell (2008) 1.80
Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection. Blood (2011) 1.76
Systemic and targeted delivery of semaphorin 3A inhibits tumor angiogenesis and progression in mouse tumor models. Arterioscler Thromb Vasc Biol (2011) 1.75
Tie2-expressing monocytes and tumor angiogenesis: regulation by hypoxia and angiopoietin-2. Cancer Res (2007) 1.70
Elusive identities and overlapping phenotypes of proangiogenic myeloid cells in tumors. Am J Pathol (2010) 1.70
In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance. Blood (2007) 1.69
Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk. Hepatology (2011) 1.66
TIE2-expressing macrophages limit the therapeutic efficacy of the vascular-disrupting agent combretastatin A4 phosphate in mice. J Clin Invest (2011) 1.66
Generation of potent and stable human CD4+ T regulatory cells by activation-independent expression of FOXP3. Mol Ther (2007) 1.64
Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. J Clin Invest (2004) 1.59
Early cellular changes after blockage of chaperone-mediated autophagy. Autophagy (2008) 1.54
Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. J Clin Invest (2006) 1.54
Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. Blood (2004) 1.53
Human T lymphocytes transduced by lentiviral vectors in the absence of TCR activation maintain an intact immune competence. Blood (2003) 1.50
Repressor element-1 silencing transcription factor (REST)-dependent epigenetic remodeling is critical to ischemia-induced neuronal death. Proc Natl Acad Sci U S A (2012) 1.48
Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice. Blood (2012) 1.46
Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors. Mol Ther (2002) 1.41
Robust in vivo gene transfer into adult mammalian neural stem cells by lentiviral vectors. Proc Natl Acad Sci U S A (2004) 1.40
Cross platform microarray analysis for robust identification of differentially expressed genes. BMC Bioinformatics (2007) 1.39
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EphrinB reverse signaling contributes to endothelial and mural cell assembly into vascular structures. Blood (2009) 1.38
Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application. Hum Gene Ther (2011) 1.37
The MET oncogene drives a genetic programme linking cancer to haemostasis. Nature (2005) 1.37
The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related enhanced lysosomal activity. Neurobiol Dis (2012) 1.35
A genomic view of estrogen actions in human breast cancer cells by expression profiling of the hormone-responsive transcriptome. J Mol Endocrinol (2004) 1.35
Attenuation of miR-126 activity expands HSC in vivo without exhaustion. Cell Stem Cell (2012) 1.35
CD44v6-targeted T cells mediate potent antitumor effects against acute myeloid leukemia and multiple myeloma. Blood (2013) 1.34
In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance. Blood (2009) 1.34
The endogenous inhibitor of Akt, CTMP, is critical to ischemia-induced neuronal death. Nat Neurosci (2009) 1.33
Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. Blood (2010) 1.33
Genetic and expression analysis of MET, MACC1, and HGF in metastatic colorectal cancer: response to met inhibition in patient xenografts and pathologic correlations. Clin Cancer Res (2011) 1.32
Role of haematopoietic cells and endothelial progenitors in tumour angiogenesis. Biochim Biophys Acta (2006) 1.30
miR-511-3p modulates genetic programs of tumor-associated macrophages. Cell Rep (2012) 1.29
Novel somatic mutations of the MET oncogene in human carcinoma metastases activating cell motility and invasion. Cancer Res (2002) 1.26
Truncated RON tyrosine kinase drives tumor cell progression and abrogates cell-cell adhesion through E-cadherin transcriptional repression. Cancer Res (2004) 1.26
Regulated and multiple miRNA and siRNA delivery into primary cells by a lentiviral platform. Mol Ther (2009) 1.25
An uncleavable form of pro-scatter factor suppresses tumor growth and dissemination in mice. J Clin Invest (2004) 1.25
Inhibition of in vivo HIV infection in humanized mice by gene therapy of human hematopoietic stem cells with a lentiviral vector encoding a broadly neutralizing anti-HIV antibody. J Virol (2010) 1.24
Neural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy. Stem Cells (2011) 1.22
In vivo targeting of tumor endothelial cells by systemic delivery of lentiviral vectors. Hum Gene Ther (2003) 1.22
Mitochondrial succinate is instrumental for HIF1alpha nuclear translocation in SDHA-mutant fibroblasts under normoxic conditions. Hum Mol Genet (2005) 1.21
Hepatocyte transplantation-induced liver inflammation is driven by cytokines-chemokines associated with neutrophils and Kupffer cells. Gastroenterology (2009) 1.21
Stability of lentiviral vector-mediated transgene expression in the brain in the presence of systemic antivector immune responses. Hum Gene Ther (2005) 1.20
Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells. Mol Ther (2002) 1.18
Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome. Mol Ther (2012) 1.18
Genome-wide discovery of functional transcription factor binding sites by comparative genomics: the case of Stat3. Proc Natl Acad Sci U S A (2009) 1.16
Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction. Blood (2006) 1.16
A role for miR-155 in enabling tumor-infiltrating innate immune cells to mount effective antitumor responses in mice. Blood (2013) 1.16
Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models. Mol Ther (2009) 1.15
Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation. Proc Natl Acad Sci U S A (2012) 1.12
Phenotype reversion in fetal human liver epithelial cells identifies the role of an intermediate meso-endodermal stage before hepatic maturation. J Cell Sci (2008) 1.12
Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector. Hum Gene Ther (2005) 1.12
A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A. Mol Ther (2011) 1.11
Hepatic targeting of transplanted liver sinusoidal endothelial cells in intact mice. Hepatology (2005) 1.11
Reprogramming T lymphocytes for melanoma adoptive immunotherapy by T-cell receptor gene transfer with lentiviral vectors. Cancer Res (2009) 1.10
Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations. J Clin Invest (2012) 1.10
Concordant morphologic and gene expression data show that a vaccine halts HER-2/neu preneoplastic lesions. J Clin Invest (2004) 1.10