Published in Blood on November 12, 2004
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
Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications. Nat Rev Genet (2009) 3.09
DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer. Nucleic Acids Res (2008) 2.64
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J Clin Invest (2007) 2.63
KRAB-zinc finger proteins and KAP1 can mediate long-range transcriptional repression through heterochromatin spreading. PLoS Genet (2010) 2.44
Ex vivo gene transfer and correction for cell-based therapies. Nat Rev Genet (2011) 2.13
Analysis of lentiviral vector integration in HIV+ study subjects receiving autologous infusions of gene modified CD4+ T cells. Mol Ther (2009) 1.97
Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection. Nat Protoc (2007) 1.73
New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy. Hum Genet (2006) 1.65
Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. J Clin Invest (2006) 1.54
Engineered T cells: the promise and challenges of cancer immunotherapy. Nat Rev Cancer (2016) 1.51
Methods for integration site distribution analyses in animal cell genomes. Methods (2008) 1.21
Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction. Blood (2006) 1.16
Hematopoietic stem cell engineering at a crossroads. Blood (2011) 1.10
Predicting preferential DNA vector insertion sites: implications for functional genomics and gene therapy. Genome Biol (2007) 1.08
Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies. Curr Gene Ther (2011) 1.08
Efficient generation of integration-free ips cells from human adult peripheral blood using BCL-XL together with Yamanaka factors. PLoS One (2013) 1.06
Towards a clinically relevant lentiviral transduction protocol for primary human CD34 hematopoietic stem/progenitor cells. PLoS One (2009) 1.03
Unaltered repopulation properties of mouse hematopoietic stem cells transduced with lentiviral vectors. Blood (2008) 1.02
Efficient reprogramming of human cord blood CD34+ cells into induced pluripotent stem cells with OCT4 and SOX2 alone. Mol Ther (2011) 1.00
Porcine endogenous retrovirus integration sites in the human genome: features in common with those of murine leukemia virus. J Virol (2006) 0.99
Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression. Mol Ther (2010) 0.99
Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases. Stem Cells Transl Med (2014) 0.97
A gene-rich, transcriptionally active environment and the pre-deposition of repressive marks are predictive of susceptibility to KRAB/KAP1-mediated silencing. BMC Genomics (2011) 0.96
Intragenic transcriptional cis-activation of the human immunodeficiency virus 1 does not result in allele-specific inhibition of the endogenous gene. Retrovirology (2008) 0.94
Live and let die: in vivo selection of gene-modified hematopoietic stem cells via MGMT-mediated chemoprotection. DNA Repair (Amst) (2007) 0.92
Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons. Hum Gene Ther (2010) 0.92
Development of lentiviral gene therapy for Wiskott Aldrich syndrome. Expert Opin Biol Ther (2008) 0.91
Evaluation of residual promoter activity in γ-retroviral self-inactivating (SIN) vectors. Mol Ther (2011) 0.87
Gene therapy and peripheral nerve repair: a perspective. Front Mol Neurosci (2015) 0.84
Streamlined design of a self-inactivating feline immunodeficiency virus vector for transducing ex vivo dendritic cells and T lymphocytes. Genet Vaccines Ther (2007) 0.84
Reducing the genotoxic potential of retroviral vectors. Methods Mol Biol (2008) 0.84
Manipulating Immune Tolerance with Micro-RNA Regulated Gene Therapy. Front Microbiol (2011) 0.83
Correction of murine Bernard-Soulier syndrome by lentivirus-mediated gene therapy. Mol Ther (2011) 0.83
Viral determinants of integration site preferences of simian immunodeficiency virus-based vectors. J Virol (2006) 0.82
Lentiviral vectors for immune cells targeting. Immunopharmacol Immunotoxicol (2010) 0.81
Platelets as delivery systems for disease treatments. Adv Drug Deliv Rev (2010) 0.81
Human immunodeficiency virus (HIV)-1 integration sites in viral latency. Curr HIV/AIDS Rep (2015) 0.79
Imaging stem cell-derived persistent foci after in vivo selection of lentiviral MGMT-P140K transduced murine bone marrow cells. Mol Ther (2011) 0.79
Production of transgenic pigs mediated by pseudotyped lentivirus and sperm. PLoS One (2012) 0.79
The application of nanoparticles in gene therapy and magnetic resonance imaging. Microsc Res Tech (2011) 0.79
Pros and Cons of Antigen-Presenting Cell Targeted Tumor Vaccines. J Immunol Res (2015) 0.77
No impact of lentiviral transduction on hematopoietic stem/progenitor cell telomere length or gene expression in the rhesus macaque model. Mol Ther (2013) 0.76
Gene delivery to rat and human Schwann cells and nerve segments: a comparison of AAV 1-9 and lentiviral vectors. Gene Ther (2015) 0.76
Lentiviral vectors can be used for full-length dystrophin gene therapy. Sci Rep (2017) 0.75
Analysis of PBase Binding Profile Indicates an Insertion Target Selection Mechanism Dependent on TTAA, But Not Transcriptional Activity. Int J Biol Sci (2016) 0.75
Lentiviral vectors can be used for full-length dystrophin gene therapy. Sci Rep (2017) 0.75
Toward Personalized Gene Therapy: Characterizing the Host Genetic Control of Lentiviral-Vector-Mediated Hepatic Gene Delivery. Mol Ther Methods Clin Dev (2017) 0.75
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol (2007) 9.23
Emergence of KRAS mutations and acquired resistance to anti-EGFR therapy in colorectal cancer. Nature (2012) 8.77
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
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
A molecularly annotated platform of patient-derived xenografts ("xenopatients") identifies HER2 as an effective therapeutic target in cetuximab-resistant colorectal cancer. Cancer Discov (2011) 3.86
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
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
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
AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet (2003) 2.81
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
Induction of MET by ionizing radiation and its role in radioresistance and invasive growth of cancer. J Natl Cancer Inst (2011) 2.47
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
Comprehensive genomic access to vector integration in clinical gene therapy. Nat Med (2009) 2.19
Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver. J Virol (2005) 2.09
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
Kinetics of liver repopulation after bone marrow transplantation. Am J Pathol (2002) 1.85
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
PDGFR blockade is a rational and effective therapy for NPM-ALK-driven lymphomas. Nat Med (2012) 1.67
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
Replacement of normal with mutant alleles in the genome of normal human cells unveils mutation-specific drug responses. Proc Natl Acad Sci U S A (2008) 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
Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. J Clin Invest (2006) 1.54
Human T lymphocytes transduced by lentiviral vectors in the absence of TCR activation maintain an intact immune competence. Blood (2003) 1.50
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
FLAME, a novel fuzzy clustering method for the analysis of DNA microarray data. BMC Bioinformatics (2007) 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
HIV-based vectors. Preparation and use. Methods Mol Med (2002) 1.39
Wiskott-Aldrich syndrome protein deficiency in natural killer and dendritic cells affects antitumor immunity. Eur J Immunol (2014) 1.39
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
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
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
Gene expression profiling uncovers molecular classifiers for the recognition of anaplastic large-cell lymphoma within peripheral T-cell neoplasms. J Clin Oncol (2010) 1.31
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
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
A novel role of the interferon-inducible protein IFI16 as inducer of proinflammatory molecules in endothelial cells. J Biol Chem (2007) 1.23
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
The MET oncogene is a functional marker of a glioblastoma stem cell subtype. Cancer Res (2012) 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
Formalin fixation at low temperature better preserves nucleic acid integrity. PLoS One (2011) 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
Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction. Blood (2006) 1.16
Stimulation of the neurotrophin receptor TrkB on astrocytes drives nitric oxide production and neurodegeneration. J Exp Med (2012) 1.15
A molecular signature for Epithelial to Mesenchymal transition in a human colon cancer cell system is revealed by large-scale microarray analysis. Clin Exp Metastasis (2009) 1.15
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
Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector. Hum Gene Ther (2005) 1.12
Clonal evolution and resistance to EGFR blockade in the blood of colorectal cancer patients. Nat Med (2015) 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
Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations. J Clin Invest (2012) 1.10