Published in Mol Ther on November 04, 2005
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Anatomically shaped tissue-engineered cartilage with tunable and inducible anticytokine delivery for biological joint resurfacing. Proc Natl Acad Sci U S A (2016) 1.51
Bone marrow stem cells expressing keratinocyte growth factor via an inducible lentivirus protects against bleomycin-induced pulmonary fibrosis. PLoS One (2009) 1.15
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The development of a novel cancer immunotherapeutic platform using tumor-targeting mesenchymal stem cells and a protein vaccine. Mol Ther (2011) 0.79
Tetracycline regulated systems in functional oncogenomics. Transl Oncogenomics (2007) 0.79
The transgenic cloned pig population with integrated and controllable GH expression that has higher feed efficiency and meat production. Sci Rep (2015) 0.78
Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compounds. Dis Model Mech (2017) 0.77
Tightly regulated 'all-in-one' lentiviral vectors for protection of human hematopoietic cells from anticancer chemotherapy. Gene Ther (2015) 0.77
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GFI1 is repressed by p53 and inhibits DNA damage-induced apoptosis. PLoS One (2013) 0.76
Enhanced MyoD-induced transdifferentiation to a myogenic lineage by fusion to a potent transactivation domain. ACS Synth Biol (2014) 0.75
A Tet-on and Cre-loxP Based Genetic Engineering System for Convenient Recycling of Selection Markers in Penicillium oxalicum. Front Microbiol (2016) 0.75
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Tissue-resident macrophages self-maintain locally throughout adult life with minimal contribution from circulating monocytes. Immunity (2013) 5.47
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Blood-derived dermal langerin+ dendritic cells survey the skin in the steady state. J Exp Med (2007) 3.64
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science (2004) 2.71
CD169⁺ macrophages provide a niche promoting erythropoiesis under homeostasis and stress. Nat Med (2013) 2.26
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Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy. Hum Mutat (2007) 1.89
Pretransplant CSF-1 therapy expands recipient macrophages and ameliorates GVHD after allogeneic hematopoietic cell transplantation. J Exp Med (2011) 1.78
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Clonal architecture of chronic myelomonocytic leukemias. Blood (2013) 1.67
Cross-presenting CD103+ dendritic cells are protected from influenza virus infection. J Clin Invest (2012) 1.57
Human endothelial cells derived from circulating progenitors display specific functional properties compared with mature vessel wall endothelial cells. Blood (2003) 1.49
AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis. Hum Mol Genet (2008) 1.44
FLI1 monoallelic expression combined with its hemizygous loss underlies Paris-Trousseau/Jacobsen thrombopenia. J Clin Invest (2004) 1.39
Thrombocytopenia resulting from mutations in filamin A can be expressed as an isolated syndrome. Blood (2011) 1.37
Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece. Am J Med Genet A (2011) 1.26
Human CD1c+ dendritic cells drive the differentiation of CD103+ CD8+ mucosal effector T cells via the cytokine TGF-β. Immunity (2013) 1.23
RUNX1-induced silencing of non-muscle myosin heavy chain IIB contributes to megakaryocyte polyploidization. Nat Commun (2012) 1.22
Systemic analysis of PPARγ in mouse macrophage populations reveals marked diversity in expression with critical roles in resolution of inflammation and airway immunity. J Immunol (2012) 1.21
Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial. Hum Gene Ther (2014) 1.19
The origin of phenotypic heterogeneity in a clonal cell population in vitro. PLoS One (2007) 1.16
Histidine-rich amphipathic peptide antibiotics promote efficient delivery of DNA into mammalian cells. Proc Natl Acad Sci U S A (2003) 1.13
Phenotypic correction of alpha-sarcoglycan deficiency by intra-arterial injection of a muscle-specific serotype 1 rAAV vector. Mol Ther (2007) 1.13
Epigenetic gene expression noise and phenotypic diversification of clonal cell populations. Differentiation (2007) 1.12
Internal ribosome entry site structural motifs conserved among mammalian fibroblast growth factor 1 alternatively spliced mRNAs. Mol Cell Biol (2004) 1.09
Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases. Nucleic Acids Res (2012) 1.09
Direct recognition by alphabeta cytolytic T cells of Hfe, a MHC class Ib molecule without antigen-presenting function. Proc Natl Acad Sci U S A (2005) 1.05
Evidence for a resident subset of cells with SP phenotype in the C2C12 myogenic line: a tool to explore muscle stem cell biology. Exp Cell Res (2004) 1.05
Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles. Mol Ther (2008) 1.04
Synthesis of linear polyethylenimine derivatives for DNA transfection. Bioconjug Chem (2003) 1.02
CD4+CD25+ regulatory T cells inhibit immune-mediated transgene rejection. Blood (2003) 1.01
SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa. Mol Ther (2010) 1.01
Genetic correction of canine dystrophic epidermolysis bullosa mediated by retroviral vectors. Hum Mol Genet (2003) 1.01
Role for the nuclear factor kappaB pathway in transforming growth factor-beta1 production in idiopathic myelofibrosis: possible relationship with FK506 binding protein 51 overexpression. Cancer Res (2005) 1.00
Crosstalk between NOTCH and AKT signaling during murine megakaryocyte lineage specification. Blood (2011) 1.00
The antibiotic and DNA-transfecting peptide LAH4 selectively associates with, and disorders, anionic lipids in mixed membranes. FASEB J (2005) 0.99
Use of a lentiviral flap vector for induction of CTL immunity against melanoma. Perspectives for immunotherapy. J Gene Med (2002) 0.99
Efficient gene targeting mediated by a lentiviral vector-associated meganuclease. Nucleic Acids Res (2011) 0.98
Quantitative proteomic analysis of lentiviral vectors using 2-DE. Proteomics (2009) 0.98
Congenital disorders of glycosylation type Ig is defined by a deficiency in dolichyl-P-mannose:Man7GlcNAc2-PP-dolichyl mannosyltransferase. J Biol Chem (2002) 0.98
RNA interference shows critical requirement for NF-kappa B p50 in the production of IL-12 by human dendritic cells. J Immunol (2003) 0.97
Comparative analysis of the CD8(+) T cell repertoires of H-2 class I wild-type/HLA-A2.1 and H-2 class I knockout/HLA-A2.1 transgenic mice. Int Immunol (2002) 0.96
Dysmegakaryopoiesis of FPD/AML pedigrees with constitutional RUNX1 mutations is linked to myosin II deregulated expression. Blood (2012) 0.96
A partial down-regulation of WASP is sufficient to inhibit podosome formation in dendritic cells. Mol Ther (2005) 0.95
Long-term immune reconstitution in RAG-1-deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity. Blood (2005) 0.95
Intranasal gene delivery with a polyethylenimine-PEG conjugate. J Control Release (2002) 0.95
DUX4 and DUX4 downstream target genes are expressed in fetal FSHD muscles. Hum Mol Genet (2013) 0.94
The cationic amphipathic alpha-helix of HIV-1 viral protein R (Vpr) binds to nucleic acids, permeabilizes membranes, and efficiently transfects cells. J Biol Chem (2003) 0.93
Optimising histidine rich peptides for efficient DNA delivery in the presence of serum. J Control Release (2006) 0.93
Mannosidase I inhibition rescues the human alpha-sarcoglycan R77C recurrent mutation. Hum Mol Genet (2008) 0.90
Femoral intra-arterial injection: a tool to deliver and assess recombinant AAV constructs in rodents whole hind limb. J Gene Med (2005) 0.90
Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency. Blood (2002) 0.90
Presence of a defect in karyokinesis during megakaryocyte endomitosis. Cell Cycle (2012) 0.89
Global transcriptional characterization of SP and MP cells from the myogenic C2C12 cell line: effect of FGF6. Physiol Genomics (2005) 0.89
Tetracycline transcriptional silencer tightly controls transgene expression after in vivo intramuscular electrotransfer: application to interleukin 10 therapy in experimental arthritis. Hum Gene Ther (2002) 0.88
Diverse genomic integration of a lentiviral vector developed for the treatment of Wiskott-Aldrich syndrome. J Gene Med (2009) 0.88
Characterization of the gene transfer process mediated by histidine-rich peptides. J Mol Med (Berl) (2006) 0.87
Cationic steroid antibiotics demonstrate DNA delivery properties. J Control Release (2005) 0.87
A deficiency in dolichyl-P-glucose:Glc1Man9GlcNAc2-PP-dolichyl alpha3-glucosyltransferase defines a new subtype of congenital disorders of glycosylation. J Biol Chem (2002) 0.86
Dilution of reporter gene with stuffer DNA does not alter the transfection efficiency of polyethylenimines. J Gene Med (2005) 0.86
Comparative proteomic analysis of HIV-1 particles reveals a role for Ezrin and EHD4 in the Nef-dependent increase of virus infectivity. J Virol (2013) 0.86
Assessment of optimal transduction of primary human skin keratinocytes by viral vectors. J Gene Med (2005) 0.86
Safety and efficacy of AAV-mediated calpain 3 gene transfer in a mouse model of limb-girdle muscular dystrophy type 2A. Mol Ther (2005) 0.85
Langerhans cell homeostasis and turnover after nonmyeloablative and myeloablative allogeneic hematopoietic cell transplantation. Transplantation (2014) 0.85
Combination of quantification and observation methods for study of "Side Population" cells in their "in vitro" microenvironment. Cytometry A (2007) 0.85
Safe selection of genetically manipulated human primary keratinocytes with very high growth potential using CD24. Mol Ther (2007) 0.85
The compartmentalisation of phosphorylated free oligosaccharides in cells from a CDG Ig patient reveals a novel ER-to-cytosol translocation process. PLoS One (2010) 0.84
Neurotropism and retrograde axonal transport of a canine adenoviral vector: a tool for targeting key structures undergoing neurodegenerative processes. Mol Ther (2002) 0.84
Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy. Pharmacogenomics (2006) 0.84
Antigen-specific Treg impair CD8(+) T-cell priming by blocking early T-cell expansion. Eur J Immunol (2010) 0.84
Proteome analysis of differentiating human myoblasts by dialysis-assisted two-dimensional gel electrophoresis (DAGE). Proteomics (2008) 0.83
Rescue of a dystrophin-like protein by exon skipping normalizes synaptic plasticity in the hippocampus of the mdx mouse. Neurobiol Dis (2011) 0.83
Rescue of a dystrophin-like protein by exon skipping in vivo restores GABAA-receptor clustering in the hippocampus of the mdx mouse. Mol Ther (2010) 0.83
Exploiting natural diversity of AAV for the design of vectors with novel properties. Methods Mol Biol (2011) 0.83
Thrombospondin-1 is not the major activator of TGF-β1 in thrombopoietin-induced myelofibrosis. Blood (2010) 0.83
The ARRIVE guidelines, a welcome improvement to standards for reporting animal research. J Gene Med (2010) 0.83
Suboptimal provirus expression explains apparent nonrandom cell coinfection with HIV-1. J Virol (2012) 0.82
Regulatable systemic production of monoclonal antibodies by in vivo muscle electroporation. Genet Vaccines Ther (2004) 0.82
Central role of conventional dendritic cells in regulation of bone marrow release and survival of neutrophils. J Immunol (2014) 0.82
FGF6 mediated expansion of a resident subset of cells with SP phenotype in the C2C12 myogenic line. J Cell Physiol (2004) 0.82
Deleterious effect of peptide insertions in a permissive site of the AAV2 capsid. Virology (2003) 0.82
A comparison of synthetic oligodeoxynucleotides, DNA fragments and AAV-1 for targeted episomal and chromosomal gene repair. BMC Biotechnol (2009) 0.81
Changes in the accessibility of the HIV-1 Integrase C-terminus in the presence of cellular proteins. Retrovirology (2010) 0.81
A high throughput method for genome-wide analysis of retroviral integration. Nucleic Acids Res (2006) 0.81
Simple conditioning with monospecific CD4+CD25+ regulatory T cells for bone marrow engraftment and tolerance to multiple gene products. Blood (2006) 0.80
Longitudinal tracking of human dendritic cells in murine models using magnetic resonance imaging. Magn Reson Med (2010) 0.79