Published in Hum Gene Ther on January 26, 2012
New applications for phage integrases. J Mol Biol (2014) 0.98
Delivery of full-length factor VIII using a piggyBac transposon vector to correct a mouse model of hemophilia A. PLoS One (2014) 0.89
Development and characterization of recombinant ovine coagulation factor VIII. PLoS One (2012) 0.83
piggyBac-mediated phenotypic correction of factor VIII deficiency. Mol Ther Methods Clin Dev (2014) 0.82
Neuroprotective therapies in glaucoma: II. Genetic nanotechnology tools. Front Neurosci (2015) 0.77
Intragenic integration in DLC1 sustains factor VIII expression in primary human cells without insertional oncogenicity. Gene Ther (2014) 0.77
Hybrid lentivirus-phiC31-int-NLS vector allows site-specific recombination in murine and human cells but induces DNA damage. PLoS One (2014) 0.76
Genome Integration and Excision by a New Streptomyces Bacteriophage, ϕJoe. Appl Environ Microbiol (2017) 0.75
Simplified mammalian DNA isolation procedure. Nucleic Acids Res (1991) 12.56
A phage integrase directs efficient site-specific integration in human cells. Proc Natl Acad Sci U S A (2000) 5.13
In vitro site-specific integration of bacteriophage DNA catalyzed by a recombinase of the resolvase/invertase family. Proc Natl Acad Sci U S A (1998) 5.04
Hepatotoxicity and mechanism of action of haloalkanes: carbon tetrachloride as a toxicological model. Crit Rev Toxicol (2003) 4.25
Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet (1995) 3.74
Site-specific genomic integration in mammalian cells mediated by phage phiC31 integrase. Mol Cell Biol (2001) 3.39
Analysis of the integration function of the streptomycete bacteriophage phi C31. J Mol Biol (1991) 2.82
A generic intron increases gene expression in transgenic mice. Mol Cell Biol (1991) 2.75
Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol (2002) 2.54
Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. Mol Ther (2000) 2.04
Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med (2001) 2.01
Integration specificity of phage phiC31 integrase in the human genome. J Mol Biol (2005) 1.95
Enzyme-free cloning: a rapid method to clone PCR products independent of vector restriction enzyme sites. Nucleic Acids Res (1999) 1.69
Structural analysis of the actinophage phi C31 attachment site. Nucleic Acids Res (1991) 1.68
The factor VIII Structure and Mutation Resource Site: HAMSTeRS version 4. Nucleic Acids Res (1998) 1.66
In vivo correction of murine hereditary tyrosinemia type I by phiC31 integrase-mediated gene delivery. Mol Ther (2005) 1.59
Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood (2003) 1.47
Prevention and treatment of factor VIII inhibitors in murine hemophilia A. Blood (2000) 1.36
Hydrodynamic gene delivery to the pig liver via an isolated segment of the inferior vena cava. Gene Ther (2007) 1.31
Naked DNA transfer of Factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice. Mol Ther (2004) 1.24
Mutational derivatives of PhiC31 integrase with increased efficiency and specificity. Mol Ther (2008) 1.23
Mechanism of the immune response to human factor VIII in murine hemophilia A. Thromb Haemost (2001) 1.10
Clinical gene transfer studies for hemophilia A. Semin Thromb Hemost (2004) 1.07
Abnormal hemostasis in a knock-in mouse carrying a variant of factor IX with impaired binding to collagen type IV. J Thromb Haemost (2009) 0.99
Biosafety assessment of site-directed transgene integration in human umbilical cord-lining cells. Mol Ther (2010) 0.94
Factor VIII inhibitors: structure and function in autoantibody and hemophilia A patients. Semin Hematol (1994) 0.92
Creation of a mouse expressing defective human factor IX. Blood (2004) 0.90
Preclinical and clinical progress in hemophilia gene therapy. Curr Opin Hematol (2010) 0.87
Tolerance to factor VIII in a transgenic mouse expressing human factor VIII cDNA carrying an Arg(593) to Cys substitution. Thromb Haemost (2006) 0.87
HLA class II genotype and factor VIII inhibitors in mild haemophilia A patients with an Arg593 to Cys mutation. Haemophilia (2004) 0.86
Gene therapy strategies for hemophilia: benefits versus risks. J Gene Med (2010) 0.85
Critical amino acid residues within the φC31 integrase DNA-binding domain affect recombination activities in mammalian cells. Hum Gene Ther (2010) 0.83
A factor VIII minigene comprising the truncated intron I of factor IX highly improves the in vitro production of factor VIII. Thromb Haemost (2001) 0.80
Factoring nonviral gene therapy into a cure for hemophilia A. Curr Opin Mol Ther (2008) 0.79
Molecular genetics of familial venous thrombosis. Br Med Bull (1994) 0.77
Construction of transgenic Drosophila by using the site-specific integrase from phage phiC31. Genetics (2004) 9.98
Phage integrases: biology and applications. J Mol Biol (2004) 3.53
Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol (2002) 2.54
KLF2 provokes a gene expression pattern that establishes functional quiescent differentiation of the endothelium. Blood (2006) 2.41
Stable nonviral genetic correction of inherited human skin disease. Nat Med (2002) 2.20
Integration specificity of phage phiC31 integrase in the human genome. J Mol Biol (2005) 1.95
Dynamics and plasticity of Weibel-Palade bodies in endothelial cells. Arterioscler Thromb Vasc Biol (2006) 1.88
Exosomes derived from IL-10-treated dendritic cells can suppress inflammation and collagen-induced arthritis. J Immunol (2005) 1.74
Uptake of blood coagulation factor VIII by dendritic cells is mediated via its C1 domain. J Allergy Clin Immunol (2011) 1.60
In vivo correction of murine hereditary tyrosinemia type I by phiC31 integrase-mediated gene delivery. Mol Ther (2005) 1.59
Phage TP901-1 site-specific integrase functions in human cells. J Bacteriol (2002) 1.57
Functional architecture of Weibel-Palade bodies. Blood (2011) 1.55
Altered glycosylation of platelet-derived von Willebrand factor confers resistance to ADAMTS13 proteolysis. Blood (2013) 1.55
Preferential HLA-DRB1*11-dependent presentation of CUB2-derived peptides by ADAMTS13-pulsed dendritic cells. Blood (2013) 1.49
Creating transgenic Drosophila by microinjecting the site-specific phiC31 integrase mRNA and a transgene-containing donor plasmid. Nat Protoc (2007) 1.47
The macrophage mannose receptor promotes uptake of ADAMTS13 by dendritic cells. Blood (2012) 1.46
PhiC31 integrase-mediated nonviral genetic correction of junctional epidermolysis bullosa. Hum Gene Ther (2003) 1.45
The spacer domain of ADAMTS13 contains a major binding site for antibodies in patients with thrombotic thrombocytopenic purpura. Thromb Haemost (2005) 1.39
Biogenesis and exocytosis of Weibel-Palade bodies. Histochem Cell Biol (2002) 1.32
A link between meiotic prophase progression and crossover control. PLoS Genet (2006) 1.29
An autoantibody epitope comprising residues R660, Y661, and Y665 in the ADAMTS13 spacer domain identifies a binding site for the A2 domain of VWF. Blood (2009) 1.27
A diversity of serine phage integrases mediate site-specific recombination in mammalian cells. Mol Genet Genomics (2006) 1.26
phiC31 integrase confers genomic integration and long-term transgene expression in rat retina. Invest Ophthalmol Vis Sci (2005) 1.24
Mutational derivatives of PhiC31 integrase with increased efficiency and specificity. Mol Ther (2008) 1.23
Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration. Proc Natl Acad Sci U S A (2005) 1.22
Analysis of the storage and secretion of von Willebrand factor in blood outgrowth endothelial cells derived from patients with von Willebrand disease. Blood (2013) 1.18
Nucleofection of muscle-derived stem cells and myoblasts with phiC31 integrase: stable expression of a full-length-dystrophin fusion gene by human myoblasts. Mol Ther (2004) 1.15
Amino acid regions 572-579 and 657-666 of the spacer domain of ADAMTS13 provide a common antigenic core required for binding of antibodies in patients with acquired TTP. Thromb Haemost (2006) 1.13
Phage integrases for the construction and manipulation of transgenic mammals. Reprod Biol Endocrinol (2003) 1.12
Real-time imaging of the dynamics and secretory behavior of Weibel-Palade bodies. Arterioscler Thromb Vasc Biol (2003) 1.11
Site-specific integration with phiC31 integrase for prolonged expression of therapeutic genes. Adv Genet (2005) 1.08
Essential domains of a disintegrin and metalloprotease with thrombospondin type 1 repeats-13 metalloprotease required for modulation of arterial thrombosis. Arterioscler Thromb Vasc Biol (2011) 1.08
Residues Arg568 and Phe592 contribute to an antigenic surface for anti-ADAMTS13 antibodies in the spacer domain. Haematologica (2011) 1.02
Factor VIII C1 domain spikes 2092-2093 and 2158-2159 comprise regions that modulate cofactor function and cellular uptake. J Biol Chem (2013) 1.01
Establishment of outgrowth endothelial cells from peripheral blood. Nat Protoc (2012) 1.01
PhiC31 integrase mediates integration in cultured synovial cells and enhances gene expression in rabbit joints. J Gene Med (2006) 1.00
IgG subclasses of anti-FVIII antibodies during immune tolerance induction in patients with hemophilia A. Br J Haematol (2008) 0.99
Intracellular cotrafficking of factor VIII and von Willebrand factor type 2N variants to storage organelles. Blood (2008) 0.99
Guanine exchange factor RalGDS mediates exocytosis of Weibel-Palade bodies from endothelial cells. Blood (2008) 0.99
Evidence for a role of anti-ADAMTS13 autoantibodies despite normal ADAMTS13 activity in recurrent thrombotic thrombocytopenic purpura. Haematologica (2011) 0.99
Phage phiC31 integrase-mediated genomic integration of the common cytokine receptor gamma chain in human T-cell lines. J Gene Med (2006) 0.98
Development of a novel helper-dependent adenovirus-Epstein-Barr virus hybrid system for the stable transformation of mammalian cells. J Virol (2004) 0.98
Storage and regulated secretion of factor VIII in blood outgrowth endothelial cells. Haematologica (2009) 0.94
Dynein-dynactin complex mediates protein kinase A-dependent clustering of Weibel-Palade bodies in endothelial cells. Arterioscler Thromb Vasc Biol (2005) 0.93
HLA-DR-presented peptide repertoires derived from human monocyte-derived dendritic cells pulsed with blood coagulation factor VIII. Mol Cell Proteomics (2011) 0.93
Site-specific chromosomal integration mediated by phiC31 integrase. Methods Mol Biol (2008) 0.93
DICE, an efficient system for iterative genomic editing in human pluripotent stem cells. Nucleic Acids Res (2013) 0.92
Low density lipoprotein receptor-related protein and factor IXa share structural requirements for binding to the A3 domain of coagulation factor VIII. J Biol Chem (2003) 0.92
Therapeutic applications of the ΦC31 integrase system. Curr Gene Ther (2011) 0.92
Safe genetic modification of cardiac stem cells using a site-specific integration technique. Circulation (2012) 0.90
Long-term increase in mVEGF164 in mouse hindlimb muscle mediated by phage phiC31 integrase after nonviral DNA delivery. Hum Gene Ther (2006) 0.90
Von Willebrand factor targets IL-8 to Weibel-Palade bodies in an endothelial cell line. Exp Cell Res (2003) 0.89
Immunobiology of inhibitor development in hemophilia A. Semin Thromb Hemost (2003) 0.89
Site-specific recombinase strategy to create induced pluripotent stem cells efficiently with plasmid DNA. Stem Cells (2011) 0.89
Intracellular storage and regulated secretion of von Willebrand factor in quantitative von Willebrand disease. J Biol Chem (2011) 0.89
Modification of an exposed loop in the C1 domain reduces immune responses to factor VIII in hemophilia A mice. Blood (2012) 0.88
Small GTP-binding protein Ral is involved in cAMP-mediated release of von Willebrand factor from endothelial cells. Arterioscler Thromb Vasc Biol (2004) 0.88
The shear stress-induced transcription factor KLF2 affects dynamics and angiopoietin-2 content of Weibel-Palade bodies. PLoS One (2012) 0.87
Tolerance to factor VIII in a transgenic mouse expressing human factor VIII cDNA carrying an Arg(593) to Cys substitution. Thromb Haemost (2006) 0.87
Proteomic screen identifies IGFBP7 as a novel component of endothelial cell-specific Weibel-Palade bodies. J Proteome Res (2012) 0.86
The Epac-Rap1 signaling pathway controls cAMP-mediated exocytosis of Weibel-Palade bodies in endothelial cells. J Biol Chem (2012) 0.84
Requirements for immune recognition and processing of factor VIII by antigen-presenting cells. Blood Rev (2011) 0.84
Impact of hydrodynamic injection and phiC31 integrase on tumor latency in a mouse model of MYC-induced hepatocellular carcinoma. PLoS One (2010) 0.84
Site-specific genomic strategies for gene therapy. Curr Opin Mol Ther (2003) 0.84
Site-specific integration with bacteriophage ΦC31 integrase. Cold Spring Harb Protoc (2012) 0.83
Differential effects of the loss of intrachain- versus interchain-disulfide bonds in the cystine-knot domain of von Willebrand factor on the clinical phenotype of von Willebrand disease. Thromb Haemost (2006) 0.83
Gene transfer to rabbit retina with electron avalanche transfection. Invest Ophthalmol Vis Sci (2006) 0.82
Rational design of small molecules targeting the C2 domain of coagulation factor VIII. Blood (2013) 0.82
Kinetics and longevity of ΦC31 integrase in mouse liver and cultured cells. Hum Gene Ther (2010) 0.81
The therapeutic potential of ΦC31 integrase as a gene therapy system. Expert Opin Biol Ther (2011) 0.81
Site-specific integration of transgene targeting an endogenous lox-like site in early mouse embryos. J Appl Genet (2010) 0.81
Long-term transgene expression in mouse neural progenitor cells modified with phiC31 integrase. J Neurosci Methods (2008) 0.81
Epstein-Barr virus vectors provide prolonged robust factor IX expression in mice. Biotechnol Prog (2003) 0.81
Factor VIII alters tubular organization and functional properties of von Willebrand factor stored in Weibel-Palade bodies. Blood (2011) 0.80
Effect of laminar shear stress on the distribution of Weibel-Palade bodies in endothelial cells. Thromb Res (2012) 0.80
Storage of factor VIII variants with impaired von Willebrand factor binding in Weibel-Palade bodies in endothelial cells. PLoS One (2011) 0.80
Extrachromosomal plasmid vectors for gene therapy. Curr Opin Mol Ther (2002) 0.80
Limited promiscuity of HLA-DRB1 presented peptides derived of blood coagulation factor VIII. PLoS One (2013) 0.80
Analysis of factor VIII inhibitors in a haemophilia A patient with an Arg593-->Cys mutation using phage display. Br J Haematol (2002) 0.79
Site-specific integration for high-level protein production in mammalian cells. Methods Mol Biol (2005) 0.79
Factoring nonviral gene therapy into a cure for hemophilia A. Curr Opin Mol Ther (2008) 0.79
Efficient reversal of phiC31 integrase recombination in mammalian cells. Biotechnol J (2012) 0.79
Factor VIII inhibitor in a patient with mild haemophilia A and an Asn618-->Ser mutation responsive to immune tolerance induction and cyclophosphamide. Br J Haematol (2002) 0.78
Biogenesis of Weibel-Palade bodies in von Willebrand's disease variants with impaired von Willebrand factor intrachain or interchain disulfide bond formation. Haematologica (2011) 0.78
Phospholipid vesicles interfere with the binding of antibody fragments to the light chain of factor VIII. Thromb Haemost (2005) 0.76
Evaluation of the haemostatic potential of factor VIII-heparin cofactor II hybrid proteins in a mouse model. Br J Haematol (2003) 0.76
Getting rid of bad memory. Blood (2011) 0.75
Improving on nature: redesigning ADAMTS13. Blood (2012) 0.75
Using phage integrases in a site-specific dual integrase cassette exchange strategy. Methods Mol Biol (2015) 0.75
Storage and secretion of naturally occurring von Willebrand factor A domain variants. Br J Haematol (2014) 0.75
Antibodies against the CUB1-2 domains of ADAMTS13 in a patient with benign monoclonal gammopathy: no causal relationship. Haematologica (2007) 0.75