Published in J Biol Chem on July 23, 2002
Directed engineering of a high-expression chimeric transgene as a strategy for gene therapy of hemophilia A. Mol Ther (2009) 1.19
Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model. Blood (2009) 1.18
Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy. PLoS One (2012) 1.15
Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype. Blood (2013) 1.11
Animal models of hemophilia. Prog Mol Biol Transl Sci (2012) 1.04
Lentiviral vector platform for production of bioengineered recombinant coagulation factor VIII. Mol Ther (2010) 1.03
Non-classical anti-factor VIII C2 domain antibodies are pathogenic in a murine in vivo bleeding model. J Thromb Haemost (2009) 1.01
Comparison of factor VIII transgenes bioengineered for improved expression in gene therapy of hemophilia A. Hum Gene Ther (2009) 0.99
Gene therapy for hemophilia. Front Biosci (Landmark Ed) (2015) 0.94
Factor VIII A3 domain substitution N1922S results in hemophilia A due to domain-specific misfolding and hyposecretion of functional protein. Blood (2011) 0.91
Enhanced biosynthesis of coagulation factor VIII through diminished engagement of the unfolded protein response. J Biol Chem (2011) 0.90
The comparative immunogenicity of human and porcine factor VIII in haemophilia A mice. Thromb Haemost (2009) 0.88
Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A. Gene Ther (2012) 0.86
Development and characterization of recombinant ovine coagulation factor VIII. PLoS One (2012) 0.83
Advancements in gene transfer-based therapy for hemophilia A. Expert Rev Hematol (2009) 0.83
Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery. Mol Ther Methods Clin Dev (2014) 0.83
Expanding the ortholog approach for hemophilia treatment complicated by factor VIII inhibitors. J Thromb Haemost (2014) 0.81
Engineering less immunogenic and antigenic FVIII proteins. Cell Immunol (2015) 0.79
A major determinant of the immunogenicity of factor VIII in a murine model is independent of its procoagulant function. Blood (2012) 0.79
Helical organization of blood coagulation factor VIII on lipid nanotubes. J Vis Exp (2014) 0.79
Delivery of nucleic acid therapeutics by genetically engineered hematopoietic stem cells. Adv Drug Deliv Rev (2010) 0.79
Enhancing the pharmaceutical properties of protein drugs by ancestral sequence reconstruction. Nat Biotechnol (2016) 0.79
Engineering Factor Viii for Hemophilia Gene Therapy. J Genet Syndr Gene Ther (2011) 0.78
Lack of recombinant factor VIII B-domain induces phospholipid vesicle aggregation: implications for the immunogenicity of factor VIII. Haemophilia (2014) 0.78
Dimeric Organization of Blood Coagulation Factor VIII bound to Lipid Nanotubes. Sci Rep (2015) 0.78
A subset of high-titer anti-factor VIII A2 domain antibodies is responsive to treatment with factor VIII. Blood (2016) 0.77
Contribution of A1 subunit residue Q316 in thrombin-activated factor VIII to A2 subunit dissociation. Biochemistry (2007) 0.77
Engineered Hematopoietic Stem Cells as Therapeutics for Hemophilia A. J Genet Syndr Gene Ther (2011) 0.77
Lipid nanotechnologies for structural studies of membrane-associated proteins. Proteins (2014) 0.76
Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A. Mol Ther Methods Clin Dev (2016) 0.76
Evaluation of the biological differences of canine and human factor VIII in gene delivery: implications in human hemophilia treatment. Gene Ther (2016) 0.75
Evidence That Factor VIII Forms a Bivalent Complex with the Low Density Lipoprotein (LDL) Receptor-related Protein 1 (LRP1): IDENTIFICATION OF CLUSTER IV ON LRP1 AS THE MAJOR BINDING SITE. J Biol Chem (2016) 0.75
Ectopic platelet-delivered factor (F) VIII for the treatment of Hemophilia A: Plasma and platelet FVIII, is it all the same? J Genet Syndr Gene Ther (2011) 0.75
Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models. JCI Insight (2016) 0.75
Structure of the factor VIII C2 domain in a ternary complex with 2 inhibitor antibodies reveals classical and nonclassical epitopes. Blood (2013) 1.47
Expression and characterization of recombinant murine factor VIII. Thromb Haemost (2002) 1.47
High-resolution mapping of epitopes on the C2 domain of factor VIII by analysis of point mutants using surface plasmon resonance. Blood (2014) 1.46
Use of blood outgrowth endothelial cells for gene therapy for hemophilia A. Blood (2002) 1.38
Identification of porcine coagulation factor VIII domains responsible for high level expression via enhanced secretion. J Biol Chem (2003) 1.21
Targeting tissue factor-expressing tumor angiogenesis and tumors with EF24 conjugated to factor VIIa. J Drug Target (2008) 1.17
Reduction of the inhibitory antibody response to human factor VIII in hemophilia A mice by mutagenesis of the A2 domain B-cell epitope. Blood (2004) 1.10
A1 subunit-mediated regulation of thrombin-activated factor VIII A2 subunit dissociation. J Biol Chem (2006) 1.07
High-level expression of porcine factor VIII from genetically modified bone marrow-derived stem cells. Blood (2006) 1.05
Lentiviral vector platform for production of bioengineered recombinant coagulation factor VIII. Mol Ther (2010) 1.03
High-dose factor VIII inhibits factor VIII-specific memory B cells in hemophilia A with factor VIII inhibitors. Blood (2005) 1.02
Nonclassical anti-C2 domain antibodies are present in patients with factor VIII inhibitors. Blood (2008) 1.02
The diversity of the immune response to the A2 domain of human factor VIII. Blood (2013) 0.96
Factor VIII A3 domain substitution N1922S results in hemophilia A due to domain-specific misfolding and hyposecretion of functional protein. Blood (2011) 0.91
The comparative immunogenicity of human and porcine factor VIII in haemophilia A mice. Thromb Haemost (2009) 0.88
Characterization and solution structure of the factor VIII C2 domain in a ternary complex with classical and non-classical inhibitor antibodies. J Biol Chem (2013) 0.88
Factor VIII expression in azoxymethane-induced murine fulminant hepatic failure. Blood (2002) 0.82
Decreased factor VIII levels during acetaminophen-induced murine fulminant hepatic failure. Blood (2003) 0.82
A quantitative measure of the efficacy of factor VIII in hemophilia A mice. Thromb Haemost (2003) 0.81
A major determinant of the immunogenicity of factor VIII in a murine model is independent of its procoagulant function. Blood (2012) 0.79
Epitope mapping via selection of anti-FVIII antibody-specific phage-presented peptide ligands that mimic the antibody binding sites. Thromb Haemost (2014) 0.78
Contribution of A1 subunit residue Q316 in thrombin-activated factor VIII to A2 subunit dissociation. Biochemistry (2007) 0.77
Visualizing cancer and response to therapy in vivo using Cy5.5-labeled factor VIIa and anti-tissue factor antibody. J Drug Target (2014) 0.76