A high efficient FVIII variant corrects bleeding in hemophilia A mouse model.

[1]  Guangwei Gao,et al.  A new mouse model for wound healing in hemophilia A. , 2015, International journal of clinical and experimental pathology.

[2]  John E. Murphy,et al.  Noncovalent stabilization of the factor VIII A2 domain enhances efficacy in hemophilia A mouse vascular injury models. , 2015, Blood.

[3]  Li Gui-lin Huang Jia-yu Li Da-wei Gao Guang-wei Study of the Effects of A and C-Domain Glycosylation Sites on the Secretion and Activity of Recombinant Factor VIII , 2014 .

[4]  L. Nemes,et al.  Interim data on long‐term efficacy, safety and tolerability of a plasma‐derived factor VIII concentrate in 109 patients with severe haemophilia A , 2012, Haemophilia : the official journal of the World Federation of Hemophilia.

[5]  D. Lillicrap,et al.  Mutation-specific hemostatic variability in mice expressing common type 2B von Willebrand disease substitutions. , 2010, Blood.

[6]  C. Négrier,et al.  Activated factor X cleaves factor VIII at arginine 562, limiting its cofactor efficiency , 2010, Journal of thrombosis and haemostasis : JTH.

[7]  J. Powell Recombinant factor VIII in the management of hemophilia A: current use and future promise , 2009, Therapeutics and clinical risk management.

[8]  P. Fay,et al.  Combining mutations of charged residues at the A2 domain interface enhances factor VIII stability over single point mutations , 2009, Journal of thrombosis and haemostasis : JTH.

[9]  P. Fay,et al.  Generation of enhanced stability factor VIII variants by replacement of charged residues at the A2 domain interface. , 2008, Blood.

[10]  Jason R. Cruz,et al.  Detailed Mechanisms of the Inactivation of Factor VIIIa by Activated Protein C in the Presence of Its Cofactors, Protein S and Factor V* , 2008, Journal of Biological Chemistry.

[11]  D. Jaeck,et al.  Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human , 2008, Cancer Gene Therapy.

[12]  Lemin Wang,et al.  The protein structure and effect of factor VIII. , 2007, Thrombosis research.

[13]  J. Oldenburg,et al.  Haemophilia A: from mutation analysis to new therapies , 2005, Nature Reviews Genetics.

[14]  S. Pipe,et al.  1 Bioengineering of Coagulation Factor VIII for Improved Secretion , 2004, Pediatric Research.

[15]  P. Fay Activation of factor VIII and mechanisms of cofactor action. , 2004, Blood reviews.

[16]  R. Montgomery,et al.  Expression of human factor VIII under control of the platelet-specific alphaIIb promoter in megakaryocytic cell line as well as storage together with VWF. , 2003, Molecular genetics and metabolism.

[17]  Y. Oh,et al.  Prolonged organ retention and safety of plasmid DNA administered in polyethylenimine complexes , 2001, Gene Therapy.

[18]  K. Loeb,et al.  Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

[19]  J. Behr,et al.  Systemic linear polyethylenimine (L‐PEI)‐mediated gene delivery in the mouse , 2000, The journal of gene medicine.

[20]  M. Shima,et al.  Role of activation of the coagulation factor VIII in interaction with vWf, phospholipid, and functioning within the factor Xase complex. , 1999, Trends in cardiovascular medicine.

[21]  R. Hynes,et al.  A mouse model of severe von Willebrand disease: defects in hemostasis and thrombosis. , 1998, Proceedings of the National Academy of Sciences of the United States of America.

[22]  R. Kaufman,et al.  Mutagenesis of a Potential Immunoglobulin-binding Protein-binding Site Enhances Secretion of Coagulation Factor VIII* , 1997, The Journal of Biological Chemistry.

[23]  H. Schwarz,et al.  Assessment of Bleeding for the Evaluation of Therapeutic Preparations in Small Animal Models of Antibody-Induced Hemophilia and von Willebrand Disease , 1997, Thrombosis and Haemostasis.

[24]  R. Kaufman,et al.  A 110-amino Acid Region within the A1-domain of Coagulation Factor VIII Inhibits Secretion from Mammalian Cells (*) , 1995, The Journal of Biological Chemistry.

[25]  A. Giles,et al.  Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII. , 1993, Blood.

[26]  R. Kaufman,et al.  The relationship of N-linked glycosylation and heavy chain-binding protein association with the secretion of glycoproteins , 1987, The Journal of cell biology.

[27]  E. Jaffe,et al.  Synthesis of factor VIII antigen by cultured guinea pig megakaryocytes. , 1977, The Journal of clinical investigation.