Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype

Protein Replacement Therapy and Gene Transfer in Canine Models of Hemophilia A, Hemophilia B, von Willebrand Disease, and Factor VII Deficiency

(2013) T. C. Nichols et al.   ILAR JOURNAL

How we choose factor VIII to treat hemophilia

(2012) P. M. Mannucci et al.   BLOOD

Centralspindlin: At the heart of cytokinesis

(2012) Erin A. White et al.   Cytoskeleton

Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy

(2012) Brandon K. Sack et al.   PLoS One

Most factor VIII B domain missense mutations are unlikely to be causative mutations for severe hemophilia A: implications for genotyping

(2011) K. OGATA et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Gene therapy for haemophilia: a long and winding road

(2011) K. A. HIGH   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

A zymogen-like factor Xa variant corrects the coagulation defect in hemophilia

(2011) Lacramioara Ivanciu et al.   NATURE BIOTECHNOLOGY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

In vivo efficacy of platelet-delivered, high specific activity factor VIII variants

(2010) T. K. Greene et al.   BLOOD

Codon optimization of human factor VIII cDNAs leads to high-level expression

(2010) N. J. Ward et al.   BLOOD

Improvements in factor concentrates

(2010) David Lillicrap   CURRENT OPINION IN HEMATOLOGY

Immunoglobulin isotypes and functional anti-FVIII antibodies in response to FVIII treatment in Balb/c and C57BL/6 haemophilia A mice

(2010) M. QADURA et al.   HAEMOPHILIA

Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs

(2010) Virginia Haurigot et al.   MOLECULAR THERAPY

Lentiviral Vector Platform for Production of Bioengineered Recombinant Coagulation Factor VIII

(2010) H Trent Spencer et al.   MOLECULAR THERAPY

Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors

(2010) Denise E Sabatino et al.   MOLECULAR THERAPY

Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model

(2009) D. E. Sabatino et al.   BLOOD

The molecular basis of factor V and VIII procofactor activation

(2009) R. M. CAMIRE et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Strategies to Modulate Immune Responses: A New Frontier for Gene Therapy

(2009) Valder R Arruda et al.   MOLECULAR THERAPY