Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies

Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII

(2015) O. A. Marcos-Contreras et al.   BLOOD

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

(2015) J. M. Crudele et al.   BLOOD

Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A

(2015) B. A. Konkle et al.   BLOOD

Recombinant activated factor VII: 30 years of research and innovation

(2015) Ulla Hedner   BLOOD REVIEWS

Alloantibodies to therapeutic factor VIII in hemophilia A: the role of von Willebrand factor in regulating factor VIII immunogenicity

(2015) J. Oldenburg et al.   HAEMATOLOGICA

Changes in the amino acid sequence of the recombinant human factor VIIa analog, vatreptacog alfa, are associated with clinical immunogenicity

(2015) J. N. Mahlangu et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

(2015) Alessio Cantore et al.   Science Translational Medicine

Alloantibodies to therapeutic factor VIII in hemophilia A: the role of von Willebrand factor in regulating factor VIII immunogenicity

(2015) J. Oldenburg et al.   HAEMATOLOGICA

Translational Data from Adeno-Associated Virus-Mediated Gene Therapy of Hemophilia B in Dogs

(2015) Timothy C. Nichols et al.   Human Gene Therapy Clinical Development

Omental implantation of BOECs in hemophilia dogs results in circulating FVIII antigen and a complex immune response

(2014) M. C. Ozelo et al.   BLOOD

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2013) J. I. Siner et al.   BLOOD

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

(2013) J. McIntosh et al.   BLOOD

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

Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A

(2013) A. Tiede et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Endogenous factor VIII synthesis from the intron 22–inverted F8 locus may modulate the immunogenicity of replacement therapy for hemophilia A

(2013) Gouri Shankar Pandey et al.   NATURE MEDICINE

Animal Models of Hemophilia and Related Bleeding Disorders

(2013) Jay N. Lozier et al.   SEMINARS IN HEMATOLOGY

Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A

(2013) Lily M. Du et al.   Nature Communications

Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs

(2012) J. A. Dumont et al.   BLOOD

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy

(2012) J. D. Finn et al.   BLOOD

F8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysis

(2012) S. C. Gouw et al.   BLOOD

Pharmacokinetics and pharmacodynamics of turoctocog alfa and N8-GP in haemophilia A dogs

(2012) H. Agersø et al.   HAEMOPHILIA

Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity

(2012) E. L. KUETHER et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage

(2011) C. Binny et al.   BLOOD

Pharmacokinetics, pharmacodynamics and safety of recombinant canine FVIIa in a study dosing one haemophilia A and one haemostatically normal dog

(2011) T. KNUDSEN et al.   HAEMOPHILIA

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

(2011) Amit C Nathwani et al.   MOLECULAR THERAPY

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

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

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

(2010) J. D. Finn et al.   BLOOD

Intravascular inhibition of factor VIIa and the analogue NN1731 by antithrombin

(2010) Lars C. Petersen et al.   BRITISH JOURNAL OF HAEMATOLOGY

Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B

(2010) T. C. NICHOLS et al.   HAEMOPHILIA

Characterization of canine coagulation factor VII and its complex formation with tissue factor: canine-human cross-species compatibility

(2010) T. KNUDSEN et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application

(2010) Paul E Monahan 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

Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy

(2008) G. P. Niemeyer et al.   BLOOD

Successful treatment of canine hemophilia by continuous expression of canine FVIIa

(2008) P. Margaritis et al.   BLOOD

Optimization of Self-complementary AAV Vectors for Liver-directed Expression Results in Sustained Correction of Hemophilia B at Low Vector Dose

(2007) Zhijian Wu et al.   MOLECULAR THERAPY