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

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

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

(2012) G. Buchlis et al.   BLOOD

Gene therapy for haemophilia: a long and winding road

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

Sensitivity of whole blood clotting time and activated partial thromboplastin time for factor IX: relevance to gene therapy and determination of post-transfusion elimination time of canine factor IX in hemophilia B dogs

(2011) T. C. NICHOLS et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

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

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

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

(2010) V. R. Arruda et al.   BLOOD

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

(2010) Virginia Haurigot et al.   MOLECULAR THERAPY

FIX-Triple, a gain-of-function factor IX variant, improves haemostasis in mouse models without increased risk of thrombosis

(2010) Chung-Yang Kao et al.   THROMBOSIS AND HAEMOSTASIS

Bioengineered Factor IX Molecules with Increased Catalytic Activity Improve the Therapeutic Index of Gene Therapy Vectors for Hemophilia B

(2009) Nicola Brunetti-Pierri et al.   HUMAN GENE THERAPY

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

(2009) Valder R Arruda et al.   MOLECULAR THERAPY

X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua)

(2009) Paolo Simioni et al.   NEW ENGLAND JOURNAL OF MEDICINE

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