Concurrent with the development of recombinant factor replacement products, the
characterization of the F9 and F8 genes over 3 decades ago allowed for the development of …

Preclinical studies and initial clinical trials have documented the feasibility of
adenoassociated virus (AAV)–mediated gene therapy for hemophilia B. In an 8-year study …

Hemophilia A and hemophilia B are both X chromosome-linked recessive bleeding
disorders that affect about 1 in 5000 males and result from a deficiency in the coagulation …

Hemophilia B is a severe X-linked bleeding diathesis caused by the absence of functional
blood coagulation factor IX, and is an excellent candidate for treatment of a genetic disease …

Continuous expression of activated factor VII (FVIIa) via gene transfer is a potential
therapeutic approach for hemophilia patients with or without inhibitory antibodies to human …

Immune responses to factor IX (F. IX), a major concern in gene therapy for hemophilia, were
analyzed for adeno-associated viral (AAV-2) gene transfer to skeletal muscle and liver as a …

Inhibitory antibodies to factor VIII (FVIII) are a major complication in the treatment of
hemophilia A, affecting approximately 20% to 30% of patients. Current treatment for …

In a phase 1 dose escalation study, 13 subjects with hemophilia A received by peripheral
intravenous infusion a retroviral vector carrying a B-domain–deleted human factor VIII …

Background Valoctocogene roxaparvovec delivers a B-domain–deleted factor VIII coding
sequence with an adeno-associated virus vector to prevent bleeding in persons with severe …

High-capacity adenoviral (HC-Ad) vectors expressing B-domain–deleted human or canine
factor VIII from different liver-specific promoters were evaluated for gene therapy of …