489. Treatment for Hemophilia B Using Self- Complimentary AAV8 Vectors
نویسندگان
چکیده
منابع مشابه
Adenoviral Vectors for Hemophilia Gene Therapy
Hemophilia is an inherited blood clotting disorder resulting from deficiency of blood coagulation factors. Current standard of care for hemophilia patients is frequent intravenous infusions of the missing coagulation factor. Gene therapy for hemophilia involves the introduction of a normal copy of the deficient coagulation factor gene thereby potentially offering a definitive cure for the bleed...
متن کاملTreatment of hemophilia A and B.
Treatment for patients with hemophilia and other bleeding disorders has evolved over the past several decades. Replacement of the specific missing plasma protein is necessary for hemostasis to occur. Lyophilized factor concentrate products contain these proteins. This chapter will address purity and viral safety issues for most of the coagulation products currently available. Dosing and nursing...
متن کاملRituximab for Treatment of Hemophilia A with High-Responder Inhibitors
Background: The development of inhibitors is a complication factor replacement therapy in hereditary factor VIII deficiency. Several management options are available for the treatment of inhibitor. Rituximab, a monoclonal antibody against CD20, reduces inhibitor level in rare bleeding disorders. The aim of this study was to evaluate the effectiveness of rituximab in lowering or eliminating the ...
متن کاملTreatment of hemophilia B: focus on recombinant factor IX
Hemophilia B is a recessive X-linked bleeding disorder characterized by deficiency of the coagulation factor IX (FIX). In hemophilia B patients the severity of the bleeding phenotype is related to the degree of the FIX defect. Hemophilia B treatment has improved greatly in the last 20 years with the introduction first of plasma-derived and then of recombinant FIX concentrates. Replacement thera...
متن کاملFederation of Hemophilia Inhibitor Treatment in Hemophilia A and B : Proceedings of the 2006
1997 (Suppl.): 162. 16 Gruppo R, Chen H, Schroth P, Bray GL. Safety and immunogenicity of recombinant factor VIII (recombinate) in previously untreated patients (PUPs). A 7.3 year update. WFH The Hague. Haemophilia 1998;
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ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2006
ISSN: 1525-0016
DOI: 10.1016/j.ymthe.2006.08.559