Discovery of an Orally Effective Factor IX-Transferrin Fusion Protein for Hemophilia B
نویسندگان
چکیده
منابع مشابه
Coagulation Factor IX for Hemophilia B Therapy
Factor IX is a zymogen enzyme of the blood coagulation cascade. Inherited absence or deficit of the IX functional factor causes bleeding disorder hemophilia B, which requires constant protein replacement therapy. Reviewed herein are the current state in the manufacturing of FIX, improved variants of the recombinant protein for therapy, transgenic organisms for obtaining FIX, and the advances in...
متن کاملMolecular Characterization of the Factor IX Gene in 28 Iranian Hemophilia B Patients
Background: Heterogeneous mutations in the human coagulation factor IX gene lead to an X-linked recessive bleeding disorder known as hemophilia B. The disease is distributed worldwide with no ethnic or geographical priority. Materials and Methods: The aim of this study was to characterize the factor IX gene mutations in 28 unrelated Iranian hemophilia B patients. Polymerase chain reaction (PCR)...
متن کاملFactor IX variants improve gene therapy efficacy for hemophilia B.
Intramuscular injection of adeno-associated viral (AAV) vector to skeletal muscle of humans with hemophilia B is safe, but higher doses are required to achieve therapeutic factor IX (F.IX) levels. The efficacy of this approach is hampered by the retention of F.IX in muscle extracellular spaces and by the limiting capacity of muscle to synthesize fully active F.IX at high expression rates. To ov...
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 کاملCirculating factor IX antigen-inhibitor complexes in hemophilia B- following infusion of a factor IX concentrate.
A persistent low-titer factor IX Inhibitor was discovered in a patIent with severe hemophllia B. The Inhibitor was very likely an immunoglobulin, since it was present in serum, was not dialyzable, retained its potency after heating to 56#{176}C, and was bound by staphylococcal protein A (SPA). When the hemophilia B patient with the inhibItor was given therapeutic infusions of factor Ix concentr...
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ژورنال
عنوان ژورنال: International Journal of Molecular Sciences
سال: 2019
ISSN: 1422-0067
DOI: 10.3390/ijms21010021