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Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. [5] [6] [7] Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. [7]
Marstacimab is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in people aged twelve years of age and older with hemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX inhibitors.
Factors VII, IX, and X all play key roles in blood coagulation and also share a common domain architecture. [10] The factor IX protein is composed of four protein domains: the Gla domain, two tandem copies of the EGF domain and a C-terminal trypsin-like peptidase domain which carries out the catalytic cleavage.
Haemophilia B, also spelled hemophilia B, is a blood clotting disorder causing easy bruising and bleeding due to an inherited mutation of the gene for factor IX, and resulting in a deficiency of factor IX. It is less common than factor VIII deficiency (haemophilia A). [3] Haemophilia B was first recognized as a distinct disease entity in 1952. [4]
It is used to treat and prevent bleeding in hemophilia B if pure factor IX is not available. [9] [11] It may also be used for reversal of warfarin therapy. [11] It is given by slow injection into a vein. [9] Another product, activated prothrombin complex concentrate or FEIBA (Factor Eight Inhibitor Bypassing Agent), may be used for acquired ...
Two complex chimpanzee blood group systems, V-A-B-D and R-C-E-F systems, proved to be counterparts of the human MNS and Rh blood group systems, respectively. Two blood group systems have been defined in Old World monkeys: the Drh system of macaques and the Bp system of baboons, both linked by at least one species shared by either of the blood group systems.
Hemophilia, or haemophilia, is an X-linked recessive disorder that impairs the body's control over blood clotting. Haemophilia A and Haemophilia B arise from mutations in the genes for factor VIII and factor IX, respectively. [6] Females with this disease are almost exclusively unaffected, obligate carriers.
Cat genetics describes the study of inheritance as it occurs in domestic cats. In feline husbandry, it can predict established traits ( phenotypes ) of the offspring of particular crosses. In medical genetics , cat models are occasionally used to discover the function of homologous human disease genes.