Hemophilia A Models

Wild Mouse 750
Wild Mouse 750
Human Xenograft Models
Cell Derived Xenograft Models
Autoimmune Disease Models
Systemic Lupus Erythematosus ModelsMultiple Sclerosis ModelsRheumatoid Arthritis ModelsPsoriasis ModelsInflammatory Bowel Disease ModelsAtopic Dermatitis Models Asthma Models
Metabolic Disease Mouse Models
Obesity ModelsDiabetes ModelsNon-alcoholic Steatohepatitis (NASH) ModelsHyperlipidemia ModelsOsteoporosis ModelsHyperuricemia Models
Humanized Immune System Models
huPBMC-NCGhuHSC-NCG-XhuHSC-NCG-hIL15huHSC-NCG
Organs or Tissue Humanized Mouse Models
Wild Mouse 765
Wild Mouse 765
Rare Disease Mouse Models
Progressive Muscular Dystrophy ModelsHemophilia A Models
Neurodegenerative Disease Mouse Models
Alzheimer’s Disease (AD) ModelsParkinson’s Disease (PD) ModelsHuntington's Disease (HD) ModelsAmyotrophic Lateral Sclerosis (ALS) ModelsTransthyretin Amyloidosis (ATTR) Models
Immuno-Oncology Mouse Models
Human Xenograft ModelsHumanized Immune Checkpoint ModelsHumanized Immune System ModelsSpontaneous Tumor Models

Hemophilia is a coagulation disorder caused by mutations in specific genes encoding clotting factors. There are two major categories of hemophilia: hemophilia A and B. 80 to 85% of patients with hemophilia are affected by hemophilia A or B. Hemophilia is characterized by either spontaneous or non-spontaneous bleeding, including uncontrollable bleeding during trauma or surgery and irreversible joint injury resulting from frequent bleeding in the joint cavity. Hemophilia A (HA) is a bleeding disorder caused by the absence of clotting factor VIII (FVIII), which is primarily produced and secreted by the liver into the bloodstream. FVIII participates in the endogenous clotting pathway, activating downstream clotting factors and prothrombin via a series of cascades with amplification effects, which activates thrombinogen, ultimately producing a hemostatic plug to stop bleeding. Its coding gene, FVIII, is located on the X chromosome, and different mutations, including point mutations, missense mutations, translocations, and insertions can lead to insufficient synthesis or malfunction of FVIII and the development of hemophilia.

GemPharmatech has developed a B6-F8-KO hemophilia A mouse model by knocking out certain exons of the C57BL/6JGpt FVIII gene, which causes a frameshift mutation that results in FVIII protein deficit. This model can simulate the pathophysiology and main symptoms of hemophilia. It can also be used to evaluate the efficacy and safety of gene therapies and screening for other types of treatments for hemophilia A.


Application

  • Screening of hemophilia A therapeutics

  • Efficacy and safety evaluation of hemophilia A gene therapies

  • Pathogenesis study of hemophilia