NIH R01 · 2025
Targeting Regulatory T cells for Factor VIII Tolerance
PROJECT SUMMARY Children and adults with the inherited bleeding disorder hemophilia A are reliant on infusions of coagulation protein factor VIII (FVIII) to treat bleeding episodes. The most significant complication of FVIII replacement therapy is the formation of neutralizing antibodies called inhibitors against the infused FVIII protein. Inhibitors increases the risk of bleeding episodes that are difficult to control, which ultimately affects quality of life. However, the advent of novel non-factor therapies used to prevent bleeding has dramatically changed the treatment paradigm for persons with hemophilia A (PwHA) and inhibitors. Despite the tremendous advancements in treatment options…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.