CORDIS Project
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This project aims to develop cell and gene therapies for Hemophilia A by exploring the potential of human liver cells and bone marrow cells to produce Factor VIII. It investigates innovative approaches to treat this bleeding disorder using patient-specific stem cells.
Currently, haemophilia A cannot be cured.
To prevent major bleeding episodes in haemophilia, human Factor VIII (FVIII) protein must be frequently administered as prophylaxis or on demand.
This treatment is complicated by its high cost and development of antibodies that neutralize FVIII activity in 20 to 30% of the patients.
Therefore, permanent solutions in the form of cell and gene therapy are very attractive for haemophilia A.
Recently, we demonstrated in a murine model that liver sinusoidal e…
UNIVERSITA DEGLI STUDI DEL PIEMONTE ORIENTALE AMEDEO AVOGADRO
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