CORDIS Project
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This project aims to improve gene therapy for Haemophilia A by developing a manufacturing platform for adeno-associated virus vectors that deliver a novel clotting factor VIII variant. The goal is to enhance the safety and efficacy of treatment for this genetic disorder.
Haemophilia is a group of hereditary genetic disorders that impair the body's ability to maintain normal blood coagulation.
Haemophilia A (clotting factor VIII deficiency) is the most common form of the disorder, occurring in ~1 in 5,000 males in Europe.
Although different gene transfer strategies for factor replacement have been evaluated, several properties make adeno-associated virus (AAV) vectors the most promising.
Most importantly, AAV directs long-term transgene expression from non-dividi…
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