DesignScience_UCL Immunity & Transplantation Open Day

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Haemophilia Researchers: Edward Tuddenham, Amit Nathwani

Haemophilia is a disease that impairs blood clotting, a process used to stop bleeding when a blood vessel is cut or damaged. Both the A and B versions of the condition result from inheriting mutations on the X chromosome. Haemophilia B results from a mutation in the gene that makes the FIX protein, which is vital for blood clotting. We are currently carrying out clinical trials to see if gene therapy is an effective way to treat haemophilia B. Gene therapy is a treatment where a new gene is inserted into cells in order to make a therapeutic protein. In this case, to make FIX proteins the person can’t naturally make.


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