857. Molecular Engineering of Factor VIII for rAAV Delivery

Protein replacement therapy for hemophilia A is very costly and has a potential side effect of developing inhibitors. Gene therapy, on the other hand, can potentially prevent these limitations of current treatments. Although recombinant adeno-associated virus (rAAV) vectors are promising for delivering factor VIII gene, applying AAV vector technology to Hemophilia A lagged behind other genetic diseases because of this size constraint (limited to |[sim]|5kb).