Published in Gene Therapy Weekly, August 12th, 1996
The risk of transmission of infectious diseases, high cost, inconvenience of therapy, and problems associated with complications of hemophilia itself all add up to make the treatment of hemophilia difficult. Gene therapy, however, may offer an efficient, safe and convenient method for the treatment of hemophilia.
Research has shown that the B domain of the Factor VIII protein (FVIII) is structurally separate, functionally unnecessary, and actually decreases the efficiency of FVIII expression in tissue...
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