ADENO-ASSOCIATED VIRUS VECTORS FOR EXPRESSING FVIII MIMETICS AND USES THEREOF

摘要

The present inventors now sought to provide for a gene therapy approach for the treatment of hemophilia A that is both safe and effective for human use and that is not affected by the presence of FVIII neutralizing antibodies. The current inventors now provide for means, methods and uses of nucleic acid sequences encoding a variant human FIX protein that mimic FVIII activity, that can be applied in a gene therapy approach suitable and effective for humans.