RECOMBINANT EXPRESSION VECTOR SYSTEM FOR VARIANTS OF COAGULATION FACTOR VIII AND VON WILLEBRAND FACTOR

摘要

PURPOSE: An expression system of blood coagulation factor VIII and mutated von Willebrand factor is provided to have a reduced size, to enhance stabilization and activation efficiency of the blood coagulation factor VIII, and to be used in gene therapy for treating hemophilia. CONSTITUTION: A mutated von Willebrand factor(vWF23) has an amino acid sequence of sequence number 2 of which exon 24-46 is deleted. The mutated von Willebrand factor has a nucleotide encoding a protein having an amino acid of sequence number 2. The gene has a nucleotide of sequence number 1. An animal cell expression vector contains a gene encoding the mutated von Willebrand factor. The animal cell expression vector is lentivirus vector. The expression vector further contains a gene encoding B domain-deleted human coagulation factor VIII. The B domain-deleted human coagulation factor VIII has an amino acid sequence of sequence number 8. The lentivirus vector is co-transfected with pGag-pol, pRev, pTat, and pVSV-G. A pharmaceutical composition for preventing and treating hemophilia contains the expression vector, mutated von Willebrand factor, and B domain-deleted human coagulation factor VIII.