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VECTOR SYSTEM WHICH IS ABLE TO EXPRESS MUTATED VON WILLEBRAND FACTOR AND BLOOD COAGULATION FACTOR VIII WHICH IS USEFUL IN TREATING HEMOPHILIA
VECTOR SYSTEM WHICH IS ABLE TO EXPRESS MUTATED VON WILLEBRAND FACTOR AND BLOOD COAGULATION FACTOR VIII WHICH IS USEFUL IN TREATING HEMOPHILIA
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机译:可以表达变异的冯·威勒伯兰德因子和血液凝固因子VIII的矢量系统,可用于治疗血友病
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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.;COPYRIGHT KIPO 2010
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