Article
Enhanced plasma factor VIII activity in mice via cysteine mutation using dual vectors.
Science China. Life sciences - 1 Jun 2012
Zhu Fuxiang, Liu Zelong, Miao Jing, Qu Huige, Chi Xiaoyan
Abstract excerpt
Hemophilia A is caused by a genetic mutation in coagulation factor VIII (FVIII) gene and gene therapy is considered to be a promising strategy for its treatment. We recently demonstrated that co-delivery of two vectors expressing M662C mutated heavy and D1828C mutated light chain genes of B-domain-deleted coagulation factor VIII (BDD-FVIII) leads to inter-chain disulfide cross-linking and improved heavy chain...
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