Development of non-viral vector for gene therapy strategy using iPS cells

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Aiming for developing a novel gene therapy of hemophilia A, we have developed non-viralbased gene delivery vectors that can maintain foreign gene expression for long term. We identified that new transposon vectors were able to maintain reporter gene expression in human iPS cells for up to 150 days. In addition, we report here for the first time that we can deliver the full-length Factor VIII cDNA with 7. 0 kb in size by using our transposon vectors. Our transposon vector is a promising technology for future gene therapy of severe hemophilia A.