News Column

Data on Gene Therapy Reported by Researchers at Jichi Medical University

January 30, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Investigators discuss new findings in Biotechnology. According to news reporting from Tochigi, Japan, by NewsRx journalists, research stated, "The use of induced pluripotent stem cells (iPSCs) as an autologous cell source has shed new light on cell replacement therapy with respect to the treatment of numerous hereditary disorders. We focused on the use of iPSCs for cell-based therapy of haemophilia."

The news correspondents obtained a quote from the research from Jichi Medical University, "We generated iPSCs from mesenchymal stem cells that had been isolated from C57BL/6 mice. The mouse iPSCs were generated through the induction of four transcription factor genes Oct3/4, Klf-4, Sox-2 and c-Myc. The derived iPSCs released functional coagulation factor VIII (FVIII) following transduction with a simian immunodeficiency virus vector. The subcutaneous transplantation of iPSCs expressing FVIII into nude mice resulted in teratoma formation, and significantly increased plasma levels of FVIII. The plasma concentration of FVIII was at levels appropriate for human therapy at 2-4weeks post transplantation."

According to the news reporters, the research concluded: "Our data suggest that iPSCs could be an attractive and prospective autologous cell source for the production of coagulation factor, and that engineered iPSCs expressing coagulation factor might provide a cell-based therapeutic strategy appropriate for haemophilia."

For more information on this research see: Production of functional coagulation factor VIII from iPSCs using a lentiviral vector. Haemophilia, 2014;20(1):E40-E44. Haemophilia can be contacted at: Wiley-Blackwell, 111 River St, Hoboken 07030-5774, NJ, USA. (Wiley-Blackwell -; Haemophilia -

Our news journalists report that additional information may be obtained by contacting Y. Kashiwakura, Jichi Med Univ, Center Mol Med, Div Genet Therapeut, Shimotsuke, Tochigi 3290498, Japan. Additional authors for this research include T. Ohmori, J. Mimuro, S. Madoiwa, M. Inoue, M. Hasegawa, K. Ozawa and Y. Sakata (see also Biotechnology).

Keywords for this news article include: Asia, Biotechnology, Japan, Tochigi, Factor VIII, Gene Therapy, Therapeutics, Bioengineering, Blood Coagulation Factors

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Source: Gene Therapy Weekly

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