Correction model of hemophilia A mice with multicopy of reprogramming factors and factor VIII gene using AC

2016 Fiscal Year Final Research Report

• Project/Area Number: 26860733
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Hematology
• Research Institution: Tottori University
• Principal Investigator: Kurosaki Hajime 鳥取大学, 医学(系)研究科(研究院), 助教 (70464295)
• Project Period (FY): 2014-04-01 – 2017-03-31
• Keywords: 血友病 / 幹細胞 / 再生医療

Outline of Final Research Achievements

Artificial chromosome (AC) vectors have some unique characteristics as compared with conventional vectors, carrying large transgenes without size limitation, showing persistent expression of transgenes, and existing independently from host genome in cells. Hemophilia A (HA) is X chromosome-linked hemorrhagic disorder caused by defects in the coagulation factor VIII (FVIII) gene. Induced pluripotent stem (iPS) cells, which can be generated from an individual’s own tissues and contribute to any tissues, have a great potential for gene therapy. Induced-PS cells were generated from HA mouse embryonic fibroblasts by introducing AC vector with four reprogramming factors. The iPS cells were corrected by transferring AC vector with FVIII gene. The AC vector system, which contains the combination of reprogramming factors for making iPS cells and complement genes, may be a promising tool for safer gene- and cell-therapy of HA.

Free Research Field

再生医科学